by Cynthia A. Bens, Senior Vice President, Public Policy, PMC

The U.S. Congress, the Food and Drug Administration (FDA), and the Centers for Medicare and Medicaid Services (CMS) recently moved to support funding for ongoing research in the field, clarify the regulatory pathway for digital health products, and collect feedback on a proposal to cover chimeric antigen receptor (CAR) T-cell therapies nationwide, respectively.

As explained below, each of these developments stands to improve the outlook for personalized medicine.

U.S. Congress’ FY 2019 Appropriations Bills Would Enhance Infrastructure for Innovation in Personalized Medicine Through NIH Budget Boost

On June 28, 2018, the U.S. Senate Appropriations Committee approved its FY 2019 spending bill. The bill would provide $39.1 billion for the National Institutes of Health (NIH), an increase of $2 billion above FY 2018. One week prior, on June 15, the U.S. House of Representatives Labor-Health and Human Services-Education Appropriations Subcommittee approved its own appropriations bill, which would provide $38.3 billion for NIH, an increase of $1.25 billion above FY 2018.

Both congressional proposals include significantly higher amounts than the $34.2 billion budget request for NIH put forward by the Trump Administration.

In addition to the increases every NIH Institute and Center would receive to advance fundamental scientific knowledge that will speed the development of personalized medicine therapies, diagnostics, and preventive interventions, the House and Senate bills make specific investments in the All of Us Program and implementation of the 21st Century Cures Act, both of which were supported in testimony PMC provided to Senate and House subcommittees earlier this year.

FDA Launch of Software Pre-Certification Model for Digital Health Tools May Help Facilitate Personalized Care Regimens

FDA released an updated working model for the Software Pre-Certification (Pre-Cert) Pilot Program on June 19, 2018. Dr. Adam Berger from FDA’s Personalized Medicine Staff within the Center for Devices and Radiological Health briefed PMC’s Public Policy Committee later that day on the changes to the model.

Software is increasingly used in health care to treat and diagnose disease, aid in clinical decision-making, and manage patient care. FDA has acknowledged that its traditional approach for the regulation of hardware-based medical devices is not well suited for software device functions that feature faster design and development timelines and require different types of validation. The Pre-Cert Program creates a voluntary pathway for digital health developers to demonstrate excellence based on software development, validation and maintenance practices and to provide faster patient access to these technologies. Version 0.2 of the working model clarifies the scope of the Pre-Cert Program, provides additional details on the program’s excellence appraisal framework, and further highlights elements of the program’s pathway determinations, streamlined pre-market review processes, and real-world performance.

FDA has an open docket for the pilot program. The agency is calling for public comment and is particularly interested in feedback from the patient and health care provider communities. Dr. Berger’s presentation to the PMC Policy Committee included asks for additional information that would be most helpful to FDA as it advances this novel approach to digital health regulation. FDA will continue developing and informally testing various components of this program through December of 2018.

CMS’ Updates to Current Coverage Policies and Consideration of National Coverage for CAR T-cell Therapies Signals Agency’s Willingness to Explore Innovative Approaches to Reimbursement for Personalized Medicines

CMS presented two opportunities this month for input on payment policy issues for CAR T-cell therapies. CMS’ FY 2019 Medicare Hospital Inpatient Prospective Payment System (IPPS) and Long Term Acute Care Hospital (LTCH) Prospective Payment System Proposed Rule included important considerations for reimbursing hospitals that administer these treatments. Updates for coverage of CAR T-cell therapies included in the proposed rule specifically related to the review of New Technology Add On Payments (NTAPs) for these treatments and the potential creation of a new Medicare Severity Diagnosis Related Group (MS-DRG) for procedures using CAR T-cell therapies.

CMS also opened a National Coverage Analysis (NCA) for CAR T-cell therapies for cancer at the request of United Health Group. The NCA is the first step in the path to developing a National Coverage Decision that would impose a uniform national coverage policy. If the process moves forward, CMS anticipates that it will release a proposed coverage decision by February 16, 2019, and a final decision by May 17, 2019.

PMC submitted comments on the IPPS Proposed Rule and comments on the NCA, both highlighting the impact CAR T-cell therapies have had in the treatment of some cancers with poor prognoses and the importance of thoughtful coverage policies to match these innovations. PMC also signed a group letter urging CMS to approach coverage of CAR T-cell therapy with vision and flexibility that can transfer to other transformative cell and gene therapies in the future.