Tysabri & Unequal Access: Nancy Berlinger Explains
24 CommentsBy Ed Silverman // October 20th, 2008 // 2:18 pm
Fred Baron is a prominent Dallas trial lawyer and Democratic Party fundraiser, who drew national headlines twice this year. First, he helped bankroll John Edwards’ presidential run and paid for the former senator’s mistress to leave North Carolina after the affair was disclosed. Last week, we learned the 61-year-old is dying and his family succeeded in gaining access to Biogen’s Tysabri to treat his multiple myeloma after reaching out to the FDA commish and getting Hillary Clinton and Lance Armstrong, among others, to contact the biotech. Why the fuss? Tysabri is approved only for MS and Crohn’s disease, and was briefly off the market after some patients developed fatal brain infections. That’s why Biogen balked at making the drug available (back story here and here). We spoke with Nancy Berlinger, a deputy director and research scholar at The Hastings Center, a non-profit bioethics research institute about compassionate use and Baron the dilemma. This is an excerpt…
Pharmalot: We know compassionate use is sometimes recognized for patients with no options, but what made this case unusual?
Berlinger: Yes, compassionate use is a recognized exception to normal research protocols. It does acknowledge that there may be something not completed in clinical trials and, therefore, is still in the investigatory phase, but there are individuals who are not research subjects who could benefit. Both the research etablishment and cancer care world recognize the situation of someone like Mr. Baron, who has a very serious illness with no further treatments available. What’s unusual here, well, he’s extremely well-connected. Not everybody has this many people who can make those phone calls. And the drug itself has been known to cause serious side effects.
Pharmalot: In explaining its decision to resist making its drug available, Biogen repeatedly stressed Tysabri is still in Phase I testing for multiple myeloma. Why should that matter?
Berlinger: You don’t want to write everything as off-label for these very serious conditions. You want to see if the benefits outweigh the burdens. Remember that Phase I involves toxicity studies. There’s a difference between Phase I and Phase II. You can’t describe it as therapeutic, because it’s about toxicity - safely putting the drug into a human body…The solution isn’t to give everyone access to a potentially harmful drug. Then we won’t understand what is safe or not safe, and effective or not effective. You can’t do an end run around trial protocols. What if everyone picked up the phone? Then there’s no trial left and you’re not able to understand how the drug workss under proper conditions. And you’ll never get to the gold standard of Phase III.
Pharmalot: So was this fair? There are probably others out there who would like the drug, too.
Berlinger: The fairness issue speaks to everyone, not just this particular case. What if Lance Armstrong can’t make a phone call for you? We can have enormous symapthy for Mr. Baron and his family. What would you do if it was you or a member of your family? For most people, you can go to the clinical trials registry but you can’t get in a trial, because you don’t fit the trial critiera or it’s Phase I and not therapeutic. It gets to the heart of a difficult issue…If he’s been given a drug, it suggests he’s not eligible for a trial. That’s becuase if it’s compassionate use, it suggests he’s not eligible. And remember, it doesn’t suddenly mean the drug is therapeutically beneficial.
Pharmalot: What about those who would argue Tysabri is already on the market and so why not?
Berlinger: This is an approved drug, yes, but it’s only in Phase I testing for his condition. One could argue this is approved and the trial is just a study for wider use, but there is the fact of those brain infections. We can’t say it’s an approved drug and that’s good enough. We have to look at the whole picture.
Pharmalot: Would you say Biogen was damned if it did and damned if it didn’t make Tysabri available?
Berlinger: The company is saying they don’t want to shut down availability for people who can benefit from an approved use. But there’s no perfect answer. I will say that it would be a shame if the lesson we draw from this is to get lots of people to make phone calls to get you a drug…The company does have a very difficult situation - there was intense and very public pressure to make a drug available…There’s a reason toxicity studies are being done under controlled circustances, because there is something to be learned about this drug after it’s given to people…There could be something that could actively harm him… They don’t want to deviate from the conditions under which the drug is available to patients with MS or Crohn’s.
Pharmalot: So should Tysabri have been made available to Baron?
