Public Comments

02/13/2015

FasterCures Comments on the 21st Century Cures Discussion Document

The Honorable Fred Upton                                        The Honorable Diana DeGette

Energy and Commerce Committee                            Energy and Commerce Committee
U.S. House of Representatives                                   U.S. House of Representatives
2125 Rayburn House Office Building                          2368 Rayburn House Office Building
Washington, D.C.  20515                                            Washington, D.C.  20515

Sent via e-mail: cures@mail.house.gov

RE: Comments on the 21st Century Cures Discussion Document                  

 

Dear Chairman Upton and Representative DeGette,

Thank you for the opportunity to provide comments on the discussion document distributed by the Chairman on January 27, 2015 under the 21st Century Cures Initiative. In the days since its release, we have reached out to our network of thought-leaders from patient organizations, industry, academia, and healthcare institutions, including our senior fellows and members of our various advisory councils, to benefit from their insights about the proposals put forward in the draft.

FasterCures shares your goal of bringing efficiency to biomedical R&D by identifying and eliminating the roadblocks that slow progress, and paving a path of meaningful engagement between patients and every sector of the research enterprise. We are pleased that our view of patients as partners is aligned with the patient-focused theme of the first title and provisions throughout the draft document. As a leading voice in bringing together patients and participants across the research ecosystem, FasterCures welcomes the opportunity to review the discussion document and outline some overarching issues, as well as provide specific comments. Given our depth and breadth of experience in patient engagement in drug development, we are also developing proposed alternative legislative language for the Committee’s consideration on Title I Subtitle A, Patient Focused Drug Development, with a goal of sharing it with the Committee by the end of February.

We have organized our attached comments by title and subtitle of the draft document, focusing on areas where our perspective and content expertise might be most useful to the Committee. Our comments fall into the following general themes:

  • FasterCures appreciates the focus on patients and would like to ensure the inclusion of patient perspectives through all aspects of medical product development and regulatory decision-making;
  • New statutory responsibilities outlined in the draft will need to be accompanied by new resources so that these proposals do not divert scarce resources from existing core responsibilities; and,
  • Provisions of the discussion document create new commissions, advisory bodies, reports, studies, and guidance documents. We are concerned that some of these requirements may overlap or be duplicative of current efforts or existing documents. We have available as a resource, should it be helpful, a spreadsheet that compiles all these new requirements.

We would like to use this opportunity to renew our call for stable and robust funding for NIH and FDA, a crucial issue for stakeholders across the research enterprise. We are active members of United for Medical Research and the Alliance for A Stronger FDA and we will be working closely with them on appropriations.

In addition, we would like to renew our proposal to the Committee submitted November 12, 2014, to form a public-private-partnership focused on advancing the science of patient input. In addition to addressing the needs implicit in Title I, Subtitle A of the discussion draft, this initiative would provide a robust forum to address challenges arising in the initial planning phases of the Precision Medicine Initiative. A workshop convened this week by NIH on “Building a Precision Medicine Research Cohort” surfaced issues very similar to those raised in the process of advancing patient-focused drug development. We believe a unified effort to develop science-based methods to engage, consent, query, and retain the ongoing participation of patients as R&D partners would strengthen the work of our federal agencies, industry, and patient organizations, and would ultimately benefit public health. Our original proposal is appended to our comments on the discussion document. We would be pleased to work with the Committee and to draft language to develop this proposal further. 

We applaud your efforts to date on this important initiative and welcome the opportunity to discuss these comments and to provide additional input as the Committee continues its path toward legislative action on a bill that will generate broad support and, when enacted, will speed medical progress.

Sincerely,

Margaret Anderson
Executive Director


 

FasterCures specific comments:

TITLE I:

We commend the specific mention of addressing unmet medical need and patient-centered benefit-risk evaluation throughout Title I.

