Need an efficient process for obtaining transformative drugs before approval

[huntingonthebluffs]

Thanks Fishermangents for your comments and feedback as always. Your perspective and experience is invaluable. On your question, his doctor indicated he would reach out to Dr. Tefferi on this case. In his last email, he indicated he had still not discussed with Dr. Tefferi but was trying to get that done and again he spoke of his very busy schedule and limited time and I'm sure the same was the case for Dr. Tefferi. I do believe his doctor was in the so called "inner circle" and worked with Dr. Tefferi on various levels based on his comments regarding Imetelstat and MF patient treatments and drugs.

[cheng_ho]

Wealthy people don't use "right to try"... they simply leave and use overseas clinics. That's why no one has set a legal precedent, those with enough lawyers use their Gulfstreams instead.

[huntingonthebluffs]

What is Compassionate Drug Use (CDU)?
Came across this information on the American Cancer Society website about compassionate drug use. It could be helpful to anyone trying to obtain access to unapproved drugs. The link was:
http://www.cancer.org/treatment/treatme ... e-drug-use

This article discusses Single patient access (SPA) versus Expanded access programs (EAP). How these work and how the patient would go about obtaining unapproved drugs in one of the two ways SPA or EAP. I think this information could begin to help people understand their options, how to proceed, timeframes, costs, etc.

The article is written to answer questions one might have about gaining access to unapproved drugs (also includes a link to information on signing an “Informed Consent” form). Some excepts included:

“A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs (EAPs) for patients who are not able to enroll in a clinical trial.

“Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access.

“Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. For people who aren’t in clinical trials, a drug company can allow use of their unapproved drug in 2 ways.

“According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions:
• Have used standard treatments and they have not worked
• Are not eligible for any current clinical trial that’s using the drug
• Have no other treatment options
• Have a type of cancer for which there’s reason to expect the investigational drug will help
• Are likely to have benefits that outweigh the risks involved

“Your doctor or one of the office staff will work with you on this process. The US Food and Drug Administration, Center for Drug Evaluation and Research is the starting point.

“Getting the drug through expanded access programs (if one is offered by the drug company) or single-patient compassionate use is possible for some people. But going through all the steps needed to get single-patient compassionate use of an unapproved drug can be frustrating and take a lot of time. For instance, drug companies have different policies and processes. And there’s no way to force the drug company to supply the drug. There may be very limited amounts of the drug available for compassionate use. Drug companies may only have enough drug for use in the clinical trial. (Producing extra medicine for people who are not in clinical trials can be costly for the drug company, especially when there’s a chance the drug might never be approved.)

Compassionate drug use can also be very confusing. There are several programs that regulate it. Many terms and definitions are used to describe how a patient may get access to an unapproved drug outside of a clinical trial. Drug companies, patient advocacy groups, and the FDA all may use different terms for the same things. The FDA provides definitions for the terms they use in their regulations. But most drug companies use different terms that are unique to their specific compassionate access programs.
Another big problem is cost. Some drug companies will supply the drug for free, but others charge patients. Most insurance companies will not pay for investigational drugs. There may also be other costs, such as the clinic’s cost of giving the drug and monitoring your response, that might not be covered by your health insurance.

Still, despite these hurdles, compassionate drug use does happen. Because actual use is not well-documented, there are no numbers or statistics on how often it’s done, who’s doing it, or how well it’s working for patients.

Where do I start if I want to apply for compassionate drug use?

Your doctor or one of the office staff will work with you on this process. The US Food and Drug Administration, Center for Drug Evaluation and Research is the starting point.

The main FDA number is 1-888-463-6332 (toll free). Select the option to “request information or speak with an FDA representative about an FDA-regulated product or program.” You may have to continue through the menu to reach someone to ask about the Emergency Use Investigational New Drug Program for Oncology Drugs.

The link to detailed information on how to ask for access to a drug is:

http://www.fda.gov/NewsEvents/PublicHea ... efault.htm

Your doctor should contact the FDA first regarding compassionate use of an investigational drug.

To learn more

We have a lot more information that you might find helpful. Explore www.cancer.org or call our National Cancer Information Center toll-free number, 1-800-227-2345. We’re here to help you any time, day or night.

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[Fishermangents]

Hunt, thanks for this important info. Many people will be gratefull for that. Now let's see how this works in the EU...