Vital Directions for Health & Health Care An Initiative of the National Academy of Medicine (2017) / Chapter Skim
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18. Innovation in Development, Regulatory Review, and Use of Clinical Advances
18. Innovation in Development, Regulatory Review, and Use of Clinical Advances
Pages 369-394
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From page 369... ...
It provides the rationale for markedly enhanced patient input throughout the process from target identification to decisions regarding insurance coverage. It describes the role of academe–industry collaboration in speeding the translation of research findings into health benefits and emphasizes the opportunity for medical education at multiple levels to realize the value of therapeutic innovations to society.
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From page 370... ...
Eight recommendations in the President's Council of Advisors on Science and Technology 2012 report sought to "double the output of innovative new medicines for patients with important unmet medical needs, while increasing drug efficacy and safety, through industry, academia and government working together to decrease clinical failure, clinical trial costs, time to market and regulatory uncertainty" (PCAST, 2012)
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From page 371... ...
Discovery of New Therapies Opportunities abound to improve efficiency in the discovery phase of new therapy development, including the following: • Target "validation." New targets for drug development are urgently needed, and the Human Genome Project has provided thousands of potential targets. A precompetitive effort to determine which targets are most likely to produce therapeutic value would benefit all stakeholders and increase the success rate of new drug development.
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From page 372... ...
Recent examples of success in the preclinical and clinical spaces include the Accelerating Medicines Partnership (nih.gov/research-training/accelerating-medicines-partnership-amp) in the former and the Alzheimer's Disease Neuroimaging Initiative (adni-info.org)
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From page 373... ...
Inclusion of patient-reported outcomes that provide insights into benefit:risk assessment is critical. Patient focus consists not merely of anecdotes but rather of a science of patient input as described later in the section "Educating the Public, Policymakers, and the Mass Media" (see page 385)
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From page 374... ...
Moving activities away from tertiary care centers and closer to patients in their own communities has the potential to reduce the infrastructure costs associated with drug development dramatically. At the same time, such measures could broaden the participation of untapped
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From page 375... ...
Although improvements in predictive toxicology and safety assessment may mitigate the risk of adverse effects to some extent, earlier market entry of innova tive products generally means that safety and effectiveness profiles are not fully elucidated. Consequently, an understanding of a potential shift in the benefit:risk ratio in the postapproval setting requires continuous monitor ing through such mechanisms as the FDA Sentinel initiative (FDA, 2016a)
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From page 376... ...
Intensifying interest of patient groups, legislatures, and the mass media will lead to expansion of regulators' tasks in such spheres as global harmonization and "regulatory convergence," access to investigational drugs, use of real-world evidence (RWE) in regulatory decisions, clinical-trial data transparency, and response to outbreaks and pandemics.
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From page 377... ...
Many states have passed "right-to-try" laws that declare a seriously ill patient's right to request an investigational drug without government oversight. FDA approves almost all requests for patient access, but problems persist, including disparities in access to information, shortage of drug supplies, lack of access to an institutional review board, unwillingness of physicians to suggest or take responsibility for administering investigational drugs, and sponsors' inability or unwillingness to create access programs.
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From page 378... ...
Recent FDA initiatives to strengthen and improve the known issues with the regulatory review of combina tion products are a step in the right direction, such as development of the Combination Products Policy Council (FDA, 2016d) and launching of the Lean Management Process Mapping Project (FDA, 2016e)
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From page 379... ...
. The Medical Device Innovation Consortium produced a framework for incorporating patient preferences into regulatory assessments of new medical technology, and the University of Maryland's Center of Excellence in Regulatory Science and Innovation has created a patient-focused drug-development rubric (MDIC, 2015; UMCERSI, 2015)
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From page 380... ...
Despite efforts to increase patient engagement in drug development, regulatory uncertainty is a major barrier to obtaining useful input (Nordrum, 2015)
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From page 381... ...
. Our health information systems do not provide sufficient clinical support and advanced analytics to guide care or innovative care models.
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From page 382... ...
Cognitive computing has been used to identify targeted treatment options for patients who have specific variants of disease. Memorial Sloan Kettering Cancer Center, for example, has been working with IBM's Watson Health (mskcc.org/about/innovative-collaborations/watson-oncology)
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From page 383... ...
The inclusion of quality measures in the Health Information Technology for Economic and Clinical Health Act suggests that health information technology, when combined with a thoughtful approach to quality measurement, can be an important enabler of the rapid integration of evidence and new clinical standards into practice. • Institutional and clinical leadership.
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From page 384... ...
, information technology professionals who support medical practices, office-management staff, practice-based quality-improvement professionals, and payers who often set clinical standards for practice. In addition, it is critical to include future practitioners (such as medical students, residents, and subspecialty fellows)
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From page 385... ...
Educating the Public, PolicyMakers, and the Mass Media about Clinical Data and Trials Many of the efforts and suggestions presented in this paper will not be realized unless the knowledge and understanding of policymakers and the public are enhanced. We believe that strategic federal initiatives to increase understanding about the role of clinical trials, about the need to increase participation, and about the importance of clinical trials to society would constitute a worthwhile investment in the health of Americans.
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From page 386... ...
Innovative designs for clinical trials can reduce development time and expenses. Such designs are especially effective in demonstrating "proof of concept" and determining efficacy.
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From page 387... ...
There is interest in using the same huge clinical database to obtain RWE of efficacy. But, most clinical databases have flaws.
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From page 388... ...
As clinical data move toward universal storage on digital platforms, the pos sibility exists to reduce the time and expense involved in the development of evidence on the effectiveness, safety, and applicability of medical inter ventions. Priorities include initiatives to develop data and interoperability standards, and improve data quality and accessibility, capacity to facilitate protected data sharing, and regulatory policies that allow phased introduc tion with evidence generation.
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From page 389... ...
To capture the advantages of the use of patient-generated data to care man agement and of patient involvement to care outcomes, priorities include initiatives to enable and facilitate the roles of patients and families in all clinical decision making, and to enlist their guidance and involvement in the capture, design, and use of clinical data for new knowledge.
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From page 390... ...
2016f. The voice of the patient: A series of reports from FDA's Patient Focused Drug Development Initiative.
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From page 391... ...
2013. FDA and pharmaceutical companies welcome patient voices to new drug development -- but will it last?
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From page 392... ...
2015. Assessing meaningful patient engagement in drug development: A definition, framework, and rubric.
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From page 393... ...
Janet Woodcock, MD, is Director, Center for Drug Evaluation and Research, Food and Drug Administration.
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