to Eteplirsen. This will be the final call of the FDA.
Rep. Rosa DeLauro, the ranking Democrat member of the subcommittee that funds the FDA, wrote on Feb 6, 2014 to FDA Commissioner Margaret Hamburg, specifically mentioned the approval of JNJ's Sirturo as an example. Sirturo was approved by the FDA through the Accelerated Approval process using a surrogate biomarker and won 18-0 in a FDA advisory committee panel.
Rep. DeLauro said "Considering the small number of patients with multi-drug resistance TB in the United States, instead of approving the drug, availability through compassionate use would appear to be a more reasonable approach to accessibility." Same is true here with Eteplirsen. Instead of risking to set a precedent of allowing the review process being strong armed by the patient advocacy groups, FDA now has the cover to argue instead of granting accelerated approach of Eteplirsen, Sarepta should make every possible effort to make the drug available through compassionate use and FDA will not get in the way of blocking such individual INDs. Meanwhile, FDA can demand a vigorous phase 3 program to demonstrate the efficacy and safety of the drug before it grants approval.
If it is all about the DMD boys to have access to the drug, parents and doctors can apply through the individual IND process and the drug would be available in 2014 instead of wasting time to fight for accelerated approval.
iposter, have you ever met Rep DeLauro? She is bleeding liberal that would give ever single person who didn't have a job, free healthcare, living, eating, drinking and drug expenses for life; all on the backs of hard working, middle and upper class workers. She is what is wrong with politics today, just like our president, hand outs for everybody and please vote for me. Screw he tax payer.
Across the board compassionate use should be described as "MANSLAUGHTER!" If drugs are not approved and sold, there is no incentive for investors in biotech companies. There is no incentive for biotech companies to develop drugs.
Unless congress wants to provide a budget for the entire drug development process. You want to see inefficient?
Protect us from ultra liberal nut cases!
Sentiment: Strong Buy
. . . where does "across the board" come from? . . . "compassionate access" for etep would be an interim step, td, until full approval could be gained through a phase 3 trial . . . what could be simpler to understand -- even for those ultra-right-wing nut cases in congress? . . .
Rep. DeLauro should get Congress to pass a law requiring insurance companies to pay for "Compassionate Use" (CU) drugs. Of course, nobody would participate in a clinical trial if they could get CU ... so in reality it rarely happens (even if FDA approves a drug CU). Case in point, see story all over national news last week of 6-y/o Jack Fowler suffering from Hunter Syndrome and Shire has a drug that could treat but will not provide under CU. CU was established in 1987 to deal with AIDS epidemic but has been a cruel hoax in terms of truly expanding drug access to rare diseases...hence the FDASIA Act of 2012 which specifically provides for AA.
The JNJ drug Rep. DeLauro was complaining about was killing people right and left. Eteplirsen's chemistry has been proven safe for a dozen years using hundreds of patients. As the FDA has already pointed out in response to DelLauro's letter, every drug is different and AA will be granted on a case-by-case basis. In other words, DeLauro, go away. The FDASIA was approved by a full Congress and won't be eliminated by a single Congresswoman.
Do you think nobody would use CU when the clinical trial run by SRPT is only for a restrictive subset with strict inclusion criteria? Insurance paying CU or not is not an issue FDA has taken into account in any of its approval decision. The advocacy groups can now work with the insurance companies as they have with the FDA. Jack Fowler's CU was denied by the sponsor company, not the FDA. Anyone imagine SRPT would deny CU for any boys after what the advocacy groups have done? JNJ's MDR-TB drug was approved less than 180 days after the signing of FDASIA via AA. That decision is now the example being specifically mentioned on why AA using surrogate marker should not be used, but rather to provide access under compassionate use.
I am long SRPT and I am not going to let someone's YMB post scare me out of my shares. But this sounds like something straight of of Atlas Shrugged ( the movie...I never read the book) to me. Scary it the government can actually do it. That is a slippery slope.
DMD number in US ?1900+/-. Treatment is infusion and weekly; cost in in the hundreds of thousands of dollars each. NO company, especially not a biotech with poor resources can do that.
There were 72 cases of MDR-TB in US in 2012 and rx much cheaper.
Basically what the guy was saying was, let that company pay for discovery, development and approval of the TB drug and then let them go thru a time-consuming and expensively labor intensive process for compassionate use EVERY time someone needed the drug. THEN, we'd like them to give it away free!
Don't think it would take a high IQ to go 'hmmmm' about that suggestion.