Hem A Gene Therapy Roctavian More Cost-effective Than Hemlibra

ICER report says Roctavian, if approved, will provide substantial cost savings

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by Steve Bryson, PhD |

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If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra.

That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit organization, ICER evaluates data on the effectiveness and economic value of therapeutics and other medical devices.

“BioMarin is pleased that ICER recognizes the potentially transformative impact of Roctavian as possibly the first gene therapy treatment for severe hemophilia A, and potential to not only deliver profound patient benefit, but also potential long-term healthcare savings,” Jeff Ajer, executive vice president and chief commercial officer at BioMarin Pharmaceutical, Roctavian’s developer, said in a press release.

The draft evidence report, which is open for public comment, will next be assessed by one of ICER’s three independent appraisal committees before a final version is issued. That final report is expected near the end of the year.

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Roctavian’s Effectiveness Tied to Molecules that Aid FVIII Production

BioMarin stated it is still on track to resubmit, by the end of the month, an application seeking Roctavian’s approval in the U.S. Typically, approval resubmissions are followed by six months of review, but the company anticipates regulators may require an additional three months based on the large volume of data being submitted.

An initial approval request by BioMarin to the U.S. Food and Drug Administration (FDA) in 2019 was delayed by the agency the following year. The FDA cited a need for two more years of patient follow-up data from the Phase 3 GENEr8-1 trial (NCT03370913), which was used to support the company’s filing.

Cost savings expected with gene therapy

In the draft evidence report, ICER calculated the lifetime cost per patient of managing hemophilia A using a one-time dose of Roctavian versus the approved therapy Hemlibra (emicizumab). Hemlibra is a prophylaxis, or preventive treatment for averting or reducing bleeds in hem A.

Roctavian — designed to restore the production of factor VIII (FVIII), the blood clotting protein that is defective or is missing in hemophilia A — is given via a one-time infusion into the bloodstream. Hemlibra, administered as a subcutaneous (under-the-skin) injection, is an antibody-based medication that mimics the action of FVIII, but requires 1–4 monthly, ongoing doses to prevent bleeds.

The total calculated cost model included the therapy and treatment-related adverse events, as well as the cost of medicines for bleeding episodes, surgery, resulting joint disease (arthropathy), and other non-treatment costs.

ICER estimated the cost of preventive Hemlibra treatments to be $640,000 per year compared with a one-time placeholder Roctavian price of $2.5 million. The effect of Roctavian was assumed to last 12 years before patients potentially switched back to prophylaxis.

Based on these assumptions, ICER estimated more than $4 million would be saved using Roctavian instead of Hemlibra per patient over a lifetime. Gene therapy also was associated with slightly fewer bleeds and higher quality-adjusted life years (QALY) — a measure of disease burden, including both the quality and the quantity of life lived; one QALY corresponds to one year of perfect health.

Notably, ICER’s model included an outcomes-based warranty pact, an agreement between BioMarin and an insurance payer in which the company will issue a refund or rebate to the payer based on the therapy’s performance in the real world. This will allow effective risk sharing for four years and will be ready to implement with payers at Roctavian’s launch.

Roctavian recently received conditional approval in Europe for treating certain patients with severe hemophilia A. Eligible patients are those without inhibitors to standard replacement therapy or detectable levels of antibodies against the therapy’s viral vector.

BioMarin expects Roctavian to be available to patients in the EU by the end of the year.

The ICER report also contained a comparative analysis involving the experimental gene therapy EtranaDez (etranacogene dezaparvovec) meant to treat hemophilia B, which is characterized by the lack of factor IX (FIX). That analysis compared EtranaDez and FIX replacement therapy.

In that case, the gene therapy was expected to save more than $7 million during a patient’s lifetime versus FIX prophylaxis, with a higher QALY.

“The gene therapies have large cost savings associated with them with very large lifetime costs associated with both the treatments and comparators in both models,” the ICER researchers wrote in the draft evidence report. “In addition, the gene therapies are associated with higher QALYs and lower bleeds.”

The ICER report did note that “there was limited data on the efficacy of the gene therapies and limited mechanisms for projecting bleeds across time.” Nonetheless, according to BioMarin, the report found that Roctavian would provide “substantial cost savings and projected gains in quality adjusted life years.”