An Excerpt from ISPE’s iSpeak Blog
By: Sandra K. Rodriguez, Market Analyst
“The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations.” stated Scott Gottlieb M.D., 23rd Commissioner of Food and Drug Administration in a January 2019 press release.
One such new therapy is Chimeric Antigen Receptor T-Cell (CAR-T) Therapy. “The new immunotherapy treatment CAR-T represents a paradigm change in cancer therapy. For the first time, cancer treatment can be directed specifically to the cancer cells, sparing most of the normal cells, and resulting in very high remission rates in patients who have otherwise failed all other available treatment. Many patients who would have otherwise succumbed to their disease, are alive and well today, as a result of the CAR-T treatment” commented Mounzer Agha, MD, UPMC, Hillman Cancer Center.
The Centers for Medicare & Medicaid Services (CMS) recently proposed to cover this new form of cancer therapy as there is no national Medicare policy. This lapse allows Medicare Administrative Contractors (MAC) to determine whether and how much to pay. The proposed determination would require Medicare to cover the therapy nationwide which would improve access and promote better patient outcomes.
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In related news, Novartis announced the world’s most expensive drug – with a price tag of over $2 Million for a one-time treatment. See the press release.