Real-World Evidence Generation and Evaluation of Therapeutics Proceedings of a Workshop (2017) / Chapter Skim
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2 Improving Evidence Generation for Decision Making on Approval and Use of New Treatments: Some Stakeholder Priorities
2 Improving Evidence Generation for Decision Making on Approval and Use of New Treatments: Some Stakeholder Priorities
Pages 7-16
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From page 7... ...
(Califf, Carroll, Vallance) • Stakeholders lack the evidence needed to make real-world deci sions on approval, coverage, and use of treatments because current evidence generation processes focus narrowly on ques tions of safety and efficacy.
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Although this realization has not yet led to significant changes in the pipeline for the production of new therapeutics, a number of advances that are underway offer opportunities to reshape the current evidence generation paradigm. THE CURRENT LANDSCAPE FOR EVIDENCE GENERATION PROCESSES FDA Commissioner (at the time of the workshop)
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From page 9... ...
The digital capture, aggregation, and analysis of health care data with the goal of improving quality of care and cost-effectiveness represents a fundamental change in evidence generation processes, with significant implications for medical product development. Califf stressed that with this new model for knowledge generation, there would be less need for a completely separate, and consequently inefficient, clinical research infrastructure.
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From page 10... ...
Industry Patrick Vallance, president, Pharmaceuticals Research and Development, GlaxoSmithKline, observed that the medical product development industry is actually becoming less efficient. The cost of clinical trials is increasing sharply (Berndt and Cockburn, 2014)
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Regulators Addressing the escalating costs of bringing new treatments to market, Califf emphasized the need for a drug development system that winnows out failures as quickly as possible and, for the promising candidates, ensures that the right clinical trials are undertaken to inform decision making by all stakeholders. While maintaining that very early clinical trials will still need to be conducted in highly controlled environments so that the safety, pharmacology, and systems biology of new treatments can be carefully assessed, Califf stressed the later stage research can be integrated within the real world of clinical practice, the better the system will be at yielding results that give doctors and patients the information they need to understand what treatment options are best for them.
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While this can create tension between payers and industry, Rhonda Robinson Beale, chief medical officer, Blue Cross of Idaho, underscored the fact that when 3 The 21st Century Cures Act, signed into law on December 13, 2016, requires FDA to evaluate the use of real-world evidence to help support the approval of a new indication for a previously approved drug and to help support or satisfy postapproval study requirements. Under the direction of the Secretary of Health and Human Services, a guiding framework will be developed to implement a program within FDA that details the circumstances, standards, and methodologies for which real-world evidence can be used in medical product evaluation.
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From page 13... ...
Joseph Chin, deputy director, Coverage and Analysis Group, CMS, pointed out that some types of patients, such as Medicare beneficiaries, are often excluded from clinical trials, making it challenging to make coverage determinations for those populations. Chin noted that the incorporation of real-world data into evidence generation processes could assist CMS coverage determinations by rendering clinical research results more immediately translatable to the beneficiary population, both by incorporating data from a more general population than typically seen in clinical trials and by potentially creating the opportunity to apply the results obtained during approval at FDA without further need to request additional studies on efficacy for CMS.
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From page 14... ...
Carroll suggested that priority focus areas to facilitate such learning health systems include the improvement of data quality and reduction of the magnitude of effort and cost currently required to gather data and translate them into actionable information. Several individual panelists agreed with Carroll that engaging health care providers in prospective research activities will add a significant burden in addition to their clinical responsibilities and thus will present a challenge to embedding research in the clinical care infrastructure.
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From page 15... ...
Califf agreed that efforts to improve communication within and outside FDA could give companies more confidence about incorporating real-world data sources and pursuing alternative endpoints. Harmonizing Evidence Generation Processes Across Stakeholder Groups There is a great deal of interest across the biopharmaceutical industry, regulatory agencies, and payers in harmonizing evidence generation processes as a means of improving the efficiency of the drug development rocess and simultaneously generating the kinds of evidence needed to p support real-world decision making.
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From page 16... ...
Addressing Privacy and Confidentiality Concerns Embedding research into the clinical care infrastructure depends on the ability to share, aggregate, and analyze patient data. But in the current cybersecurity environment, it is not possible to absolutely guarantee the security of those data, cautioned Califf, who advocated instead for a participatory environment that is endorsed by patients and consumers and includes robust procedures for ensuring data security and protecting confidentiality.
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