Engaging Youth in Global Health

Four universities, fifty students, ten months of research and writing now lead to culmination at the annual conference of Polygeia: Students Shaping Global Health, at the Cambridge Union Society. Our key message: motivated and bright young people are not only the global health leaders of the future, but have the desire and ability to improve global health today.

An emphasis on experts and experience in global health is understandable; but there are several important reasons why engaging young people can mobilise valuable resources in the field of global health improvement, and perhaps help solve some major challenges.

We don’t do silos or boxes – young people are seeking joined up solutions

No one is born into a particular team working on a particular issue; barriers to collaboration are progressively built as experience is accrued. At the very beginning of our careers or academic lives, young people have yet to build such high walls and we therefore work exceptionally effectively in cross-disciplinary teams.

Polygeia has this year been commissioned by the Africa All Party Parliamentary Group in the UK to write a report on lessons learned for community engagement from the West Africa Ebola Crisis. Rather than using a single perspective, the team for this project included students studying public health, African studies, medicine and management, ranging in experience from undergraduate to PhD.

As another example, working this summer with the Public Health Laboratory Ivo De Carneri, myself and colleagues looked to providing new perspectives to address old problems. This included research into the effectiveness of antibiotic stewardship on Zanzibar and supporting the development of the organisation’s 5-year strategy for research and organisational development. We explored the scope for ongoing research into traditional problems such as endemic helminthiases and other neglected tropical diseases, and identified new and emerging threats to health, including diabetes. This will help the organisation adapt to cope with the double burden of disease now faced.

We understand the power of connecting – young people want to see solutions applying technology with emotional intelligence

The pace at which technology is developing means that even “older” younger people, such as myself at the age of 24, can feel like we have a less comprehensive grasp of the latest developments than younger peers. Young people are in a strong position to work collaboratively with health experts in understanding and maximising the potential applications for eHealth, for big data and for the ways that technology can connect people, ideas and progress.

eHealth is a theme we explored as part of our ongoing series of guest blogs for IFPMA, exploring health partnerships such as the mVaccination programme in Mozambique to help parents remain up-to-date with their child’s vaccination schedule, and large-scale public-private partnerships using mobile technology to promote healthy lifestyles and reduce the global burden of chronic diseases.
Not only is there a team working on eHealth for Polygeia this year, other Polygeia teams have also been exploring the power of technology in this regard. For example, our mental health team have been exploring the use of mobile apps for mental health and wellness, and how these might be made available in low resource settings.

Tomorrow, we’ll be the ones on the front line – young people want to learn through experience

Whilst Polygeia advocates strongly that young people have the power to change the face of global health today, it is still true that young people will go on to be the health leaders of the future. This is especially important as we are faced with a global shortage of healthcare workers and people going into training to be healthcare professionals.

This is why we are keen to have the opportunity to partner with established organisations in global health, such as working together with IFPMA, CRC Press and the BMJ in preparation for our annual conference. Organisations who engage young people improve the skills and knowledge base of tomorrow’s leaders and shape our ability to address the great challenges of tomorrow.

IFPMA is proud to support Polygeia’s annual conference, taking place on Saturday 14th November at the Cambridge Union Society. Tickets are available online now.

“Polygeia” on Global Health Matters

Cell and Gene Therapies – The Next Transformative Pillar of Medicine

Genes. Chromosomes. Mutations. Today, these terms are as likely to be overheard in a casual dinner conversation as they are in scientific symposia and medical congresses. While the discovery of DNA dates back to 1869 when Swiss biochemist Johann Friedrich Miescher discovered nucleic acid, it was not until James Watson and Francis Crick published in 1953 their seminal paper in Nature that the science of genetics became commonplace and our vernacular was forever changed. Their finding that DNA replicates by separating into individual strands, each of which becomes the template for a new double helix, was heralded by some to be “the secret of life.” Similar exuberance followed the mapping of human genome in 2003 with the hope that we could now identify all of the “bad actors” in our DNA and find “simple” methods to fix them. However, as history teaches us, early science is almost always confounded by frustration, controversy and yes, failure. This has certainly been true for the field of cell and gene therapies.

The early days of gene therapy were befitting a Greek tragedy. Just as the promise of this new approach was gaining acceptance in the late 80s and early 90s, hopes and confidence were dashed in the wake of some devastating clinical setbacks. It seemed that this nascent field of science was on the verge of being extinguished. However, thanks to the unwavering dedication of undaunted researchers and the remarkable bravery of clinical trial participants, the field of gene therapy not only survived, but it has experienced a remarkable resurgence – due in large part to new methods of transduction which have led to better and safer ways to modify genes.

One of those intrepid pioneers, Dr. Carl June from the Perlman School of Medicine at the University of Pennsylvania, developed an approach which utilizes a disabled lentiviral vector to insert a new gene into a patient’s T cells, producing chimeric antigen receptors (CAR) T cells. These reprogramed T cells aim to hunt, bind to and eliminate cancer cells that have a specific antigen on their surface – such as CD19.

The results from Dr. June’s early trials in blood cancer were impressive. They have garnered headlines in some of the world’s foremost peer reviewed journals. They also led to Novartis’ interest in forming an innovative collaboration with University of Pennsylvania to develop and commercialize CART19 (now called CTL019) and conduct joint research on new CAR T cell therapies. In 2014, Novartis established a dedicated Cell & Gene Therapies Unit to streamline this complex process by combining and leveraging our deep capabilities in Clinical Development, Technical Operations, environment to our own broad scale manufacturing facility in Morris Plains, N.J. The cell reprograming center is the first US Food and Drug Administration (FDA) approved Good Manufacturing Practices quality site for a cell therapy. The facility is currently supporting our Phase II multi-center global study at clinical trial sites in the US, and we expect to expand those trials to the EU by the end of the year.

Novartis is leading the vanguard of investment in cell and gene therapies, and since we announced our commitment to this emerging new field, interest has spread like wildfire across the entire industry, energizing others to join the effort and build a new ecosystem. Additional players and different approaches will only lead to more and better therapies, and I am confident that our collective efforts will have a lasting impact on the lives of many who today have limited, if any, treatment options.

Clearly, many challenges still need to be overcome. Cell and gene therapies are not without side-effects or risks, and defined regulatory frameworks are needed to ensure the safety and efficacy of new products and provide sponsors with a clear path to making new therapies available to patients.

So what will the future hold for cell and gene therapies? Could Miescher have imagined where we would be today? We are tantalizingly close to treating hematologic malignancies with a brand new cell and gene-based therapeutic approach, and learning every day how to harness this potential and apply it to solid tumors, organ transplant and genetic disorders, to name a few. Whatever its shape or form, the recent renaissance in this field strengthens my belief that cell and gene therapies will be the next transformative pillar of medicine – becoming as impactful and disruptive as small molecules and biologics once were.

Usman Azam, MD
Global Head of the Cell & Gene Therapies Unit at Novartis Pharmaceuticals

– Click on the image to enlarge –


Today visionary, tomorrow down to business


As our heads of state prepare to meet later this month in New York, at the United Nations General Assembly (UNGA) to hopefully adopt the Sustainable Development Goals (SDGs), including the vision of Universal Health Coverage (UHC), I will allow myself a moment to appreciate the magnitude of the promise we are making to future generations.

The adoption of the SDGs gives governments and those of us involved in the health community a chance to embrace a vision as fundamental as the Declaration of Human Rights when it was adopted by the United Nations General Assembly in 1948. It is one of the few occasions where we are not over-dramatizing the point – the next generation will judge us on whether we measured up to the challenge.