  • We support the development of a framework to develop methods and means to collect and apply patient perspectives in the assessments of benefits and risks. We endorse collaborative opportunities outlined in Subtitle A: Patient Focused Drug Development (pp. 8-15) to shape guidance on this topic. However, the specific language of this subtitle does not fully meet the intended objective of achieving patient focused drug development and could be strengthened in order to provide greater regulatory certainty about the collection, application, and integration of information about patient experiences, expectations, and tradeoffs. In addition to the recommendations provided below, FasterCures is developing a proposal for alternate legislative language that may better achieve what we understand to be the intended objective. These comments and our more detailed proposal are based on work through our Benefit-Risk Program and a one-day meeting of experts we convened last fall at our Benefit-Risk Boot Camp.
    • Sec. 1001 suggests that the structured assessment of benefit-risk informed by “patient experience data” will be utilized by the agency only for regulatory decision-making following a sponsor’s submission of a New Drug Application (NDA), a relatively late stage in the drug development process that follows the completion of multiple clinical trials. This application of “patient experience data” is too narrow, and applied too late in the approval process. Rigorously collected patient perspectives from representative populations have the potential to inform the entire drug development spectrum. Consistent with FDA’s repeated statements that “… the medical product review process could benefit from a more scientific, systematic, and expansive approach to obtaining input from patients who are experiencing a particular disease condition,” the proposal should reflect that patient perspectives can also inform:
      • the earliest steps of target identification and preferences for benefits and tolerances for harms;
      • testing of new agents in humans to evaluate safety;
      • clinical trial design, including the selection of endpoints, comparators, and exclusionary criteria, as well as to evaluate the burdens of clinical trial participation;
      • analysis of study data to shape further development steps; and
      • post-market review including ongoing safety surveillance, risk communications, and consideration of label changes.
    • The use of the term “patient experience data” is open to confusion with other, similar terms. For example, patient experience surveys developed by the Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) under direction from the Centers for Medicare and Medicaid Services (CMS) collect information about the patient’s experience of hospital care. In other settings the term “patient experience data” is used to refer to retrospective capture of patient experience through diaries and self-monitoring methods.
    • To ensure that the type of data covered by the process and guidance documents outlined here, both the term and definition used should encompass a broad range of information that could be beneficial to drug development and regulatory decision-making, including the methods outlined in sections (C) and (D) on page 12, lines 19-25, which are considered separate from patient experience data. We can help to clarify this section in our proposal.
    • Regardless of the final definition of “patient experience data,” we recommend expressly naming industry as one of the parties collecting such data in the definition of “patient experience data” (p. 10, beginning line 18) and as a participant in the workshops described (pp. 13-14). We believe that a multi-stakeholder process that includes FDA, patient organizations, clinicians, academic researchers, and industry would facilitate development, adoption, and refinement of standards and procedures for capturing patient perspectives and integrating them into medical product development.
    • The report required five years following enactment (p. 14) should not be limited to addressing the use and potential improvement of specific measures outlined in the provision. We believe it should be expanded to include an assessment of the agency’s overall progress toward patient-focused drug development.
    • Finally, FDA may require additional expertise from social scientists, health economists, and outcomes researchers familiar with this type of data in order to appropriately incorporate patient experience data (broadly defined) into medical product reviews and throughout all stages of regulatory decision-making. This notation relates to placeholder provisions in the draft document related to FDA staffing and means by which might be authorized to access and attract specialized expertise.
  • We commend inclusion of patient perspectives on benefit-risk as a factor in decision-making in Subtitle E, “Priority Review for Breakthrough Devices,” (pp. 72-81) and recommend that it also be included in Section F, “Accelerated Approval for Breakthrough Devices” (p. 81).
  • To facilitate greater understanding about expanded access programs maintained by sponsors of medical products with active development programs, we support the transparency requirements, including publicly named points of contact and information about decision-making timelines, outlined in Subtitle G, “Expanded Access” (pp. 82-83).
  • We support “SubtitleK, Cures Acceleration Network” to provide the director of the National Center for Accelerating Translational Science (NCATS) with more flexibility to fund projects consistent with the purpose of the Cures Acceleration Network (p. 99). We are also in favor of an increased emphasis on awarding grants and contracts by NCATS for drug repurposing as described in this subtitle (pp. 100-101).

TITLE II:

