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.

References

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

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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 !

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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?

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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.

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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

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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

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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.

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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

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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 

The Vaccine Revolution: How Innovation Will Change the World…Again

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by Emilio A. Emini

Over 200 years ago, the first vaccine was created to protect against smallpox, an infectious disease that had afflicted humanity for centuries. That discovery opened the door to a golden period of vaccine development that has led to extraordinary improvements in worldwide public health. Today, vaccine development continues to thrive as scientists advance technologies and approaches to reach more people in new ways. Over the years, our understanding of the immune system has grown considerably. We are now learning how to not only prevent infectious diseases, but also how to train the immune system to assist in fighting non-infectious diseases. The possibilities are boundless as vaccines can be potentially used to treat allergic asthma, to help smokers kick their habit, and to fight off cancerous cells. Here’s a helpful video on the evolution of vaccine research

As vaccine technology continues to advance, we are working towards the development of novel vaccines in areas of continued medical need. Patients being hospitalized represent a particularly vulnerable population. People often arrive at the hospital feeling preoccupied with the medical need that brought them there in the first place, hoping their surgeries or treatments go well and anxious to get better. What they don’t expect is to leave the hospital with an additional ailment. Each year, hundreds of millions of patients around the world are affected by hospital-acquired infections[i]. These infections, caused by bacteria such as Staphylococcus aureus and Clostridium difficile, are highly debilitating and often life threatening. To make matters worse, hospital-acquired infections are becoming increasingly resistant to antibiotics. Therefore, in addition to inventing new ways to treat these deadly infections after they occur, we must also look to developing vaccines to prevent the infections in the first place. As an example, a patient planning an elective surgery would be given a S. aureus vaccine within a defined period prior to surgery to allow protective immune responses to develop. These immune responses could then prevent the patient from contracting the infection during surgery by killing any bacteria that may enter the body during the surgical procedure. If effective, such a vaccine has the potential to greatly reduce the burden of the infection in hospitals, and would potentially save numerous lives of patients who experience infection with treatment-resistant bacteria. When I began my career 30 years ago as a virologist, it was just becoming clear that AIDS was caused by the HIV virus. I had the opportunity to work on developing one of the first highly active anti-HIV therapies that protected infected individuals against disease progression.  The experience of seeing the impact of these therapies on those afflicted by the infection was gratifying.  In a similar way, over the years, I have been fortunate to experience again and again the immediate and profound impact that newly developed vaccines have had on infectious disease burdens, both on the individual and population levels. As the potential for vaccines continues to expand with scientific and technological advances, I hope to continue these experiences well into the future.

To read more about Pfizer’s vaccine R&D: http://www.pfizer.com/research/therapeutic_areas/vaccines [i]

http://www.who.int/gpsc/country_work/gpsc_ccisc_fact_sheet_en.pdf

Constantly Innovating

Andrew JennerAndrew Jenner

Pushing the boundaries of innovation is at the core of the pharmaceutical industry. However, it’s also the “business model” for many diseases. Microbes, including bacteria and viruses, are constantly evolving to cloak themselves from being affected by antimicrobial medicines.  Similarly, aggressive cancers are increasingly becoming unresponsive to first line treatments.  In order to stay ahead of the curve, R&D pharmaceutical companies are rethinking the way they innovate.

Medicines discovery is no longer the step-by-step process that it may have been in the past. Historically, pharmaceutical innovation followed an in-house high risk model.  R&D pipelines were reliant on their own company expertise and compound libraries.  This was largely due to how innovation was conducted: a single company would see through a medicine’s journey from experimental compound to approved medicine all under the same roof.  That process was linear, and a hiccup anywhere along would create inevitable downstream delays.

Today’s innovation model is becoming increasingly networked where partnerships are crucial. Through partnerships, companies can segment innovation into discrete phases.  For instance, early stage molecule discover may be done in collaboration with academia.  Testing the safety and efficacy of a promising compound may be coordinated with firms specializing in conducting clinical trials. The goal of today’s networked innovation model is simple, create an innovation ecosystem to provide patients with more lifesaving medicines, faster.