I am sure many of us will feel the magnitude of this moment. However, I am also in no doubt that after the meeting we will have to roll up our sleeves and get down to work. From the innovative pharmaceutical industry’s perspective, we are in a good shape to lead the way in some areas; in others we will need to get stuck-in and work through the difficult questions together with others.

Since the launch of the Millennium Development Goals (MDGs) in 2000, those of us working in global health have learned a lot.

We have learned it is possible for the world to come together in the pursuit of shared goals for a common good. Our sector has a considerable track record in this area and we can measure up with the best of them, but we won’t be able to rest on our laurels.

We have learned that just in fifteen years we can halve the global deaths attributed to some of the world’s biggest killers such as TB or malaria. However, with chronic diseases increasingly threatening low- and middle-income countries, these advances may be short-lived. In addition to discovering and researching new treatments, innovative pharmaceutical companies are developing creative ways of leveraging the knowledge and expertise of local governments, industry and civil society, using new technologies to reach the greatest amount of people possible. We are also contributing to global partnerships pooling multiple stakeholders and technologies, such as Be He@lthy, Be Mobile, a partnership that uses mobile phones to tackle chronic diseases, spearheaded by the International Telecommunications Union and WHO, and supported by NGOs, the private sector and the African and Asian development banks.

Very recently, with the Ebola outbreak, we have also learned what it means to be unprepared, to come up short in the face of a crisis and see health systems collapse under pressure, and how quickly the repercussions can be felt around the world. While our pipelines are yielding new treatments and vaccines to contain this disease, we are reminded once again of the importance of resilient health systems and the need to ensure people have access to the health care they need without experiencing financial hardship. This is what Universal Health Coverage (UHC) addresses –and it is powerful stuff. UHC means a parent living in a rural India can reliably provide insulin for their child living with diabetes, it means a woman in Colombia will be screened for breast cancer so she can begin treatment before it is too late, it means a health center in Guinea that receives a patient infected with Ebola virus has the capacity to prevent further transmission in the community and to its health care workers. It means more than access to medicines alone.

With 400 million people lacking access to basic health care and more than 2 billion people lacking regular access to essential medicines, no one would say guaranteeing health as a right for all is an easy feat. The pharmaceutical industry is a strong advocate for UHC. In 2014 we identified a set of guiding principles in the areas we believe our industry can contribute, and with the adoption of the SDGs we are galvanized.

What the SDGs are demanding of us for the next 15 years goes far beyond fragmented improvements. The SDGs will require pooling resources, expertise and working together across sectors as well as with governments and civil society like never before.

It is no accident that the 17th goal of the SDGs is one dedicated entirely to the promotion of partnerships. Whereas partnerships in the health sector are by no means a new phenomenon, partnerships looking forward will be characterized not only by their ability to sustainably serve the needs of populations, but also by an increased linking up of diverse sectors.

To promote a mission as grand as UHC, we will likely find ourselves in partnerships that move beyond the vision of the SDG for health and the global health community, also tackling challenges around water and sanitation, urban living, education and climate change through convening fora such as the United Nations Global Compact.

There is increasing recognition that providing quality health coverage is a key contributor to the wealth and economic productivity of countries. As I look ahead to this next generation of targets, I am confident that though challenges remain, we are well placed to rise to the occasion. Achieving these new goals and closing the gaps in health coverage will take commitment and creative thinking, as well as cross-sectoral partnerships, and this is enthusiastically welcomed by myself and my colleagues in this industry.

mHealth: Responding to Global Health Emergencies

World Population Day, on the 11th July, was established by the Governing Council of the United Nations Development Program in 1989. Its aim is to raise awareness of global population problems, with this year’s focus on vulnerable populations in emergencies.

This theme is timely—the past 12 months have seen natural disasters strike the populations of Nepal and the Philippines, as well as the spread of Ebola virus in Guinea, Liberia and Sierra Leone and MERS in South Korea. Faced with such acute emergencies, this year has often felt like the global development community was always playing catch-up.

However, even just in the past 10 years, emergency responders and the global health community more broadly have been able to use a new tool: mobile technology. mHealth applications and communication tools are increasingly being developed to combat a range of issues; with most of the world’s population having mobile access, including 5 billion users in developing countries, it is now completely possible to reach those in the remote areas of the world.

mHealth in practice

Pharma companies are increasingly turning to mHealth programs to connect with difficult to reach communities. As an example, the Pediatric Outreach Telehealth project in Mali and Burkina Faso allows community health workers to input patient information into a centralized database to be analyzed by local doctors in real time. As a result, it is 3x easier for children to access healthcare through this system when compared to non-users.

Another example: the mVaccination program in Mozambique uses mHealth technology to increase coverage of lifesaving vaccines, monitoring vaccine stocks and alerting new parents via SMS when they are scheduled to visit the clinic.

mHealth is clearly beginning to impact upon everyday medicine worldwide – but can it be useful in a crisis?

mHealth and vulnerable populations in emergencies

After the 2010 earthquake in Haiti, trial mHealth programs were launched as parts of the official response.  One of the unique functions of the system was that assignment of a barcode to each patient, creating a centralized digital system for critical information through the disaster response. This was a marked improvement to the common practice of writing down a patient’s vitals in marker on their skin.

Where mHealth is not used to its full potential, for example during initial response to the Ebola Virus epidemic, when international partners were heavily criticized for a lack of effective collaboration, an absence of data sharing between governments, international organizations, private companies and NGO’s was pinpointed as in part responsible for the upwardly spiraling case load of Ebola virus disease (EVD). mHealth would have been an effective way to bridge these gaps and thus bring the epidemic to an earlier conclusion.

Still playing catch-up – mHealth and NCDs

In 2011, the United Nations General Assembly met for only the second time in its history to agree to an action plan in face of a health crisis: the first was to combat the transmission of HIV/AIDS in sub-Saharan Africa; this second meeting was on the rise of non-communicable diseases (NCDs).

Crises are not restricted to natural disasters and acute outbreaks. As stated by Dr. Margaret Chan, Director-General of the WHO, “the worldwide increase of non-communicable diseases is a slow-motion disaster.”[1]

Driven by globalization, urbanization and unhealthy lifestyles, NCDs may pose a threat of magnitude that we have never encountered before.

Luckily, mHealth is already being utilized in a number of ways to combat this serious problem. One such program is the International Telecommunications Union’s “Be He@lthy, Be Mobile”, which aims to disseminate health promotion messages on NCD risk factors, survey the epidemic and persuading users to change unhealthy behaviors.

The flexibility and penetrating potential of mHealth applications offer a unique way of safeguarding vulnerable populations, whether they are revealed as a result of natural disasters, infectious disease outbreaks or a surge in non-communicable diseases.

[1] Address at the High-level meeting on non-communicable diseases, United Nations General Assembly, 2011. Director-General of the WHO, Dr Margaret Chan. http://www.who.int/dg/speeches/2011/un_ncds_09_19/en/

4th African Regulatory Conference: Promising outcome

P_DThe 4th African Regulatory Conference generated unprecedented consensus on a shared vision to bring about genuine regulatory harmonization in Africa. Discussions built on the strong foundations laid by African Medicines Regulatory Harmonization (AMRH) that will ultimately shape a new regulatory landscape for Africa. This is a giant leap from when the first African Regulatory Conference took place in 2008 as no meaningful dialogue was yet in place among the different stakeholders. Dakshina Reddy and Paul Dearden, members of IFPMA’s Africa Regulatory Network (ARN), share some of the insights gained.

