FYI: Compassionate Access to Investigational Drugs
FYI: "Compassionate Use"
Given the recent discussions about access to cyclodextrin, we thought you might like to read more about compassionate access to drugs. The term "compassionate use" refers to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available.
Drugs that are being scientifically tested but have not yet been approved by the United States Food and Drug Administration (FDA) are called investigational drugs. Being able to use one of these drugs when you are not in a clinical trial specifically designed to study that drug has many names, but is most commonly referred to as compassionate use .
Compassionate use of an investigational drug is very carefully regulated and the approval process to use such drugs for an alternate purpose is rigorous. The FDA has developed a patient- and family-friendly Web site that provides information about access to investigational drugs under several circumstances, as well as information that might help you make a decision about whether or not to seek compassionate access.
Remember that this information applies only to drugs that are considered investigational. If there is no current clinical trial and the drug is not approved for treatment of any condition, it would not fall under this approach to drug access.
The press release about the FDA Web site can be accessed at:
http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm176845.htm .
From the above page, there is a link to the actual site, or you can go directly to:
Another significant part of thinking about requesting compassionate access to a drug is the ethical decision-making that parents, care givers and regulators must negotiate as they decide about an unapproved use of a drug. A recent article published in the Archives of Disease in Childhood entitled, “Compassionate and innovative treatments in children: a proposal for an ethical framework,” by Drs. Joe Brierley and Vic Larcher, outlines a series of questions that can be considered when deliberating about compassionate use. Issues include potential for effectiveness, how to obtain informed consent, access to the drug by the patient population in question, and others. Please see the full article (linked above, and here ) for more details.
If there are any questions about the information contained on the Web sites or in the article, please don’t hesitate to contact the NNDPF Coordinator of Education Referral and Advocacy, Cate Walsh Vockley, MS, CGC .
Cate Walsh Vockley, M.S., C.G.C
NNPDF Coordinator of Education, Referral and Advocacy
Senior Genetic Counselor
Medical Genetics
Children’s Hospital of Pittsburgh
One Children's Hospital Drive
4401 Penn Avenue
Pittsburgh, PA 15224
Tele: 412-692-7349; Fax: 412-692-7073
[email protected]
[Oct 16. 2009 mem]
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