An R&D ecosystem accelerates innovation because research can be coordinated to include numerous experts and stakeholders. There is a vast amount of research and knowledge inherent in any therapeutic area, yet communication between discrete research groups is often minimal and may result in redundant work.  Public institutes, academic research groups, and even competing firms can benefit from one another by coordinating research efforts.  Through such various partnerships, R&D costs are reduced because they are borne by multiple partners, knowledge and know-how is transferred, and medicines innovation is accelerated.  This leads to increasing healthcare providers’ arsenal of innovative medicines to treat more patients in the most effective manner.

In an interview with the IFPMA President, John Lechleiter (see link) explains what a sustainable ecosystem looks like for innovation to happen in the field of medicines and vaccines. A must watch!

Smart testing in personalised healthcare – ctDNA is set to change the companion diagnostic landscape

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AST111_blood_Vessel_cancer_cell_and_DNA

 

In the pursuit of truly personalised healthcare, AstraZeneca has entered into partnerships with Roche and QIAGEN for the clinical development of novel diagnostic tests based on circulating tumour DNA (ctDNA). These tests will move us to the next generation of companion diagnostics by allowing detection of recognised tumour mutations based on accurate analysis of minute quantities of tumour DNA found circulating in cancer patients’ blood.

AstraZeneca is a pioneer in the cutting edge technology of ctDNA, which uses “smart tests” that can pick out specific signals from a background of normal DNA noise. I have been involved with this exciting development for many years, working with technical experts who have pushed the boundaries of science to take ctDNA testing from a highly exploratory science to a robust technology that can be used in clinical practice for our companion diagnostic projects.

Our first research programme in ctDNA started with a clinical fellowship in 2006. In particular, our experience with EGFR testing in non-small cell lung cancer provided the clinical setting in which to correlate ctDNA to tumour samples. We recognised the potential that ctDNA technology has to improve diagnosis and treatment in this type of cancer, where many patients have limited or no tumour samples for testing.

From my perspective this technology is most exciting because it makes it possible for innovative drugs to become available to patients where no suitable tumour sample is available. Blood can be taken at a medical centre quickly and easily, since it doesn’t require hospitalisation or the use of an expensive diagnostic imaging suite.

Regulators have also been quick to recognise the promise of these novel techniques for patients, and have worked actively with the diagnostics and pharma industries. Of course it is the priority of the regulators to protect patients, but I’ve seen that the authorities were keen to review data and discuss what evidence would be needed to approve companion diagnostics based on ctDNA.

As with most scientific developments, we often forget how many challenges were involved. I am very proud of the tenacity of our scientists; the years of work that went into selecting the best techniques and preparation methods for ctDNA.  To start with, we were even unsure if oncologists would trust test data from blood, when they were so used to dealing with tissue samples. All these obstacles have been overcome.

In the future, I would like to see these tests developed so that they could monitor the progression of cancer and even detect if cancers return. Currently, companion diagnostic testing is done using an individual sample based on the assumption that tumours are static. In reality, however, cancers evolve over time in response to treatment. Some experts have speculated that a more sensitive ctDNA technology could be developed to monitor cancer as it changes its molecular nature, allowing us to prescribe the right medicines throughout the course of therapy. This would represent a real clinical advance and confirm ctDNA as a cornerstone of personalised healthcare.

These partnerships on ctDNA are a significant step in Personalised Healthcare scientific innovation. They move us one step closer to achieving our bold ambition of transforming patients’ lives through personalised healthcare by ensuring that innovative treatments are matched to those patients who will benefit most.

Ruth March is Vice-President and Head of Personalised Healthcare & Biomarkers at AstraZeneca

To read more about AstraZeneca science: http://www.labtalk.astrazeneca.com/

Biotherapeutic Medicines: Putting Patients First

eduardo-pisani

As experts in the field of biotherapeutic medicine meet in Rio de Janeiro on 24th-25th August in the run-up to the 16th International Conference of Drug Regulatory Authorities (ICDRA), they should capitalize on the opportunity to discuss the future of biotherapeutic therapies, as well as address the risk that non-comparable biotherapeutic products pose for patients and health systems globally

Undoubtedly, one innovation that has radically transformed medicines is the development of newer, more sophisticated type of drugs produced by living organisms called biotherapeutics. More than 350 biotherapeutic medicines have benefited to date some 400 million patients worldwide.

