Gene therapies are in the vanguard of medicine and can potentially provide a single-use option for patients with rare genetic diseases. But the treatments are costly: one particular one-time gene therapy for spinal muscular atrophy (SMA) exceeds $2 million.
However, a poll from the Pacific Research Institute (PRI) shows that high cost isn’t always a deterrent. Data show that Americans overwhelmingly support new gene therapies that are regarded more as possible cures rather than long-term treatment. More than 78% of respondents believe expensive gene therapy treatments are worth it, because they could bring about cures.
As the goal of gene therapies is to address underlying disease causes rather than to treat symptoms, more people are supportive of the possibilities. According to the poll, 80% of respondents agree that focusing on cures is more important than chronically treating them. The favorable response is particularly interesting in light of the recent approval of Novartis’ Zolgensma, which costs $2.125 million for SMA treatment, and Spark Therapeutics’ Luxturna, with its $850,000 cost for a potential one-time treatment for a rare form of blindness.
High prices are common for orphan disease drugs. Each of the top 10 most expensive drugs in the U.S. has a list price of more than $500,000 per year.
Major findings of the PRI poll include the following:
Source: BioSpace, June 27, 2019