Expanded Access to investigational Drugs for Treatment Use

[akita]

How can i get a permit for extended access to drugs who are not yet approved by the FDA? This is possible in the cases described in the different documents, listed in this thread.

If the FDA says yes, the other question: who pays for the drug, must be solved. Medicaid does not pay for it. As to read in the discussions indicated in the legal documents 4. and 5., it was supposed that people with low income should get the drugs for free as some drug oompanies perhaps give them for free to some sort of needy people. Perhaps there are special funds that help you. Maybe not successfull applying there, maybe an illusion..?

But beside the cost of drugs are expenses also for the doctor who has to come in for the FDA-special permission for the adminmistration. By law rules the drugproviding company can decide freely if it would give the drug for extended use. The treating doctor is fully responsible for the treatment, the patient must provide informed consent. In the documents and informations you can read more detail. In addition it is possible to learn more by free webinars provided by ASCO.

If somebody has special questions, i hope that i or anybody else in the forum can answer them.

Practically it would cause a lot of paper-work for applying to that extended use e.g. for Nplate for an individual person in emergency health situationö. One must consider the special local situation in the treating center, who makes the treatment decisions, where are the patients data needed for the application stored, how can they be approached.. etc. (for example: When i want to get my datas, i have to apply to a special Center in vienna which organizes that. Patients datas 1 year old oder older are not stored in the ward or ambulance any more.. And you need perhaps such datas for argumentation, that your disease and treatment history demands the special medication now..).

In the appendix of this posting you find the mentioned links.

Hope i can help,

Margarete


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1. IDE Early / extended access

http://www.fda.gov/MedicalDevices/De.../ucm051345.htm

2.Information for Consumers: Access to Investigational Drugs

http://www.fda.gov/ForConsumers/ByAu...gs/default.htm

3. Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use

http://www.fda.gov/Drugs/Development.../ucm107434.htm

4. Expanded Access Training Webinar

http://www.fda.gov/ForConsumers/ByAu.../ucm217583.htm

Legal Materials:

5. http://edocket.access.gpo.gov/2009/pdf/E9-19005.pdf

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
21 CFR Parts 312 and 316
[Docket No. FDA–2006–N–0238] (formerly Docket No. 2006N–0062)
RIN 0910–AF14

Expanded Access to Investigational Drugs for Treatment Use
AGENCY: Food and Drug Administration,
HHS.
ACTION: Final rule.

6.. http://edocket.access.gpo.gov/2009/pdf/E9-19004.pdf

Register, August 13, 3009 Part II
Department of Health and Human Services
Food and Drug Administration
21 CFR Part 312 and 316

Charging for Investigational Drugs Under
and Investigational New Drug Application;
Expanded Access to Investigational Drugs
for Treatment Use; Final Rules
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[akita]

Promacta has been approved by FDA under some restriction as a drug for ITP.

This is not an extended access- program.

If a patient with a non-ITP diagnosis wants to get Promacta, he/she must go the way i described above .

"Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use" and some extra things. They way in which forum members hat success in obtaining the medicament remains unrevealed to me. But it could be possible for some reasons.

Kind regards,

Margarete

[CindyW]

How does one get into test groups for newer drugs and treatments? Does your doctor have to be involved in the study or are the studies done at research hospitals? I've always wondered how people get into the test groups that happen before the FDA approval.

[Neil Cuadra]

If you can identify a trial that might suit you, you can apply to be in it. Every trial has its own rules about who can participate, called the inclusion/exclusion criteria, based on age, disease condition, previous treatments used, and other factors. Your own doctor doesn't have to be affiliated with the study but you usually have to be at one of the facilities running the trial. Many trials are held at the National Institutes of Health in Bethesda, MD.

The best source of clinical trial information is clinicaltrials.gov.