Did you know that many rare disease patients enrolled in Medicaid cannot access lifesaving and life improving Food and Drug Administration (“FDA”) approved therapies? This is a result of barriers designed to make it impossible for rare disease patients to benefit from medical innovation.  As organizations committed to advancing innovative technologies that can diagnose, treat, and potentially cure rare diseases, we also need to ensure  patients have access to these innovations. We are united in asking Congress to use 21st Century Cures to prohibit Medicaid plans from rationing access to rare disease therapies. Without access, innovation means nothing for patients.

States are claiming conflicting federal statutes give them authority to use step therapy protocols as part of prior authorization to deny access to medically necessary therapies.  Despite recognizing the significant risk that the forced use of alternative treatments poses to patient health outcomes and the resulting increased costs to the health care system, the Centers for Medicare & Medicaid Services (“CMS”) has not intervened.  CMS also in some instances has enabled rationing through Medicaid waivers.  Simply put, congressional intervention through legislation is needed to expand the “path” to 21st Century Cures, to ensure “access” to 21st Century Cures.

We are proposing a solution that is based on our understanding of the direct correlation between innovation and access, which has been gleaned from decades of our efforts advocating for new and improved technologies for the nearly 7,000 identified rare diseases and conditions.  Before restricted access to rare disease therapies becomes a public health crisis for not only millions of Medicaid beneficiaries, but also every individual and family member coping with a rare disease, we urge Congress to address this issue through 21st Century Cures. More specifically, Congress must consider enacting legislation that will prohibit discriminatory utilization control practices by state Medicaid plans – “epic” legislation that would ensure:

  • Equity for individuals with rare diseases and conditions;
  • Physician and patient treatment determination;
  • Innovation of and access to individualized treatment; and
  • Continuity of care.

Please sign onto the attached patient letter in support of this initiative.  We would like to have the letter ready to go during rare disease week.  Even if this issue doesn’t directly affect you at this moment, please sign on in support.  We have seen more and more of this over the past few years, and this trend needs to stop.

Download the letter here!

Read the full proposal here.

This information comes from The National MPS Society.  The National MPS Society exists to find cures for MPS and related diseases. We provide hope and support for affected individuals and their families through research, advocacy, and awareness of these devastating diseases. Learn more here. 

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