Biotherapeutics lead to more targeted therapies, better diagnostics and improved outcomes following a patient’s course of treatment for many of the world’s most prevalent diseases, as well as for less common conditions. Biotherapeutics include innovative products for the treatment of chronic diseases such as cancer, diabetes and rheumatoid arthritis, as well as for acute conditions such as myocardial infarction and stroke – and have two common denominators: biological origin and structural complexity. These products bring genuine differences in saving lives, reducing hospitalization and ultimately, contributing to healthier societies.

 

The challenges of manufacturing biotherapeutic medicines

Biotherapeutics are very complex molecules made in living organisms by genetically engineering DNA. As such, every step we take in the production of these medicines requires highly controlled testing to ensure consistent quality, safety, and efficacy. On average they undergo 5 times more testing compared to chemically-synthesized or conventional medicines. They are usually proteins that can be up to 1,000 times bigger than conventional drugs and are normally given by injection or infusion.

How Biotherapeutics are made

Step 1: insert the DNA into host cells. Once the DNA is inside the cells, it uses the cells’ machinery to transcribe and translate the DNA message into the biologic.

Step 2: The most effective cell line is selected for expansion and is grown in bioreactors.

Step 3: The biologic is purified and tested to ensure its function and quality criteria are met.

Biotherapeutics, Biosimilars and Non Comparable Biotherapeutics

Once the data exclusivity and patent expires on an innovative biotherapeutic medicine, follow-on products can now appear on the market: these types of medicines are known as biosimilars and non-comparable biotherapeutic products. Let me try to give you a quick overview.

Biosimilar medicines, on the one hand, are developed and assessed through comprehensive clinical testing to demonstrate their therapeutic equivalence to the reference product. Basically biosimilars are highly similar to a biotherapeutic that has already been authorized by a national drug authority after the review of a full regulatory dossier. They are subject to tailored regulatory data package with side-by-side analytical and clinical testing and may have minor variations compared to original biotherapeutic reference product, but with no clinically meaningful differences identified.

On the other hand, non-comparable biotherapeutic products are medicinal products that are developed with limited or no side-by-side comparability exercises and have not been approved via a regulatory procedure in alignment with World Health Organization (WHO) principles. In other words, these products may or may not be similar to the original biologic medicine that they claim to be.  When such medicines are used, patients could potentially be put at risk due to the unknown safety and efficacy of the product. The potential threat to public health is worrisome as continued use of these medicines could reverse years of hard-won progress in the research and development of safe and effective biotherapeutic medicines.

Call to action for biotherapeutic medicines

Government officials and drug regulatory authorities meeting at the ICDRA conference in Rio de Janeiro this week should capitalize on the opportunity to discuss the future of biotherapeutic therapies, considering that manufacturing a safe and effective biotherapeutic medicine requires tremendous effort and vigilance. Our industry is rigorously committed to quality, safety and efficacy as this is the only way to ensure that each dose will be to the patients’ benefit. Governments should adopt appropriate regulations and stringent regulatory processes in harmony with WHO principles to ensure quality, safety, and efficacy are achieved at all levels. This would help in addressing the risk that non-comparable biotherapeutic products pose for patients and health systems globally.  What’s crucial in medicine is to ensure they are safe, improve patients’ health and do not pose any unnecessary risk.

For more, click on everything you need to know about ICDRA

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An Infographic on Biotherapeutics – View full size

If hit by a chronic disease, fighting the flu with a shot is your best shot!

Otmar-Kloiber-(headshot)150-x150

Most people think about the seasonal influenza (more commonly known as flu) as a bad cold that one can recover from within a week or two. Flu is not a simple cold. It’s a highly contagious disease caused by a virus and does not spare anyone. WHO estimates that there are as many as 3 to 5 million cases of seasonal influenza each year, resulting in 250,000 – 500,000 deaths worldwide.

