Access to T DM-1

schoonder · 10-20-2011, 12:54 PM

http://southshorexpress.com/whitman-...ncer-drug.html

A recent WSJ article reported new FDA programs are being implemented by FDA Commissioner Margaret Hamburg to make the approval process clearer and more predictable for applicants. In particular elements in need to be improved upon were:.

1 - Timelines are long.
2 - Lack of clarity and consistency from FDA reviewers.
3 - Early dialogues between company founders and the agency will help smooth the regulatory process.
4 - New emphasis on personalized medicine, a burgeoning area that needs an updated approval strategy.
5 - Expedited drug development pathway for drugs that target threatening conditions and illnesses for which there is no great alternative already on the market.
6 - Unpredictability at the FDA.

http://blogs.wsj.com/venturecapital/2011/10/05/fda-tries-to-mend-fences-with-med-tech-start-ups-investors/?mod=google_news_blog
Since drug approval process is so closely tied to actual life and death issues, one would think Congressional Oversight Committee would assure immediate steps be taken to set right what now has been accepted to be a somewhat flawed approval practice, especially with personalized (targeted) medicine.
The T-DM1 refuse to file letter send to Genentech IMO was a direct consequence of a number of the shortcomings identified in that WSJ article.
There’s little to no risk involved for FDA to retract that RTF decision and to instantly start with examination of T-DM1 phase II results. Roche and partners are currently conducting THREE large, international phase III trials in 1st, 2nd and 3rd line HER2+ MBC setting. These data results when they are filed, the 2nd line study expected to complete by mid-2012, will guide FDA in further decision making.

phil · 10-21-2011, 07:21 AM

Thanks Schoonder. Several of you have e-mailed me , asking if Lorraine got to switch tx. to Boston . She did NOT !! Genentech decided to close access , and with it the flexible tx. plan we had been working with in Va. That plan reduced the dose , because of low platelets. The cancer shrank more, and plats , still low , stabilized over the past 3 months. Now Genetech says Lorraine must meet " new criteria " for a " new study ". We were shocked. It had been portrayed as a simple roll-over , to a " maintenance " study. And Farber , the t dm-1 trial hospital in Boston, said that since it wasnt a full-fledged trial we could get treated by our doc at MGH. But she has never handled t dm-1. Partners IRB, which owns MGH, got involved. And we are stuck between the two. First the FDA, now this ! So, we have met w/ our Congressional reps, and had to go back to Va. this week. To stay in the flexible plan that Gen. and Va. worked out.
The south shore express article you included as a link, is better on paper than on-line. It was shrunk to fit on-line.
A few in-accuracies, and the pictures should be captioned : Before T DM-1 ( on adria last Nov., w/ our youngest grand dau. ), and After T DM-1 ( last week ). Those pics are worth the whole article !!
We fight on , David fighting Three Goliaths !