A wave of new, expensive gene therapies are starting to change patients’ lives—and stress test healthcare systems. To Steve Pearson, M.D, president of the Institute for Clinical and Economic Review, working out fair prices for these one-time treatments is key to ushering in wider acceptance of the growing drug class.
The U.S. healthcare system has successfully absorbed the sticker shock of Novartis’ $2.1 million spinal muscular atrophy gene therapy Zolgensma. But more gene therapies—with even higher price tags—have arrived or are close to market, and the cumulative cost could once again agitate payers.
While these gene therapies come with the promise of potential cures, not all have clinical data to back their cases, Pearson said. Therefore, not all are worthy of multimillion-dollar price tags, he argued.
ICER, an influential U.S. drug cost watchdog, is continuing to work on gene therapy price evaluations. To Pearson, it’s critical for the healthcare system to differentiate between programs that represent true advances from those that “just make things a little bit better.”
“We have to figure out how we don’t fall into the same hole where every orphan drug is $300,000,” Pearson said in an interview with Fierce Pharma. “I don’t want it to be every gene therapy is $3 million. We have to be able to distinguish what’s really bringing value and what’s not.”
ICER has already drafted reports on some new gene therapies, including bluebird bio’s $2.8 million beta thalassemia treatment Zynteglo, CSL’s $3.5 million hemophilia B drug Hemgenix and BioMarin’s Roctavian for hemophilia A, which is still under FDA review. The organization has also started assessing a couple sickle cell disease candidates—Zynteglo’s sister med lovo-cel and Vertex and CRISPR Therapeutics’ exa-cel.
For the hemophilia treatments, ICER’s draft report, released in November, put the maximum suggested price for Hemgenix at slightly below $3 million and for Roctavian at below $2 million. The organization made the recommendations based on the drugs' potential to offset costs associated with prophylactic treatment such as regular blood clot factor infusions.
But ICER maintains that drugmakers should share some of the savings with other segments of the healthcare system, a point pharma companies may contest.
Inflation Reduction Act response
Elsewhere on ICER’s to-do list, the organization wants to update its drug pricing models in the wake of the Inflation Reduction Act (IRA). The new law caps how fast companies can raise drug prices and will eventually allow Medicare to negotiate prices for some of the costliest drugs with no available copycats.
ICER is evaluating the effects from IRA and plans to update its methodology this year or next, Pearson said.
The model update could be complex, particularly because it will involve many uncertain assumptions such as the availability of generics down the line, Pearson explained.
The good thing is, drugmakers are much more cooperative with ICER than in the past, Pearson said, possibly because of the group’s growing influence among payers.
Nowadays, companies sometimes approach the group for pricing input even before launching. That’s a change from when ICER first started doing drug reviews and companies often contested its conclusions.
For instance, Pearson complimented Eisai for sharing details of the company’s cost effectiveness analysis of Alzheimer’s disease drug lecanemab, which is now commercially available under the brand name Leqembi.
Although ICER doesn’t agree with some of the assumptions Eisai made in pricing the drug at $26,500 per year, having an open discussion is “a healthier approach” to the overall drug pricing system, Pearson said.
In addition to reviewing traditional drugs, ICER is considering branching its analysis to digital therapeutics, which are currently mostly used for neurological or behavioral disorders. As Pearson noted, there’s a lack of understanding of the adequate evidence needed introduce digital therapies into practice and around their fair price.