Dear Addy: A Session To "Ask The Experts"

HFA’s Dear Addy Session Allows Patients and Caregivers to Ask the Experts

HFA Symposium (Las Vegas, April 1, 2016) – Attendees at the HFA Symposium asked a panel of experts (live and by Twitter) about advocacy, policy, insurance and much more. One question was why is factor so expensive?  As a biologic product developed for a rare condition, there are many processes that need to be developed to manufacture the product to test in animals, then in clinical trials, then in larger quantities once approved, all under tight scrutiny; in addition, clinical trials must be developed that meet regulatory agency requirements.  Not all products that are developed make it to market either, even in hemophilia and bleeding disorders. Another question was about what HFA thinks about inhibitor development and the SIPPET results?  While SIPPET preliminary presentation of results in December showed higher rate of inhibitors with recombinant than plasma derived factor VIII products, the full results are not published yet including the genetic mutations of the patients in the study, many of the newer US FVIII products were not included, and none of the extended-half-life FVIII products were studied. HFA is following the issue closely and awaiting publication of the results hopefully this spring. Another attendee asked for tips on understanding confusing new products. Panelists recommended accessing drug reps for information, but also depending on the HTC staff for guidance.  Advice for college-bound teens was another focus.  Panelists recommended connecting with the chapter or HTC nearest the college, develop a support structure, focus on sticking with your treatment regimen, and working on communication about hemophilia with the student health center and your roommate.