Hi--Just wanted to introduce myself.  I joined PPMD in January as the Research and Advocacy Director after 11 years at MDA running their translational research and drug development programs.  At PPMD I'll be further developing work with drug companies, reviewing and growing the main research program and participating in advocacy intiatives.  I will also try to jump in as much as possible to answer questions about research and track down information about new treatments/therapies that anyone hears about.  One of my first projects along this line is to get to the bottom of this stem cell report from Costa Rica.  If anyone posts something that needs a response from me and you don't hear from me, you can always send me a nudge at sharon@parentprojectmd.org.  I'm really excited about the chance to focus exclusively on Duchenne and am looking forward to hearing from all of you.

 

Best Wishes,

Sharon Hesterlee

Views: 225

Reply to This

Replies to This Discussion

The painful truth is that my son will not live to have his own children w/o a treatment. He might not even live to see one of the Project Catalyst drugs approved. So I need to take my chances and trust that EMEA is doing its job and does not approve a drug that could kill him before DMD does. Isn't that the job of regulatory agencies, EMEA included?



Sharon Hesterlee said:
All I can tell you is that the FDA is a different agency from the EMEA-they may be asking for slightly different measurements or maybe not. There is no straightforward mechanism for the FDA to accept data from a study conducted under the auspices of the EMEA. It is crazy...but there is not a lot of harmonization and there's no doubt that the lack of coordination slows progress and dramatically increases expenses. At the same time, keep in mind that thalidomide was approved in Europe but rejected in the U.S. saving thousands of U.S. families the devestation of having a baby born with deformities. Of course that was many years ago and both agencies are probably using higher safety standards now. It is a problem.
Hi Ofelia,
I was wondering if you had any idea about Canada making Exon skipping available if it were to be approved in Europe. Canada has made Deflazacort available when the States did not.

Jennifer

Ofelia Marin said:
The painful truth is that my son will not live to have his own children w/o a treatment. He might not even live to see one of the Project Catalyst drugs approved. So I need to take my chances and trust that EMEA is doing its job and does not approve a drug that could kill him before DMD does. Isn't that the job of regulatory agencies, EMEA included?



Sharon Hesterlee said:
All I can tell you is that the FDA is a different agency from the EMEA-they may be asking for slightly different measurements or maybe not. There is no straightforward mechanism for the FDA to accept data from a study conducted under the auspices of the EMEA. It is crazy...but there is not a lot of harmonization and there's no doubt that the lack of coordination slows progress and dramatically increases expenses. At the same time, keep in mind that thalidomide was approved in Europe but rejected in the U.S. saving thousands of U.S. families the devestation of having a baby born with deformities. Of course that was many years ago and both agencies are probably using higher safety standards now. It is a problem.
No Jennifer, I have no idea. I assume that they will have canadian sites in the trials and apply for approval there as well. It needs to be approved by the canadian regulatory agency. My guess is that it will be approved sooner than in the US (if everything goes well).

Jennifer said:
Hi Ofelia,
I was wondering if you had any idea about Canada making Exon skipping available if it were to be approved in Europe. Canada has made Deflazacort available when the States did not.

Jennifer

Ofelia Marin said:
The painful truth is that my son will not live to have his own children w/o a treatment. He might not even live to see one of the Project Catalyst drugs approved. So I need to take my chances and trust that EMEA is doing its job and does not approve a drug that could kill him before DMD does. Isn't that the job of regulatory agencies, EMEA included?



Sharon Hesterlee said:
All I can tell you is that the FDA is a different agency from the EMEA-they may be asking for slightly different measurements or maybe not. There is no straightforward mechanism for the FDA to accept data from a study conducted under the auspices of the EMEA. It is crazy...but there is not a lot of harmonization and there's no doubt that the lack of coordination slows progress and dramatically increases expenses. At the same time, keep in mind that thalidomide was approved in Europe but rejected in the U.S. saving thousands of U.S. families the devestation of having a baby born with deformities. Of course that was many years ago and both agencies are probably using higher safety standards now. It is a problem.
So, the FDA decides to throw our babies out with the Thalidomide "bath water". The end doesn't justify the means and vice versa. That leaves us with no other option then to trust the EMEA. I was told by a very reliable source the FDA can choose to accept data from another regulatory agency, such as the EMEA, for treatment under compassionate use law here in the US. What I've been told sounds the exact oppisit of what you are saying Sharon. How did you come to conclude the FDA has no mechanixm to accept data? Confusion!

Sharon Hesterlee said:
All I can tell you is that the FDA is a different agency from the EMEA-they may be asking for slightly different measurements or maybe not. There is no straightforward mechanism for the FDA to accept data from a study conducted under the auspices of the EMEA. It is crazy...but there is not a lot of harmonization and there's no doubt that the lack of coordination slows progress and dramatically increases expenses. At the same time, keep in mind that thalidomide was approved in Europe but rejected in the U.S. saving thousands of U.S. families the devestation of having a baby born with deformities. Of course that was many years ago and both agencies are probably using higher safety standards now. It is a problem.
Hi Cheryl--sorry for the confusion. I said there was no straight forward mechanism for the FDA to accept data from the EMEA--I should have added "routinely." You are talking about a very specific situation in which a treatment is made available in the U.S. through an expanded access program (as regular trials are still ongoing in the U.S.)--these are not common and typically can't accomodate very many people. The need for better coordination between the two agencies will be a bullet on the letter we are preparing for congress to send to the FDA. If clinical studies didn't need to be repeated in duplicate in the U.S.and Europe we would see many drugs available in both places more quickly. It would probably dramatically reduce the cost of drug development and help with the spiraling healthcare costs in the U.S. Here is my personal hit list of problems with the process:

