Value-Based Agreements On The Cards For UniQure/CSL’s $3.5m Hemophilia B Gene Therapy
On Top Of Up To 30% Discounts
Executive Summary
While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.
uniQure N.V./CSL Limited’s hemophilia B gene therapy Hemgenix’s (etranacogene dezaparvovec) $3.5m price tag could be softened through discounts and value-based agreements (VBAs) intended to parse out risk and enhance accessibility.
CSL revealed Hemgenix’s hefty upfront cost after it won US Food and Drug Administration approval for severe hemophilia B on 23 November. The price surpassed bluebird bio’s $3m active cerebral adrenoleukodystrophy gene therapy, Skysona (elivaldogene autotemcel), setting Hemgenix up to become the most expensive drug on the US market.
The Australian firm said it was confident the price would generate significant cost savings for the healthcare system. Unlike standard-of-care clotting factors or prophylactic drugs for hemophilia B, Hemgenix is a one-time gene therapy that offers a long-term cure for the rare disease. (Also see "UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price" - Scrip, 23 Nov, 2022.)
Even so, the cost exceeded the Institute for Clinical and Economic Review’s (ICER) health benefit price benchmark of $2.9m. ICER’s advisory committee expressed mixed views during a recent vote on Hemgenix’s long-term value for money with one voter describing the treatment as “outrageously overpriced.”
However, CSL has said it plans to provide discounts for Hemgenix, including VBAs with commercial payers. Girisha Fernando, CEO of value-based contracting platform Lyfegen, told Scrip there was potential for 20%-30% discounts as well as separate value-based mechanisms. VBAs facilitate staggered payment models tied to a drug’s real-world performance.
For instance, Spark Therapeutics, Inc. (now part of Roche Holding AG) and health services company Harvard Pilgrim entered into a VBA in 2018 for the former’s retinal dystrophy gene therapy Luxturna (voretigene neparvovec-rzyl), which was priced at $850,000. Under their contract, a portion of the cost was made contingent upon Luxturna’s short-term efficacy and long-term durability in real-life patients.
Fernando said, “The price tag of $3.5m for Hemgenix is pressuring insurers and drug companies to work together to create VBAs for high-cost life-saving treatments.” There are several aspects to consider when negotiating these agreements.
“First, there’s value for patients which is determined through health outcomes, such as the extent to which the disease has been improved or cured and what impact this has on the patient’s quality of life,” Fernando explained. Hemgenix’s value for patients is quite clear based on its ability to reduce bleeding episodes, he added.
In the 54-patient pivotal HOPE-B trial, Hemgenix reduced the adjusted annualized bleed rate by more than 64% over months seven to eight. The therapy also reduced all Factor IX-treated bleeds by 77% over the same timeframe.
“The second consideration is value for the healthcare system which is determined through how much the treatment helps save on total cost of care for a patient and whether the healthcare system can deliver more efficient care as a result,” Fernando continued, adding bleeding episodes are associated with significant costs of hospitalization, treatment and follow-up.
Indeed, Cantor Fitzgerald analyst Kristen Kluska said in a recent note that patients with the most severe form of hemophilia B often need prophylactic or clotting factor treatment worth at least $1m a year throughout their lifetime, adding Hemgenix could eliminate these costs entirely.
“Even considering this, you could ask ‘does that still warrant a $3.5m price tag?’ and that is a difficult question with no straightforward answer,” Fernando admitted. If Hemgenix’s real-world performance does not match up to its pivotal trial and a patient experienced a bleeding episode, its cost would not be justifiable even with the anticipated 20-30% discount, he concluded.