Letter from the Department of Health and Human Resources

Dear Mrs. (name)

Thank you for your letter to Representative Bob Inglis, regarding the approval of Telaprevir for the treatment of Hepatitis C.  Representative Inglis requested that we respond directly to you.

Please be assured that the Food and Drug Administration (FDA or the Agency) is very much aware of, and sympathetic to, the desperate situation of patients who would like access to new therapies that they believe will improve their conditions.  The agency is committed to providing timely access to potentially useful medical treatments for seriously ill patients, as well as working for speedy approval for new drug products while maintaining high, scientifically based safety and efficacy standards.

FDA regulates the manufacture, sale, and distribution of drugs in the United States under the authority of the Federal Food, Drug and Cosmetic ACT (FD&C Act or the Act).  In order for a new drug to be marketed in the United
States, it must be shown to be safe and effective for its intended use through the submission of an Investigational New Drug (IND) Application and a New Drug Application (NDA).  Drug manufacturers are required to file IND with FDA.  Once accepted, the IND allows the sponsor to ship the drug in interstate commerce for research purposes only.  The responsibility for the sponsor determines that adequate and well-controlled studies showing the drug is safe and effective have been carried out, that information, coupled with information on the manufacturing procedures and controls used in producing the drug, is submitted to FDA in the form of NDA.  After comprehensive review by the FDA, the NDA is either approved or not approved; upon approval, the drug may be marketed.

In your letter you ask that FDA speed up the approval of Telaprevir for the treatment of Hepatitis C.  Confidentiality laws prohibit FDA from disclosing information about a drug product that is not approved unless the manufacturer has already made that information public.  In the case of Telaprevir, certain information was make public by the manufacturer, Vertex Pharmaceuticals Incorporated.  On November 23, 2010, Vertex announced that they had completed the submission of an NDA to FDA.  In addition, they also announced they requested a six-month priority review.  Additional information can be found at:  htpp://investors.vrtx.com/releasedetail.cfm?ReleaseID=532555.  You may also wish to contact the manufacturer directly to determine whether they wish to reveal any more specific information about their product under development.

In situations where a patient might benefit from unapproved product and there is no alternative product already approved on the market, single patient INDs are a means of facilitating the availability of potentially promising new drugs to desperately ill patients for whom no other therapy is available.  Drugs under single patient INDs must be distributed from the manufacturer to a licensed physician.  The initial step in obtaining a single patient IND is for the physician to contact the manufacturer.  FDA does not become involved until the appropriate arrangements are made between the physician and the drug manufacturer.  In addition, FDA cannot intercede on behalf of the patients for the release of experimental drugs.  However, single patient INDs do allow products undergoing testing in humans to be legally distributed to appropriate patients before approval for general marketing.  For more information, please visit the FDA's website at http://www,fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/defaul.htm. for consumers containing information on access to investigational drugs.
We assure you that FDA is concerned about the treatment option for patients who suffer from illnesses, and the Agency is continually striving to expedite the approval of safe and effective treatment for those conditions.  FDA will expedite review time lines for drugs that have the potential to meet an unmet medical need or that may provide a significant improvement over existing therapy.  The Agency encourages the development of additional treatments for individuals with Hepatitis C and believes that it is important to have effective alternatives to the current available therapies.

Thank your for contacting Representative Inglis regarding this matter.

Sincerely,
Karen Meister
Supervisory Congressional Affairs Specialist

[FlGuy]

Gee, you sure learned a lot with that reply.  The written version of the annoying humming sound that emanates from DC.

[Trinity4]

I know, much to do about nothing but I thought I'd share it.  The response took so long I'd actually forgot I'd written a letter.  ;)

Trin

[mikesimon]

Maybe the executor/executrix should respond thanking Karen but informing her that the patient didn't survive long enough to read her helpful response. It wasn't timely enough.

Mike

[child24angel]

Maybe Michael is right !
FDA....hurry the heck up and get these PI's to market !!

[scratchinghead]

Ditto!!!

[desrt]

Six months to approve a drug, that took Vertex over 6 years to decide whether it would be safe/effective (profitable) enough a product to even ask for approval?
Bad, bad, naughty gov't.

http://www.medhelp.org/posts/Hepatitis-Social/Targeted-Therapies-and-FDA-Approval/show/1409708

From the link posted in that thread:

"Tannock's team examined so-called targeted therapies ...... that work by interfering with specific molecules......"

"He said doctors had assumed the medicines would be safer than traditional chemotherapy, but that the data suggested otherwise."

Vertex is still the stumbling block to "single patient INDs". If you think Vertex should be able to charge for unapproved drugs, maybe they can change that while they're revising/repealing the new healthcare law. I'm pretty sure that would open the floodgates on INDs.

.

[fretboard]

Well I'm glad you took the time to write them bozos a letter but I surely don't like their empty response at all.  It's such a crock especially since they included this part:

"In situations where a patient might benefit from unapproved product and there is no alternative product already approved on the market, single patient INDs are a means of facilitating the availability of potentially promising new drugs to desperately ill patients for whom no other therapy is available.  Drugs under single patient INDs must be distributed from the manufacturer to a licensed physician".

Talk about throwing water in the wind!!  No company in their right mind would ever use their drug in a single patient IND scenario!  I can't even believe the fda would include such nonsense!!  If they really cared about the HepC population then they would allow a 6 month fast track approval time.  If they do it their way, which they are many ppl die every year because they can't get tx'ed with the best drugs.  Just think, if society loses half of the infected HepC population, they could save billions of dollars.  Yes, they have thought this out completely!!!  Could it be possible?  Absolutely!  All they gotta do is act dumb and we all know the gov't does that very well!!