Berlinger: Well, you don’t want to create work-arounds for the process of determining whether a drug is safe and effective, because if you do, you’ll leave the clinical trial process in tatters… This raises the question of unequal access - who is going to be able to make those petitions? People with means and clouts…. But when something is in Phase I trials, you don’t know if it’s the best therapy or any good at all…We want people to be advocates and knowledgeable, but what happens when someone crosses the line and asks for something way up the pipeline?
The only thing I can say is the answer is unfettered access to any drug at any stage in the pipeline - it undermines the idea of a research protocol. And we have to find evidence of safety and effectiveness. If we say anybody gets access to anything at anytime, how will we ever know? The way to satisfy fairness is to have the strongest and most expeditious trial protocols we can find.
rocky roquemore
so you are not going to answer the question of unequal access, only admit it exists.
WE HOLD THESE TRUTHS TO BE SELF EVIDENT.
also note ammendment 14 to the US constitution
Steve
This lady would make a good politician as she seems to circumvent the case at hand.
She says, “There could be something that could actively harm him”. At this stage of the matter, who cares? This man has tried everything and this may be his salvation. Otherwise, death is the outcome. She scares me. Very cold!
All I see is a woman (the writer) as going “by the book”, no matter what.
Former pharma Marketing Exec
This is a good interview.
We do not know for certain that Mr. Baron wouldn’t be a candidate for the trial, because we do not know the trial protocol. Usually, Phase I - toxicity studies (in cancer anyway) enlist patients who have nothing left to try. Makes sense for ethical reasons. Nancy Berlinger should have made this point more clear, because this is a critical point.
We would all probably find more balance to this story if we knew how many patients were being accrued for the phase I trial (all obviously unknown’s?) Given what we know about Phase I, why was Mr. Baron not considered to be a candidate under the auspices of the trial protocol?
Actually, I only agree to unfettered access to any drug when there are no other options to try. It is an urgent matter because time is of the essence.
The other side of the story is this: we do not know the safety of this drug for MM. So, Mr. Baron could actually be doing more harm than good to himself. His money and fame could have bought him the fast track to bigger harm…
Enfermera
I’m on Tysabri for my MS. I went through a lot of red tape to obtain the drug, which Mr. Baron obviously has not. Would the average Joe with multiple myeloma get this medication? Can the average Joe get politicians and celebrities to call on his behalf? Hell no he can’t! I don’t have a problem with people who desperately need the medication getting it. What I have a problem with is some receiving more equal treatment than others.
Steve
Former,
You say, “could have bought him the fast track to bigger harm”. Do you mean something worse than death?
Trish
I have no problem with this man receiving the drug. At the same time, if it turns out to be a successful treatment, then I would hope we create a path for others like him to be afforded the same benefit. In this case, I doubt an insurance company would cover the drug for a non-approved use, so the other requirements is big bucks. Tysabri runs about $30k per year just for the drug, never mind the infusion process, etc. Now that to me is an even bigger problem. There are organizations that can help those who can’t pay for the big-ticket drugs, but I feel not enough people know about them. I also don’t know that they will cover something that is an off-label use.
I’m on Tysbabri for MS.
Trish
That would be “Tysabri”!
Paul B.
The problem here is the restrictive use monitoring program for Tysabri, TOUCH, prohibits the normal off-label use other drugs have. Otherwise Baron would have gotten the drug off label and no one would have heard of the case.
Because of the rocky road Ty has had to trod, Biogen was very guarded about its off label use. Ironically, I understand that Fred Baron made his living as a trial lawyer by suing drug companies just like Biogen.
And so it came down to preferential treatment by the FDA because Baron is connected. Ain’t favoritism grand? Surely this is how the Founders envisioned this country to operate.
As Ms. Berlinger explains, if everyone could do what Baron did the clinical trials would collapse, because no one wants placebo. The only answer I see is what reform organizations have called for - get the FDA to do a good job on drug safety, and get them out of the drug efficacy game altogether. Leave that to the docs and the patients. Within broad safety parameters, we don’t need the government telling us which drugs we can or cannot have.
Harold
I read an article today that stated “The Mayo Clinic is allowed to prescribe the drug as part of their clinical trials.” It would be very interesting if you could get to the bottom of that comment. It is from the URL I listed above.
