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Biotech / Medical : IDPH--Positive preliminary results for pivotal trial of ID -- Ignore unavailable to you. Want to Upgrade?

To: DMB who wrote (1435)	1/18/1998 2:38:00 PM
From: Bob L	Read Replies (2) \| Respond to of 1762

As long as you are dropping in on this conversation, I thought I would too. You raise a very interesting point on "compassionate use" approvals. I have a different perspective. My father has NHL. After the favorable FDA advisory committee recommendation for Rituxan in July, he inquired about treatment IND ("compassionate use") at both the large HMO where he is a patient, and a regional university medical center that had participated in the Rituxan trials. After some hemming and hawing, it became clear that none of the doctors were interesting in tackling the paperwork. One doctor said by the time they got approval for an individual protocol for my father, Rituxan would probably be on the market anyhow. These are compassionate and helpful doctors, not unreasonable people. It took almost 4 more months for Rituxan to get final approval. I'm sure it wouldn't have taken that long for IND approval. But it shows the practical obstacles a patient faces in finding a willing doctor, a willing drug company, and getting FDA clearance. Coulter, for example, says they are a small company and not interested in the burden of getting treatment IND approval for Bexxar. Perhaps this is justified by the manufacturing difficulties of a radioisotope-labeled product. I don't know. Eventually, my father got in the trial for Bexxar, but only after waiting months for his platelet level to creep from 95K to the required 100K. Any reasonable person in his position would have wanted to take the risk of treatment earlier. We understand the need for controlled studies, but not the need for dying while waiting for the results of the studies. I wonder if there are any statistics for the number of seriously ill people who have successfully obtained treatment IND. I suspect it is the proverbial drop in the bucket. And many of those are probably AIDS patients, who are an organized group that has been pounding on the FDA for years.

To: DMB who wrote (1435)	1/18/1998 10:28:00 PM
From: Maurice Winn	Read Replies (3) \| Respond to of 1762

DMB, thanks for your comments and welcome to the world of Silicon Investor - I see you are a new entrant. I can tell you that after almost two years, this has been a very helpful "place" to be. More than that, a sociable place. I hope you get a lot from it. Giving more gets more - a single post can initiate a swarm of expert commentators, such as yourself and all can share. It's great. Synergy. Synchronicity, if that's a word. As you can see from the previous posts, some patients are not finding the approvals system user-friendly. A little paperwork can be daunting. What seems simple to the bureaucratic mind is not so simple to we ignorant users of such systems because our lives don't revolve around the systems. Bureaucrats love their bits of paper, systems, protocols, permits, approvals, licenses, and other unknown, undiscoverable and capricious barriers to the full expression of an independent mind. I can tell you that anger, frustration and fear is what the person being "helped" by the bits of paper feels. At least the trend is towards independence, but it seems that they don't yet have a bunch of happy campers. My son has newly diagnosed NHL and getting past the bureaucratic barriers seems to be the main occupation in getting appropriate treatment. Everything I have read leads me to believe that Rituxan treatment in conjunction with the old-style cures I mentioned would improve his chances of living with few side effects. But will he get the improved chances? No. No approvals you see for Rituxan in stage 1A, intermediate grade diffuse large B-cell lymphoma, 2cm cervical tumor excised. What do you think my opinion of medical systems and approvals will be if in 5 years time, my son is dead and they have discovered after many approvals and chi-squared investigations that the very benign side effects of Rituxan would have been the way to maximize his chances? Dead people make good press because the press readers are fearful of being dead, fearful of authority, untrusting of compassionate bureaucrats and the bodies are the proof of the problem. As two correspondents here have said, FDA compassion doesn't seem to get the right goods delivered to those who might die. We get a double whammy in New Zealand, because after the FDA has had a chew on the product, the New Zealand authorities want to give their stamp of approval. After lunch and the backlog has been dealt with of course. I want Rituxan available now. There is plenty of clinical evidence which shows it to be effective and fairly safe. Who is stopping it? Is it possible to go to the USA and get off-label treatment with Rituxan? IDEC has gone to the trouble and expense of developing a way to save lots of people from the scourge of lymphoma linked to CD20 and whatever other apparently unknown factors determine its success. They have the product on the shelf. There is a customer sitting here waiting. Now we watch and wait? The Rituxan compassionate use program I guess was for people near death's door rather than those who were heading that way who might have chosen to use it in its relatively unproven state. Thanks for your outlook on the approvals process and compassionate use. Okay, away to soak up some sunshine and maybe give grist to the mill of the melanoma researchers. It's such a tough life, trying to balance enjoyment with risk. I wish we had cast iron DNA! Please excuse my, umm, intensity? I guess this is as hot a button as I have [my child in real danger] and I'm usually more benign. Though some would disagree. Anyway, happy hunting on SI. Maurice