Sure it’s possible. Biogen can go ahead and prove it. The FDA is giving them until 2030 to submit their final report, meanwhile we’ll be wasting billions paying for a medication that doesn’t work.
I get what you’re saying but in my opinion the crucial distinction is that the pathophysiology of hypertension leading to end organ damage is much better understood than the role that amyloid plays in Alzheimers.
Because of that I think the burden of proof should be on Biogen to prove efficacy and they shouldn’t be entitled to the benefit of the doubt no matter how awful the disease in question is.
It was apparent quickly that imatinib was a miracle drug. In Brian Druker’s phase I trial at Oregon back in 1998 that imatinib was effective. I can’t remember if the first arm had 30 or 31 patients but these were patients with very abnormal blood counts. ALL of them normalized within a few months. These patients were having neutropenic infections, requiring frequent blood transfusions, continuous bleeding problems because of low platelets and that all disappeared in a few months. A requirement to demonstrate survival would have been insane. Nearly dead patients were being restored to 100% normal function in a few months. Imatinib was clearly efficacious and I’m pretty sure approved by the FDA in record time in 2001.
This isn’t true. We were definitely treating CML prior to imatinib’s approval. The treatments didn’t work great, often had severe toxicity, and often involved allogeneic stem cell transplant but we were definitely treating (and every now and then curing) CML prior to imatinib.
I think you have raised valid points in this thread. But, I think the moral hazard that FDA has created by this approval is appalling.
I can follow Biogen’s model and pick an awful disease with few treatments options (and there are a ton of these unfortunately) and develop some innocuous phytonutrient or monoclonal antibody, do a few short trials to prove its reasonably safe, create some plausible biomarker, and ask the FDA to approve my drug while I’m running the efficacy trials. Five years down the road then the phase III trial is negative and my drug is yanked from the market - who cares, I’ve made a bundle.
In the oncology space, some pharmaceutical companies have been abusing the system for a long time and getting approval for drugs which sure showed some small statistically significant benefit but the clinical impact was meaningless. This Biogen debacle has now removed the efficacy barrier, all you need now is a plausible theory.
Just some back of the napkin math: There are 6 million patients with Alzheimers in the US. Let’s say that just 20% of them decide to seek out this drug or are candidates for this. It’s reportedly $56k for a year of treatment and that doesn’t count all the ancillary costs (imaging/infusion port placement/nursing administration).
So: 1.2M patients x $56,000 per patient = 67 billion dollars for just one year of treatment. Some patients receive this for years. It is just plain irresponsible to say we’re OK with spending that kind of money until 2030 without knowing if it is actually helping.
One of the guys who resigned consulted on my sister-in-law’s diagnosis of extreme early-onset a while back. (She was 55 when she was diagnosed, and didn’t make it to 59; hugely rapid decline in a very short span of years.
Dr. Cavazzoni worked in the pharmaceutical industry for several years and held senior executive positions in clinical development, regulatory affairs, and safety risk management in large companies across multiple therapeutic areas.
She’s corporate pharma. Maybe we shouldn’t have a fox in the hen house?
I’d bet everything I have that when she’s done with this position, she ignores academia and runs straight back to pharma.
From what other background would you draw qualified people? Serious question. If you make a rule for all top scientists that any involvement with corporations means they can never work in government, then all the top people will shrug their shoulders and . . . . never work in government. How would that improve things?
Like everyone in academia? You know how many MD PHd’s are not only developing the basis for new drugs, but running the controlled studies? There’s a lot of people who are a hell of a lot more qualified for this than corporate pharmacy execs. This is akin to having Boeing do their own regulation, and we all know how that worked out.
The person you were just complaining about had a background in academia as well. Academia doesn’t have a ton of high paying secure gigs for top people. The bright ones who want to retire with a good nest egg and stuff usually end up taking stints in the corporate world.
To re-iterate, a “no corporate background people in government” rule is functionally equivalent to a “no top people in government” rule.
Most of them are doing those studies after getting a grant from the pharmaceutical company.
Industry support is a major plus on an academic CV. I remember watching a lecture from a prominent researcher that started with something along the lines of, “I have no conflicts of interest to disclose today. Unfortunately. Hopefully soon I will, though!”
No. Past association with pharmaceutical companies does not mean there is a conflict of interest any time any drug is under consideration. That’s as ridiculous as saying that a Harvard graduate has a COI when reviewing an application from any Harvard professor.
Typically a COI means a drug you specifically worked on is involved. In this case there was only one person with a potential conflict of interest, and he was recused.
I think there’s so much money involved here it’s pretty obvious.
When was the last time 3 FDA members resigned out of protest over a potentially useless drug being approved that will cost the country billions?
Never.
That’s not what I’m saying at all. What I’m saying is it’s an issue this time. Tom Wheeler was an broadband industry rep before heading to the FCC. He used his knowledge from that to try and pass measures that were beneficial to the American people. This in its worst form will could cripple the healthcare industry and opens the pathway to no drug company needing to prove their drug makes marked clinical differences in their patients.
Then let me ask you a question. What’s wrong with Biogen doing another study before getting broader approval? If we’re going to be fair, then there’s 3 other drug companies who had similar or greater plaque reductions who are equally eligible to release their drugs on the market as well.
Mark my words there will be a huge uproar when insurance companies deny covering this that will put immense pressure on the system.
Because the FDA believes that reducing amyloid, all by itself, is likely to be helpful to patients. They are not alone in this belief, though obviously not everyone agrees with them.
And if that’s what they believe, then another study will unnecessarily delay benefit to a rapidly graying population.
If this were true, then why has every other study done by drug companies that reduce amyloid not shown improvement in the quality of life in their patients? Why has every other drug been shut down that does the exact same thing?
This is trump era FDA legislation being used to push this out. While you underhandedly call me a conspiracist theorist, you’ve dismissed every single individual who’s brought legitimate arguments that also say this should never have been approved including a brilliant neuro-oncologist.
I want Alzheimer’s patients to have the best chance possible to extend their lives and retain precious memories and function. But we’re talking about breaking the healthcare system and selling false hopes. This is NOT the way to do this. If people want to contribute their minds to try the drug and continue the study that’s wonderful. But that should be on Biogen to support not everyone else.
