Public comments


FasterCures Comments on PDUFA VI Commitments Letter

Divisions of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, Maryland 20852

RE: Federal Register Notice FDA-2016-N-1895
Prescription Drug User Fee Act; Public Meeting; Request for Comments


FasterCures, a center of the Milken Institute, is a non-profit, non-partisan action tank driven by a singular goal -- to save lives by speeding up and improving the medical research system. We thank you for the opportunity to submit these comments in response to Federal Register Notice FDA-2016-N-1895, Prescription Drug User Fee Act; Public Meeting; Request for Comments.

At FasterCures, we believe it is critical for stakeholders to develop more systematic ways of capturing and integrating patient perspectives into medical product development and regulatory decision-making. Since its inception in 2003, FasterCures has been working to put patients forward as partners in the biomedical research enterprise. To that end, through our Patients Count program, we are focused on expanding opportunities for patients’ perspectives to shape the processes by which new therapies are discovered, developed and delivered. It has been a privilege to work on behalf of patients throughout the stakeholder process for PDUFA VI. As part of that process, in November 2015, FasterCures submitted comments outlining our recommendations for how to meaningfully advance the science of patient input in PDUFA VI and are pleased to see many of those priorities reflected in the Proposed Commitment Letter.

Overall, FasterCures is very pleased with the recommendations put forward by FDA.  We believe the proposals will continue to enhance and improve the drug review program as a whole. Our comments focus on four enhancements outlined in the goals letter, that we believe are specifically relevant to enabling FDA to advance its efforts to deepen and expand engagement with patients, patient advocates and family caregivers.

1. Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making (pg 27-29)

We commend FDA for its renewed commitment to “enhance the incorporation of the patient’s voice in drug development and decision-making.” The Patient-Focused Drug Development initiative that was launched under PDUFA V has helped give more prominence to the patient voice in the drug development process. However, as the PDUFA VI goals letter acknowledges, more work is needed to advance the science of patient input and integrate the patient voice in a more meaningful and effective way. We believe the overall recommendations laid out in this section will help all stakeholders be better positioned to effectively integrate patient perspectives into drug development by providing needed clarity, tools, and resources. We highlight a few specific elements below.

  • To ensure that the goal outlined in this section of achieving more effective and meaningful integration of the patient voice is realized, it is critical that FDA have appropriate and qualified staff to execute the underlying work. Accordingly, we are pleased to see that the first activity outlined in this section involves a commitment to “strengthen the staff capacity” to develop and use patient-focused methods. The goals letter goes on to identify specific areas of subject matter expertise that will be targeted to fill these roles. We believe the goals letter identifies the right areas of subject matter expertise, but note the importance of ensuring that projects are staffed with the appropriate mix of such expertise.
  • We are pleased to see that these new staff will be assigned to review teams as “core members” with the responsibility to “engage patient stakeholders.” Such direct engagement is critical to ensure effective and early integration of patient perspectives and we are pleased to see this commitment to better understand and learn from patients.  We encourage FDA to communicate regularly with stakeholders about how these activities are being integrated into the review process. The patient community looks forward to engaging with FDA on this initiative to help maximize the value of patient engagement in the review process.
  • We believe each of the four guidance documents proposed in this section, explores a critical question that must be addressed to meaningfully advance the science of patient input. We applaud FDA for requiring public engagement around each guidance development as it is important to ensure that experts – including patients themselves – have an opportunity to provide input. We respect and appreciate the intense amount of effort, time, and resources that FDA must devote to developing each one of these guidance documents. However, we are concerned that the extended guidance development timelines and phased guidance plans laid out in the goals letter could delay industry and patient stakeholders from advancing some of these methodological tools and measurements while they wait for guidance from FDA. We encourage FDA to explore whether there is an interim step that can provide clarity for the community as a whole, to ensure we don’t lose valuable ground on advancing these issues.  If resource constraints limit the ability to accelerate timelines, we recommend that the agency work actively with stakeholders to ensure that this work does not stall as the guidance process moves forward. FasterCures stands ready and willing to work with FDA and other organizations to advance these tools and methods.
  • We applaud FDA’s proposal to create a “repository of publically available tools on FDA’s website.” At FasterCures, we have compiled well over 90 existing science of patient input resources and hope this can be a useful resource for FDA and other stakeholders. We look forward to creatively exploring with FDA and other organizations how best to maximize our collective reach and output to ensure that stakeholders can learn from, and build upon existing tools and resources to advance the science of patient input. 

