- The Italian Medicines Agency (AIFA) has informed GlaxoSmithKline that it will only pay for Strimvelis, a gene therapy for a very rare disease that affects children, if the treatment works, reports Technology Review. Strimvelis, which is intended to be curative, comes with a price tag of $665,000.
- Using its proprietary gene and cell therapy platform, GSK developed Strimvelis for treatment of ADA-SCID, a disease in which children are immune-compromised to the point that even everyday infections are a threat. ADA-SCID is caused by a faulty gene inherited from both parents which prevents the body from producing a critical enzyme. Without this enzyme, toxins build up and destroy infection-fighting lymphocytes.
- GSK initially tried to price Strimvelis at $1 million in the Italian market, but AIFA pushed back---hard.
ADA-SCID is extremely rare,but treating these patients is costly. Treatment often requires a bone-marrow transplant that can cost as much as $1 million and injectable enzyme-replacement therapy of $250,000 per year.
Glaxo's therapy is developed from the patient's own bone marrow cells, which are extracted and then inserted with the missing enzyme. This decreases the risk of rejection significantly. The outlook for ADA-SCID patients treated with Strimvelis is good. All 12 people who participated in the Phase 3 trial that led to the approval of Strimvelis are still alive, with an average follow-up time of seven years. The first patient received the gene therapy more than 13 years ago.
AIFA has not singled GSK out. In fact, the agency regularly imposes pay-for-performance rules on companies and has so far collected €250 million in refunds. As for GSK's profit motives around Strimvelis, the company claims not to be focused on profits, but rather advancing the broader interests of gene therapy R&D and the establishment of its gene and cell therapy platform.
Pya-for-performace models are increasing becoming for popular —even in the U.S. Several big pharmas have inked deals with payers exploring these programs and basing price on how a drug performs in a specific patient population.