Theresa Toigo, RPh, MBA Director
Office of Special Health Issues
Food and Drug Administration (FDA)
5600 Fishers Lane
HF-12, Room 9-49
Rockville, MD 20857
Dear Ms. Toigo,
We are people with Hemophilia and their supporters.
We recognize and appreciate the recent FDA publication of rules to clarify and expand the process whereby some patients may obtain access to drugs in development (Expanded Access to Investigational Drugs for Treatment Use – Federal Register August 13, 2009) . However, many people with Hemophilia have run out of approved treatment options for hepatitis C (HCV) and are rapidly running out of time. Meanwhile, promising drugs for HCV are under investigation. We request that FDA focus on the urgent problem of access to life-saving HCV drugs for our community.
In summary the petition says:
People with Hemophilia, particularly those with advanced HCV and/or co-infection with HCV/HIV, desperately need access to promising investigational HCV therapies. We call on FDA to:
Coordinate a public hearing for all stakeholders, including consumers, healthcare providers, medical industry representatives, and public officials to facilitate approval of promising HCV drugs and drug combinations;
Provide an ‘expanded access’ pathway to combinations of two or more promising HCV drugs for people infected with HCV who are poor candidates for P-IFN/RBV therapy;
For persons who have HCV which is ‘difficult to treat / low likelihood of treatment success,’ and for whom P-IFN/RBV may be indicated, provide access to regimens which add promising (preferably oral) agents to the standard therapy. Priority should be given to protocols which add two or more such promising agents.
The petition requests that FDA recognize the crisis of HCV in People with Hemophilia and take definite steps to allow them access to promising HCV investigational drugs, beyond the scope of any current clinical trials.
We, the petitioners, look forward to engaging with you on this urgent concern.
Mark Antell, Person With Hemophilia (PWH)
Paul Brayshaw, PWH
Richard M. Lawrence
L. Sabrina Adams
Florence and Jim Hellenbrand
Jeff and Judy Hellenbrand
Terry and Jamie Hellenbrand
Sue and Chuck Ziegler
Kevin and Sue Hellenbrand
Paul and Julie Hellenbrand
Jay and Michelle Hellenbrand
Jodi and Jeff Hellenbrand
Franklin Wayne Roberts
Kim and Martin Wood
Hemophilia Federation of America
Frank Burroughs, Abigail Alliance for Better Access to Developmental Drugs
Tim Duncan, RN, CCRN, CRTT, Account Executive Grifols
Mandi White, Wyeth Hemophilia Specialty Manager
Sandy Deierhoi, Director of Marketing, Factor Support Network
Responses should be addressed to:
*Mr. Antell is a hemophiliac who also has HCV. **Mr. Brayshaw is a person with hemophilia and is employed by a home health care company. Other than an interest in saving our lives, and the lives of other people with hemophilia and HCV, neither of us have a conflict of interest in the matter of expanded access to HCV therapies.