Researchers predict ‘friction’ between payers, physicians, patients over cost of B-VEC

Key takeaways:

• Beremagene geperpavec (B-VEC) treats autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa.
• Based on manufacturer reports, the estimated total annual expenditure of B-VEC is $286 million.

The FDA’s broad indication for the use of beremagene geperpavec therapy for the treatment of dystrophic epidermolysis bullosa may have large financial implications for payers, according to a study.

In May 2023, the FDA approved the first topical gene therapy, beremagene geperpavec (B-VEC; Vyjuvek, Krystal Biotech), for the treatment of autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB).

According to filings B-VEC manufacturer Krystal Biotech disclosed to the U.S. Securities and Exchange Commission, the likely annual price of therapy could be between $200,000 and $400,000 per patient.

Additionally, the FDA did not limit the approval of B-VEC to just autosomal recessive DEB, but extended it to autosomal dominant DEB, even though the pivotal trial supporting the approval only had one patient in this indication. Because autosomal dominant DEB comprises approximately 50% of all DEB cases, this can have a large financial impact on payers.

“Even if payers try to limit coverage of B-VEC therapy to only patients with severe forms of DEB, the potential budget impact of this new gene therapy could be sizeable,” Adam J. N. Raymakers, PhD, of the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital, and colleagues wrote.

This begs the question: How sizeable is this impact?

According to Raymakers and colleagues, the estimated number of U.S. patients with DEB eligible for treatment with B-VEC in the first year after FDA approval is 894. If therapy for each patient costs $300,000 annually, then the estimated total annual expenditure for B-VEC is $268 million.

Over a 3-year period, the estimated expenditure is $805 million, whereas the lifetime total costs per patient with autosomal recessive DEB and autosomal dominant DEB averaged $15 million and $17 million, respectively.

“These estimated costs would be more than double the expected costs of treatment had the FDA approved the gene therapy only for the more severe variant,” the authors stated. “The wider indication granted by the FDA may lead to friction between payers on the one hand and patients and physicians on the other.”