Massachusetts is Looking for Ways to Help Patients Receive Expensive Gene Therapy

State is working with Novartis' AveXis unit to work out an installment plan.

 

Spinal muscular atrophy (SMA) type 1 (also known as infantile onset or Werdnig–Hoffmann disease) is the most common type of SMA. Symptoms, the most significant of which is respiratory muscle weakness, appear at birth or within the first six months of life. Use of portable ventilation devices can help in some cases; in others, a tracheotomy is necessary. Insufflator–exsufflator and high-frequency chest wall oscillation devices may also be required.

Novartis’s new gene therapy Zolgensma (AVXS-101, onasemnogene abeparvovec) is designed to address the genetic cause of spinal muscular atrophy. The U.S. Food and Drug Administration recently accepted  Novartis’ Biologics License Application for Zolgensma and granted the drug priority review. 

If approved, the drug, which is a one-time treatment, will be the first of any kind available for this rare, devastating disease. However, it will come with a hefty price tag of up to $5 million per treatment. Massachusetts wants to ensure that residents of the state suffering from the disease have access to the drug once it reaches the market, despite the high cost.

The state is taking a novel approach –– introducing an installment plan so that health insurers can pay for the treatment over several years.

Although installment plans are commonly used to pay for big-ticket items, this payment approach has never been used in the healthcare sector. The hope by organizers of the program is that success would lead to its use across the United States.

Novartis’ AveXis unit is working with the state to develop a potential solution. In addition to the installment approach, the payment plan would include a clause regarding the effectiveness of the treatment. If it does not work for a patient, Novartis does not get paid. The discussions are being mediated by the MIT Center for Biomedical Innovation's NEWDIGS program, a group that brings organizations together to discuss how the U.S. health system can pay for costly cures.

 

David Alvaro, Ph.D.

David is Scientific Editorial Director for That’s Nice and the Pharma’s Almanac content enterprise, responsible for directing and generating industry, scientific and research-based content, including client-owned strategic content. Before joining That’s Nice, David served as a scientific editor for the multidisciplinary scientific journal Annals of the New York Academy of Sciences. He received a B.A. in Biology from New York University and a Ph.D. in Genetics and Development from Columbia University.