The FDA and Life Saving Drugs - Redux
This post is not directly about investment - but read it anyway, I had a lot of coffee this morning and it shows.
Earlier in the week a Federal circuit court reversed a decision that would have allowed a dying woman - now dead - access to drugs not yet approved or rejected by the FDA. The legal logic was the FDA had the statutory authority to do so. The court seems to have forgotten a central underpinning of our Constitution, our legal system and, if you push back a bit, English common law - the natural rights of man, which, while not included explicit in the Constitution, are assumed in the Constitution, and this is the right to live.
An op end piece in today's Wall Street Journal covers the legal and constitutional side of this issue far better than I can - but I have been preaching about this longer than the guy form the Cato Institute - not a favorite of mine, but this was a good piece nonetheless, you should read it - and my concern here is the more tactical issue of drug approvals.
Simply put, if a patient is dying, and a drug has trial data that shows, with statistical certainty that it is safe, effectiveness should be measured by the patient's willingness to use the drug and eventually the marketplace. I am not talking Chinese herbal remedies here folks - I mean advanced cancer and other therapies rejected by the FDA because the arrogant scientists (that is a multi-syllabic phrase for morons) running many biotechs have screwed up their clinical trial design and process. Notice I am not blaming the FDA here 100% -- it is 50% -- an that really is the right number. My degree is in philosophy from Georgetown and I could design clinical trials better than more than half the biotechs I cover. But the 50% the FDA is responsible for is a regulatory pathway that puts far too much emphasis on statistics, ignores patients and is going the wrong way from the creative and innovative leadership of Dr. Mark McClellan, who left the FDA a few years ago and things have not gone right since.
Let the market decide - am I not a total, Cato Institute free market, screw the great unwashed, compassion has no role in society type - and it will. A cancer treatment may work for 5% of potential patients - and will probably fail in trial. Why should those 5, or 500, or 5,000, depending on the kind of cancer, die because of statistical metrics? What happened to Oliver Wendell Holmes (actually, a real nasty piece of work but a great writer and jurist) when you need him - I paraphrase here - "I would rather let a thousand guilty men go free than convict one innocent man?" (might have been a hundred, Google is slow today). How about the FDA saying "I would rather have one thousand patients waste some money and save one dying human soul." Or something like that. Actually one in a thousand is not practical.
Ah, here is the rub. The market really cannot be allowed to determine everything - there needs to be some reason in this - but right now all reason is thrown out the window by FDA statistical fascists who do not have the moral courage to take a chance, to stray beyond the fence of statistical certainty, to save a life. Let me brutal here - the staff decision to reject prostate cancer treatment Provenge, if it worked on just 10% of patients, will kill in one year twice as many Americans than have died in Iraq, showing Shia and the Sunnis the beauty of democratic government in a tribal society, since George the Light (his father was the real George or George the Heavy, a very fine public servant, independent of your views of his politics) began the war.
The solution? A streamlined regulatory pathway for drugs for the terminally ill that, if proven safe, can come to market based on efficacy data less rigorous than what is demanded today. And this will need to be put in place by force of law. The FDA staff is morally and professionally incapable of acting this aggressively to create its own regulatory pathway and will not be led by the current head of the FDA Andrew von Eschenbach. He is one of the most ineffective and reviled bureaucrats in all of Washington, just as we was when running the National Cancer Institute, the primary notch on his resume that landed him on the jobs his friendship with George the Light and being from Texas.
Did I wake up on the wrong side of the bed this morning? No, but if Provenge works on ten percent of patients, then a few more fathers and sons and husbands and heroes --- just like our boys and girls in Iraq - will not wake up at all this morning. Let us hope this circuit court decision, which is about a different drug entirely, is appealed to the Supreme Court and the great, fighting protectors of human life - who oppose abortion but are also ardent supporters of the death penalty - reverse this reversal in the name of the sanctity of any human life. And the power of the patient and the marketplace.
Comments (2)
Hello Mr. Michael Shulman.
Nice job on your article covering the FDA shortcomings.
There is a Pulitzer prize waiting for journalist/commentators looking into the Provenge saga - I think you are aware of this revolutionary treatment for prostate cancer made by a Seattle company named Dendreon.
I'm concerned about how the FDA handles its job. We need direct and very bright sunlight into the FDA back rooms.
I know that many people requested that the FDA explain to the people and their medical doctors why it chose not to approve Provenge, even though it was advised by its appointed panel of Oncologists, Urologists, and Immunologists, all experts in the fields of immunotherapy and oncology, that Provenge is unquestionably safe (100% of the experts voted yes on safety) and that there was 'substantial evidence' of the drug's effectiveness as required by FDA regulations (76% of the experts voted yes on substantial evidence of effectiveness).