I am also on Tysabri for MS and I have finally been able to put down my cane, after 13 years, and my cognitive problems are improving as well
I have no problem with Mr. Baron receiving Tysabri. In fact, the main reason Tysabri is available to MS and CD patients is because a large group of highly motivated MS patients, and their caregivers, descended on the FDA and basically said, We want our Tysabri and we want it Now!
There was no big money behind there effort, no politicians, and no well known superstars. It was a grassroot effort coordinated by people in need of this Wonder Treatment.
If it weren’t for the efforts of these courageous people, Tysabri might not even be here for MS and CD.
Jacquelyn
What many of you may not know is that Fred Baron spent a lifetime fighting for the rights of the underdog and common man. Yes he has made a fortune for himself along the way, but you will never find a person who works harder. While he is certainly controversial, hated by some and revered by others, one thing that is unquestionable is his tenacity and caring spirit. Fred acts from his heart, which more often than not gets him into trouble.
If Fred is somehow able to beat his cancer through the help of this medicine, he will most certainly make a second career fighting again for the rights of the underdog and common man, this time with respect to health care.
Like it or night, this is how change happens. For one, I think we are lucky to have him and I feel fortunate to have had the privilege of working with him.
He is a good man and I wish him the best of luck in beating this terrible disease.
Former pharma Marketing Exec
Steve,
Fast track to bigger harm, in all honesty means changing his current dismal situation for one that is even more dismal, if you can imagine that.
Paul B. You sound like you have additional information. Who says’ they are comparing this to a placebo in the phase I trial? Why are you implying that Mr. Baron “jumped” the trial so that he can avoid being randomized into a placebo? How do we know that? Where is that information?
I can’t imagine they would use a placebo in the case of MM.
Most Phase I trials that I know of for cancer are open label, it is to establish toxicity.
I agree with Trish - if this works for him, then I hope they get through the Phase I very quickly and provide expanded access to those who have no other options left.
Just A Thought
The thing that strikes me is that it has become a one man clinical study for the public view. I understand the drug company dragging their feet here. If their product is a dismal failure in this one man it could damage the drug’s future prospects and the chance for others to receive therapy, if only because doctors would have no faith to prescribe it. At a glance it seemed a chance to know the product without any risk exclusions that might color the clinical studies in a favorable light. Bet the drug company would have said no, despite the campaign from Washington, if the drug maker thought Baron was not a good candidate.
I don’t see Ms. Berlinger as cold, Steve. She was damned no matter what she said. Either she says ‘we cater to an elite crowd and will suspend our own procedures for them’ or ‘we are too heartless to offer treatment to a dying man’. It’s a lose/lose- unless it is wildly successful.
Just A Thought
Good interview, Ed. It’s always interesting to see how things will go from the hot seat.
Steven Walker
Good comments. It is important to note that Nancy Berlinger misrepresented (or just didn’t know) quite a lot in her interview responses. First, it has been reported that Fred Baron is not eligible for the clinical trial. The trial is listed along with its eligibility criteria, on http://www.clinicaltrials.gov. His docs say he is close to death. The trial criteria state that eligibility requires a life-expectancy of 24 weeks.
The Phase I trial is being conducted at the Mayo Clinic (where Fred is apparently being treated - probably by the same docs conducting the trial) and at one other center, so it is likely he was evaluated for entry into the trial and didn’t qualify. Everyone gets the drug in the trial, so had he qualified, that would have been the quickest (by far) way for him to receive it, and it would have been free at one of the best hospitals in the world. Second, it is a two-arm dose finding trial (two dose levels being tested) where everyone gets the drug. This is not a typical toxicity Phase I design in which groups of patients receive increasing doses until the maximum tolerated dose is acheived (characterized by the emergence of unacceptable toxicity), then they are cut off even if they are benefiting. This Phase I/II trial design for tysabri in MM is usually intended (and was in this case) to compare both toxicity and efficacy at two different doses. Bear in mind that a lot is already known about the safety of this drug, and the brain infections referenced occurred in only 3 of 3,000 patients with MS. Its occurrence has continued to be extremely rare after thousands more patients have received it as an approved drug for MS and Crohns. So far, over more than two years, no new cases have been reported in the US, and only a few in Europe where the use of other drugs for MS is somewhat different, and may be to blame for the rare PML cases they have seen there. The serious side effect is in fact so rare that by oncology standards, Tysabri is a very safe drug.
