The EveryLife Foundation Responds to Comments on Proposed Rules (FDA and CMS)

The EveryLife Foundation for Rare Diseases has responded to two requests for comments on proposed rules. The first is from the Food and Drug Administration (FDA) setting recommendations for diversity plans that aim to increase clinical trial diversity and the second, from Centers for Medicare and Medicaid Services (CMS) annual update of the Inpatient Payment Prospective System (IPPS) that sets payment policies for inpatient hospital-based care.  

Ensuring Inclusion and Representation for All in Clinical Trials

In April, the FDA released a draft guidance entitled Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials. The guidance provided recommendations to sponsors developing medical products on how to design a Race and Ethnicity Diversity Plan to recruit underrepresented racial and ethnic populations in the United States. We are pleased to see our Diversity, Equity, Inclusion, and Accessibility (DEIA) priorities reflected in these recommendations and are grateful for the opportunity to engage in this policy discussion. In our comments, we highlighted additional ways the FDA could enhance clinical trial diversity including:

• expanding their focus to ensure study teams are representative of the community they are serving;
• recommending the implementation of DEIA training programs for clinical research teams;
• focusing on recommendations specific to the conduct of decentralized clinical trials;
• clarifying and expanding the expectations for reimbursement of research participant’s related expenses;
• providing recommendations and training to sponsors on how to establish and build relationships with diverse community organizations;
• requiring more frequent reporting on progress and supporting greater sharing of best practices and learnings from efforts to increase clinical trial diversity; and
• expanding focus to encourage clinical trial participation of people from the LGBTQIA+ community, multiple geographic locations, diverse socioeconomic statuses, pregnant and lactating women, immigrants, and more.

Read the full FDA comments here.

ICD Codes and Strategies for Ensuring Adequate Payment for Rare Disease Therapies in the Hospital

The EveryLife Foundation also responded to a request for information that was embedded in the FY23 IPPS rule released by CMS last April.  While the rule is largely focused on technical payment policy and programs, CMS included a comment acknowledging the challenges in creating appropriate payment mechanisms for many rare disease related hospitalizations, especially those that include higher cost therapies that must be delivered while the patient is hospitalized. Notably, CMS acknowledged the negative implications of having few established ICD codes for rare diseases, a longtime focus for EveryLife Foundation policy efforts.  In response to CMS’ request for policy ideas, the EveryLife Foundation, with input from its Community Congress members, provided suggestions including:

• Redoubling efforts to support development of well-evidenced ICD-10 codes for rare diseases;
• Consider approaches that would enable a rare disease modifier or suffix that would indicate presence of a rare disease, triggering a payment adjustment to account for more intensive costs of treatment;
• Require hospitals to have plans in place to ensure they can promptly obtain any FDA-approved medications, even when it is not practical to have on-site access to every medication; and
• Provide clear and accessible ways for patients and families who are experiencing trouble accessing therapies in the hospital to report and resolve the issue.

Read the full CMS comments here.