Innovation in Development, Regulatory Review, and Use of Clinical Advances: A Vital Direction for Health and Health Care

Rosenblatt, M; Ventures, Flagship; Austin, Christopher P.; Boutin, Marc; Chin, William W.; Galson, Steven K.; Jain, Sachin H.; McMurry-Heath, Michelle; Nussbaum, Samuel R.; Orloff, John J.; Weinberger, Steven E.; Woodcock, Janet

doi:10.31478/201609u

Cited by 3 publications

(6 citation statements)

References 6 publications

(5 reference statements)

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“…The use of biomarkers has been encouraged by the FDA, which has now established means by which to qualify biomarkers for use in drug development, but there is a lack of validated biomarkers at this time 14, 15, 16. Commercial development and validation of biomarkers is slow, because development of robust and meaningful biomarkers is both time-consuming and extremely expensive (Figures 1 and 2) (17).…”

Section: Why Drugs Fail In Clinical Trialsmentioning

confidence: 99%

Phase II Trials in Drug Development and Adaptive Trial Design

Norman

2019

JACC: Basic to Translational Science

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Summary Phase II clinical studies represent a critical point in determining drug costs, and phase II is a poor predictor of drug success: >30% of drugs entering phase II studies fail to progress, and >58% of drugs go on to fail in phase III. Adaptive clinical trial design has been proposed as a way to reduce the costs of phase II testing by providing earlier determination of futility and prediction of phase III success, reducing overall phase II and III trial sizes, and shortening overall drug development time. This review examines issues in phase II testing and adaptive trial design.

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Section: Why Drugs Fail In Clinical Trialsmentioning

confidence: 99%

Phase II Trials in Drug Development and Adaptive Trial Design

Norman

2019

JACC: Basic to Translational Science

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“…More collaboration among the government, academia, and industry scientists will be necessary to advance innovation, including in the most challenging therapeutic areas such as autoimmune, neurodegenerative, and inflammatory diseases. 44 Exploration of multifocal public-private research partnerships has been the focus of initiatives at the NIH and FDA, including those related to the programs expanding brain and cancer research. Pharmaceutical and device companies are exploring sharing trial data in the interest of advancing very-large-scale clinical databases to facilitate discovery.…”

Section: Advance Science-forge Innovation-ready Clinical Research Pro...mentioning

confidence: 99%

“…Foster cross-disciplinary and public-private partnerships. More collaboration among the government, academia, and industry scientists will be necessary to advance innovation, including in the most challenging therapeutic areas such as autoimmune, neurodegenerative, and inflammatory diseases . Exploration of multifocal public-private research partnerships has been the focus of initiatives at the NIH and FDA, including those related to the programs expanding brain and cancer research.…”

Section: Essential Infrastructure Needsmentioning

confidence: 99%

Vital Directions for Health and Health Care

Dzau

McClellan

McGinnis

et al. 2017

JAMA

148

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IMPORTANCE Recent discussion has focused on questions related to the repeal and replacement of portions of the Affordable Care Act (ACA). However, issues central to the future of health and health care in the United States transcend the ACA provisions receiving the greatest attention. Initiatives directed to certain strategic and infrastructure priorities are vital to achieve better health at lower cost.OBJECTIVES To review the most salient health challenges and opportunities facing the United States, to identify practical and achievable priorities essential to health progress, and to present policy initiatives critical to the nation's health and fiscal integrity.EVIDENCE REVIEW Qualitative synthesis of 19 National Academy of Medicine-commissioned white papers, with supplemental review and analysis of publicly available data and published research findings. FINDINGSThe US health system faces major challenges. Health care costs remain high at $3.2 trillion spent annually, of which an estimated 30% is related to waste, inefficiencies, and excessive prices; health disparities are persistent and worsening; and the health and financial burdens of chronic illness and disability are straining families and communities. Concurrently, promising opportunities and knowledge to achieve change exist. Across the 19 discussion papers examined, 8 crosscutting policy directions were identified as vital to the nation's health and fiscal future, including 4 action priorities and 4 essential infrastructure needs. The action priorities-pay for value, empower people, activate communities, and connect care-recurred across the articles as direct and strategic opportunities to advance a more efficient, equitable, and patient-and community-focused health system. The essential infrastructure needs-measure what matters most, modernize skills, accelerate real-world evidence, and advance science-were the most commonly cited foundational elements to ensure progress. CONCLUSIONS AND RELEVANCEThe action priorities and essential infrastructure needs represent major opportunities to improve health outcomes and increase efficiency and value in the health system. As the new US administration and Congress chart the future of health and health care for the United States, and as health leaders across the country contemplate future directions for their programs and initiatives, their leadership and strategic investment in these priorities will be essential for achieving significant progress.

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“…Nonetheless, there are several underlying conditions which also suggest that the apparent regulatory delays are unintended. For instance, the upshot of the knowledge gap between innovators and reviewers is the additional time regulators may require in assessing and extending their understanding of the technology underpinning the NPI under review in order to arrive at a decision (Rosenblatt et al, 2016;Carpenter, 2004). This phenomenon is largely due to the pace at which technologies evolve as well as the complexity and sophistication they come with.…”

Section: Speed and Delaysmentioning

confidence: 99%

“…The ever-increasing numbers and complexity of data required for regulatory approval imposes a severe financial burden on innovating firms, and this is leading to the collapse of relatively smaller innovating firms in the pharmaceutical industry (Rosenblatt et al, 2016;Dimasi et al, 2003;Thomas, 1990). In recent times, several scholars have observed that in addition to loss in early entrant profit and competitive advantage that may be accrued to the innovating firm (Prieger, 2008;Carpenter, 2002), regulatory delay imposes high opportunity costs for investment, and research and development (R&D) time (DiMasi et al, 2010;Dimitri, 2010;Vernon et al, 2009;DiMasi, 2002).…”

Section: Cost and Uncertaintymentioning

confidence: 99%