The skeptics may say these numbers don’t seem too high when compared to the disease burden and deaths caused by other communicable and non-communicable diseases. There’s a catch though: Flu aggravates the underlying medical conditions, leading to increased hospitalization and rising healthcare costs. Complications arising from influenza are particularly frequent and severe in adults with chronic conditions, such as chronic obstructive pulmonary disease, cardiac disease, cerebrovascular conditions and diabetes. If two comorbid conditions are present (old age and high-risk), influenza-related deaths are over 100 times greater than in healthy adults.

Other high risk groups include the elderly, the immunocompromised (e.g. HIV/AIDS), pregnant women and young children (under five years of age).Healthcare workers are also considered a vulnerable group at high risk of both acquiring and transmitting the virus.NCDs & Influenza Infographic-Flickr

The good news is that the vaccine to prevent influenza is available, safe and inexpensive. It is also effective: According to WHO, vaccination against influenza can reduce influenza-related morbidity by 60% and mortality by up to 80%. And there are even more public health and economic benefits. For example, vaccination reduces healthcare costs by reducing influenza-related hospitalizations and staff absenteeism. Vaccinating pregnant women helps protect not only expectant mothers but also their newborn children up to 6 months of age. Immunization of school children has a fringe benefit of protecting their households and even reducing mortality rates among elderly individuals, since children are commonly the virus transmitters. Immunization of people with chronic diseases is an effective way to reduce the high disease burden among them. One study found that vaccination against influenza was beneficial for old people with cardiac disease and reduced the adjusted risk of winter mortality by 37% during four influenza seasons.

Immunization of health workers is particularly important not only for occupational health reasons but also for protecting their patients and families. There is evidence that vaccination of healthcare workers decreases influenza infections among them by 88% and reduces patient mortality by as much as 50%. Immunization of home-carestaff has been found to reduce mortality in at-risk groups. Despite these benefits, immunization rates of healthcare workers globally remain unacceptably low. The US CDC recommends 100% vaccination coverage of all healthcare workers, while in Europe, most countries recommend the immunization of healthcare professionals without a minimum coverage rate and it is not mandatory with some exceptions.

Not surprisingly, the vaccination coverage in many European countries is very low, ranging from 48% in France to 14% in the UK. Lack of knowledge has been cited as one of the possible explanations for the low coverage of healthcare workers. Restricted access to vaccination guidelines and other relevant information among healthcare workers in hospitals was found to contribute to their not recommending the vaccine to elderly patients.

To overcome the barriers and increase the immunization coverage among healthcare workers and particularly physicians, who can act as role models to their patients, the World Medical Association (WMA), with the support of IFPMA, launched a global campaign in 2013 to increase the awareness of physicians on the importance of the topic and help fill knowledge gaps to improve the immunization practices. The campaign information and materials can be found at:

http://www.wma.net/en/20activities/60campaigns/10immunization/

The campaign has just entered its second phase and aims to enhance physicians’ communication skills to promote influenza immunization among vulnerable groups (the elderly, people living with chronic conditions, pregnant women and children). As part of this campaign, IFPMA and WMA produced an infographic that demonstrates the linkages between influenza and non-communicable diseases (NCDs) and beneficial effects of immunization in people with NCDs. For example, evidence indicates that flu vaccination reduced the risk of hospital admissions of patients with diabetes by 79%. The infographic is available at:

http://www.ifpma.org/resources/infographics.html

It is clear that the implementation of a targeted vaccination strategy towards NCD patients and other vulnerable groups is very complex and will require a commitment of many stakeholders at multiple levels. Currently, no WHO targets have been set for the vaccination coverage level for patients with chronic conditions or other vulnerabilities, and health promotion campaigns are also lacking for healthcare professionals to council those patients and encourage them get vaccinated. The WMA campaign is just a start, and by involving physicians as role models in the campaign, the possibility of achieving adequate vaccination coverage, particularly in high risk groups, can become a reality.