Gene therapies may cure disease but can we afford them?
For most of his life Rob Schroeder had to regularly inject blood clotting factor to stop the uncontrolled bleeding caused by the genetic disorder haemophilia B.
But his life was transformed four year ago after he volunteered to become one of the first patients to trial an experimental gene therapy called Hemgenix.
“I haven’t had a bleed since I had the treatment. Nothing. I effectively reached my wildest dream goals with this therapy,” Schroeder, a pharmaceutical rep from Monclova, Ohio, told the Financial Times.
AHIP, a group representing more than 1,300 companies that provide health insurance to 200mn Americans, said its members were not averse to paying for gene therapy drugs, as long as they proved to be effective and value for money.
Insurers would probably set restrictions to ensure only those who were most suitable for the treatments had access to them and co-payments from patients would be common, it added.
“In the case of the Hemgenix drug, it has got a lot going for it. It’s a cure. It’s one and done. It happens to be helping a population, people with haemophilia B, that doesn’t have a lot of options for treatment,” said Sergio Santiviago, vice-president of drug policy at AHIP.
Santiviago said insurers are exploring risk-based payment approaches to overcome the challenges of covering costly therapies that in most cases have not proven their durability over longer timeframes. For example, members have agreed a coverage deal for Zynteglo, which provides for Bluebird Bio to pay 80 per cent refunds if patients continue to require blood transfusions within two years of treatment, he said.
But Santiviago said the economics of covering gene therapies becomes more difficult when large numbers of patients could become eligible for multimillion-dollar treatments, particularly if there was not strong evidence supporting its effectiveness.
An important test is on the horizon with the anticipated approval next year in the US of a gene therapy targeting sickle cell disease, which has been developed by Vertex Pharmaceuticals and Crispr Therapeutics. About 100,000 Americans live with sickle cell disease in the US, compared to only around 30,000 people with haemophilia B.
A study assessing the impact of gene therapies on state budgets in the US, which was published last year in the journal Jama Pediatrics, concluded that a successful gene therapy for sickle cell disease would present affordability problems for several Medicaid insurance plans.
“While therapies with huge price tags garner a lot of attention, what tends to be more consequential from a payers’ perspective is the patient population size,” said Patrick DeMartino, a physician specialising in care for children with blood disorders and co-author of the Jama study.
“From a patient access and advocacy perspective, if there were delays in access from a payer constrained side, that would be a PR nightmare. I think it would harm people but it would also be very bad optics for the payer.”
This story originally appeared on: Financial Times - Author:Jamie Smyth