With the growing need for medicines every day, regulatory systems in Africa must improve if patients want to receive their medicines in a timely manner. With limited resources, it is essential that nations work together, share efforts, and look for efficient ways to assess the quality, safety, and efficacy of medicines. Referencing the African saying, Mr Ibrahima Wone, Secretary General of the Ministry of Health and Social Action of Senegal, opened the event with, “If you want to go fast, go alone; if you want to go far, go together.” This spirit was increasingly present throughout the conference, with a huge emphasis on collaboration and work-sharing, not only amidst regulators but also between industry, national regulatory authorities, including the World Health Organization (WHO).

It was clear from the outset that in an environment where many counterfeit and poor quARC2ality medicines exist, a strong need and commitment for trust was emphasized. By working together and supporting the capacity development of regulators, appropriate levels of trust can be built and national regulatory authorities will be equipped to make decisions in the interest of the health of their communities.

During the first sessions of the meeting, Dr Margareth Ndomondo-Sigonda, from the New Partnership for Africa’s Development (NEPAD), which is the technical arm of the African Union coordinating the African Medicines Regulatory Harmonization (AMRH) project,  explained that the aim for AMRH is to have one single agency for Africa through a step-wise approach, i.e. 54 national agencies harmonizing to five regions. In addition to regulatory harmonization of registration procedures, NEPAD wishes to pursue harmonization of clinical trial requirements, particularly as Africa is becoming increasingly involved as a location for clinical studies.

Dr John Patrick Mwesigye, East African Community (EAC), representing Tanzania, Kenya, Uganda, Rwanda, and Burundi pointed out that “Harmonization is underway in our region since November 2014; although launched with interest in selective products, the EAC is keen to invite other submissions and committed to further work on their guidelines, including variations procedures which affect the product’s availability during its lifecycle. The first two EAC approvals were achieved using a prequalification-type review with WHO support. The following five approvals were achieved by joint review by EAC agencies.”

Dr Luther Gwaza, from the Zimbabwe regulatory agency explained that the ZaZiBoNa initiative (short for Zambia, Zimbabwe, Botswana, and Namibia) has taken a pragmatic approach. They demonstrated the effectiveness of regulators taking a bold attitude towards changing the ‘business as usual’ approach and wanting to make progress and achieve real positive change. There is a strong drive from this group to ensure medicines are equally available in participating countries. Regarding the Southern African Development Community (SADC) region, there remains a question as to how much progress is ongoing, and this needs further consideration.

Professor Amadou Dieye, Director of the Pharmacy and Medicine Directorate of the Senegal Ministry of Health together with Dr Corneille Traoré, Director of Health, Social Protection and Mutual Insurance of the Commission of the West African Economic and Monetary Union (WAEMU)—an organization that supports West African harmonization for Benin, Burkina Faso, Cote d’Ivoire, Guinea Bissau, Mali, Niger, Senegal, and Togo—gave updates from their region which has least progressed in its discussions. They provided a strong commitment to the new work underway and the importance of seeking industry collaboration in drafting regulatory legislation and guidelines. In addition, WAEMU representatives were highlighting their local challenges – and stressed the political will required to implement these agreements in national law. Dr Traoré was passionate in his presentation stressing that “For regulatory agencies, working together is no longer an option – it is the only way forward.”

Throughout the two-day conference, there was lively discussion and debate around regulatory systems and pathways – important considerations when developing new regional frameworks. An interesting area of discussion is around options for achieving regulatory ‘review’ i.e. how agencies can reach the approval decision. Examples of other agency work-sharing e.g. EMA systems were highlighted followed by discussion surrounding the reliance on one’s own or others expert reviews, and the reliance on reference countries e.g. the use of Certificate of Pharmaceutical Products (CPPs), etc. Participants also questioned the value of sample analysis during registration review and WHO commented that “such work by agencies rarely adds value,” highlighting that this resource and effort could be re-prioritized elsewhere.

Building Regulatory Capacity for Success

The overarching message from the conference was that training and capacity building are key drivers for change in this area – a number of options already exist and stakeholders like the WHO, the World Bank Group play a key role, as do NEPAD and the Regional Centres of Regulatory Excellence (RCORES). Still, there is a significant amount of capacity development needed. Industry acknowledged it must consider how to further engage in this area, including RCOREs.

There was a special workshop on this topic and it concluded that capacity building for harmonization needs to be across all levels, not just one aspect of registration. There needs to be capacity building in many areas: 1) across the entire life cycle of a product (eg. clinical trials, pharmacovigilance, variations etc.); 2) funding is essential yet funding only the project itself will not help; 3) career progression for regulators is important (eg. courses, training, regional opportunities etc.); and 4) continuous professional development is essential to develop further competencies. In essence, there needs to be an approach towards educational harmonization too.

To conclude, the Conference represents a major milestone as there are now real harmonization efforts underway and agreement that industry is considered an important contributor. The Conference allowed national regulatory authorities to share examples of real progress and intentions. As trust develops between stakeholders it is clear industry is expected to play a growing role in AMRH. To do so it will need to re-focus efforts towards the specific regional activities: prioritize where we can input, establish mechanisms to engage and contribute to harmonization and capacity building activities.

For more information, click here: http://www.ifpma.org/quality/regulatory-conferences/africa-regulatory-conference.html

Together, Taking on Drug Resistant Malaria

The human cost of malaria affects countless families worldwide – including my own. As a young man, my grandfather suffered with the disease whilst serving with British troops in India. He described it as having ‘stolen a year of his life’, but that his long illness paled in comparison to the suffering of the local population.

Today, almost half of the world’s population is at risk from malaria infection, which is the case of 600,000 deaths each year. Whilst successful treatments are available, the spectre of parasite resistance is emerging.

As an early-career scientist, I am fascinated by the biological challenges in tackling resistance – however, I feel that scientific development can only take us so far, and that many of the key challenges we face are in the sharing of data, resources and the logistical burdens of delivering treatment. When faced with the emerging public health threats that arise from globalization, such as the quick way resistance to a disease spreads across international borders, it is clear to many of us new researchers that global coordination and collaboration across sectors is key to moving forward.

On World Malaria Day, I want to highlight the vital work of some global health partnerships are already doing to prevent the spread of antimalarial resistance.

The Emergence of Resistant Malaria Parasites

The most lethal form of malaria is caused by infection with the parasite species Plasmodium Falciparum. The current frontline treatment for this infection is artemisinin combination therapy (ACT). Worryingly, resistance to ACT has emerged in the Mekong region of Southeast Asia and its continued spread is monitored rising cause of concern for scientists and the global health community. Resistance affecting other lethal parasite species such as Plasmodium Vivax has also been documented.

Defined as the ability of the parasite strain to survive or multiply despite the successful administration of a drug, resistance may occur due to a variety of causes. These range from specific quirks of parasite genetics, to the practical realities of incorrect drug use and administration and the proliferation of counterfeit and substandard medicines.

In order to effectively tackle the range of causes of resistance, the World Health Organization (WHO) used its 2011 Global Plan for Artemisinin Resistance to call for a five-pillar strategy to reduce resistance spread. I think there are two main themes that are of particular importance: research and development to allow the release of new, effective antimalarial medicines; and simultaneously monitoring drug use effectively, to ensure proper use of anti-malaria treatments and to increase early detection of drug resistance spread.

Prevention through Partnership

I do not believe that isolated research groups, NGOs, public sector initiatives or companies can tackle the pervasive issue of resistance. Much of the recent groundbreaking progress to defeat malaria and counter resistance has been made through the efforts of several important partnerships working in afflicted countries. I found two of these partnerships particularly striking: the Novartis Malaria Initiative and the Medicines for Malaria Venture.

