NHIA to assess paying hefty price for new hemophilia B gene therapy

Taipei, Aug. 19 (CNA) The National Health Insurance Administration (NHIA) will evaluate including a one-time gene therapy to treat individuals with hemophilia B in the NHI drug reimbursement scheme after it is approved in Taiwan, an NHIA official said Monday.

The U.S. Food and Drug Administration (FDA) has approved the gene therapy fidanacogene elaparvovec (Beqvez), which has a hefty US$3.5 million (more than NT$100 million) price tag, for adults with hemophilia B, a rare bleeding disorder.

The therapy has only received conditional marketing authorization from the European Commission under the moniker Durveqtix because it was tested on a small number of individuals, Tai Hsueh-yung (戴雪詠), an official from the NHIA's Medical Review and Pharmaceutical Benefits Division, said in a joint media interview.

Tai added that the Beqvez manufacturer is required to continue providing relevant data on the treatment's long-term efficacy.

Applications for importing the one-time hemophilia B gene therapy product have been submitted to Taiwan's Food and Drug Administration, according to Tai.

If the suppliers submit reimbursement applications to the NHIA after an import permit is granted, the agency will begin evaluating the efficacy of the treatment, its cost-effectiveness and the extent to which it enhances a patient's quality of life, Tai said.

Hemophilia B is a rare inherited disease caused by a deficiency or defect in blood clotting factor IX (FIX) meaning the blood cannot clot properly to control bleeding.

Individuals with severe hemophilia B often receive regular infusions to prevent bleeding episodes. The disease affects 196 patients in Taiwan and the average annual cost of treatments is about NT$5 million-NT$7 million (US$15.63 million-US$21.88 million).

Although Beqvex costs around NT$100 million, the one-time gene therapy is intended to help patients produce factor IX rather than needing infusions multiple times a week.