Narrowing the Gap. Clinical Development and Regulatory Pathways for Medical Technologies for Neglected Diseases. Tom Bollyky

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1 Narrowing the Gap Clinical Development and Regulatory Pathways for Medical Technologies for Neglected Diseases Tom Bollyky Stanford Law School Drug Development and International Access Conference April 9, 2010

2 Overview Disclaimer Health Product Development for Neglected Diseases Tremendous Recent Progress; Looming Challenges Potential Funding Gap The Clinical Development Pathway for Medical Technologies for Neglected Diseases Moving towards a Sustainable Strategy for Bringing Clinical Trial Costs and Finances into Better Balance SLS - 2 4/9/10

3 Neglected Diseases Disclaimer Chronic infectious diseases that affect rural and urban poor in low and middle-income countries 1 billion people, including 400 million children, suffer from one or more neglected tropical diseases (NTDs). If HIV/AIDS, Malaria, and TB included, affected population is much larger. Millions of deaths per year, many more disabled or deformed, undermines economic development, education, and security. SLS - 3 4/9/10

4 Historically, Few Products for NDs Disclaimer Many NTDs have no viable treatment or prevention options The 10/90 gap only 10% of worldwide expenditure on health research and development has been devoted to the problems that primarily affect 90% of the world's population. Of the 1393 new chemical entities approved between 1975 and 1999, only 46 (3.3%) were for neglected diseases (including HIV/AIDS, Malaria, and TB). SLS - 4 4/9/10

5 Increased Recent Attention and Funding Disclaimer Between 1999 and 2009, annual funding for neglected diseases rose 25-fold ($2.5 billion in 2007) 60% of the total spending on neglected diseases was from the Bill and Melinda Gates Foundation and U.S. National Institutes of Health Biopharmaceutical industry provided 9% of the total spending plus in-kind contributions of expertise and services 80% of the funding has gone to HIV/AIDS, Malaria, and TB SLS - 5 4/9/10

6 Push Disclaimer and Pull Mechanisms Efforts to create incentives for health product discovery, development, and delivery for neglected diseases can be divided into two general categories Push mechanisms -- subsidize or lower costs of R&D Grants, tax credits, and subsidies Expedited regulatory review Liability Protection Pull mechanisms increase potential reward for successful R&D and delivery Advance market commitments Prizes Intellectual Property and market exclusivity incentives Priority Review Vouchers Most funding to date has been push self-funded research or to Public-Private Product Development Partnerships (PDPs) and other intermediaries SLS - 6 4/9/10

7 Tremendous Progress; Looming Challenges Disclaimer Tremendous progress over the last decade in building the pipeline for health products for neglected diseases Led by efforts of PDPs, dozens of products in now pipeline Challenges loom: potential shortfall in financing for clinical development limited research and regulatory capacity for late stage trials SLS - 7 4/9/10

8 Challenges of Clinical Development Generally Disclaimer Clinical trials for drugs and vaccines have become increasingly time consuming and expensive Clinical development can be as much as 70% of the total cost of developing a medical technology Process can last between years Success rates low SLS - 8 4/9/10

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10 Challenges of the Clinical Development of Products Disclaimer for Neglected Diseases Trials must occur in disease-endemic countries, which often have limited clinical research capacity and regulatory infrastructure Difficult regulatory and ethical challenges posed by the study products and subjects Trials are often large-scale, multi-center, and multicountry SLS /9/10

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13 Challenges of the Clinical Development of Products Disclaimer for Neglected Diseases Trials must occur in disease-endemic countries, which often have limited clinical research capacity and regulatory infrastructure Difficult regulatory and ethical challenges posed by the study products and subjects Trials are often large-scale, multi-center, and multicountry SLS /9/10

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16 Challenges of the Clinical Development of Products Disclaimer for Neglected Diseases Trials must occur in disease-endemic countries, which often have limited clinical research capacity and regulatory infrastructure Difficult regulatory and ethical challenges posed by the study products (often vaccines) and subjects (frequently pediatric, in devastatingly poor settings) Trials are often large-scale, multi-center, and multicountry SLS /9/10

17 Basic Elements of the Clinical Trial Process Pre-Trial Establish working group to develop idea Estimate costs & obtain financing Develop trial protocol, patient information sheet, consent form Record trial on an international clinical trials register Trial Set -Up Protocol authorization from regulatory authority Obtain ethical approval from Institutional Review Board (IRB) Implement procedures for drug handling Develop the case report forms Negotiate the necessary agreements and contacts Obtain approval from each site Set up site assessment & initiation Activate sites During Trial End of Trial Conduct trial; monitor progress in sites Independent Data Monitoring Committee Send annual safety report to regulatory authority Send Annual Progress & Safety report to Ethics Committee Lock database & Close trial (inform regulatory authority) Published first efficacy and safety results Long-term follow up (efficacy and/or safety)

18 PATH/GSK Disclaimer RTS,S Phase III Trial 11 clinical trial sites 7 countries 40 IRBs 16,000 children and infants SLS /9/10

19 A Sustainable Strategy for Bringing Clinical Trial Disclaimer Costs and Finances into Better Balance Increased or innovative financing important, but reducing clinical trial cost and risks must be part of the solution Improvement in clinical development pathway necessary for increased private investment and scientific rigor, quality of trial data, and protection of subjects SLS /9/10

20 Sensible Clinical Trial Research to Neglected Disclaimer Disease Pipeline Emerging research on sensible approaches to reducing cost and risks of large scale, randomized, commercial clinical trials Potential application to products or settings most relevant for neglected diseases Vehicles for assembling and encouraging adoption of sensible clinical trial guidelines SLS /9/10

21 Regional Disclaimer Regulatory Streamlining Regional pathways with integrated regulatory and ethics reviews could have significant benefits here: Pool regional regulatory resources and share expertise Reduce unnecessary costs, delays, and risk Link to existing networks or regional economic or public health entities Potentially generate private sector funding or cross subsidies SLS /9/10

22 Thank you SLS /9/10

Bridging the Gap: Improving Clinical Development and the Regulatory Pathways for Health Products for Neglected Diseases

Bridging the Gap: Improving Clinical Development and the Regulatory Pathways for Health Products for Neglected Diseases Thomas J. Bollyky Abstract There has been tremendous progress over the last decade