We appreciate the opportunity to provide comments on EEOC’s FY 2022-2026 Draft Strategic Plan and look forward to working together to end the discriminatory practices of employer-sponsored health plans, ensuring that rare disease patients have access to the treatments and therapies they desperately need.

It is vital that EEOC exert its proper authority within its jurisdiction to prevent employers from intentionally or unintentionally discriminating against rare disease patients based on their disabilities and, in so doing, preventing those patients from accessing the transformative treatments that have become available through decades of healthcare innovation.

image of RDCC Letter - EEOC Draft FY22-26 Strategic Plan for Public Comment

When the pandemic began in March 2020, the scientific community reacted with a dramatic increase in COVID-19 clinical trials, but progress in other therapeutic areas stalled due to safety concerns. The new safety and logistical challenges added by the pandemic resulted in increased costs, delays, and uncertainty in trial initiation and completion for rare disease drug manufacturers. Rare disease patients have conditions that make them more susceptible to and higher risk for COVID-19, making it especially difficult for this population to participate in clinical trials.   

By October 2020, an estimated 80 percent of non-COVID trials were stopped or delayed during the pandemic. Over 700 trials experienced disruptions since the beginning of the pandemic, with over 250 suspending enrollment due to the pandemic. In May 2020, NIH Director, Dr. Francis Collins estimated that $10 billion of research outside of COVID-19 had been lost. Given the tumultuous two years that have followed, the amount of lost research has continued to multiply.

Many of the companies investing in rare disease research are small biotechnology companies that face particularly stark financial challenges, leaving them with exceedingly difficult choices on whether to resume their trials or stay in business altogether. Much of the critically necessary rare disease clinical research remains stalled, while millions of patients are waiting for breakthrough treatments and cures for the diseases that threaten their lives.

The RDCC is grateful for the leadership of Representative Josh Gottheimer (D-NJ) and Representative Don Bacon (R-Neb) to co-sponsor The Orphan Drug COVID-19 Mitigation Act and push for its inclusion in the next available legislative vehicle. Learn more on this important issue which will help to bring hope and needed cures to the at least 95 percent of rare diseases currently lacking an FDA-approved treatment in this letter.

Click here to download this overview.

The Rare Disease Company Coalition (RDCC) submitted comments on S. 4348, the Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act of 2022. In a follow-up letter to the House Energy & Commerce (E&C) and Senate Health, Education, Labor, and Pensions (HELP) Committees, the RDCC writes to commend the work done to date while strongly encouraging the timely reauthorization of the user fees to ensure continuity of critical funding for the Food and Drug Administration. Timely action will avert a delay in the rare disease drug review process.

Please see here for the RDCC’s letter.

Newborn screening is a vital and proven public health program that screens approximately four million U.S. newborns each year. In the first 24 to 48 hours of a baby’s life, a small blood sample is taken to detect serious, often fatal, genetic conditions that can be treated if diagnosed early. We believe equitable access to newborn screening can ensure that all babies have the best chance for a healthy life. Join us in working towards a modern newborn screening system that provides equal access to a timely diagnosis for all babies.

Learn more in this educational resource.

RDCC submitted comments to Board Members of the Colorado Prescription Drug Affordability Review Board (PDAP) concerning the impact a proposed rule could have on advancing research and development for new and innovative rare disease therapies. The Coalition asserted that the draft proposed rule as written could “in the long run harm more patients than it helps.”

In this letter, the RDCC strongly urges the PDAB to consider the unique circumstances of rare disease patients and therapies as it crafts its policy proposals, and cautions against punitive measures that would have an outsized impact on rare diseases. The impact of misguided policy proposals could stall future investment and development of rare disease treatments that would further disadvantage rare disease patients.

A “one-size-fits-all” approach to drug pricing and value assessment frameworks undermines development of therapies for people – very often children – with rare diseases. As rare disease companies, we champion policies that help people with high unmet needs by developing innovative cutting-edge medicines and working with federal and state policymakers to address access and affordability.

Learn more about the importance of restoring equity and patient focus when valuing rare disease treatments in this overview.

The Orphan Drug Tax Credit is a vital lifeline for rare disease patients and should be protected to preserve hope for the rare disease community. Given that approximately 95% of rare diseases of rare diseases lack an FDA-approved drug, any proposal to reduce the ODTC would have a devastating impact on orphan drug development in the U.S. and on millions of Americans living with a rare disease. We must preserve innovation and not lose the gains that have been made since the passage of the ODA.

Learn more about the Orphan Drug Tax Credit and its role in rare disease treatment development in this overview. 

FDA’s accelerated approval pathway recognizes that a “one-size-fits-all” traditional model cannot always work – especially for serious and unique rare diseases with very small population sizes that progress slowly and variably from patient to patient. Therapies approved through the accelerated approval pathway are subject to the same stringent, evidence-based clinical review and approval standards as the traditional FDA approval process, as well as to post-approval confirmatory trials to verify clinical benefit. 

While less than 10% of all accelerated approvals in its 30-year history have been used for nononcology rare diseases, today’s increased understanding of rare disease and advances in targeted drug development are making it possible to realize the promise of accelerated approval for rare diseases where current treatments don’t yet exist.

As policymakers consider policy proposals that impact the trajectory of rare disease treatment development, we advocate for an approach that considers the unique circumstances of rare diseases and caution against punitive measures that could chill future investment and development of rare disease treatments.

Click here to download this overview on the benefits of the accelerated approval pathway for rare diseases.

Building on our letter submitted to the Oregon Health Authority (OHA) on the proposed renewal and amendment of its Section 1115 waiver, RDCC submitted comments to the Department of Health and Human Services Secretary Becerra and Centers for Medicare and Medicaid Services (CMS) Administrator Brooks-LaSure on the OHA Section 1115 Demonstration Waiver. In this letter, the RDCC expresses concern with the potential impact that waiving certain provisions of Section 1927 of the Social Security Act would have on the discovery and development of new medicines, particularly on treatments and cures for patients with rare diseases who currently have limited or no treatment options.

Limiting Medicaid coverage for viable treatments would have a devastating impact on patients benefiting from this innovation and create a greater healthy equity divide, all while resulting in limited budget savings. This proposal is the wrong solution for patients.