Friends and supporters,
We should all be enormously encouraged by the increasing cascade of promising results from investigational HCV drug trials. A couple of years ago, greatly improved therapy was a dream. Now, some investigational drug combinations show good cure rates with low side effects and greatly reduced treatment timeframes. Pharmaceuticals are engaged in a race to test and document such combinations. Many expect that hard work and market incentives are likely to result in prescription-availability of much better therapies in the next three years. But we, people with bleeding disorders, do have specific needs, and specific reasons to push for the fastest possible access to promising investigational therapies.
● In the first place, we are medically different from the average HCV community as a whole. We are more susceptible to side effects affecting hemostasis (clotting ability), plus, we’ve all had HCV for a very long time, often starting early in childhood. Because we’re different, some clinical trial data for people with bleeding disorders and HCV would help inform therapeutic decisions immediately after new drugs are approved.
In this regard, one pharmaceutical firm has agreed to provide “People with Bleeding Disorders and HCV” with timely notice on clinical trials for their particularly promising drug. We will circulate all notices to our mailing list and to the national bleeding disorder organizations, with the hope that trial participation from the bleeding disorders community may provide our treaters with information on community-specific side effects/efficacy when therapies first become available for prescription.
Better notification of upcoming clinical trials may be helpful for individuals who are eligible, but who have found trials to be fully subscribed by the time they hear of them. However, this notification activity will probably not be helpful to those with advanced disease because often they do not meet trial eligibility requirements.
● In the second place, a substantial percentage of our adult population (25% according to one recent paper*) has advanced liver disease. These people need therapy the most, but they are the worst candidates for current standard therapy, and they are frequently excluded from trials because of the severity of their condition. Under normal procedures this group (or whatever percentage of them survives) will see no relief till much better new drugs are fully approved. In circumstances such as ours, FDA has told us** that they will encourage pharmaceuticals to provide special relief via curative protocols***.
We are in communication with several pharmaceutical firms about curative protocols. We think that we would be a little more effective with pharmaceutical firms and with FDA if we could state that we have a list of individuals (perhaps several dozen) who are endangered by advanced HCV disease and poor medical options, and would be willing to consider a curative protocol. Please get back to us if you would like to be added to this list. The list will never be released, but we will send you an email if a pharmaceutical firm offers a curative protocol … so that you can contact them. Provide your name, address, and email. Kindly note that this list is designed to include only people with bleeding disorders who are both:
– at substantial health risk without therapy within the next few years, and,
– poor candidates for existing approved therapies.
● Finally we’d like to mention a more long term effort. Pharmaceutical innovators tell of substantial disincentives and weak incentives to pursue curative protocols. We hope to discuss this with FDA and prescription drug policy groups.
Mark Antell and Paul Brayshaw for
People with Bleeding Disorders and HCV