No one gets a placebo in the Phase Tysabri trial, which is described as a Phase I/II, meaning that this trial is designed to continue on right into Phase II efficacy testing. It is possible that Biogen and the investigators have very high hopes for this drug in this indication. Perhaps they have seen compelling anecdotal data from treating MS and Crohn’s patients who also happened to have multiple myeloma.
The Risk Management Plan (RMP) imposed on Biogen by the FDA prohibits off label use of the drug, which is unusual for a drug that has actually proven to be quite safe when compared to the risks of the diseases it treats; so why the strict restrictions on prescribing? The three cases of PML (the brain infection) arose after the drug was approved for MS but shortly after the Vioxx scandal surfaced, so this highly effective MS drug was taken away from patients who were benefitting, and held away from them for about 1.5 years. Their MS progressed while they waited, and progression is usually not reversible in MS. With Tysabri, sometimes it is - that is how effective Tysabri is for MS. When it came back on the market, no off label prescribing was allowed under the new RMP because FDA was in a get tough mode in response to media and Congressional pressure springing from Vioxx. So Biogen rightfully has concerns about FDA over-reaction to any stray safety signal based on direct experience. Remember, we are talking about three cases of PML out of 3,000 patients treated for an extended period in a carefully controlled clinical trial - about 0.1 percent. Experts admit they are not even sure the PML is caused by the drug. Yet, Tysabri was severely restricted by the FDA.
Nancy Berlinger also parroted some of the standard talking points about access crashing the clinical trials system. These arguments are simplistic and inherently false. Mr. Baron did not qualify for the trial. Is he and others like him to be abandoned to die because of that? He faces precisely the same risk of death from his multiple myeloma as the patients who qualify (the reason he didn’t qualify could be something like he did or didn’t have some prior treatment, or he had it too recently, or his docs didn’t think he had 24 weeks left to live unless he received successful treatment). He will not be allowed to participate in the clinical trials for this drug, and he will be long dead before the FDA gets around to approving it in a few years, should it meet the FDA’s standards. So how does allowing him the drug now leave the clinical trial system in tatters? It doesn’t. It is a ridiculous premise, and there are thousands of others out there with various serious and terminal diseases in the same boat, mainly because of an intransigent, obsolete, stagnant and unresponsive FDA.
A blog really isn’t the place to cover this very complex subject, but when looked at factually and objectively, the arguments being made against changing our system to allow access to investigational drugs for patients who have no approved options left and can’t qualify for a clinical trial simply don’t hold up.
Finally, this drug is simply not dangerous enough to merit the severe RMP it has. It is a drug that should be available for off label use. It is safer than a lot of approved oncology drugs that are not restricted in any way, and demand for the drug is not likely to climb until evidence of efficacy begins to emerge (if it does) - but ask yourself why oncology docs at the Mayo clinic (one of the premier medical facilities in the world) think Fred Baron should get this drug. They have started treating patients in the trial, and oncology docs are not generally taken to flights of fancy when it comes to aggressively trying to get a drug for a patient who doesn’t qualify for a trial. Maybe they are seeing something positive with the drug, or maybe they simply feel giving Baron the same chance their qualifying patients are getting is moral, ethical and medically reasonable. If it is the latter, they are right. We will know soon enough if it is the former.
So Mr. Baron is famous and has connections. So what? A life is a life and Mr. Baron’s is as inherently valuable as anyone elses. The real question is why the FDA doesn’t jump to the aid of non-famous people in his situation. Trust me. They don’t. We at the Abigail Alliance have learned this the hard way through the direct experience of trying to help our own loved ones and many others. That is the real problem. The FDA is supposed to work for us, not people like Nancy Beringer (who I am sure is a very nice, well-intentioned person who really cares, but who also is simply repeating the clinical trialing industry’s nonsensical talking points).
