Rare & Ready: A Genetic Condition Coalition

Rare & Ready: A Genetic Condition Coalition believes state policies must make sure that patients with rare or genetic conditions can get the care they need. These patients deserve access to new FDA-approved therapies as soon as they are available. We need to mitigate state Medicaid program hurdles that limit access.

Patients with Rare & Genetic Disorders Deserve Timely Access to New Treatments

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  • 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.

Patients with Rare & Genetic Disorders Deserve Timely Access to Care by Medical Experts

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  • 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.

Patients with Rare & Genetic Disorders Deserve Timely Access to Gene and Cell Therapies

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  • For many rare conditions, medical progress is moving from treating symptoms to actually addressing the underlying cause of the disorder through gene and cell therapies.
  • To ensure patient access, we need reimbursement models - like outcomes-based arrangements (OBAs) - that are equally as innovative as the therapies themselves.
Get Involved
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Every voice matters! Share your experience and contact us to join our coalition. Help us make progress in having an impact on rare disease policies. Attend our educational and collaborative webinars on a variety of topics with experts and like-minded individuals.

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Follow us on Twitter to get weekly updates on rare disease policy issues and coalition members, and to join a community of similarly impassioned people. Staying update-to-date is a big part of advocacy and, through @rare_ready on Twitter , you can do just that!