One of the key problems with drug administration is the provision in countries with inadequate health systems of artemisinin drugs at either an incorrect dosage or without the accompanying combination therapies. The Novartis Malaria Initiative, comprised of partners that span advocacy NGOs, research institutes from Switzerland to Kenya, national governments and multilateral donors, focuses on improving access to treatment, working to provide access to quality-assured ACTs in malaria endemic regions, alongside education and training to allow local communities to deliver the drugs effectively.

This partnership has also historically delivered anti-resistance products, with the first of its kind release of Coartem® in 1999. This is a fixed-dose artemisinin-based therapy, which is provided free-of-charge for public sector use in Africa. The treatment combines an artemisinin derivative with another compound, which in combination acts to reduce the risk of resistance compared with other treatments. It is also available as Coartem ® Dispersible, a sweet-tasting, easily dissolvable form to improve delivery of the bitter-tasting therapy to children. Since 2001, over 800 million treatments have been distributed across Africa, Southeast Asia and the Pacific.

Another partnership driving product development is the Medicines for Malaria Venture (MMV). This is a product development partnership (PDP)that involves both public and private partners in research, developing and facilitating delivery of new malaria treatments. MMV is working with hundreds of dedicated experts to build the largest pipeline of antimalarials in history.

One key aim of this partnership is to provide a drug portfolio broad-enough to outflank emerging resistance. MMV supports in the early discovery phases of drug research which has led to screening of over 5 million compounds for their potential to act against malaria parasites. MMV-supported projects have also taken a clear stance on accessibility and have released data on active molecules into the public domain.

The Novartis Malaria Initiative and MMV[1] show that by acting across several different strategies to combat malarial resistance, cross-sectorial partnerships can break down barriers between organizations. They can provide a forum to safely share data and research materials for the benefit of the entire research community and, ultimately, people the world over in need of innovative treatments.

Ultimately, I believe that the success of releasing of new treatments currently in the R&D pipeline and ensuring their effective delivery will largely depend on such partnerships. Working together, we may prevent the further spread of resistant malaria, and in doing so, prevent the countless deaths and suffering from continuing down the generations.


Abigail Wood is a researcher with Polygeia, a global health think-tank based at University of Cambridge, where she is studying biochemistry. Her research interests focus on infectious diseases and she has project experience in strategic policy work for Lepra, a UK-based charity tackling leprosy, tuberculosis and other major infectious diseases. Abigail also serves as President of BlueSci, the Cambridge University Science Communications Society.


World Health Organization, Global plan for artemisinin resistance containment (GPARC), January 2011

IFPMA Health Partnerships Directory

Traffic in counterfeit medicines must be curbed through international cooperation and regulatory harmonisation


For all the advances it has produced, globalisation also has its downsides. One such example, the counterfeiting of medical products, represents a major threat to our societies, in particular the poorest ones.

The data is rare and difficult to verify with certainty. According to the World Health Organisation (WHO), about 15% of medicines in the world are counterfeit, and in certain regions, this rate may easily exceed 60%! This scourge primarily affects the most disadvantaged regions, because of their impoverished populations and failing government institutions.

While these populations are already severely affected by transmissible and non-transmissible pandemics, producers and traffickers of fake medications are committing a two-fold crime: cheating patients out of their hope of being treated with quality medicine, and endangering the lives of those taking medicine that at best will have no effect, and at worst will be toxic or even fatal.

Mobilisation of all parties is essential in the face of this insidious menace, which evades the vigilance of the various links in the medicinal product chain and serves the financial interests of highly organised criminal groups.

First and foremost, this will require greater accountability on the part of political decision-makers who have shown little engagement, as well as greater risk awareness on the part of populations who know little about this new form of criminality. All links in the health chain must be mobilised to take part in an unrelenting battle. This means mobilisation of the authorities, doctors, healthcare professionals, pharmacists, manufacturers, civil society and all citizens concerned.

In 2006, the Conférence Internationale des Ordres de Pharmaciens Francophones [International Conference of Chambers of Francophone Pharmacists] made a first declaration in Beirut (Lebanon), directed at pharmacists as well as public authorities and patients. These pharmacists highlighted the paradox of investing in the development of medicinal products beneficial to health when the distribution systems are not sufficiently controlled and are increasingly in the hands of organised crime.

The Cotonou Declaration, issued on 12 October 2009 by French President Jacques Chirac in the presence of numerous heads of state and government, notably African (Africa being the continent the most harmed by counterfeit medicines), introduced the political dimension necessary to win this fight. The objective was to rally political decision-makers to fight this menace and urge them to provide more help to the public health community.

This political advocacy is not intended to replace the health community but to involve political decision-makers in combating this traffic, which is doing more and more harm to their populations.

Since then, several “booster shots” have been given, such as the Declaration of Niamey, signed in November 2013 by five African First Ladies (Burkina Faso, Central African Republic, Guinea, Mali, Niger) who commit to continue this political advocacy with the heads of state and government. This month marked the launch of the international awareness campaign Fight The Fakes, which now brings together over 25 partners (representatives of healthcare professionals, product development partnership, foundations, financing institutions, wholesalers, mobile application, coalitions for consumer protection and the generic and R&D pharmaceutical industry). This campaign constitutes a concrete example of shared awareness.

Another critical need is reinforced international cooperation among states and expanded regulations harmonisation. Impunity in the face of the near absence of coercive and penal measures in certain regions, along with the disconcerting profitability of this traffic, makes it more and more attractive for certain criminal groups. A response is urgently needed in order to fill the legal vacuums taken advantage of by criminals.

In 2011, during a conference in Moscow, the Medicrime Convention of the Council of Europe was opened to the signature of all states. This convention protects public health by criminalizing and sanctioning the production, traffic and sale of counterfeit medicines, while ensuring cooperation among states to tackle this scourge.

The Medicrime Convention is the first international legal instrument available to every state as it is open to signature and ratification by all states – members or not of the Council of Europe. If offers an opportunity to establish international cooperation, which has been sorely lacking in the fight against counterfeiting of medicine, and currently counts 23 signatory states, including 3 that are not members of the Council of Europe (Guinea, Israel, Morocco). Only one more signatory state must ratify in order to bring the convention into force, which will certainly renew mobilisation.

Regional initiatives are also essential, because they involve responsible parties, elected officials and individuals closer to the reality in the field. Like the European Union, which armed itself with a directive on these lines in 2011, the African states should also invest in harmonising their regulations.

In Africa, authorities and professionals are expressing increasing interest in being better informed on the regulations and initiatives that may be transposed into their health systems.

In this framework, the DIA (Drug Information Association), the Fondation Chirac and the IFPMA (International Federation of Pharmaceutical Manufacturers & Associations) are working together to organise a workshop on 29 April 2015 in Dakar with the theme “Integrated Approach against Counterfeit Medicines.” This event will bring together regulatory affairs professionals, representatives of health authorities and other professionals involved in combating counterfeit medicinal products.

The discussions will deal with the current regulatory landscape in Africa, the initiatives and measures capable of strengthening the integrity and inviolability of the distribution chains, and will end with exploration of possibilities for inter- and intra-state cooperation.

This reflection and approach should create a more widespread and intense awareness in support of a general mobilisation against the counterfeit medicines targeting the most impoverished. Do you want to be a part of this fight? Join us on 29 April 2015!

Le trafic des faux médicaments doit être endigué par une coopération internationale et une harmonisation règlementaire !


Bien que source de nombreux progrès, la mondialisation présente également ses dérives. La contrefaçon de produits médicaux est l’un de ces phénomènes faisant peser une menace majeure sur nos sociétés, singulièrement les plus pauvres.

