Life Science Compliance Update

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July 05, 2016

Bipartisan Policy Center Holds Briefing on Health Innovation

At a briefing hosted by the Bipartisan Policy Center (BPC), Senate Health, Education, Labor, and Pensions (HELP) Committee Chairman Lamar Alexander confirmed that negotiations over the Senate "Innovation Initiative" are still underway. Senator Alexander noted that the Committee is "in the midst of discussions" with House Speaker Paul Ryan and Senate Majority Leader Mitch McConnell on how to advance the package.

Specifically, Senator Alexander highlighted a conversation he had with Senator McConnell, where both senators agreed that the medical innovation package could possibly be "the most important legislation that Congress acts on this year."

In his remarks, Chairman Alexander described the Innovation Initiative as a proactive investment to help cut down healthcare spending, citing the forward-looking success of President George W. Bush's President's Emergency Plan for AIDS Relief (PEPFAR) in helping to control the HIV/AIDS epidemic abroad.

Senator Alexander highlighted the need for a smooth transition to electronic health records, as precision medicine will not work without using advanced health data. He does not believe that the federal government's investment in EHR has not been spent very well, and that Congressional leaders are working together to help ensure future efforts are more efficient.

Senator Alexander addressed the critics of the Innovation Initiative, stating that the Senate needs to take steps to "make sure the horror stories don't derail the success stories."

Senator Alexander also used his time to highlight other issues, including combating the opioid abuse epidemic and how to improve opportunities for Americans to be treated with regenerative medicine.

New BPC Report

In connection with the briefing, the BPC published a new report, "Using Real-World Evidence to Accelerate Safe and Effective Cures." The report provides a set of recommendations on improving the use of data in data development and strengthening the Food and Drug Administration's (FDA's) ability to oversee the progress.

The recommendations focus on different ways the current drug development process could be modernized and improved through the use of real-world evidence, as well as policy action suggestions to implement those suggestions.

Among the proposals are measures to: improve the medical product development process, increase regulatory clarity, use health information technology to improve health care, and increase investment in medical products to address unmet and public health needs.


The recommendations are as follows:

Recommendation I: Improve Regulatory Clarity Regarding Use of Real-World Evidence

Current evidence requirements date back to 1962, when amendments to the Federal Food, Drug and Cosmetic Act (FDCA) included provisions requiring manufacturers of drug products to establish a drug's effectiveness by substantial evidence and adequate and well-controlled investigations before it could be approved for marketing. Since that time, several regulations and guidances have been published.

Included in the policy ideas for this recommendation were suggestions that the FDA should: develop formal guidance regarding the use of real-world evidence to inform regulatory decision-making, including the circumstances under which real-world data could be used as well as the types of real-world data, methods, and the levels of evidence that would be acceptable for use in regulatory review and decision-making. The guidance should include, but not be limited to, new drug approvals (including approvals under expedited programs), label expansions, new indications, post-market commitments, and post-market study requirements; and engage representatives of regulated industry, patient and disease research organizations, academia, experts in the use of electronic data, experts in statistical methods, and experts in privacy policy in the development of the guidance.

Recommendation II: Improve Methods and Data Quality for the Generation and Use of Real-World Evidence

The scope and amount of real-world data potentially available is rapidly expanding, as are the methods to effectively use and interpret the data for regulatory decision-making purposes. The methods and interpretations that have traditionally been used may not be the most appropriate methods to use for understanding larger data sets, or data drawn from across a network of disparate databases. It is therefore imperative that regulatory agencies and others who rely on the data educate themselves about best practices in methods of use and interpretation of real-world evidence for decision-making purposes.

This recommendation included suggestions for both the FDA and HHS, including:

  1. the FDA should establish a program to promote sharing and evaluation of methods used in the evaluation of real-world evidence for regulatory decision-making. The FDA should invite a broad spectrum of researchers who are active in the generation and use of real-world evidence and methods development, as well as leaders who rely upon such real-world evidence—including regulators and payers—to participate in this program.
  2. The U.S. Department of Health and Human Services (HHS) should support research to improve methods for the use of real-world evidence, which take into account the much larger samples of electronic data now available and enable high-throughput methods that produce accurate and well-calibrated inferences that quantify levels of uncertainty more accurately. Such research should focus on issues that include, but are not limited to, mitigating bias, obtaining solutions to better refine outcomes definitions, understanding implications to analyses for integrating observational data across a number of disparate sources, and understanding the contributions of real-world evidence to causal reasoning.

Recommendation III: Improve Policies for Information Sharing to Support Clinical Research

Under current law, in order to conduct one real-world study across multiple health care systems, multiple institutional review board (IRB) approvals are required. Given the differences in how IRBs view their remit and what constitutes a clinical trial, a unique and individualized approach is often needed to seek approval from each, which can lead to delays in trial execution and increased costs.

In light of that, two suggestions were made to Congress, including the idea that Congress should require the HHS Secretary—through the OHRP and the FDA—to issue regulations and guidance to facilitate the broader use of centralized IRBs within 36 months, by clarifying the roles of IRBs in multi-site studies and the risks and benefits to human subjects, standardizing informed consent, and incorporating community values through the use of local IRBs while continuing to use central IRBs.

Recommendation IV: Explore New Adaptive Pathways to Modernize Drug Development and Support a New Era of Personalized Medicine

As medicine continues to become more personalized and drugs become targeted for smaller populations, traditional, large-scale RCTs will become increasingly less feasible and additional approaches will be needed to assure safety and efficacy and protect the public's health. Rapid advances in technology and personalized medicine will allow for more-close monitoring to be easier, cost effective, and accurate. To advance the exploration of a new, more flexible adaptive approach to drug approval, several steps must be taken.

Included in those steps are the following suggestions:

  1. The FDA should develop a new program to develop and test a new adaptive pathway approach to expand the capacity for drug development that has the following key attributes: iterative phases of development, beginning with initial marketing authorization to a restricted patient population, then expanding to wider populations based on risk-benefit ratios; gathering evidence through close-monitoring and other real-world evidence, to supplement RCTs; and early involvement of stakeholders who have a role in determining patient access to the drug, including industry, payers, regulators, clinicians, and patients.
  2. The FDA's new program to develop and test a new adaptive pathway approach for drug development should include the following elements: qualifying criteria for the program, which will determine which types of drugs at what stages could be considered for the adaptive pathway approach; types and levels of evidence required for initial approval and expansion, including evidence generated from close-monitoring, other real-world evidence, and randomized controlled trials, as appropriate; methods for early involvement of patients, clinicians, payers, industry, and regulators; and methods for assuring market removal or label modification of products when follow-up studies and monitoring are not completed or when an unfavorable risk-benefit ratio for certain populations is demonstrated.


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