Presidential Tech Council Urges Changes At FDA as Part of Rx Innovation Upgrade

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By Brian Broderick    

The United States should set a goal of doubling the output of innovative new medicines that meet critical public health needs over the next 10 to 15 years, while continuing to increase drug safety, a presidential advisory council  said in a report issued Sept. 25.

The report from the President's Council of Advisors on Science and Technology (PCAST) includes several recommendations for changes at the Food and Drug Administration, including improved drug evaluation and improving the agency's management.

"While basic biomedical sciences have seen stunning progress in past decades, challenges remain in translating those scientific advances into practical solutions," the White House said in a release announcing the PCAST report. The report's title is Propelling Innovation in Drug Discovery, Development, and Evaluation.

To support innovation and accelerate development of new therapies, the report makes a number of detailed recommendations aimed at bolstering the discovery and development of new therapeutic compounds. It calls for "optimizing" the processes used by FDA to evaluate the safety and efficacy of candidate drugs, enhancing the long-term monitoring of approved medicines, and improving the public's understanding about the benefits and risks of medicines.

PCAST said that all three major components of what it calls the "drug development ecosystem"--basic biomedical research in universities and research institutes, clinical research in hospitals, and drug discovery and development in the biopharmaceutical industry--are facing growing challenges as the time, complexity, and cost of developing drugs have gone up.

The report said that the rate of new drug applications submitted by industry to FDA, as well as new drug approvals, has remained relatively constant for 20 years. But in what it called an encouraging sign, PCAST noted that FDA "approved 35 new medicines in the past year--among the highest totals in the past decade."

The report said there are two critical needs related to drug discovery and development that must be addressed to advance innovation. First, scientists need better methodologies and tools for translating basic biological insights into validated therapeutic targets and leads. Second, the report said that pharmaceutical developers and regulators need to incorporate new efficiencies into clinical trials of candidate medicines.

To achieve some of the report's broader goals, PCAST recommended the creation of a public-private "Partnership to Accelerate Therapeutics," involving representatives from the biopharmaceutical industry, academia, physician societies, pharmacists, patient-focused research foundations and advocacy groups, providers and insurers, and the federal government. PCAST said this partnership "would help identify and plan collaborative actions to speed drug development while balancing competing stakeholder interests and minimizing duplication of efforts."

Recommendations for FDA.  

In the report, PCAST laid out several recommendations for change at FDA.

Among the recommendations is that FDA should make "full use of accelerated approval for all drugs meeting the statutory standard of addressing an unmet need for a serious or life-threatening disease, and demonstrating an impact on a clinical endpoint other than survival or irreversible morbidity, or on a surrogate endpoint, likely to predict clinical benefit." In addition, the report said, FDA should "fully enforce its requirement for post-approval confirmatory studies demonstrating that the drug indeed results in desired long-term clinical benefit."

Another PCAST recommendation is for a new pathway for initial approval of drugs shown to be safe and effective in a specific subgroup of patients. "This would be an optional pathway under which sponsors could propose early in the development process to study a drug for a narrow population," the report said, adding that such drugs would be approved under a designation of "Special Medical Use, signaling strongly to payors and prescribers the limited population that should be prescribed a drug."

PCAST said that FDA should run pilot projects to explore adaptive approval mechanisms to generate evidence across the life cycle of a drug from the pre-market through the post-market phase. "Legislation to create a formal adaptive pathway or model for such approval, however, would be premature at this time, and PCAST advises against it."

The council said FDA's tools for monitoring and communicating the risks and benefits of drugs need improvement. "FDA should strengthen capabilities for post-marketing surveillance, and the Congress should authorize line-item appropriations of $40M per year to expand the Sentinel System accordingly." Sentinel, a rapid-response electronic safety surveillance system for drugs and other medical products, was created as part of the FDA Amendments Act of 2007, and FDA officially launched the program in 2008.

Another recommendation from the council is to reform FDA's management practices. "The FDA should implement a range of reforms, including the use of pre-market review leaders to oversee each drug candidate application from its investigational stage through final marketing decision," the report said. Other reforms should include establishing a regulatory innovation program, overhauling the IT systems, and establishing a commissioner's Advisory Board for Medical Products to improve management and ensure consistent implementation of reforms, PCAST said.

Industry Response.  

In a Sept. 28 statement, Pharmaceutical Research and Manufacturers of America Senior Vice President Matthew Bennett noted that one of the recommendations in PCAST's report proposes a public-private partnership to support the continued evolution of the discovery process. "It is a promising concept but currently lacks sufficient details for a full evaluation or endorsement," Bennett said.

Bennett added that PhRMA "welcomes the opportunity to work with PCAST and the other members of the innovation ecosystem to further explore the issues raised in the report, including defining the specifics of a proposed new public-private partnership to support the evolution of the drug discovery process."

 By Brian Broderick  

The PCAST report can be viewed at or