Earlier today, the House Energy and Commerce Committee capped almost a year of hearings, roundtables, and stakeholder outreach with the release of draft legislation focused on improving the process of developing life-saving medical products and technologies. This effort – the 21st Century Cures initiative – was a rare bipartisan bright spot during the closing days of the 113th Congress, and we commend Chairman Fred Upton (R-MI) and Representative Diana DeGette (D-CO), who spearheaded this work, as well as their fellow committee members and the Energy and Commerce staff for producing a timely, thoughtfully-considered set of legislative proposals.
We also look forward to providing input and additional commentary as these proposals are refined and strengthened over the coming months. Ensuring that legislation is addressing the most pressing issues facing Federal agencies, drug and device manufacturers, academic researchers, and – most importantly – the patients in need of improved treatments should be a common animating goal for all stakeholder groups working to bolster biomedical innovation. Doing so efficiently and without undue burden on a biomedical innovation ecosystem already subject to stressors and constraints will be an important part of moving the discussion forward.
The draft legislation released today takes steps to address some of the challenges inherent in drug and device development in a number of key areas:
It proposes pathways for expediting clinical development and FDA regulatory review while maintaining standards of safety and efficacy. Whether through establishing a broader push toward using novel statistical methods and trial designs during clinical development, giving the Agency the ability to approve the most promising new drugs based on early clinical evidence, creating new pathways for approving game-changing medical devices and diagnostics, or streamlining the data required to expand the number of FDA-approved uses for a drug already on the market, the draft legislation provides industry and FDA greater flexibility for quickly and collaboratively working together to move innovative products from bench to bedside.
It provides multi-stakeholder opportunities for developing new tools and methodologies to support research. The process by which drugs and devices are developed – early-stage scientific discovery, clinical trials, and regulatory review – is highly dependent on accurate, actionable, scientifically-validated ways of measuring a product’s clinical impact. This may be through tracking a drug’s effect on biomarkers like genetic mutations, or the amount of virus contained in a patient’s bloodstream; finding effective ways of measuring outcomes that matter to patients but are not at first easily quantified, such as pain or changes to quality of life; or developing surrogate clinical endpoints that can allow for faster development and approval of a promising treatment. This draft legislation tasks both FDA and external stakeholders with developing and qualifying such tools for use through updated guidance documents, detailed evidentiary and data standards, and, in some areas, dedicated public-private partnerships.
It supports comprehensive development and use of clinical and outcomes data throughout the full product life-cycle. We live in an age of data, and health care is no different. Data and evidence from basic scientific research and randomized clinical trials, real world data on outcomes and patient use from Electronic Health Records and payer claims, and evidence that accrues during post-market safety surveillance or a patient’s participation in registry studies all contribute pieces to a much larger health care puzzle – pieces that need to fit together and be rigorous enough for use in further research and development. The proposed legislative language outlines potential steps for moving toward a more systematic and standardized process for collecting and integrating such disparate data sources in the service of improving treatment.
It begins to address some structural barriers to coverage of new medical technologies. Quite often, FDA approval of a medical product or technology is not the final hurdle manufacturers must clear – they must also meet the evidentiary standards for coverage from private and public health insurers. Draft legislation aims to ease this process for a small number of priority areas, such as Medicare coverage for truly breakthrough medical devices or statutory improvements to the Centers for Medicare & Medicaid Services’ Coverage with Evidence Development policy, which allows for provisional Medicare coverage of a service or product while additional evidence on the effectiveness of the intervention in the Medicare population is gathered and analyzed. These are key steps toward improving the transition from development to patient use.
It doubles down on the importance of incorporating the patient voice during clinical development and regulatory review. The proposed legislation contains many opportunities for patients to be directly engaged with the drug development and regulatory review process through meetings and workshops, development of guidance documents, and public-private partnerships. It also contains specific proposals for the development and use of patient experience data in evaluating a medical product’s benefits and risks. These and other ongoing efforts to make the patient perspective central to biomedical innovation are among the most important areas where progress needs to be made.
While our team here at Brookings continues unpacking the complete raft of legislation and its likely impacts on biomedical innovation over the coming days and weeks, we plan to publish additional blog posts highlighting where language could be tightened or improved, where individual efforts could be given more time to come together so that they are achieving the desired aim, and – in the case of a concerted strategy for improving the post-market evidence development space – where more proposals for progress are needed. The process of refining these proposals and moving them toward passage will take time, additional expertise, and a continued push from leaders in Congress and executive agencies, and we look forward to doing our part by joining this important discussion.