Les chiffres sont parcimonieux et difficiles à vérifier avec certitude. Il convient de retenir que selon l’Organisation Mondiale de la Santé (OMS) environ 15% des médicaments dans le monde sont des contrefaçons, et que dans certaines régions ce taux peut facilement dépasser les 60% ! Ce fléau touche avant tout les régions les plus défavorisées car ce sont celles constituées par des populations démunies et aux structures étatiques défaillantes.

Alors que les pandémies transmissibles et non transmissibles affectent déjà gravement ces populations, les producteurs et trafiquants de médicaments falsifiés commettent un double crime : celui de tromper les patients dans leur espoir de pouvoir se soigner grâce à une prise médicamenteuse de qualité et celui de mettre en danger la vie de ceux recourant à un médicament au mieux sans effet, au pire toxique, voire mortel.

Face à cette menace insidieuse trompant la vigilance des différents maillons des chaînes d’approvisionnement de médicaments et servant les intérêts financiers de groupes criminels très bien organisés, la mobilisation de tous est indispensable !

Celle-ci passe avant tout par la responsabilisation des décideurs politiques insuffisamment engagés et par la sensibilisation des populations quant aux risques encourus, rarement conscientes de cette nouvelle forme de criminalité. Tous les maillons de la chaîne de santé doivent se mobiliser afin de contribuer à une lutte sans merci. Il y va de la mobilisation des autorités, des médecins, des professionnels de santé, des pharmaciens, des industriels, de la société civile, des patients et de tout citoyen se sentant concerné.

En 2006, la Conférence Internationale des Ordres de Pharmaciens Francophones a fait une première déclaration à Beyrouth (Liban) s’adressant aussi bien aux pharmaciens qu’aux pouvoirs publics et aux patients. Les pharmaciens y dénonçaient le paradoxe d’investir dans la recherche de médicaments utiles pour la santé alors que les systèmes de distribution ne sont pas assez contrôlés et de plus en plus souvent dans les mains de mafieux.

L’Appel de Cotonou lancé le 12 octobre 2009 par le Président Jacques Chirac, en présence de nombreux Chefs d’Etat et de gouvernement, notamment africains (l’Afrique étant le continent le plus meurtri), a introduit la dimension politique nécessaire afin de mener à bien ce combat. L’objectif était de rassembler les décideurs politiques contre cette menace et les inciter à aider davantage les acteurs de la santé publique.

Ce plaidoyer politique n’a pas pour vocation à remplacer les acteurs de la santé mais d’impliquer les décideurs politiques contre ce trafic portant un préjudice croissant à leurs populations.

Depuis, plusieurs « injections de rappel » ont eu lieu à l’instar de la Déclaration de Niamey en novembre 2013 de cinq Premières Dames africaines (Burkina Faso, Centrafrique Guinée, Mali, Niger) dans laquelle elles s’engageaient à poursuivre ce plaidoyer politique auprès des Chefs d’État et de gouvernement. Au cours de ce mois a été lancée la campagne internationale de sensibilisation Fight The Fakes, rassemblant aujourd’hui plus de 25 partenaires (représentants les professionnels de la santé, les grossistes, des associations, des partenariats de développement de médicaments, des fondations, des institutions de financements, les grossistes , des services d’application mobiles, des coalitions de protection des consommateurs ainsi que l’industrie générique et de recherche et développement du médicaments. Cette campagne ) constitue un exemple concret d’une prise de conscience commune.

L’autre impérieuse nécessité qui s’impose est de renforcer la coopération internationale entre Etats et de développer une harmonisation des règlementations. L’impunité résultant de / découlant de à la quasi absence de mesures coercitives et pénales dans certaines régions, ainsi que la déconcertante rentabilité de ce trafic le rendent de plus en plus attractifs pour certains groupes criminels. Il y a donc une réelle urgence à réagir afin de combler ces vides juridiques profitant aux malfaisants.

En 2011, lors d’une conférence à Moscou, la Convention Médicrime du Conseil de l’Europe était ouverte à la signature de tous les Etats. Cette convention protège la santé publique en criminalisant et sanctionnant la production, le trafic, et la vente de faux médicaments, tout en assurant une coopération entre les Etats pour lutter contre ce fléau.

La Convention Médicrime est le premier instrument juridique international mis à la disposition de tous les Etats car ouverte à la signature et à la ratification d’Etats non membres du Conseil de l’Europe. Elle est aujourd’hui l’opportunité d’instaurer cette coopération internationale dont l’absence fait cruellement défaut dans le combat contre la falsification de médicaments et compte actuellement 23 Etats signataires, dont 3 Etats-Tiers au Conseil de l’Europe (Guinée, Israël, Maroc), et il ne lui manque plus que la ratification d’un seul État signataire pour entrer en vigueur, ce qui relancera indéniablement la mobilisation.

Les initiatives régionales sont également primordiales, car elles impliquent des responsables, des élus et des individus plus proches de la réalité du terrain. Tout comme l’Union européenne qui s’est dotée d’une directive en ce sens en 2011, les Etats africains devraient également s’investir dans une harmonisation de leurs règlementations.

En Afrique, un intérêt croissant des autorités et des professionnels se manifeste pour être mieux informés sur les régulations et initiatives pouvant être transposées dans leurs systèmes de santé.

Dans cette lignée, DIA (Drug Information Association), la fondation Chirac et l’IFPMA (International Federation of Pharmaceutical Manufacturers & Associations) s’associent pour organiser un atelier le 29 avril 2015 à Dakar dont le thème sera «Pour une approche intégrée contre les faux médicaments». Cet événement rassemblera des professionnels des affaires règlementaires, de représentants des autorités de santé et d’autres professionnels impliqués dans la lutte contre la falsification de produits médicaux.

Les discussions porteront sur le paysage règlementaire actuel en Afrique, les initiatives et les mesures permettant de renforcer l’intégrité et l’inviolabilité des chaînes de distributions, et termineront sur les possibilités de coopération inter et intra étatiques.

Cette réflexion et cette démarche doivent déboucher sur une prise de conscience plus générale et plus intense au service d’une mobilisation générale contre les faux médicaments qui s’en prennent aux plus pauvres. Vous voulez prendre part à ce combat ? Rejoignez-nous le 29 avril 2015 !.

What is needed to accelerate access to quality medicines & vaccines in Africa?


In a globalized world, the regulatory landscape is changing every day to address, in tandem, old and new challenges. With regulatory systems increasingly under pressure globally, what will be the most promising weapons in the fight to make regulatory systems in Africa work more efficiently? And how to pay the bill for the extensive regulatory capacity building needed for the region? What is required so that current and future generations in Africa can access quality innovative medicines and vaccines in a timely manner to sustain healthier societies? These questions are precisely what the IFPMA, the Drug Information Association (DIA), in sponsorship with the Bill & Melinda Gates Foundation and the World Bank have on the agenda for the 4th Africa Regulatory Conference taking place on 27 – 28 April in Dakar, Senegal.

New technology and progress, breakthrough medicines and vaccines are entering the healthcare system every day at a different stage of maturity. In response to this, national regulators on every continent, including Africa, are adjusting their regulatory paradigm with new mechanisms for the timely and effective assessment of benefit-risk of such products to public health. What is very challenging from an industry perspective is simply put this way: regulatory requirements can be very different from one country to another, which may jeopardize timely access for patients to the new treatments we deliver.

A case in point is the redundant testing and delayed access to medicinal products in general, with a special emphasis, as illustrated here for vaccines. As one may know, the quality of vaccines is confirmed via the extensive testing that is performed during the manufacturing process. The product is also tested for lot release by the official control laboratory in the country of manufacture. Additional testing in the importing country may be required which will then delay further the availability of the vaccine in-country. Typically these tests are performed consecutively. The remaining shelf life of a vaccine is directly affected by the time taken for testing. Prolonged testing means less time for the distribution and administration of the vaccines for patients.


