Rare & Ready: A Genetic Condition Coalition
The Rare & Ready Coalition advocates for policies that ensure those living with rare or genetic conditions get the care they need, when they need it. The rare disease community deserves access to FDA-approved therapies as soon as they are available. We work together to mitigate state hurdles that limit access.
Timely Access to New Treatments
- Patients with rare conditions served by Medicaid must have timely access to new FDA-approved therapies.
- Medicaid fee-for-service (FFS) and Medicaid managed care organizations (MCOs) must have a specific timeframe and transparent processes for clinical review and setting coverage criteria for new drugs.
- Patient access in Medicaid FFS and Medicaid MCOs must not be limited by coverage criteria that are more restrictive than the FDA label.
- Exceptions for step therapy for rare disease therapies are needed.
Approving Telehealth Across State Lines
- Many states do not allow patients with rare conditions to use telehealth to access physician specialists who practice medicine in other states.
- With a limited number of medical experts in rare conditions, telehealth across state lines is critical to accurate diagnosis and good disease management.
Newborns Deserve a Timely Diagnosis
- Scientific breakthroughs have led to treatments for previously untreatable rare conditions.
- Delays in diagnosis have severe consequences. Nearly two-thirds of Americans living with a rare disease are children, and 30% of these children do not survive past their fifth birthday — lives that could have been saved through screening.
- Early diagnosis leads to timely interventions, improving quality of life, and survival rates.
Prescription Drug Affordability Boards (PDABs) Deter Access
- State efforts to create Prescription Drug Affordability Boards (PDABs), while intended to make drugs more affordable for health plans, can deter the development of and access to critical medical innovations.
- PDAB reimbursement caps could result in less rare disease research, fewer new treatments for patients, and restricted patient access to medicines.
Get Involved
Every voice matters! Share your experience and contact us to join our coalition. It costs nothing to join, and your level of involvement is completely up to you.
Follow us on (@rare_ready) to get weekly updates on rare disease policy issues.
