The Cure CMD Advocacy Team has set these priorities to guide our legislative advocacy efforts.

  • Awareness of CMD and the unique needs of the CMD community
  • Access to affordable and equitable healthcare, employment, education, and travel
  • Funding for research that will lead to treatments and cures for CMD

We continue to watch, support, and add our voice to proposed legislation that will further our priorities and goals. We encourage you to contact your Members of Congress, share your CMD story, and explain how proposed legislation will help your family. If you aren't sure how to do this, check out these tips from Rare Disease Legislative Advocates (RDLA).

  • ABLE Age Adjustment Act: This bill will expand access to ABLE accounts by increasing the age by which an individual must have established a qualifying disability from 26 to 46.
  • Air Carrier Access Amendments Act: This bill will protect and improve the rights of people with disabilities in air travel.
  • SSI Savings Penalty Elimination Act: This bill increases resource limits used to determine eligibility for the Supplemental Security Income (SSI) program.
  • STAT Act: This bill, created with the input of the rare disease community, aims to improve the development of and access to therapies for the rare disease community.
  • BENEFIT Act: This bill will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the risk-benefit assessment. 
  • Prescription Drug User Fee Act: PDUFA was created by Congress in 1992 and authorizes FDA to collect fees from companies that produce certain human drug and biological products. Since the passage of PDUFA, user fees have played an important role in expediting the drug approval process. This act will expire if it is not reauthorized this year.
  • Telehealth: These services have expanded past couple years thanks to revised rules under the COVID-19 Public Health Emergency. Without new legislation, we return to pre-COVID rules for telehealth. There are several proposed bills at the federal level that will make telehealth a more permanent service. Many states have similar legislation in the works.
  • Project RDAC: The National Organization for Rare Disorders (NORD) launched Project RDAC to establish a Rare Disease Advisory Council (RDAC) in all 50 states. An RDAC is an advisory body that gives the rare community a stronger voice in state government. RDACs address the needs of rare disease patients and families by giving stakeholders an opportunity to raise awareness and make formal recommendations to state leaders on the most important issues they face.

What are we missing? We want to hear from you! Email Lani Knutson with your feedback.
July RDLA Monthly Webinar: Thursday, July 28th at 9 am PT/12 pm ET

Rare Across America: August 8 – 19, 2022

Global Gene's Patient Advocacy Summit, September 12-14 in San Diego
News of Note
Center for Drug Evaluation and Research (CDER) launched the Accelerating Rare disease Cures (ARC) Program in May 2022. This program's mission is to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.

The EveryLife Foundation for Rare Diseases is offering the Young Adult Representatives of RDLA (YARR) Leadership Academy, a series of on-line courses offered to a select group of young adults in the rare disease community (ages 18-30). Academy students will learn about the roles and opportunities for patient representation in policy-making, drug development, and the regulatory process and the steps it takes to get there.
Know someone who would like to join the Cure CMD Advocacy Team? Email us and we'll add them to the list!