Gene Therapies In Hemophilia

By David Carr & Larry Blandford

Potentially curative gene therapies for hemophilia are getting closer to market, representing a transformation in care for many thousands of patients. But all is not so simple, and the fact that these gene therapies could positively impact so many people presents a new challenge for the growing impact of advanced therapies. So far, gene therapies have targeted smaller, rare, or ultra-rare indications with a high disease burden, which means access stakeholders have been more forgiving on the evidence requirements, and the high treatment costs haven’t had major impacts on budgets. Download the available white paper to find out more.