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Global Dementia Legacy Event: Raj Long, Senior Regulatory Officer


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Session Two: Barriers to investment in research to find a disease modifying therapy or cure for dementia.

Raj Long, Senior Regulatory Officer – Integrated Development, Global Health at the Bill & Melinda Gates Foundation & World Dementia Council Member

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Global Dementia Legacy Event: Raj Long, Senior Regulatory Officer

  1. 1. Global Dementia Legacy Event – London, 2014 Dementia Treatment in Pharmaceutical Research Regulatory Challenges & Imperatives Raj Long Senior Regulatory Officer - Integrated Development Global Health Bill & Melinda Gates Foundation - London 1
  2. 2. Pharmaceutical R&D Regulatory Decision Points LabelDose Safety SurveillancePK* initial human safety Animal model efficacy Safety and Efficacy Drug and target Efficacy DevelopmentResearch Full DevelopmentEarly Development LaunchDiscovery Post Launch Approval Phase III Phase IIb Phase IIa Phase I PoC Pre-Clinical Phase Candidate Selection Phase Every Step is a Regulatory Hurdle to Overcome – Chronological, Duration >12 years & £££ R. Long - June 19th, 2014 2
  3. 3. Regulatory Variances Example - Intravenous Dx Agent for AD Development Rationale Same Indication US FDA • Single Application • Review Dx Division • Covered primarily by Dx and medicines regulations • PDUFA coverage 1 dose 2 Pivotal Studies EMA • Single Application • Review CHMP ( same as for medicines) • Covered by Medicines Regulations 2 doses 2 Pivotal Studies Japan • 2 Applications • Device and Medicine, 3 Pivotal studies Same Molecule US EU Japan R. Long - June 19th, 2014 3
  4. 4. Regulatory Challenges Key Challenge Impact Ageing – prolonged time process. Disease prediction, diagnosis, treatment , monitor  Too long for traditional R&D – high costs with high risks  Should the regulatory environment reflect this …. Why? Dementia  Still somewhat of an unknown ‘quantity’ for both regulators & industry  Defining the regulatory framework comes from ‘experience’  Experience is limited and dispersed – many unknowns Regulatory Variances by Geography  Not an Incentive for developers  Costs /Risks Regulatory/Clinical Guidelines  Have we got the science correct ? Need to focus more on translational research or disease modelling ? Technology in Dementia  Technology can play a key role – prediction, diagnostics, & monitoring. Need awareness that technology is a moving goal post – potential challenge for regulators 3 1 4 5 2 R. Long - June 19th, 2014 4
  5. 5. Dementia Regulatory Landscape • Attrition is high in discovery & early development - RISK • Clinical ‘unknowns’ poses regulatory strategy issue – RISK – a single disease or multiple – ‘black box’ of disease progression – known ‘unknowns’ resulting in a lack of target mode of action • Highly regulated platform and with variances –requiring tailoring of ‘global’ R&D to a ‘local’ geography – RISK High Risks + High Costs = Investment Deterrent R. Long - June 19th, 2014 5 Insight as to why only 3 drugs were launched in 15 years !!!
  6. 6. Innovative Regulatory Imperatives 1. Can Dementia merit a special regulatory category – e.g. HIV, Oncology, Orphan drug designation. Target – expedited review 2. Explore ‘adaptive development design’ potentially leading to adaptive licensing. E.g. Target approval with a robust Ph2 + dynamic disease modelling. Follow up with a post approval commitment and bridge with a rigorous risk management plan to safeguard efficacy/safety. 3. G7 Regulators sharing resource – split/twin/share logical components of the review process. Confluence of resource, expertise and expedite the review time 4. Develop a series of regulatory incentives e.g. fee waivers/reduction, use of priority vouchers e.g. Knight Therapeutics for Impavido (Leishamaniosis Disease) 5. Proof of Concept Study Pilot – Conduct a tripartite partnership of manufacturer, regulators, developers and WHO to work together through the entire development cycle e.g. Meningitis Vaccine in Africa 6. Global Fund – enable private/public partnership to catalyse G7 ambition to deliver a cure/disease modifying therapy by 2025. Cannot be Business as Usual … R. Long - June 19th, 2014 6
  7. 7. Disclaimer •The views and opinions expressed in the above PowerPoint slides are those of the individual presenter and should not be attributed to affiliates, or any organization with which the presenter is employed or affiliated. •These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United Kingdom and other countries. Used by permission. R. Long - June 19th, 2014 7