2. Improving FDA Hiring and Retention of Review Staff (pg 39-41)

To carry out the ambitious goals articulated in the PDUFA VI commitment letter, FDA must be able to hire and retain staff with appropriate skills and expertise. Commissioner Califf has cited strengthening the FDA workforce as one of his top priorities and we applaud both FDA and industry for allocating resources towards this critical component to the success of the drug review program. The commitment to dedicate user fees to the breakthrough therapy designation program, originally introduced as part of FDASIA, will also help address some of the staff and resource constraints the review divisions have faced implementing this program. The workforce analyses to be performed by the new unit within the Office of Medical Products and Tobacco will be essential for the agency and stakeholders to fully understand the competitive hiring landscape and then identify and overcome barriers to successful hiring and retention. While we recognize that the PDUFA commitments are focused on enhancements to the drug review program, we hope that this new unit will be equipped to take a broader view of workforce issues beyond CDER.

We also applaud the inclusion of a “Comprehensive and Continuous Assessment” of the hiring and retention program. Simply adding new employees will not guarantee a meaningful improvement or enhancement to the capacity of the agency to meet its programmatic goals. Ongoing monitoring is critical to ensure that the right people are hired (or retained) and integrated appropriately into the existing workforce and FDA structure. This will hopefully result in more sustainable and long-lasting improvements to bring stability to FDA’s workforce.

There is more that can be done outside of the boundaries of the PDUFA process to strengthen the agency’s workforce, but we are grateful to see this issue addressed so explicitly in the performance goals letter. We hope to have continuous and ongoing dialogue with stakeholders at FDA and beyond to explore and evaluate the outcomes of these enhancements to hiring and retention.

3. Enhancing Benefit-Risk Assessment in Regulatory Decision-Making (pg 29-30)

We applaud FDA’s commitment to further the agency’s implementation of structured benefit-risk assessment. Patient perspectives are critical to making informed benefit-risk assessments and the proposed enhancements and guidance documents will further the effective integration of those perspectives. In particular, we are pleased to see a specific recognition that the benefit-risk framework be applied throughout the human drug lifecycle and not just at the time of approval. We commend FDA’s focus on identifying appropriate approaches to communicate to the public FDA’s thinking on a product’s benefit-risk assessment.  Too often, the public – including patients – are left out of these discussions with no way of evaluating whether or how patient perspective information impacted regulatory decision-making.

4. Enhancing Use of Real World Evidence for Use in Regulatory Decision-Making (pg 26-27)

We applaud FDA’s commitment to explore how real world evidence (“RWE”) can be more effectively incorporated into regulatory decision-making. Real world evidence is an area with huge potential to enhance the patient experience. There are however, many challenges associated with collecting and analyzing RWE and it is critical for FDA to engage with external experts as they explore these challenges. We commend FDA for committing to hold at least one public workshop bringing together patients and other key stakeholders for input on how to best move forward. We think it is also important for FDA to collaborate with other government agencies (e.g. ONC, CMS, etc) where appropriate, to share learnings and discuss what systems are needed to collect RWE. FasterCures looks forward to sharing outputs of its own work in this area in the coming months, as we engage with patients and other stakeholders to build resources to help patients understand how their health related data may be used as part of Real World Evidence decision-making.

We thank FDA and industry for the tremendous time and effort put forward to develop this comprehensive and thoughtful commitment letter. We are grateful for the opportunity to submit these comments, and to participate in the formal stakeholder process throughout the course of the PDUFA VI negotiations. The PDUFA VI commitment letter contains ambitious and laudable goals and FasterCures is committed to being a ready, reliable, and informed partner with the agency to help realize the promise of greater patient participation in regulatory decision-making as a means to improve public health.  We welcome the opportunity to explore these comments in greater detail with agency staff.



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Margaret Anderson
Executive Director

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