Biogen, by law, has to work for and protect its shareholders, and their view of the FDA at this point has got to be one of legitimate distrust (as is the case with most companies out there developing highly promisinig new drugs). So should they have allowed Fred Baron to have the drug when he asked? Yes. But their fears of the FDA are well-justified, and under the restrictions now hamstringing Tysabri, making it available outside trials gets complicated - not for the reasons described by Ms. Berlinger - but because they simply can’t trust the FDA to act in the best interests of patients and progress against disease. The real blame for why Fred Baron’s family had to call in some powerful chips so he could get his chance at continued life lays squarely at the doorstep of a stumbling, bumbling FDA.
Steven Walker
Abigail Alliance for Better Access to Developmental Drugs
Ed Silverman
Hi Steve,
Just to clarify, I may not have been clear when I spoke with Nancy about the fact that Baron was not eligible for the trial. If so, I will take responsibility for that. And while I won’t speak for her, to an extent, some of her remarks were aimed at such unfortunate situations, in general, not just Baron.
Also, I would disagree with the notion that a blog isn’t the place to cover this or any complex subject. This amounts to the third post here on Pharmalot about the Baron episode and these have managed to inform the audience about the issue and provided an opportunity to discuss it as well. The overriding issue has been on full display here.
In any event, thanks much for stopping by and filling us in some of the details and your views. The participation is always appreciated.
Ed
Nathan
Steven,
Thanks for your well-written post. It made me appreciate the “other side” of the arguement. I do have one question for you: One patient (Mr Barron) isn’t likely to have an effect one way or the other on the outcome of a clinical trial. But what about the other 10,000 people a year that die from MM? What if a significant percentage of them decided to request Tysabri? What happens to all that data? It amounts to an “uncontrolled clinical trial” in which we (in the industry) have almost no control over the experimental design. Don’t you fear that such a scenario could significantly skew the results as compared to a carefully controled study on a (relatively) homegeneous population that recieves the drug?
As I’m sure you are well-aware, there is a significant amount of mistrust of the pharma industry right now. The scientific method requires a carefully designed study that is geared towards answering a specific question. Only then can we really have confidence in the efficacy results. I feel that a system in which people are allowed to take a drug prior to proper experiments being done creates an uncontroled experiment which may undermine what little trust people still have in the pharma industry.
will create an environment that easily could create even more mistrust and ultimately delay the development of approved medications.
Steve
Nathan,
Good comments. I say, both sides have good arguments. I think your bottom line is correct but maybe, more compassion on the part of those that say, live by the rules and make no exception. I just can’t go along with that. I have had personal experiences and, to see someone die (denied the drug) that “may” have saved them with just maybe a miracle, is very painful.
Amgen did it with their Parkinsons study and the repercussions to this day still ring, including law suits, because they stopped the trials.
Maybe, someday we will have a meeting of the minds and work out a better system.
Paul
Randomized controlled clinical investigations are critical to the operation of our domestic drug regulatory system’s pre-market clearance process. Ordinarily, the system ensures a supply of human candidates for clinical trials by restricting access to investigational drugs, at least early on in the course of a drug’s development, to those individuals willing to participate in clinical studies where there is a chance, but no guarantee, that they will receive an active investigational agent. To allow selected individuals guaranteed access to an investigational drug while others (patients in trials) are not provided equivalent access is fundamentally unfair. Hardly surprising–just another illustration of how those with influence get what they want while those without it don’t!
Former pharma Marketing Exec
To Steven Walker,
I really admire the work you are doing with the Abigail Alliance.
Thanks for the informative post and clearing the air.
The world would be a much better place if our current system could be updated to allow every person with no other options left an opportunity to try experimental drugs. The information it provides could be invaluable and although it would not be from within a study protocol, it could be considered (bad or good) and add to the data.
I agree with what you are saying regarding Oncology doctors, they are not prone to flights of fancy and wouldn’t suggest a drug like this unless they felt it would help at all.
While I do believe the FDA has fumbled more often than not, I cannot let the role pharma has to play off the hook. There are famous cases of data suppression, manipulation, missing. So it’s happening on both sides and now neither one completely trusts the other. Because whether we agree or not, our systems are based a lot on trust and we have seen how the actions of one or the other has eroded that trust.
When both sides start dealing fairly and honest with one another, maybe the system will improve.
