FDA approves therapies for rare and genetic conditions
Your state Medicaid program may set eligibility criteria which make it more difficult to access the latest therapies for rare and genetic conditions
Patients with rare or genetic conditions may face significant delays before FDA-approved therapies are covered
What is the Rare & Ready Coalition?
The Rare & Ready Coalition ensures that patients with rare and genetic conditions get access to the care they need, when they need it. Rare & Ready welcomes EVERYONE to join us! Whether you are a patient, caregiver, healthcare provider or member of a patient advocacy group, your voice matters. It costs nothing to join, and your level of involvement is completely up to you.
Our Members
Current Advocacy Group Coalition Members
Our Priorities
Time to Treat
Many state Medicaid programs are developing detrimental strategies to limit access to newly approved rare disease therapies. Rare & Ready: A Genetic Condition Coalition supports four priorities to mitigate the programmatic hurdles to access put into place by Medicaid fee-for-service (FFS) and Medicaid managed care organizations (MCOs).
- PATIENTS WITH RARE CONDITIONS SERVED BY MEDICAID MUST HAVE TIMELY ACCESS TO NEW FDA-APPROVED "QUALIFYING RARE DISEASE THERAPIES"
- Approved by the Orphan Drug Designation; or
- Approved for the treatment of rare pediatric diseases; or
- Designated as a breakthrough therapy; or
- Designated as a regenerative medicine advanced therapy.
- MEDICAID FFS AND MEDICAID MCOs MUST HAVE A SPECIFIC TIMEFRAME AND TRANSPARENT PROCESSES FOR CLINICAL REVIEW AND SETTING COVERAGE CRITERIA FOR NEW DRUGS
- Within 60 days of FDA approval, Medicaid must conduct clinical review as well as development (and publishing) of coverage criteria for qualifying rare disease therapies.
- Manual reviews must be available to ensure coverage immediately after approval and during the 60-day window.
- PATIENT ACCESS IN MEDICAID FFS AND MEDICAID MCOs MUST NOT BE LIMITED BY COVERAGE CRITERIA THAT ARE MORE RESTRICTIVE THAN THE FDA LABEL
- Inclusion/exclusion criteria from clinical trials must not be permitted as a basis for coverage criteria.
- Coverage criteria must be consistent with the medically accepted indication.
- Coverage must not be more restrictive than the indications and usage section of the drug's label approved by the FDA.
- EXCEPTIONS TO STEP THERAPY FOR QUALIFYING RARE DISEASE THERAPIES ARE NEEDED
- A clear exceptions process to override a step therapy protocol imposed for certain drugs must be in place.
- Medical necessity of a drug must be determined by providers - not Medicaid.
- A response to an exceptions request must be provided within 24 hours for an urgent request and 72 hours for a non-urgent request.
Telehealth
- PATIENTS WITH RARE CONDITIONS NEED EXPERT MEDICAL CARE
- If you or your loved one is living with a rare condition, you know finding healthcare providers who really understand the disease is difficult.
- Often, good treatment is far from home and may even be in another state.
- TELEHEALTH IMPROVES PATIENT ACCESS TO CARE
- The COVID-19 pandemic ushered in state flexibilities with regard to telehealth, and we learned just how much virtual medical visits help patients - especially those with rare conditions - get the care they need.
- Laws that expanded the availability of telehealth should be extended past the pandemic to ensure these services continue to be covered.
- WHERE YOU LIVE SHOULD NOT BE AN IMPEDIMENT TO EXPERT CARE
- Some states make it hard for out-of-state providers to become licensed to provide care to in-state residents.
- Without a license to practice medicine in the state where the patient lives, a physician cannot provide care and consult (even virtually).
- Since physician licensure rules are not uniform, patient access to virtual physician visits across state lines varies dramatically across the country.
Expand Newborn Screening
- What is newborn screening?
- Newborn screening is a public health program that involves collecting a few drops of blood from a newborn’s heel to test for serious medical conditions that have effective, FDA-approved treatments.
- The number of conditions screened varies from state to state.
- Why Expand Newborn Screening?
- 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.
- The Urgency to Act
- We are in an era of remarkable medical progress with advanced and even curative treatments for genetic conditions.
- The current state-specific newborn screening system has not kept pace with medical advancements, constrained by a slow and fragmented expansion process—screening for fewer than 1% of rare diseases, with many states omitting conditions that have FDA-approved treatments.
- On average, a rare disease diagnosis takes more than six years and nearly 17 medical encounters after symptoms start, creating a significant economic burden—exceeding $517,000 per patient.
PRESCRIPTION DRUG AFFORFABILITY BOARDS (PDABs)
Some states legislators are setting up unelected boards called Prescriptions Drug Affordability Boards or PDABs to cap prescription drug reimbursement for certain health plans. PDABs may help insurers, but those savings are not seen by patients.
- PBABs DO NOT REDUCE COSTS TO PATIENTS
- While the intent is to contain costs, PDABs do not lower patient copayments, reduce premiums, create health system transparency, or increase access to care for rare patients.
- Rather, PDABs could restrict access to innovative therapies.
- OTHER POLICY SOLUTIONS CAN LOWER COSTS TO PATIENTS
- Co-pay accumulator bans can limit patients’ out-of-pocket costs.
- Reforms should address policies of health plans and pharmacy benefits managers (PBMs).
- Outcomes-based arrangements, also called value-based purchasing arrangements, can be adopted to lower payers’ risks and costs while facilitating patient access.