What is needed for securing timely access to life-saving treatments is building the foundation of a sustainable regulatory environment that may include the following: moving towards convergence of standards, collaborating with well-resourced regulatory authorities with relevant expertise to rely on assessments already made, leveraging appropriate use of World Health Organization Prequalification to accelerate local review and authorization, and enhancing regional cooperation to leverage complementary expertise. All this is with the aim of making best use of limited resources while still supporting other essential regulatory functions to ensure adequate quality, safety and efficacy oversight.

So if we look closer to the upcoming ARC, the 2-day event will kick off with a special training course on pharmacovigilance on 26 April. The main conference will then run from 27-28 April, followed by a one-day workshop on counterfeit medicines on 29 April. The ARC will attract a broad cross section of key regulatory stakeholders committed to improving access to safe and quality medicines in Africa, including industry, policy-makers, researchers, governmental and non-governmental organizations.
The conference will provide participants with an opportunity to get an update from regulatory agencies on the African Medicines Regulatory Harmonization (AMRH) program which is leading the regulatory convergence efforts within the continent. Good review practices and regulatory convergence; improvement in Good Manufacturing Practices compliance; opportunity for pragmatic dialogue between medicines regulatory authorities and industry; how to tackle the challenges encountered in clinical trials and related capacity building in Africa, are all topics on which the ARC aims at providing comprehensive elements of information, and much more.

If you are interested to join us, get registered here.
About the IFPMA Regulatory Conferences here

About African Regulatory Network
The ARN is an ad-hoc network of the Regulatory Policy and Technical Standards Committee (RPTS) of IFPMA. The Network works in partnership with regulatory authorities and the pharmaceutical industry in Africa to encourage greater harmonization of regulatory requirements with the aim to help enable faster and expanded access to good quality innovative medicines for patients.

Patient Centricity is Essential to Modern Day Drug Development


Providing new therapies to patients living with unmet needs faster and more efficiently is an industry-wide challenge. A major key to improving the speed cost and quality of drug development is harnessing the insights of the customer – patients themselves.

The traditional model of clinical development has looked at drug development through the lens of a scientist, often primarily focused on a mechanism of action and subsequent clinical endpoints. This model has resulted in larger, longer and more expensive trials. What we are just beginning to understand is how to identify, target and develop in a manner that can most profoundly impact patients and their day to day lives, which may or may not be related to the clinical endpoint.  To better design our trials, research questions and the outcomes studied must be prioritized by patients.  To more efficiently recruit, retain and ultimately deliver clinical trials we must understand the patient perspective. Finally, to better characterize the benefits and risks of our products for health authorities, we must understand how patients’ perceive the burden of the disease and the standard of care.  Patients play the most important role in clinical trials, and therefore are in a position to help make a difference.

Successful clinical development will be found at the intersection of great science and patient centricity.

Merck Serono has chosen to engage with patients and patient advocacy groups during clinical development through a focus group approach. Patients in the focus group represent a cross section of patients living with the disease. Patients afflicted with life-threatening diseases are often motivated by the knowledge that they are making a difference for people with the same condition. Taking this focus group approach allows us to understand the needs and wants of the patients when it comes to clinical trial design, inclusion/exclusion criteria as well as day to day study impact on patients (e.g. site selection, scheduling, etc). Such an approach allows us to learn the individual needs and motivations by region in a given therapeutic area and then incorporate in our clinical trial designs.

This type of patient centricity is only the beginning. New technologies will eventually make it possible to isolate the patients for whom the drug in development will work.  This will be a paradigm shift in the way clinical development is conducted. Patient centricity will give way to “individual centric” clinical trials with the development of and proliferation of predictive biomarkers and tumor profiling through the collection and testing of cancer cells to determine their molecular and genetic signatures at tumor banks. Predictive biomarker data along with information collected on the tumor can then be applied to determine the best possible treatment that works with the specific type of cancer cell leading to stratified medicine which can, in fact, replace the clinical development model that is in place today. New technologies have the potential to drastically reduce the number of patients required to participate in clinical trials, making it possible to have trials tailored to individuals.  Ultimately, patient centricity will lead to a more accurate diagnosis, and better treatment selection, which has a much greater potential for a successful outcome for patients.

I am an academic researcher by background, and helping people with unmet needs has always been my main driver. Working with patients directly to improve how clinical trials are run is another way to be closer to the patient and understand their needs to help design the best possible therapy. As an industry, we cannot assume we know what patients need – we need to involve their voice in our efforts.  Clinical development that is built on solid science and supported by principles of patient centricity, which includes simplicity of execution, is likely to lead to getting new therapies to patients in need sooner. That is what the scientists and researchers are looking for: science that can be translated into potential new medicines to serve the patients in need.

To learn more about our R&D strategy, please visit http://www.merckserono.com.

European perspective for effective cancer drug development and new forms of partnerships for managing uncertainty.

The aims of the European Organisation for Research and Treatment of Cancer (EORTC) are to develop, conduct, coordinate, and stimulate translational and clinical research in Europe to improve the management of cancer and related problems by increasing survival but also patient quality of life. Extensive and comprehensive research in this wide field is often beyond the means of individual European hospitals and can be best accomplished through the multidisciplinary multinational efforts of basic scientists and clinicians.

The ultimate goal of the EORTC is to improve the standard of cancer treatment through the testing of more effective therapeutic strategies based on drugs, surgery and/or radiotherapy that are already in use. The EORTC also contributes to the development of new drugs and other innovative approaches in partnership with the pharmaceutical industry. This is accomplished mainly by conducting large, multicenter, prospective, randomized, phase III clinical trials. In this way, the EORTC facilitates the passage of experimental discoveries into state of the art treatments.

Through translational and clinical research, the EORTC offers an integrated approach to drug development, drug evaluation programs and medical practices.

EORTC Headquarters, a unique pan European clinical research infrastructure, is based in Brussels, Belgium, from where its various activities are coordinated and run.

The EORTC is both multinational and multidisciplinary, and the EORTC Network comprises over 300 hospitals and cancer centers in over 30 countries which include some 2,500 collaborators from all disciplines involved in cancer treatment and research.

The 170 members of the EORTC Headquarters staff handle some 6,000 new patients enrolled each year in cancer clinical trials, approximately 30 protocols that are permanently open to patient entry, over 50,000 patients who are in follow-up, and a database of more than 180,000 patients.

Health care systems and the clinical research landscape evolve continuously owing to increased risk aversion, scrutiny by funding bodies, and costs of clinical trials. In this context, however, current drug development procedures are far from optimal, as exemplified by the late-stage failure of several drugs. The identification of new drugs urgently requires approaches based on a solid understanding of cancer biology, and that will support the design of robust confirmatory trials. The complexity and the costs of drug development are now beyond the knowledge and operational capacity of single organisations, therefore, a drastic deviation from the traditional path of drug discovery and new forms of multidisciplinary partnerships are needed to succeed in this sector. The European Organisation for Research and Treatment of Cancer (EORTC) proposes the use of collaborative molecular screening platforms (CMSPs) as a new approach to tackle this issue. These CMSPs have the advantage of optimizing the expertise of several partners and combining efforts alongside with cost-sharing models for efficient patient selection.

EORTC has developed a CMSP called Screening Patients for Efficient Clinical Trials Access (SPECTA). The EORTC SPECTA is a European screening programme that aims to ensure efficient clinical trial access for patients with a range of tumour types. SPECTA is an integrated and cost-sharing model developed to address the described concerns relating to the current drug development process. This programme coordinates several SPECTA platforms on a pan-European level with the aim of identifying, at an early stage, specific druggable aberrations and, therefore, to offer specific targeted treatment to patients within clinical trials.

