Public comments


Maureen Japha's oral comments to PDUFA Reauthorization Public Meeting

Good morning. My name is Maureen Japha and I am Legal Counsel to the Milken Institute and a member of the policy team at FasterCures. For those of you who may not be familiar with our organization, FasterCures is a nonprofit, nonpartisan DC-based center of the Milken Institute. We work to bring greater efficiency to the biomedical research process across diseases, and look to find ways to reduce the time it takes to move promising discoveries from lab to patients. Our mission is to save lives by speeding up and
improving the medical research system.

We work with all sectors of the medical research and development ecosystem – from patients to academia, to government and industry. Prior to joining FasterCures, I worked for a law firm where we regularly advised clients on how to navigate the regulatory landscape at FDA. In my new role at FasterCures, I have the opportunity to work with our colleagues in industry, academia, and government to explore how that landscape can be improved, and to identify ways to implement those changes in a productive way.

PDUFA V - Important Steps Forward
An area of increased focus for FasterCures and many patient advocacy organizations – some of whom have already touched on this today – centers around enhancing the science of patient input to more effectively incorporate the patient voice into all aspects of the drug development process.

It’s important to understand that efforts to enhance the science of patient input, should not be viewed as a feel good exercise to simply give patients an honorary voice in the process. Incorporating patient perspectives into regulatory decision-making has real utility for all stakeholders.

This utility was highlighted for me as I reviewed materials from a recent meeting of FDA’s Oncology Drugs Advisory Committee where the advisors were asked to discuss – not simply vote yes or no – on the key questions of whether study data supported a positive benefit/risk assessment for an experimental therapy to treat squamous non-small cell lung cancer. This deadly condition has seen no new treatments in over 15 years. As advisory committee members wrestled with data that showed a statistically significant, yet arguably small improvement in overall survival, it was striking to me how much this discussion could be enhanced with data showing the affected patient community’s range of minimum expected benefit and maximum threshold for harm. Such information can inform the dialogue, and ultimately the regulators’ decision as to whether to approve this therapy.

Traditionally, opportunities for patients to inform regulatory decisions regarding medical products have been limited to participation by single individuals who may not represent the range of perspectives and expectations of the broader patient population.

PDUFA V started to shift this traditional paradigm in significant and important ways, by establishing new approaches to more effectively integrate patient perspectives into the regulatory process. Specifically, FDASIA, the authorizing legislation for PDUFA V, included a directive for FDA to establish a structured benefit-risk framework to improve the transparency and clarity of FDA’s benefit-risk decisions. In 2013 FDA released a proposed 5-year implementation plan, and has begun piloting that structured benefit-risk framework. In addition, as part of PDUFA V, FDA committed to host at least 20 patient-focused drug development meetings. As discussed here today, each meeting is focused on a different disease or condition, and is designed to give FDA reviewers a better understanding of the patient experience of the disease, and patient perspective on current treatment options.

The introduction of the structured benefit-risk assessment framework and the launch of the PFDD initiative have been huge catalysts for change and I want to commend CDER and FDA for the advancements achieved under PDUFA V. These efforts sent a signal to the entire ecosystem that FDA was willing and ready to explore more patient-centered drug development and have helped advance the discussion in meaningful ways.

PDUFA VI and Opportunity for Advancing Science of Patient Input
Today, as we look toward the reauthorization of PDUFA VI, we have the opportunity to improve upon these advancements and integrate patient input into regulatory decision-making more effectively.

I want to highlight three actions that, if implemented, we at FasterCures believe can meaningfully advance the way patient input is incorporated into the drug development process.

  • First, patient perspectives should be integrated throughout the drug development pipeline, not just at the time of approval as contemplated by the current benefit-risk framework. Although the public hasn’t yet had an opportunity to see FDA’s use of the benefit-risk assessment framework in connection with a drug approval, our understanding is that it will be used primarily as a communications tool. This framework has the potential to be much more, and could have real value as a tool to guide decision-making throughout the drug development process. Patients would benefit from mechanisms that allow regulators and sponsors to explore and address patient perspectives much earlier in the pipeline.
  • Second, transparency is essential to advancing the science of patient input. For approved drugs, FDA should clearly explain how patient perspective data guided its regulatory decisions. For drugs that are not approved, it is important for stakeholders to understand how the benefit-risk framework influenced decisionmaking. Recognizing that sponsors have legitimate concerns about maintaining confidentiality, a compromise approach would be to release appropriately redacted versions of the structured-benefit risk assessment as part of the Advisory Committee process or after a complete response letter has issued.
  • Third, we need to develop appropriate, scalable, and sustainable analytical methods and practices that will more effectively integrate patient perspectives into all aspects of drug development and delivery – from pre-clinical through phase III trials and beyond. Although the PFDD meetings have been an important and critical step forward, they have real limitations. Specifically, it is unclear how FDA is using the information generated from PFDD meetings to inform regulatory decisions, or how patient perspectives regarding diseases or conditions that are not the subject of a PFDD meeting can be meaningfully collected and submitted to FDA. Patients, companies, academics, and regulators are poised to co-develop appropriate methods and practices, but guidance and direction from FDA about its evidence requirements will accelerate and enhance this process.

In addition to improving the use and implementation of the benefit-risk framework, PDUFA VI also presents an opportunity to re-visit the procedure for validating patient-reported outcomes, or “PROs”. At present, only one PRO instrument has been qualified by FDA. FDA’s efforts to develop a Compendium of Clinical Outcomes Assessment is a commendable and important step toward bringing more transparency to the process of qualifying PROs and other drug development tools. However, a more workable standard that facilitates approval of well-defined and reliable PROs can and should be established. Additional resources for SEALD and FDA’s review divisions could lead to enhanced communication between these groups, as well as increased capacity to coordinate effectively with sponsors regarding PROs.

In closing, it’s important to note that to realize the changes suggested today, FDA must have sufficient resources to effectively evaluate patient-driven information. This includes ensuring that reviewers have the training and tools required to properly assess and analyze patient perspectives. Public-private partnerships could be a useful mechanism to enhance this regulatory science, but we must adequately fund these initiatives to ensure they advance.

I’d like to thank FDA for the opportunity to participate in this meeting and present a patient-centered perspective of PDUFA and its programs. FasterCures looks forward to working with FDA and other stakeholders to enhance the existing benefit-risk framework, improve the science of patient input, and ensure FDA has the resources and tools it needs to effectively incorporate patient preferences into regulatory decision-making.

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