On March 24, 2022, the Rare Disease Legislative Advocates hosted its monthly webinar to provide updates on legislation and public policy that is relevant to the rare disease patient community. Speakers for this month’s webinar included:
- Marc Yale, International Pemphigus & Pemphigoid Foundation
- Jamie Sullivan, EveryLife Foundation for Rare Diseases
- Nisha Quasba, Faegre Drinker Consulting
- Baillie McGowan, EveryLife Foundation for Rare Diseases
- Shannon von Felden, EveryLife Foundation for Rare Diseases
Marc Yale provided some updates on a piece of legislation called the Access to Rare Indications Act of 2021 (H.R 6160). This legislation is designed to prevent patients from losing access to vital treatments by getting denied coverage from their insurance company. Some of the proposed changes in this bill include:
- Aligning Medicaid and Medicare coverage protocols for off-label use of a drug for rare disease treatment with the current policy used for deciding off-label drug coverage for cancer treatment.
- Implementing requirements for private insurers to provide denial appeals and reconsideration mechanisms for treatment coverage, as well as expedited formulary exception policies.
Jamie discussed the latest news regarding recent Congressional hearings which took place. A wide variety of bills were discussed, including the STAT Act, legislation of significant importance to the rare disease community. The discussion around the STAT Act was encouraging, with many legislators being in broad agreement about the bill’s provisions. She also recognized the achievements of the community’s advocacy efforts. The number of co-sponsors has been increasing rapidly. The Accelerated Approval pathway, a program that has been implemented for some years, was also under discussion so that it could be made more effective.
Nisha touched on the FY22 appropriations bill. The omnibus bill calls for an $11.3 billion increase in funding for HHS in comparison to last year. This includes $1 billion to establish the Advanced Research Project Agency for Health (ARPA-H), which falls under the HHS Office of the Secretary. The goal is to increase the pace of research progress for major diseases such as cancer, Alzheimer’s, ALS, and more. The NIH would also see a $2.25 billion in funding from last year. The FDA could see a $102 million increase.
Baillie gave an overview of 1115 waivers. These waivers allow states to alter their Medicaid programs from the federal standard and normally last five years. These can allow states to do several beneficial things, like:
- Expand access
- Provide behavioral and substance abuse services
- Reallocate funds to new, community level programs
However, in the state of Oregon, there is a 1115 waiver being proposed that would halt the coverage of treatments passed under Accelerated Approval that have limited evidence of efficacy. The justification of this is allegedly due to the high costs of the drugs that are passed under the protocol, but data shows that these therapies account for less than one percent of actual spending. If the waiver is implemented, it could seriously hinder treatment access for some rare disease patients in the state. There is concern that other states may try similar measures if it goes through.
Shannon talked about participation during Rare Disease Week and also announced a new program called Rare Advocacy Learning. This is a six week seminar series focused on local and state level rare disease advocacy. There are a limited number of slots for attendees, and applications must be submitted by April 1. Click here to learn more.
