Innovation in Drug Research and Development for Prevalent Chronic Diseases Proceedings of a Workshop (2021) / Chapter Skim
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6 Lessons Learned for the Future
6 Lessons Learned for the Future
Pages 51-62
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From page 51... ...
• Managed care payers are reticent to pay for new drugs if generic classes of drugs are available, which contributes to the lack of innovation in drug R&D for prevalent chronic diseases. (Manolis)
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From page 52... ...
Michelle Rohrer, global head of product development regulatory and policy at Roche, provided an industry perspective, offering a suite of suggestions to foster more innovative trial design and better incorporate patient input. Joseph Menetski, associate vice president of research partnerships at the Foundation for NIH, spoke about ways the foundation is helping to open up new possibilities for understanding and treating prevalent chronic diseases.
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From page 53... ...
. Specifically, she described a few challenge areas that will be of particular importance in developing treatments for prevalent chronic diseases in the future: • Trials are growing more complicated, with more procedures and more endpoints, with data from the Tufts Center for the Study of Drug Development indicating that between 2005 and 2020, the average number of data points for a phase III trial grew from 494,000 to 3.56 million (Tufts Center for the Study of Drug Devel opment, 2021)
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From page 54... ...
These include model-informed drug development, adaptive clinical trials, Bayesian approaches, the inclusion of novel and digital end points, platform studies, testing multiple agents, the use of external controls, and the use of real-world data in assessing quality, safety, and efficacy. There may be resistance from regulators and inves tors, Rohrer acknowledged, but adopting new approaches will be crucial "because it is just not sustainable to insist on randomized clinical trials for absolutely every condition when we have these other perfectly valid innovative approaches to use." 5.
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From page 55... ...
SOURCES: Presented by Chronis Manolis on March 1, 2021, at the Innovation in Drug Research and Development for Prevalent Chronic Diseases Workshop; Pharmaceutical Research Manufacturers of America, 2020.
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From page 56... ...
This, in turn, impacts whether a company might be willing to support research programs for prevalent chronic diseases. Another issue is what Manolis called the "pharmacy benefit managers rebate machine" -- the current practice of pharmaceutical companies paying rebates to pharmacy benefit managers to favor their drugs, which can skew the choice of drugs put on a formulary.
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From page 57... ...
The Accelerating Medicines Partnership AMP launched in 2014 as a public–private partnership among NIH, FDA, multiple biopharmaceutical and life science companies, nonprofits, and other organizations to transform the current model for developing new diagnostics and treatments Based on input from NIH director Francis Collins and representatives from biopharmaceutical companies, AMP has focused on identification of drug targets. In particular, Menetski said, AMP focused on making sure that targets that had been identified in animal models translated to humans.
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From page 58... ...
The project on type 2 diabetes produced a publicly available repository of genetic data to help researchers identify genes of interest and validate drug targets. The Biomarkers Consortium The Biomarkers Consortium, by contrast, has focused on generating drug development tools that can be used to enhance the results from clinical trials.
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From page 59... ...
A REGULATORY PERSPECTIVE Smith highlighted the heterogeneity of chronic diseases from a drug development perspective, stating that, "with respect to available therapies, some disorders have hardly any truly effective available therapies, whereas others have multiple classes, sometimes with substantial availability of generic products." He recognized there could be variability across chronic diseases in terms of the pathophysiology and how well the underlying disease biology is understood, and in terms of the degree of regulatory precedent with respect to suitable endpoints for clinical trials. On top of these considerations, Smith suggested that in many cases, drug R&D for prevalent chronic diseases may require large clinical trials to detect the treatment effect or ensure an adequate safety database, particularly if existing safe and available therapies are available.
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From page 60... ...
"We have been doing cardiovascular outcome trials for decades," he said, "and there might be ways to innovate here that would actually provide more outcome events" and thus improve the efficiency of the trial. The implementation of decentralized clinical trials, in which some or all trial-related procedures and data acquisition take place at locations remote from the investigator, could also offer new opportunities to reach patient populations who might not otherwise be included in clinical trials.
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From page 61... ...
2020. Net spending on retail specialty drugs grew rapidly, especially for private insurance and Medicare Part D
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