The specta collaborative platform

The SPECTA programme is a fully integrated business model that, as well as comprising disease‑oriented screening platforms also embeds specific initiatives to address both the regulatory challenges and the molecular pathological complexity of cancer. A collection of fora has been created for all stakeholders to meet and discuss for optimal mutualization of knowledge.

  • SPECTApath is intended to address quality-assurance programmes across the disease platforms for optimal biomarker qualification and validation, and is crucial for the involvement of pathologists and molecular biologists.
  • SPECTAreg is a research project that addresses the evolution of the forms of partnerships, looks into optimizing regulatory procedures for these new forms of clinical research and explores the routes to new forms of licensing.

specta a collaboration platform

SPECTA is now currently recruiting patients and EORTC is seeking partnership with the commercial sector to explore new models of drug development.

Four priorities for regulatory convergence in Asia

Caroline MendyCaroline Mendy

What can be done to speed up access to new medicines for patients and improve quality, safety, and efficacy of medicines in Asia

The 2015 Asia Regulatory Conference (ARC) on February 4-5 in Chinese Taipei brought together over 250 stakeholders from regulatory, pharmaceutical, academic, civil society, international and non-governmental organisations, including policy experts from Asia. They met on the understanding that no single state, regulatory authority or company within the pharmaceutical industry can meet current regulatory challenges alone. Cooperation, coordination, and convergence on priority issues are important to ensure a pragmatic, effective, and rapid response to facilitate access to new medicines. The conference’s goal was to discuss how to advance best practices for regulatory review and submission in Asia, ultimately expediting access to medicines for patients across the region.

This blog summarizes keynote remarks made by former chair of ARC 2013, A/Professor John C.W. Lim, Deputy Director of Medical Services (Industry & Research Matters) Ministry of Health, Singapore, and current Executive Director of the Centre of Regulatory Excellence, Duke-NUS Singapore, on ‘regulatory convergence and cooperation to improve public health’. Professor Lim is uniquely placed to bear witness to the path travelled since 2013 and today’s challenges.


1. Science-based regulatory decisions enhance collaboration and priorities

To keep up with the rapid advancements in science and technology, today it is all the more essential to ground our regulatory pathways on sound science. Consequently, it is only sensible that collaboration increases amongst regulatory authorities, industry, and academia – working together to address the challenges presented by this fast changing environment. Regulatory frameworks must be based on sensible decisions and sound understanding, and require making judicious judgments by prioritizing which scientific advances are the most relevant and applicable to specific settings. Professor Lim applauded the move since 2013 towards practical convergence over ideal harmonization. But for the future, given the limited resources facing countries in the region, he emphasized that “we also need to tackle which are the priority issues that we need to converge on”.

2. Addressing capacity gaps is a challenge across the board

A review conducted by Centre of Regulatory Excellence at Duke-NUS at the end of 2014, showed a strong desire by key regulatory stakeholders to see the capacity and capabilities of national regulators built up further and move towards greater convergence in the Association of Southeast Asian Nations (ASEAN) and South East Asia. Interestingly, “we found out that industry’s own regulatory affairs professionals in the region also needed capacity development, including scientific capabilities needed to address the rapid growth of the biomedical sector in Asia” said Professor Lim. Finally, because of the increasing activities involving companies, organizations, clinical trials, and biomedical research in Asia, there is a need for greater regulatory leadership. Without effectively addressing these gaps, the full potential of the many biomedical initiatives in Asia will not be achieved in the years ahead.

3. Boldness to challenge old regulatory paradigms

Professor Lim called for more boldness in challenging old paradigms and promoting innovation in both drug development and regulation. This includes developing sound regulatory approaches to manage benefit-risk, health technology assessment, and dynamically monitor as well as prioritize scientific advances and their impact. He added that it was “highly commendable how things have moved on with two good complementary approaches: good review practices being developed by the World Health Organization (WHO) and regulators, and good submission guidelines developed by industry is in itself an example of how convergence and stakeholder cooperation have advanced and continue to advance”. This is clearly mirrored in the region as the Asia-Pacific Economic Cooperation (APEC) Regulatory Harmonization Steering Committee works with WHO on the review aspect, whereas Asia Partnership Conference of Pharmaceutical Associations (APAC) serves as the representative of industry on submission guidelines. Instead of working in isolation and at cross purposes, they share expertise and demonstrate how regulatory stakeholders can build on their expertise and strengths towards convergence, coordination, and communication to address capacity issues in a rational and complementary manner.

4. A healthy dose of pragmatism to achieve tangible outcomes

The last priority exposed in Professor Lim’s remarks is “the need to support our aspirations with a healthy dose of pragmatism and reality”. He noted that gatherings like the ARC enable regulatory stakeholders to gain a better understanding of the many issues different players have to deal with. These include ongoing public health challenges, ethical issues, commercial realities as well as public and political pressures. However, he drew the audience attention to the fact that understanding and shared commitments alone are not very useful unless they are translated into finding practical approaches and meet tangible outcomes that address significant regulatory issues.
Parting thoughts – making a genuine difference to the quality of regulation in Asia
Today, all the various regulatory stakeholders, including national agencies, industry associations, international organisations and regional groups, show a commitment to regulatory excellence, which Professor Lim believes is key to effectively meet the range of regulatory challenges unfolding around us. This involves professional development through:

  • targeted education initiatives;
  • greater transparency;
  • collaboration for consistent monitoring and follow-up;
  • advancing best practices for regulatory reviews and submissions.

Regulatory excellence will help ensure regulatory authorities and industry has the necessary legitimacy, credibility, and moral standing to carry out their responsibilities. The more so as all these stakeholders are under greater scrutiny from the public and governments, and need to meet increasingly high expectations from civil society and patients.

Professor Lim concluded his remarks by stressing the importance of keeping the momentum going and the dialogue open with all key stakeholders. As he put it, “all stakeholders in the region have a shared goal: to make a genuine difference to the quality of regulation in Asia”.

ARC2015: Regulatory convergence and best practices are next week’s hot topics in Asia

The clock is ticking! In just few days, we will be joining friends and colleagues in Taipei for the Asia Regulatory Conference (4th and 5th of February). The programme was crafted by a Programme Committee, which consists of regulatory professionals from regulatory authorities, industry and trade associations including the Taiwanese Food and Drug Association (TFDA), Singapore’s Health Science’s Authority, Switzerland’s Swissmedic, International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), Japan Pharmaceutical Manufacturers and Associations (JPMA), the International Research-Based Pharmaceutical Manufacturers Association (IRPMA) and the R&D-based Pharmaceutical Association Committee (RDPAC). The Programme Committee has held regular ‘check-ins’ with an Advisory Committee, consisting of experts from regulatory authorities both within and outside the region. And, rather excitingly, the conference has been endorsed by Asia Pacific Economic Cooperation (APEC).
So what should be expected from the conference?

On day one, there is a focus on good review practices: thought leaders such as John Lim from Duke-NUS Graduate Medical School (former of the HSA, Singapore) and Lembit Rago of the World Health Organization (WHO) will provide keynote speeches followed by a panel discussion between regulators inside and outside the region. These will provide answers to critical questions: what more is needed to make good regulatory practices a reality for the region? Does it necessarily need regulatory amendments or just changes in working practice? WHO has recently developed guidance on ‘good regulatory practices’ so it seemed timely to hear more about this from the regulatory authorities, such as Taiwan’s TFDA, who were closely involved in developing it.

