Lucas Kempf Associate Director Rare Diseases Program at Food and Drug Administration

The FDA is approving treatments for orphan diseases at an unprecedented rate. Which in itself is great for rare disease patients who have been waiting for a treatment. However, FDA approval does not equal access, as the reimbursement community is demanding demonstration of value to justify the high cost. Absence of strong confirmatory evidence means no access for some patients. Many times, coverage policies are limiting access based on arbitrary requirements, misunderstanding of the accelerated approval pathway, or worse, no understanding of the disease intended to treat. This process results in sick patients seeking treatment only getting sicker and costing more to treat and provide care. Creative solutions should be discussed and explored to this critical issue.