Nov24 Regulatory approaches

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Information about Nov24 Regulatory approaches

Published on April 16, 2008

Author: Alien


PRIORITY MEDICINES FOR EUROPE AND THE WORLD :  PRIORITY MEDICINES FOR EUROPE AND THE WORLD A report prepared by WHO for the Netherlands Government by Warren Kaplan Richard Laing and Saloni Tanna Marjolein Willemen Eduardo Sabaté Monique Renevier Joyce Wilson Lisa Greenough Ann Wilberforce Kathy Hurst Objectives of Priority Medicines Project:  Objectives of Priority Medicines Project Provide a methodology for identifying pharmaceutical “gaps” from a public health perspective, for Europe and the World . Provide a public-health based pharmaceutical R&D agenda for use by the EU in the 7th Framework Programme, “Good public policy should spend public funds on areas of greatest public needs” Generating a Preliminary List of Diseases and Gaps:  Generating a Preliminary List of Diseases and Gaps Pharmaceutical "Gap":  Pharmaceutical "Gap" Example of an absent pharmaceutical gap:  Example of an absent pharmaceutical gap "Commonality of interest" :  "Commonality of interest" Special Needs for Women, Children, and the Elderly:  Special Needs for Women, Children, and the Elderly All groups neglected in drug development Complicated by different physiology & metabolism Recent improvements in situation of women and children Considerable gaps remain for the elderly who use the most medicines Promoting Innovation and Removing Barriers:  Promoting Innovation and Removing Barriers Public Private Partnerships may be a vehicle to address market failure Pricing issues are critical to the future of the European pharmaceutical industry. Propose investigating differential pricing based on GNI per capita and efficacy measures. Reseach prospective price setting. EMEA, FDA, Rawlins and Industry have all proposed similar measures to remove barriers Comparative trials provide critical information on head to head comparisons. Use of European databases may facilitate such studies Differential Pricing: Indicative prices in US$/annum of highly active antiretrovirals (HAART) and a new hypothetical regimen in countries of variable wealth :  Differential Pricing: Indicative prices in US$/annum of highly active antiretrovirals (HAART) and a new hypothetical regimen in countries of variable wealth Barriers to Innovation: Background:  Barriers to Innovation: Background Cost and duration of medicines development have increased Many projects in early development stages, but not enough of this potential innovation is being translated into market approvals. For some diseases, there remains an inadequate understanding of basic science, and potential targets for medicines development have not been identified. Recent Publications:  Recent Publications Four papers published almost simultaneously, plus commentary on Priority Medicines website EMEA: Discussion Paper: The European Medicines Agency Road Map to 2010: Preparing the Ground for the Future  FDA: " Challenge and Opportunity on the Critical Path to New Medical Products" Perspectives – "Cutting the Cost of Drug Development?" M.D. Rawlins Sue Middleton: GSK Commentary on "Barriers to Innovation" Barriers to Innovation: Suggested Solutions:  Barriers to Innovation: Suggested Solutions All authors agreed that every aspect of the regulatory process should be re-examined and that the evidence base for regulatory practices should be critically analysed using modern methodologies. Preclinical studies Many old tests and methods New analytic tools needed Validation of old methods required Clinical Studies Regulatory authorities are risk averse so the RCT has become the gold standard Other approaches even using historical controls may be possible and desirable Neglected Areas: Post marketing Surveillance and Patients:  Neglected Areas: Post marketing Surveillance and Patients All papers neglect Phase IV studies as a part of the innovation process. Great opportunities for Europe from use of electronic data bases for Phase IV studies on both efficacy and safety Apart from the industry paper, none of the three other papers mention any role for patients in this review. They are referred to as beneficiaries of the process but never as contributors to the decision-making. Role of Patients remains unclear:  Role of Patients remains unclear Patients have speeded innovation e.g. AIDS and Orphan diseases Valuable role in treatment guideline development emerging e.g. NICE Patients play important role in ethical & hospital committees e.g. IRB & DTCs Will now be part of CSM in UK Future role likely to be important and growing A "barriers to innovation" Research Agenda:  A "barriers to innovation" Research Agenda Bottlenecks and barriers in the current medicines development process are to be identified and solutions elaborated to overcome them. Range of stakeholders required: academia, clinicians, patient organizations, large and small industry, regulatory and ethics specialists Every requirement within the medicines development process whether clinical or preclinical should be questioned for its regulatory relevance, costing, and predictive value. The involvement of the EMEA and the various European national regulatory agencies and their scientists is critical to this initiative. 6th Framework already has issued a call closing November 16th PRIORITY MEDICINES FOR EUROPE AND THE WORLD :  PRIORITY MEDICINES FOR EUROPE AND THE WORLD Comparative Clinical Trials Chapter 8.4 Warren Kaplan Comparative Trials: Background:  Comparative Trials: Background For most medications at launch, we know little about whether or not a given medication is better value for the money than alternatives. This knowledge gap exists because most drug regulatory authorities do not examine "value for money" For most medications at launch, clinical trials are not designed to look at whether or not a given medication is better than another drug. Typically, efficacy is measured versus a control which is often, but not invariably, a placebo lacking any active ingredient. Many registration authorities do require comparative benefit studies To estimate comparative benefit between two medicines, large (time consuming and expensive) trials are needed since there are often small differences between treatment outcomes when comparing the interventions. Comparative Trials: Who should pay for them and who conducts them?:  Comparative Trials: Who should pay for them and who conducts them? Industry? Government? Or… Private insurers and the government could set aside some fraction of their annual medicines spending to endow an organization to provide an independent source of reputable research into comparative effectiveness and cost. Electronic prescription and medical databases to conduct Phase IV and/or pharmacoepidemiologic studies in place of controlled, comparative clinical trials. EU has a great comparative advantage over the USA as this kind of electronic linkage of prescribers and medical records is still fragmented in the USA Phase IV studies could add a randomization step ("randomized epidemiology") Comparative Trials/ Comparative Effectiveness:  Comparative Trials/ Comparative Effectiveness Europe 2005: "An information society for all", available at This Action plan on "electronic health" should be used as a way of creating post-marketing studies to better understand comparative effectiveness and cost-effectiveness. General Conclusions:  General Conclusions Commonality of interest exists for chronic diseases between Europe and the World Priorities can be set based on evidence, trends and projections and social solidarity Pharmaceutical gaps exist as a result of biological challenges and market failure Highest priorities are antibacterial resistance, influenza, smoking and neglected diseases Pricing issues and barriers to innovation strongly affect the European industry The EU needs to find a way to support translational research for market failure pharmaceutical gaps Regulatory Conclusions:  Regulatory Conclusions Common view as to how regulatory obstacles serve as a barrier to innovation Different solutions proposed by different stakeholders. All should be considered Striking lack of attention to Phase 4 activities. Vioxx and FDA hearings may change everything Strong interest in routine use of large (even complete) patient data bases A researchable topic! Priority Medicines Project :  Priority Medicines Project For further questions, please contact: +41-22-791-4533

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