The second session of day one will draw on the key themes and outputs of the previous ARC (which took place in Singapore in 2012): “Co-operation, Convergence, Competency, Capacity, and Communication.” Cordula Landgraf from Swissmedic will explain how this soundbite has been brought to life through work she has done in collaboration with regulators in Australia, Canada and Singapore.

As it would be hard to hold a regulatory conference discussing the current topic of ‘innovative and alternative’ regulatory pathways, we will hear, in the afternoon of the first day, about progress in this area in United States of America (USA), European Union (EU), and Japan. This session will be hosted by John Skerritt from Australia’s Therapeutic Goods Administration (TGA).

On day two, we will enter a day of discussion focusing on good submission practices. Asia’s trade associations have come together to develop good submission guidance, which they plan to showcase in the opening panel discussion. This will be followed by a reflection focusing on industry’s experience of submitting dossiers in the Association of Southeast Asian Nations (ASEAN) – this being of particular interest in light of the ASEAN harmonization initiative which has, in theory, led to harmonization of dossier content and format.

In the afternoon, we will again look to step beyond the main themes of the conference and hear about initiatives that APEC is driving in relation to sustainable regulatory convergence. We will conclude the conference with a panel discussion reviewing the overall outcomes from the conference and teeing up areas for further action and discussion.

We do believe that the programme we have put together is relevant to both the novice and the expert of the Good Regulatory Practices concept.

Have your say and join us and all stakeholders to help contribute to the definition of pragmatic regulatory approaches for improving access to innovative therapies in Asia!

A smart dose of flu shots is direly needed worldwide

The case has been made time and again: the annual flu shot saves lives. Around 3 to 5 million people suffer from severe flu each year and it is estimated 5% to 10% of people die as a result. Children, pregnant women, healthcare workers and the increasing number of people who live with chronic diseases such as heart disease, diabetes, and asthma are most vulnerable and therefore at risk of suffering from flu.

Impfung bei einem ArztSome countries are making great strides to ensure that each flu season more people get vaccinated. However, many countries are still failing to meet the most basic levels of services: vaccine coverage rates of 75% of the elderly, as recommended by the World Health Organization. During the 2010-2011 flu season, coverage rates among the elderly were reported to be as low as 10% or less in some European countries (Poland, Estonia, and Latvia).[1]

In a period when there is so much talk of Universal Health Coverage and resilient health systems, why is there little focus on reaching the 75% influenza coverage rate agreed a decade ago by all 194 countries at the World Health Assembly?

Systematic global data on influenza vaccine coverage rates do not exist. Therefore, since 2008 we periodically analyze seasonal influenza vaccine distribution. We know it’s not perfect, but it gives a very good year on year gauge of the extent to which flu vaccines are used in 157 countries. The latest study “Seasonal influenza vaccine dose distribution in 157 countries (2004–2011)” shows a huge variation and disparity between countries and regions. It does not automatically boil down to differences between richer and poorer countries, as there are also huge disparities within the same WHO regions.

While different countries have different health priorities and associated policies, including those for influenza prevention, the health benefits of influenza vaccines should be not ignored during policy planning. Policy measures include increasing knowledge, addressing attitudes and practices among doctors, nurses, and other healthcare providers, and designing communications programs targeted to those who are the most vulnerable to seasonal flu.

An interesting finding from the study tells us that current distribution rates of influenza vaccines are in striking contrast to the global efforts for pandemic preparedness. Only about half of the global vaccine capacity for a northern hemisphere seasonal influenza vaccine was being utilized in 2011, and even less for a southern hemisphere vaccine. This could potentially compromise pandemic flu preparedness, as the logistic and manufacturing capacity of the countries remains untested.[2]

Global efforts to prepare the world to deal with a new pandemic (including manufacturing, stockpiling, and system preparedness) require huge investments and considerable resources allocated to multi-year projects. These efforts and investments are indeed necessary, and as a result health advocates analyze whether all those involved in these efforts are meeting their obligations. But how can we square this focus on emerging influenza pandemic with the lack of attention given to underutilized, cheap and existing seasonal influenza vaccines that protect vulnerable patients against an annually recurring high burden of disease?  In terms of the impact to people’s lives and reducing the economic burden of flu, season flu vaccination is essential and worthy of more attention than it receives. What’s more, if we don’t have resilient seasonal flu vaccination programs in place; we won’t have the necessary systems in place to manufacture and roll out vaccination programs in the eventuality of a flu pandemic.

Given the annual burden of seasonal flu, the ample scientific evidence on the safety and benefits of seasonal influenza immunization and the recommendations by the World Health Organization, it would make sense for the public health community to take stock and re-evaluate how best to implement seasonal flu vaccination programs to reduce suffering and costs at a huge scale on an annual basis.

[1] Eurosurveillance 2014 – http://www.eurosurveillance.org/ViewArticle.aspx?ArticleId=20780)

[2] Palache et al. 2014; Partridge et al. 2013

Forging new approaches to biopharmaceutical innovation in neuroscience

SM logo

By Anke Post

As a psychiatrist, I have long worked to address mental diseases such as schizophrenia and depression. Experiencing first-hand the suffering and heavy burden these ailments place on people motivated me early on in my career to better understand the underlying mechanisms of these diseases and to help find new treatment possibilities. This is why I have devoted the majority of my professional life to neuroscience preclinical research, patient care, and clinical drug development.

Major depressive disorder (MDD) is the most common mood disorder, with life-changing impact on people such as decreased quality of life, functional impairment, and increased mortality rate.  In fact, suicide related to depression is a major cause of death in industrialized countries.

While we have had antidepressants since the 1950s, in many cases people with depression still are not correctly diagnosed and treated. This can be due to stigma as well as perceived efficacy.

Biopharmaceutical research continues to improve care by developing novel antidepressants but periodically their benefits in daily practice are questioned. Skeptics sometimes argue that new medicines provide only limited additional efficacy over current therapies. These challenges can complicate biopharmaceutical research in psychiatric disorders but also provide me and others encouragement to take R&D to new levels.

CNS neurons

We’ve long known the pathophysiology of these conditions involves many biological aspects such as mono-aminergic neurotransmitter changes, stress circuits dysregulation, and many other related disturbances. New approaches and new targets are needed. Innovation is not only associated with testing new targets but also with developing new ways to test. We need to identify clinical signals earlier, with more certainty, and in the right populations.

That is what we, at Lilly, are doing – investigating new targets related to those fundamental changes associated with depression. We have discovered molecules which target new mechanisms of action and might lead to treatment advancements for patients with mood disorders and other psychiatric diseases.

Our development work is still early, but current clinical data for one target are exciting enough that we hope to translate some of our preclinical findings and outcomes from animal models into practical clinical knowledge. To understand the potential benefits of this compound, we applied established technologies such as PET and fMRI. These helped us understand our target receptor and functional implication. Moreover, we used behavioral assessments and neurophysiological tools (clinical biomarkers) –new research tools for us – to identify treatment responses earlier.

The basis for the latter approach is that patients suffering from MDD often have cognitive negative biases. That is, they are more likely to remember negative information and pay attention to negative stimuli than to non-depressed subjects. To identify these markers early, we use behavioral paradigms based on psychological testing, and neuroimaging techniques such as fMRI. Using these experimental tools allowed for more effective signal detection and gave new insights in the mechanism of our compound.

Besides such clinical tools, genetics may help us in the next round of discovery and development to identify new targets in psychiatry and overcome the stagnation in the field.

Do you want to learn more about Mental and Neurological Disorders? Check out the DoYouMind? Campaign

For more information about Lilly’s research, visit this website