BIO Calls for Increased Emphasis on Patient Perspectives by Biopharma, FDA

Regulatory NewsRegulatory News | 17 June 2015 |  By 

Biopharmaceutical companies should seek out and incorporate the perspective of patients earlier on in the drug development process, a new white paper from the Biotechnology Industry Organization (BIO) argues.


The call comes in the midst of a sustained shift by both companies and regulators to incorporate the views of patients into the regulatory process. Starting in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), FDA has begun to meet with more than a dozen patient groups in the hopes of better understanding the diseases afflicting them and the risks of a drug they would be willing to tolerate in return for specific benefits.

The hope, regulators and companies have said, is that regulators can tailor approvals to narrower groups of patients who might be sicker, and therefore more desperate to access cutting-edge therapies that otherwise would not be approvable.

For more on FDA's patient centered drug development efforts, please see Focus' tracker here.

BIO's Whitepaper

But as BIO's new white paper, A Lifecycle Approach To FDA's Structured Benefit-Risk  Assessment Framework, explains, FDA isn't the only entity that should be embracing patients into the fold.

Companies, too, need to do more to incorporate patients into a structured benefit-risk (sBR) framework to reflect the views of patients early on in the product development process—not just at its conclusion.

To do so, BIO recommends borrowing FDA's established sBR framework, which is now used to evaluate all New Molecular Entities (NMEs) and as of 2017 will be used to review all new drugs.

FDA's Structured Benefit-Risk Framework
Decision FactorEvidence and UncertaintiesConclusions and Reasons
Analysis of Condition  
Current Treatment Options  
Risk Management  
Benefit-Risk Summary Assessment

"Much of the information available in the Investigator's Brochure should be directly transferable to the sB/R framework and thus could serve as an easily referenced foundation for the evolution of all downstream documents that rely on this information during investigational product development," BIO wrote.

Indeed, several pharmaceutical companies "have already begun proactively completing the sBR framework and submitting it to FDA as part of the NDA/BLA submission," the group explained.

BIO's white paper goes on to list specific steps companies should follow during the development lifecycle, including pre-Investigational New Drug Application (IND), clinical development and regulatory submission. For example, companies are advised to begin discussions with patient groups even before they begin testing "to engage in dialogue around the burden of the condition and the current armamentarium of treatment options."

"Systematic and early use of FDA's structured benefit-risk framework can be a powerful tool to achieve common alignment among sponsors, patients, and FDA on the benefit-risk considerations underlying an FDA approval decision," BIO wrote. "Rather than being used just to communicate FDA's decision in later review stages, the framework should be viewed as an iterative, evolving document that can serve as a decision-support tool to help guide benefit-risk discussions across a product's lifecycle."

Need for FDA Clarification

The paper also describes a lack of guidance on the part of FDA, which the paper argues could do more to help companies incorporate patient preference information.

"While FDA has expressed support for this [sBR] practice, it is currently unclear whether FDA will commit to reviewing a sponsor-proposed sBR framework or discussing it with the sponsor during review."

Another problem, BIO said, was that FDA currently has provided "little formal guidance" on the content, format or use of this benefit-risk information when it is included in regulatory submissions. "In some instances, FDA has provided no feedback to sponsors about submissions that include the use of quantitative benefit-risk modeling methods, including formal studies of patient preferences."

"The lack of information … provides opportunities to define a clear pathway to increased collaboration between sponsors and FDA to enable consistent, efficient and transparent benefit-risk assessment," BIO said. "Given that the benefit-risk assessment is complex and includes many factors, including patient input, sponsors and patients would benefit from clear guidelines on how the Agency will synthesize patient input, how the review division should use this information at the reviewer level, and how this would be systematically implemented across the Agency."

The information, BIO continued, "holds the potential to serve as a bridge between Patient Focused Drug Development and individual product approvals so that regulatory decisions would be informed by and grounded by patient preference data and other data-driven perspectives on benefit-risk."


BIO Whitepaper


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