1. Lack of coordination between FDA and EMEA
2. Requirement by institutions to have individual IRBs for each trial site in a multicenter trial (MAJOR time sink); this isn't an FDA requirement but rather that of the lawyers at all the individual hospitals and univeristies
3. Lack of resources from a budgetary standpoint at FDA
4. Lack of authority for Office of Orphan Product Development
4. Need for mechanism to expedite review of new technologies (eg., exon skipping)

Additional things that reduce costs and increase speed of clinical trials: having EVERY child genotyped and in a single updated registry; developing and validating "surrogate markers" for clinical studies (that is, things you can measure quickly and cheaply that reliably predict a functional improvement); and newborn screening and early childhood screening to identify children as early as possible.

That's my wish list. These things will likely find their way onto our advocacy agenda and the strategic research plan I'm drafting for PPMD.
Thanks Sharon!!

Less confused now. Still can't spell worth beans but less confusion is good. I like your list...especially #4. Orphan status makes it sound like things will be on a fast track when in reality we are on a "slow boat to China". No offense intended to our Chinese friends.
cheryl, tell me more about compationate use...whom do i contact about such things, doctor wong, prosensa...?
Jenn
my email is cherylcliff@gmail.com, please contact me and I'll give what info I have. :)

jenn said:
cheryl, tell me more about compationate use...whom do i contact about such things, doctor wong, prosensa...?
Hi

This is a great thread. I have several thoughts. Prosenea is probably doing a non-ambulatory trial because the subjects can be over 18 years old and can give consent. Clinical trials on children have more hoops and they can get the info they need from the older guys (what happens to the drugs over time. what is the mechanism).

My second thought is that the PPMD conference has had a representative from the FDA present for the past 7-8 years. We can bend his/her ear.

My opinion is the RAC process is a diversion, because FDA doesn't want to deal with it yet. Perhaps that question could be asked at the conference.
Karen
Sharon,

I think your list is well focused, and in most instances, these are issues common to all genetic diseases. As I see it, one of the problems is that FDA personnel feel accountable for instances in which harmful drugs and devices are approved when they shouldn't have been or aren't removed from the market fast enough. Unfortunately, they don't feel accountable for instances in which they are too slow to approve life saving medications. With apologies to Ofelia, my father and all the other professional statisticians I have known, there are lies, damned lies and statistics, but I expect it would be easy to demonstrate that more boys have died from Duchenne, to say nothing of the other diseases out there for which there is no treatment, in the last ten years then died from Vioxx, phen-fen (sp?) and all the other inappropriate life style drug approvals combined.

It would seem too late this year, but perhaps next year we could build on the success of the MD Care Act which made the NIH accountable for translating basic research into usable drugs and standards of care for neuromuscular diseases. Lean on Congress to hold hearings to have the head of the FDA come down and identify which conditions have orphan drugs in the approval process, where they are in the approval process and how many people died of that condition since the last hearing. That would get old real fast for our friends at the FDA, and I'm sure that they would be a little less cautious and a little more generous with their funding and time.
well said, i find myself telling people all the time, when they ask "what if the drug causes harm?" i tell them, i already know what will happen if we do nothing, there are plenty of THOSE trials going on every day with FATAL results, im willing to take the chance in trying something new.

Paul Cliff said:
Sharon,

I think your list is well focused, and in most instances, these are issues common to all genetic diseases. As I see it, one of the problems is that FDA personnel feel accountable for instances in which harmful drugs and devices are approved when they shouldn't have been or aren't removed from the market fast enough. Unfortunately, they don't feel accountable for instances in which they are too slow to approve life saving medications. With apologies to Ofelia, my father and all the other professional statisticians I have known, there are lies, damned lies and statistics, but I expect it would be easy to demonstrate that more boys have died from Duchenne, to say nothing of the other diseases out there for which there is no treatment, in the last ten years then died from Vioxx, phen-fen (sp?) and all the other inappropriate life style drug approvals combined.

It would seem too late this year, but perhaps next year we could build on the success of the MD Care Act which made the NIH accountable for translating basic research into usable drugs and standards of care for neuromuscular diseases. Lean on Congress to hold hearings to have the head of the FDA come down and identify which conditions have orphan drugs in the approval process, where they are in the approval process and how many people died of that condition since the last hearing. That would get old real fast for our friends at the FDA, and I'm sure that they would be a little less cautious and a little more generous with their funding and time.
Paul--I agree that we need to ramp up the focus on FDA process as part of our legislative agenda. It's also important that Congress be more generous with their funding of FDA for orphan products. We should couple something like your suggestions for increased accountability with the means to carry out the program. We're planning to send a letter to the FDA from Congress as part of this year's agenda--perhaps that can serve as a "shot across the bow" in preparation for ramping up.

Reply to Discussion

RSS

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2020   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service