Public Comments

02/08/2016

FasterCures Comments on Senate Innovation Agenda

The Honorable Lamar Alexander
Chairman
Committee on Health, Education, Labor, & Pensions
United States Senate
Washington, DC  20510

The Honorable Patty Murray   
Ranking Member
Committee on Health, Education, Labor, & Pensions
United States Senate
Washington, DC  20510

Dear Chairman Alexander and Ranking Member Murray, 

FasterCures’ mission is tightly aligned with the goal of the committee’s bipartisan biomedical innovation initiative, to accelerate the pace of cures. FasterCures, a center of the Milken Institute, is a nonprofit, non-partisan action tank driven by a singular goal: to save lives by speeding up and improving the medical research system. We have appreciated the opportunity to work with you and your staff on these vitally important issues and would like to provide additional comments on the legislative agenda that the committee announced on Jan. 19, 2016. 

FasterCures will focus our comments on two topics:

  • Expanding opportunities for patients and patient perspective data to inform drug development and regulatory decisions.
  • Ensuring that the U.S. Food and Drug Administration (FDA) and National Institutes of Health (NIH) have the tools, people, and resources needed to advance medical innovation and deliver new cures.

Expanding opportunities for patients and patient perspective data to inform drug development and regulatory decisions 

The current set of bills under consideration does not directly address patient engagement in regulatory decision-making. Existing patient-focused drug development programs at FDA focus on the very last stage of regulatory decision-making, many years into a product’s development program. Ongoing user-fee negotiations between FDA and industry are exploring next steps for patient engagement in the regulatory process. Consistent with section 1137 of the Food and Drug Administration Safety and Innovation Act, FasterCures strongly advocates that patient input be utilized across the full spectrum of regulatory activity, from early-stage clinical programs to post-market surveillance.

Specific comments for consideration during Senate deliberations:

  • The health information technology (HIT) discussion draft (released Jan. 20, 2016) contains many important reforms with respect to empowering patients:
    • Section 6 seeks to incentivize interoperability of HIT products with registries (i.e., data repositories used in medical research and development). This is a worthy goal; however, as written, the incentive appears to apply only to “clinician-led” registries. Registries organized by patients and related organizations can make equally valid contributions to research,[1] and FasterCures recommends that they be included in this provision. To be clear, we strongly support high standards for technical interoperability and data quality, regardless of the person or organization managing the registry or other HIT product. FasterCures will release a report on patient registries next week, which we will make available for background.
  • Providing FDA with a limited waiver from the Paperwork Reduction Act when seeking input from individual patients and patient organizations would enable the agency to engage more effectively with patients at the speed and frequency required to inform regulatory decision-making.
    • Concern has been expressed about the breadth of the Paperwork Reduction Act waiver provided by H.R. 6 section 2284 and the impact the waiver could have on communications with the regulated industry. To address this issue, we recommend that final legislation narrow the scope to focus on patients and patient organizations.
  • H.R. 6 section 2001 defines a new type of “patient experience data” that can supplement the data submitted to FDA to facilitate or enhance the agency’s benefit-risk assessment.
    • To reduce potential confusion with the Centers for Medicare and Medicaid Services’ use of the term “patient experience data” (for a wholly different type of data than that envisioned by section 2001), FasterCures proposes using the term “patient perspective data” in its place.
    • FasterCures recommends the definition for these data include burden of disease, structured approaches to gathering patient preference data, and information on patients’ minimum expectation of benefits and maximum tolerable harms or risks that patients might be willing to accept. The Prescription Drug User Fee Act (PDUFA) negotiation meeting minutes reflect a similarly expansive view of the types of patient perspective data that can inform drug development and regulatory review. As part of the PDUFA stakeholder engagement process, we have submitted formal comments to FDA that go into more detail on this issue.[2]

Ensuring that FDA has the people and resources needed to advance medical innovation and deliver new cures

The current set of bills being considered by the committee does not directly address FDA resource and workforce issues. FDA needs strong, sustained appropriations, and the $132 million (5 percent) increase in FY2016 was an important step that FasterCures would like to see continue. However, given the pressures to meet performance goals, agency leaders must make tough choices allocating limited people and funds among product reviews and broader initiatives, such as advancing regulatory science and developing guidance documents that fall outside of the user-fee agreements. There has been a growing drumbeat of concern – starting with the FDA Science Board’s “Science and Mission at Risk” report in 2007 – over the agency’s persistent challenges to building and maintaining its workforce of highly trained scientists, clinicians, and engineers. Even with additional funding (through appropriations or user fees), FDA will need new tools to maintain a workforce that is able to most effectively accomplish the agency’s life-saving work.

Specific comments for consideration during Senate deliberations:

  • Establishing new, flexible hiring authorities and salary caps tailored to the agency’s needs would enable FDA to better compete with industry in the extremely small pool of highly skilled talent. FasterCures supports the hiring authority reforms in H.R. 6:
    • H.R. 6 section 2281 would reform the existing Senior Biomedical Research Service to widen the scope of eligible candidates, remove the hiring limit, and boost the salary cap.
    • H.R. 6 section 2285 would grant three FDA centers (Center for Drug and Evaluation Research, Center for Biologics and Evaluation Research, and Center for Devices and Radiological Health) a new, flexible direct-hire authority with an increased salary cap.
  • Enhancing the ability of FDA (and NIH) staff to travel to scientific conferences and technical training is mission-critical to maintain staff fluency in cutting-edge science.
    • Travel was curtailed in 2012 by Office of Management and Budget memorandum M-12-12 mandating 30 percent cuts to travel budgets in all federal agencies. This memorandum could be rescinded or revised to explicitly include FDA (and NIH) staff travel to scientific conferences and technical training in the list of mission-critical exemptions to the travel restrictions.
    • H.R. 6 includes a “Sense of Congress” that participation in or sponsorship of scientific meetings is mission-critical for FDA (sec. 2282) and NIH (sec. 1025). 
  • As a regulatory agency, effective conflict-of-interest (COI) mitigation policies are essential for FDA. That said, we recommend exploring ways to streamline COI policies for FDA staff and members of advisory committees.
    • Slow, cumbersome COI review during recruitment can reduce the chances of a successful hire, especially for experienced, mid-career candidates coming from industry.
    • There may also be opportunities for COI mitigation through use of Qualified Blind Trusts or Qualified Diversified Trusts, as defined by the U.S. Office of Government Ethics.

We look forward to working with you on any additional pieces of legislation that will be introduced as part of the committee’s innovation agenda. In particular, we are pleased that the committee is considering legislation to support the Precision Medicine Initiative and the National Cancer Moonshot as the NIH and FDA launch these exciting research enterprises.

We applaud your efforts to date on this important initiative and very much welcome the opportunity to discuss these comments and to provide additional input as the committee continues its work. If there is anything that we can do in terms of convening to discuss this, we are happy to help.

 

Sincerely,

MA sig

Margaret Anderson

Executive Director

FasterCures, a center of the Milken Institute

 

cc: Members, Senate HELP Committee 

 


[1] The Patient-Powered Research Networks are prime examples of how patient-led registries can contribute to the research enterprise: http://www.pcornet.org/patient-powered-research-networks/

[2] Federal Register Notice FDA‐2010‐N‐0128. FasterCures comments: http://www.regulations.gov/#!documentDetail;D=FDA-2010-N-0128-0079 

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