Next Push – Advanced Therapy

Our Citizen Petition to the FDA in 2009 (with amendment), asked for recognition of the HCV crisis in our community and it requested the earliest possible access to advanced HCV therapies.聽 We have made substantial progress in moving regulatory policy toward appropriate urgency.

However, as advocates and patients, we are impatient for therapeutic improvements.

We therefore intend to find industry and clinicians who will support our use of advanced investigational agents in curative therapy protocols.
For those people with bleeding disorders and HCV who are less threatened, we will continue to lobby for early access to improved therapy via the clinical trial pathway.

Why this push?
The crisis is now.聽 Around 80% of those who received factor through the mid 1980s have chronic HCV.聽 HCV often advances slowly, but we were infected 30, 40, or 50 years ago. After decades of slow progression, HCV is now the leading cause of death in our community.

Problems with current medical therapies
Current therapies are inadequate.聽 The only currently approved medicines available for curative therapy of HCV are:
– Ribavirin (RBV) plus pegylated Interferon (p-IFN).聽 This combination is also known as Standard of Care (SOC) therapy, and,
– SOC plus a first generation protease, either telaprevir or boceprevir.

As to SOC, cure rates for our community are likely lower than average due to known factors including: age, co-infection, advanced disease, period of infection, genotype, etc.聽 This has been recognized by the NHF in MASAC bulletin 203.聽 We, People with Bleeding Disorders and HCV, recommend that people considering SOC should consult with their treater about clinic-specific cure rates for people with similar disease and co-infection status.聽 Further, we challenge the commonly held paradigm that failed therapy is benign.聽 Instead, we suspect that it frequently is harmful, particularly for those with advanced disease, based on anecdotal evidence and recently published papers generated by the NIH HALT-C program.

As to therapy with the first generation protease inhibitors, these require a concurrent long course of the SOC cocktail, plus they bring their own side effects including a high likelihood of viral resistance.聽 The NHF in MASAC bulletin 203 cautions that the new protease inhibitors are also anti-hemostatic and concludes that they cannot (yet) be recommended for our community.

Better treatment alternatives on the horizon
The newest investigational therapies are extremely promising.聽 The recent purchase by Gilead Pharmaceuticals of Pharmasset Pharmaceuticals is an indicator of how close we are to a better cure.聽 The sale valued Pharmasset at $11 billion based on investor expectations that an advanced investigational drug (Pharmasset’s PSI 7977) will be part of much improved therapy.聽 Many firms are developing their own agents, leading to a complex scene of optimistic claims and reports for investigational drugs.聽 We attach a report on the many drugs discussed at the American Association for the Study of Liver Disease conference meeting held in early November in San Francisco.

Possible Options
We think that the medical options for individuals with bleeding disorders and HCV are:
1. Continued hopeful waiting for far better HCV therapy, or
2. Taking a course of SOC or enhanced SOC therapy (not recomended where contraindicated), or
3. Aggressively seeking out access to better therapy (substantiated by fairly early safety and efficacy data).聽 This is particularly justified for people with bleeding disorders and HCV because we tend to have advanced disease (and thus a substantial potential for adverse medical events) and because we have lower- than-average likelihoods of good outcomes from currently approved therapy.

With whom we will work
– Regulators.聽 We have received substantial encouragement from FDA for this approach, but open doors don’t remain open forever.聽 We will continue to seek rapid action.
– Industry.聽 We have made preliminary contact with several drug companies which seem interested, perhaps because current FDA guidelines encourage them to provide compassionate access.聽 Certainly, successful therapy for ‘difficult to treat’ cases can only improve chances for early drug approval.
– Clinicians.聽 We hope some clinicians will be willing to step forward and work for curative protocols.
– Advocates.聽 We see increased crisis recognition and increased demand for urgency among bleeding disorder advocacy organizations.聽 We will solicit continuing outspoken support from HFA, NHF and COTT.聽聽 We will also continue to interact with other HCV advocacy organizations including the Hepatitis C Community Advisory Board (HCAB), and the Forum for HIV Collaborative Research, HCV Drug Advisory Group (HCV DrAG).聽 We will also continue to work with the Abigail Alliance for Better Access to Developmental Drugs.

Obtaining drug access via participation in clinical trials, or via compassionate access protocols will be not be easy or without risk.聽 We will share our experience among the bleeding disorders community so that others can consider joining a trial or curative protocol. We are hopeful that our experience will help others determine a course of action best suited for their individual situation.

Mark Antell and Paul Brayshaw for People with Bleeding Disorders and HCV
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