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Flatiron Health Comment on PDUFA VII Commitment Letter

September 28, 2021


On September 28, 2021, Flatiron Health provided remarks about the U.S. Food and Drug Administration's release of their proposed terms to the Prescription Drug User Fee Act (PDUFA) VII agreement. Brad Jordan, Flatiron's Senior Director of Regulatory Policy, applauded the FDA's recommendations to support real-word data and called for the Agency to accelerate new programs that have the ability to improve the quality and acceptability of real-world evidence. His full remarks follow here:

Good afternoon. My name is Brad Jordan. I am the Senior Director of Regulatory Policy at Flatiron Health, an independent affiliate of the Roche group. I am a Roche and Amgen shareholder. On behalf of Flatiron, I would like to thank you for the opportunity to comment today on the success of the PDUFA VII negotiations and to provide our recommendations for potential enhancements that will support the future use of real-world data (RWD) in the final agreement.

Flatiron Health is a healthtech company dedicated to helping cancer centers thrive and deliver better care for patients today and tomorrow. We translate patient experiences into real-world evidence (RWE) to improve treatment, inform policy and advance research.

RWD and RWE can complement evidence from clinical trials to evaluate the safety and effectiveness of drugs and devices. It can help fill critical evidence gaps by capturing the experiences of patients who are not typically included in clinical trials, such as people with rare conditions or whose cancers possess rare genetic mutations. There has been substantial growth in the use of RWE for regulatory purposes, and Flatiron has been proud to play a role in this growth and in pushing the frontiers of RWD sourcing, collection and analysis. W e expect the use of RWE to continue to expand as new sources of data become available, and methods to collect, analyze and derive insights from these data continue to evolve.

As outlined in the FDA PDUFA VII commitment letter, the Advancing RWE Program pilot for drug sponsors will establish programs that can improve the quality and acceptability of future RWE by sharing learnings from RWE submissions and providing targeted regulatory guidance. Flatiron appreciates FDA's efforts to advance the use of RWE, and we encourage the Agency to consider accelerating its efforts to establish these new programs. In doing so, these programs could more rapidly lead to new understandings, new use cases for RWE, and more rapid incorporation of RWE into regulatory decisions. Additionally, we strongly support FDA's efforts to establish a Digital Health Technology Framework as now, and in the future, a variety of data sources and technology platforms may be combined to generate evidence to support drug approvals, endpoint development and innovative trial designs. A comprehensive approach to technology modernization is needed and should include input from stakeholders from across the health and technology sectors.

As FDA considers public comments on PDUFA VII, Flatiron respectfully requests that the Agency establish a pathway to consult with data organizations such as ours, who are generators of RWE, to gain a greater understanding of methodological and technological considerations as the role of RWE is further advanced for use in regulatory decision making. We welcome how the use of RWD is woven into many of the goals outlined in the FDA PDUFA VII commitment letter. In addition to the broader goal of advancing the use of RWE in regulatory decision-making through the Advancing RWE Program, FDA's goal to optimize the Sentinel initiative and develop the DHT Framework will make use of RWD acquired through EHR systems or from other DHT-based measurements. Also, in the future, well-designed observational studies may be used to support post-approval requirements -- for example, with drugs approved through the Accelerated Approval Pathway.

RWD/E organizations, like Flatiron, are crucial to advancing the use of RWD in ways that can contribute to the PDUFA VII goals and eventually realize the full potential of RWE, as we play an active role in the collection and use of healthcare data. Routine engagement with these organizations will enhance FDA's insights into the feasibility of data submission, quality, management and analysis in the near term, as well as help the Agency respond to changes in technology over time. Through broad stakeholder engagement and the Agency's transparency, organizations like Flatiron and sponsors that utilize RWD, can better focus their development efforts to help ensure the success of FDA's modernization efforts and use of RWE to improve patient outcomes.

At Flatiron, we are constantly optimizing the collection, curation and use of RWE intended for regulatory decision making. It is therefore extremely important to have a mechanism for data organizations, like Flatiron, to engage directly with FDA outside the scope of a sponsor's drug development program. In these cases, participation of a data provider in meetings with the Agency may be at the discretion of the drug sponsor, and therefore the ability to extract learnings and input from FDA on optimizing the use of RWD/RWE may progress at a slower pace. We, therefore, respectfully request that FDA consider a framework by which these direct engagements with data organizations can occur.

Thank you again for this opportunity to contribute our input, and we look forward to additional opportunities for feedback in the PDUFA VII authorization process.