Dive Brief:
- A draft report from the Institute for Clinical and Economic Review (ICER) found that Roche AG's recently approved hemophilia medicine Hemlibra would save the healthcare system money compared with current therapies, despite the drug's high price tag.
- Budgetary cost savings could be as much as $1.9 million per year for patients aged 12 years or older with inhibitors to factor treatment and $700,000 for those under 12 years old, compared with patients receiving prophylaxis with bypassing agents or no prophylaxis.
- This review is the first budget impact report carried out by ICER for emicizumab for hemophilia A. The report is open for public comments until Feb. 23, and the final report will be posted in mid-March.
Dive Insight:
The approval of Roche AG's Hemlibra (emicizumab) in the U.S. last November as prophylaxis for bleeding episodes in patients who have hemophilia A with Factor VIII inhibitors promises to shake up the market based on its once-weekly prophylactic dosage schedule. However, one sticking point could be the wholesale acquisition price, costing $482,000 for the first year of treatment, and around $448,000 (depending on weight) for subsequent years.
Assessments from the cost-effectiveness watchdog ICER are often viewed as challenging for the industry, with calls for steep reductions in prices for PCSK9 inhibitors, multiple myeloma medications and gene therapy over the last few years.
However, the message for this one is looking brighter for Roche. The draft evidence report from ICER assessed the potential total budgetary impact of emicizumab in hemophilia A patients with inhibitors in the U.S. based on the wholesale acquisition price. This assessment included hemophilia A patients under 12 years, and 12 years of age or older, both with inhibitors, and looked at how Hemlibra might take market share from existing drugs.
This news will be a welcome boost for Roche, which is facing biosimilar threats to blockbuster drugs Rituxan (rituximab), Avastin (bevacizumab) and Herceptin (trastuzumab). The drugs made up around half of the big pharma's income in the third quarter of 2017, and Hemlibra has been touted as a potential blockbuster that could help offset the revenue loss of generic competition.
Hemlibra, which has been shown to have a statistically significant and clinically meaningful reduction in the number of treated bleeds compared with patients on no prophylaxis, has been recommended for approval in Europe. The thumbs up by the EU Committee for Medicinal Products for Human Use (CHMP) was for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors. The Hemlibra marketing authorization application is being reviewed under accelerated assessment, and a final decision is expected "in the near future."