Regulatory Focus™ > News Articles > New Report Calls for Overhaul of Regulatory Processes with Goal of Doubling Drug Approvals by 2027

New Report Calls for Overhaul of Regulatory Processes with Goal of Doubling Drug Approvals by 2027

Posted 27 September 2012 | By Alexander Gaffney, RAC 

A new report released by the President's Council of Advisors on Science and Technology (PCAST) has called for the US Food and Drug Administration (FDA) to approximately double the number of pharmaceutical products it approves each year by the end of 2027.

The report, Propelling Innovation in Drug Discovery, Development and Evaluation, comes on the heels of two reports released earlier in 2012 which called for, respectively, a more refined regulatory approach for nanotechnology products and a restructuring of the wireless spectrum to accommodate, among other things, medical devices.

Unlike the previous two reports, PCAST's drug discovery report focuses solely on the life sciences industry, noting that the slow pace of therapeutic development has created, "Serious stresses throughout the biomedical ecosystem."

The good news for FDA regulators: The group found the agency is largely operating within the expected confines of its resource allocations and legislative authority. Regardless, the group said a "wide range of issues" continue to plague the larger biomedical system. Among the most troubling of those issues, the report's authors explained, was that the pace of development has not kept up with the pace of advancements in biomedical research.

Two Essential Needs

The report concludes that the drug development ecosystem needs two essential developments for it to realize its full potential: better translational tools with which to validate therapeutic leads and targets and new and more efficient models for conducting clinical trials, which the report notes consumes approximately 40% of each drug's development budget.

The report's authors said both of these problems suffer from a lack of investment and energy, largely because they are difficult to solve, require the cooperation of numerous actors within the development ecosystem, and because the payoff for any one company is unlikely to be worth the investment costs.

To surmount this investment challenge, the report recommends the formation of a new public-private partnership it's calling the "Partnership to Accelerate Therapeutics (PAT)," a consortium between industry, academia, patient advocacy groups, researchers, insurers and government agencies aimed at identifying ways in which stakeholders could collectively tackle shared problems.

In addition, the report recommends that the US Department of Health and Human Services (DHHS) should study ways to incentivize important but financially unattractive medical challenges such as those outlined in the report.

Focus on Regulatory

Researchers also proposed a number of new regulatory considerations which they conceded might take both new funding and new legislative authorities, including FDA authority to limit off-label prescribing to ensure faster approvals to benefit narrow patient populations when broader questions about a drug's safety and efficacy are still unanswered. Stronger postmarketing surveillance, which FDA has recently taken steps to begin overhauling, could also stand to be strengthened, which could allow for faster recalls if a risky product turns out not to be safe for patients.

Another recommendation focused on something familiar to many regulatory professionals: the need for adequate clarity regarding which pathways are necessary to gain approval and what evidence is necessary to show a product is safe and effective. The proposed solution is likely just as familiar to regulatory professionals, though.

"The development of rapid, clear, and thorough guidance documents that reflect the consensus of the scientific community on new and emerging areas of scientific innovation could help address this need," the report explains. "To develop such guidances in a timely manner while reflecting high­level expertise, the FDA may need to more heavily rely upon the biomedical community to collaboratively suggest standards and pathways that the agency can then consider in developing guidance documents to clarify its policies and practices."

The report concluded by saying the authors are hopeful that their recommendations, taken as a whole, could result in the number of drugs ultimately approved by FDA-35 in 2011-could more than double in the next 10-15 years. "PCAST believes that such a goal is attainable over the next 10­15 years," they said, though they cautioned that achieving it will, "Require concerted efforts by stakeholders across multiple sectors."

The report's recommendations, they concluded, are meant to lay the groundwork for those structural efficiencies and improvements to take shape.

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