  • Subtitle A, “21st Century Cures Consortium” (pp. 131-139) proposes to create a new entity independent of the federal government to bring together stakeholders to foster collaboration, establish a strategic agenda, identify gaps and opportunities, facilitate interoperability, and to award grants/contracts to accelerate discovery and development of cures, treatments, and prevention. We believe further definition is needed to clarify eligibility for grantees/contractors as well as how private sector funds will be solicited, contributed, and restricted.
    • The independent organizational status and governance structure follow those of the Patient-Centered Outcomes Research Institute (PCORI). As such, we recommend that the learnings from PCORI be leveraged in terms of building a contract/grant-making entity from the ground up and the appropriate level of staff, infrastructure, and ongoing programmatic evaluation. “PCORI at Three Years,” published in the New England Journal of Medicine, is an introductory source of information. Further, efforts should be made to leverage effective policies created by existing private-public partnerships such as the Foundation for the NIH, Clinical Trials Transformation Initiative (CTTI), Critical Path Institute, and others. FasterCures’ analysis of 369 consortia, published in Science Translational Medicine, provides some excellent insights about ways to leverage the output of research-by-consortia, as does our Consortia-pedia report.
    • A narrowed mission focus would enhance rapid mobilization of active participants, resources, and success. We believe that the concept we proposed to the Committee in November for a public-private partnership to advance the science of patient input might provide a more focused agenda for such an effort. Our original proposal of November 2014 is appended here.
    • We also recommend including an explicit minimum requirement for patient representatives on the governing Board, as a matter of principle.
  • While we are supportive of the intended role and purpose of the proposed Medical Product Innovation Advisory Commission in Subtitle B (pp. 140-148), we are concerned about the amount of infrastructure support required to establish the commission under this model. MPIAC would review federal policies of NIH, FDA, CMS related to discovery-development-delivery of new medical products with the intent of identifying actions to speed the innovation cycle. Its agenda would be developed in consultation with Congress. However, the manner in which its recommendations might affect legislation, be utilized by the Administration, or impact the 21st Century Cures Consortium (proposed in Subtitle A) is unclear.
  • Subtitle F,“Building a 21st CenturyClinical Trial Data Sharing Framework” (pp. 162-168) proposes initiating an application process to award a contract to a “neutral third party” (unaffiliated with any clinical trials) able to provide funding from government or private sources to compile data from federally-sponsored clinical trials in standardized formats. The success – and viability – of this worthy aim will turn on the caliber of the contractor and the Department’s capacity to manage the contract in the absence of performance measures related to funding. In service of the objective for the contractor to serve as a neutral third party (p. 166, line 13), applicants should be required to disclose any interests they have with companies that could materially benefit from the applicant’s participation in building the network.
  • We support new authorities to expand the use of de-identified Medicare data to improve clinical outcomes as outlined later in Subtitle F, “Building a 21st Century Data Sharing Framework” (pp. 168-184) including providing access to this data by qualified researchers without regard to their institutional or commercial affiliation. The proposed change in the privacy standards to conform with HIPAA rather than the Common Rule will remove certain restrictive practices.
  • Subtitle G, “Utilizing Real-World Evidence” is complementary to the Patient-Focused Drug Development provisions in Title 1, Subtitle A and we encourage consultation with industry, academia, patient advocacy organizations, and disease research foundations to foster use of data from patient registries and observational studies (pp. 193-195). The definition of real-world evidence is sufficiently broad to anticipate the evolution of both the sources and types of data that have utility.
  • We support the expansion of the NIH Director’s authority under Subtitle L, “NIH-Federal Data Sharing” (pp. 206-207) to require grant recipients to share data and believe it could be more explicit to build on the 2003 rule setting an expectation for NIH grantees to share data.
  • The ability to “Access, Share and Use Health Data for Research Purposes” under Subtitle M (pp. 207-214) enhances patients’ opportunities to grant use of their data to HIPAA-covered entities for certain specified types of research and we support the intent underlying this provision.
  • Large cohort studies are at the center of several provisions in the discussion document, including Subtitle N, “21st Century Chronic Disease Initiative Act” (pp. 215-216), Title IV, Subtitle B, “Advancing Research for Neurological Diseases” (pp. 255-259), as well as with the President’s promising Precision Medicine Initiative proposed in the FY 2016 budget. We look forward to further details about the alignment of the Committee’s vision with the Administration’s vision for precision medicine, in text that would populate Title II,Subtitle Q, “Precision Medicine.” This appears to be an opportunity to bring all these cohort-based initiatives together.   
  • The aim of SubtitleP, “FosteringHigh-Risk/High-Reward Science” (p. 222) may be possible to advance with increased support utilizing existing NIH support mechanisms as was reported in 2014 with 85 awards totaling $141 million made through the NIH Common Fund using New Innovator, Transformative Research, and Early Independence Award programs.

TITLE III:

  • We support provisions in Subtitle A, “Clinical Research Modernization” (pp. 229-231) to encourage centralized institutional review boards and reduce duplicative effort for multi-site studies. We believe this will reduce administrative burdens and accelerate innovation.  We encourage the Committee to draw upon the expertise of entities including CTTI to accelerate ongoing multi-stakeholder efforts and avoid potentially confusing terminology or rules.
  • In Subtitle B, “Broader Application of Bayesian Statistics and Adaptive Trial Designs,” (pp. 232-235) we support prioritizing the issue of the final guidance from FDA in this area to provide greater regulatory certainty.

TITLE IV:

We support the strategic allocation of resources and performance evaluation and encourage the Committee to work with the Secretary and NIH and FDA leadership to enhance planning and accountability functions, as well as reduce administrative burdens.

  • We commend Section 4009 (p. 254) in Subtitle A, “National Institutes of Health” to allow NCATS to support phase IIb trials to advance promising therapies further in development.
  • We look forward to evaluating the forthcoming language for Subtitle E, “FDA Hiring, Travel and Training,” as we believe this will be crucially important to the success of many provisions outlined in the discussion document.
  • We commend the creation of opportunity for public comment in Subtitle H, “Local and National Coverage Decision Reforms” (p. 286). The author’s note to seek ways in which national and local coverage decisions can work better for the Administration and patients seeking coverage under Medicare gets to the heart of issues here.
  • Similarly, we welcome the measure in Subtitle P, “Medicare Pharmaceutical and Technology Ombudsman” to make CMS more accessible by the public, including industry (p. 322). 

TITLE V:

  • Subtitle D, “Medical Device Reforms” (p. 356) again reinforces that registry data be considered as valid scientific evidenceand defines a process to establish standards for it. To the extent possible under existing authorities, we recommend harmonizing processes to establish standards for these types of data and evidence for drugs and devices.

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