Among the NOs, Dr. Sher and Dr. Hussain.
Provenge achieved a 200% increase in overall survival measured at 3 years... (which means three times BTW) with multiple recipients alive and well over 6 years later.
27,000 men with end-stage prostate cancer will die needlessly early (some will die needlessly period) each year the Provenge is delayed by the FDA.
If FDA found the clinical trial data submitted by Dendreon (Provenge producer company) to be insufficient, why did it even submit the data to a panel of experts for their opinions?
Some of those doctors that voted "NO" as to the effectiveness evidence even had letters (urging the FDA NOT to approve Provenge) "leaked" to the media. They were Dr. Sher and Dr. Hussain. I let you judge on the ethics of this behaviour.
A couple of weeks after the FDA delaying letter regarding Provenge, on May 30, 2007, news was that Schering-Plough Corp. said it had agreed pay Novacea Inc. up to $440 million for rights to develop the smaller drugmaker's prostate cancer treatment, causing Novacea shares to double.
Both Dr. Sher and Dr. Hussain are Novacea consultants.
Do you think Schering-Plough would have paid $60 million immediately and an additional $380 million to Novacea if Provenge was on the market?
Subsequent research shows Dr. Sher is also involved in Proquest, a healthcare venture capital firm with over $875 million under management. Dr. Sher is a Scientific Advisory Board member.
Proquest owns shares in Novacea (now, an interesting question would be: Does Dr. Howard Sher own shares in Novacea?).
Why the FDA - for the first time in its history - did not approve a drug deemed safe and effective by its own panel?
How is that Dr. Sher and Dr. Hussain - with their obvious and enormous conflicts of interest - took part in the panel?
Who did choose Dr. Sher and Dr. Hussain?
We already have proof that Provenge works (two phase III trials). Many people are going to die due to the delay. Who is accountable?
Why isn't this revolutionary and extremely safe treatment available NOW?
I can't imagine the motives are about monetary gains. Otherwise, here is the Pulitzer. That would be huge.
I wish you all the best.
Posted by phil_vardena (Pharm. D.) | August 10, 2007 3:05 PM
Posted on August 10, 2007 15:05
3 cheers for your extra coffee today and your incisive article on the seemingly never-ending Provenge saga.
While you may be right on dividing the blame equally between the company and the FDA, where do you see the following "outside influence" apparent nefarious action coming into play?
Scher reported only 3 COI's to the FDA per his filing in late February available at the FDA website which appears to be composed of ownership of 1 stocks and 2 competing industry interest.
Internet research shows the following for Scher thus far:
1. NOVACEA: grants & research support; STUDY CHAIR of DN-101; Direct competitor to Provenge
2. GPB BIOTECH: financial conflict of interest per Scher in MedPage
3. PHARMION: financial conflict of interest per Scher in MedPage
4. SANOFI-AVENTIS: grants & research support
5. BRISTOL MYERS SQUIBB: consultant, grants & research
6. MILLENNIUM PHARMCEUTICALS: grant of research support
7. COUGAR BIOTECHNOLOGY: principal investigator; advisory board;
8. INNOVIVE PHARMACEUTICALS: principal investigator
9. INFINITY PHARMACEUTICALS: principal investigator
10. BIOGEN-IDEC: jointly held stock with spouse
11. PFIZER: jointly held stock with spouse
12. GENTA: scientific advisory board (as of March 6, 2007; since removed from web, but cached)
13. DEPARTMENT of DEFENSE: Principal Investigator PC Clinical Trials-P1 and P2
14. PROQUEST INVESTMENTS: consultant, scientific advisory board; Limited Partner FINANCIAL interest
15. MEDIVATION, INC: principal investigator MDV3100
16. AMBRILIA BIOPHARMA INC: Principal Investigator PCK3145, Phase I/II (Announced April 7, 2007)
There appears to be a significant and disturbing difference between his 3 disclosures to the FDA and the alleged 16 COI's found so far on the internet. It seems incomprehensible for someone with the training and education Scher has to "forget" so many apparent COIs... that is simply beyond all reasonableness and suggests intentional dishonesty or deceit.
Even more disturbing is the FDA's Office of Inspector General and others at the FDA to whom this information has been supplied and their apparent failure to investigate these charges that Scher has more COI's than he disclosed to them. When such information is provided and such allegations of potential federal law violation is involved, any ordinary citizen has the right to expect our government to investigate such allegations timely and thoroughly.
As a "temporary government employee" at the FDA, he is obligated, under Federal law, to provide full and complete disclosure. If he didn't, he may have the potential for liability for law violation(s) as well as for providing false data to the FDA under which he secured a waiver in order to participate on the Provenge AC meeting March 29th.
Posted by Tony F | August 11, 2007 12:06 AM
Posted on August 11, 2007 00:06