There is an interagency cold war between the Food and Drug Administration and the Centers for Medicare and Medicaid Services at the Department of Health and Human Services. FDA wants to approve promising treatments for patients faster.  CMS is more interested in avoiding costs and restricting patient access.

For patients with no or minimal treatment options, there is a great hunger to rapidly advance science and make new treatments available to advance our understanding of diseases and the long-term value of therapies. After years of taking heavy criticism from patient organizations, particularly in ultra-rare disease, the FDA has become more responsive to the needs of patients.