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But both public and private funders developing therapeutics consider disease burden and unmet need in the context of myriad other competing considerations and goals in determining where to invest, such as limited funding; balancing advance ments in basic, translational, and clinical science; advocacy from patient groups; the likelihood of regulatory approval and associated challenges; payer reimbursement; market competition; and overall timing and overall profits and return on investment. These competing practical and financial priorities do not always align with diseases with the highest burden and unmet needs.
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Some diseases and conditions have no available therapies, and often the existing, approved therapies are only modestly effective, have unclear clinical benefits, or have significant safety risks. Thus, the current system of drug development needs adjusting to improve the alignment of public and private investment in therapeutics with areas of unmet needs.
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5. Strengthen a fiscal and policy environment to align reimbursement policy with evidence-based therapeutic value and the extent to which products address unmet need.
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4 ALIGNING INVESTMENTS IN THERAPEUTIC DEVELOPMENT Identify misalignments between: Problem Disease burden and Current therapeutic Definition unmet medical needs investments and scientific research efforts •Information gaps •Scientific gaps Key Barriers •Market failures to Alignment •Policy and regulatory frictions •Enhanced data transparency •Targeted research funding Policy Action •Policy and regulatory investment and reform Areas •Public–private partnerships •Increased research activity in high-need disease areas Intermediate •Reduction in scientific and market barriers Outcomes to innovation •Need-aligned therapeutic development that promotes equitable health benefits •Alignment of therapeutic innovation with public health priorities Long-Term •Improved health outcomes and health equity Outcomes •Enhanced return on health research and development investments FIGURE S-1 Conceptual framework work aligning investment with therapeutic need.
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Currently, leaders have imperfect data upon which to make scientifically informed policy judgments. Limitations in the availability of data necessary to assess how investments in therapeutic development align with disease burden and unmet medical need is one factor that contributes to misalignment between investments in therapeutic development and unmet medical need during research prioritization.
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government has a responsibility to ensure that timely data on public investment and population health data be made publicly available to support research and strategic investment in areas of unmet need. Following these conclusions, the committee specifically recommends the following: Recommendation 1: Congress should establish and fund an interagency consortium charged with tracking and assessing unmet therapeutic need associated with U.S.
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One way of collecting this information could be through a survey of pharmaceutical and biotech companies, which, while imperfect, could gather a useful sampling of data to gain insight into private investment in therapeutic development. The collecting of these data could be incentivized by allowing early access to updated datasets and reports to private and public companies that have contributed to the repository, which would provide a valuable, precompetitive output that companies could use to inform business decisions.
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Similarly, a lack of plausible surrogate endpoints in some disease areas prevents drug developers from using the accelerated approval pathway, which is designed to bring therapeutics to market faster on the condition that they complete postmarketing studies to confirm clinical benefits. The committee found that investment in preclinical biomedical research is essential to driving innovation in disease areas with significant burden and unmet needs.
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efforts to facilitate and expedite development and review to address unmet need. After extensively reviewing the evidence around these programs, the committee made the following conclusion: Conclusion 5-3: Additional resources are needed for FDA programs that successfully support endpoint development and validation, innova tive trial design, and the resolution of other broad scientific challenges that impede drug development for unmet needs, as well as programs designed to support communication between sponsors and FDA to quickly resolve challenges arising in specific development programs.
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For example, regulatory flexibility may be unavoidable for certain rare diseases, where it is nearly impossible to meet standard expectations. However, regulatory flexibility that allows for approval when trials fail to show clinical benefit raise a number of concerns, including the possibility of misleading patients, inhibiting the development of the information necessary to guide clinical decision making, and impeding the conduct of clinical trials for other drugs that may be more efficacious, both by
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Recommendation 7: To ensure that regulatory flexibility is exercised in a manner that promotes the approval of drugs that are both safe and effective, the Food and Drug Administration (FDA) should uphold strong regulatory approval standards.
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One potential strategy to address market forces and promote therapeutic development is to use public–private partnerships (PPPs) , which can be particularly beneficial for innovation in areas where cost and risk sharing are valuable, such as novel drug target discovery, biomarker testing, or preclinical development models.
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Despite the successes of existing PPPs and the potential to expand these to enhance innovation in needed therapeutic areas, several barriers exist within the United States to developing more PPPs. First, PPPs are often initiated by federal agencies seeking to expand therapeutic development in a specific area.
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A revised approach to reimbursement policy could help address innovation incentives for therapeutical development by linking pricing to evidencebased value assessments that incorporate clinical effectiveness and patient perspectives. Many other countries, including the UK and Germany, offer useful models for this approach of prioritizing payment for new therapies that address unmet need and limiting reimbursement for products that offer marginal improvements or whose clinical benefit remains unproven, although the details of these programs would need to be adapted for the United States.
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. Recent evidence also suggests that as commercial products have under performed, new investments in cell and gene therapies and curative thera pies are being challenged, and drug developers have begun to retreat from this market, despite the exceptional benefits of these products for patients (especially those with very rare diseases)
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These strategic investments will drive scientific discoveries that enable more efficient clinical research that -- when accompanied by aligned market incentives -- can bring to market effective therapies that address critical unmet needs.
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