The blame for the broken system of today must be shared equally.
Tanin
Hi,
I think I disagree with a number of people who seem to believe this is a good example why compassionate use access should be more widely avialable.
To give an example say a hypothetical drug moved into a phase 2/3 trial with a placebo group and enrolled 120 patients (60 in each arm). All other patients are now ineligible for the trial. Using the compassionate use reasoning all these extra patients outside of the trial should also recieve the active treatment. This means that the only people that dont get access to the experimental treatment are the people randomised to the placebo group(not really a spectacular incentive to particpate!!!!)
My point could probably be stated better but hopefully people can understand what I am trying to say.
Steven Walker
Ed, I didn’t mean to imply that your excellent blog isn’t a good place to discuss this subject. Rather, I meant that fully explaining why all the arguments against allowing access to promising investigational drugs fail to hold water would take another 10,000 or 20,000 words, or more. Sorry for glossing over that point.
To those who think we can’t have both a robust clinical trials system and access to progress for those who can’t continue to wait, it simply isn’t true. In our Citizen’s Petition to the FDA (submited in June 2003 and to which the FDA has never responded), in our lawsuit, and in the Access Act (S. 3046) introduced in May of this year, we have always proposed that access would be available only to those patients ineligible or, for other legitimate reasons, unable to enroll in clinical trials. The protection of the clinical trials system is expressly built-in, which exceeds the protections of the system in current FDA policies and its draft regulations. Given that FDA can and as a matter of routine does, very effectively severely restrict the avilability of a drug like Tysabri, does anyone really doubt their ability to restrict what we now call Compassionate Investigational Approval (CIA) in the Access Act?
There also are some very pragmatic lessons we can take from the very few sizable compassionate use programs that have been run (and we have taken those lessons), where patients are first screened for entry into ongoing clinical trials, and offered access only if they are found ineligible. Folks this is doable and we should be doing it, but first we need a program that works for all concerned, including the sponsors. Our current programs do not, the FDA knows it and prefers it that way, and is in the process of promulgating new regulations that will cement policies into place that have failed miserably for three decades. Their message to most (in fact nearly all) people like Fred Baron who are quite forcefully abandoned by the clinical trials system is - drop dead. The FDA message is to most dying Americans is - the progress being made is not for you.
Folks, the people that is happening to is us - all of us. You and your families may be healthy now - but tomorrow? Next month? Next year? This is going to hit you one way or another, and you are going to have a different view of the faceless bureaucrat sitting at his/her desk near the beltway north of Washington who has decided you (and thousands like you) should die prematurely, untreated, because the FDA wants something called a p-value generated from statistical measurements of the accrual rate and height of two piles of bodies built in a double-blind, randomized, placebo-controlled trial using a drug that was already compellingly proved effective in earlier trials. Sounds like rhetoric, I know (and it is a bit by the choice of words), but it is also true much more often than most people realize.
Also, we are not and never have asked for unfettered access (a favorite disingenuous tactic used by opponents to attack our proposals). CIA drugs would become available only to patients who have run out of approved options and can’t get into a clinical trial, and only after the sponsor filed an application with the FDA for approval of their drug as a CIA treatment and received restricted approval in accordance with the approval standards set forth in the Access Act. Patients receiving the drug could get it only through and under the care of a qualified physician, and would be required to provide informed consent. Folks, this ain’t crazy. It’s rational, humane, and very much needed.
We are not proposing a wild west scenario and never have. I suggest to anyone interested in this issue to take the considerable time required to understand how our system is currently set up at the detail and implementation level, understand why it doesn’t work (these first two steps are critical and what you will learn will probably infuriate you), and then look carefully at our proposals to convert the existing access mechanisms into programs that will in fact work. The “understanding the status quo part is critical to understanding how minimalist and incremental our proposals really are. We are not crazy and we are not trying to crash the clinical trials system.
That said, there is broad and growing agreement that we have a 50-yaer-old, ignorance-based clinical trials and regulatory system that functions as the enemy of delivering progress to patients who have the diseases we hope to better treat. As for randomized trials being the only way to test drugs - we started doing them in 1962 (when Congress directed FDA to start regulating efficacy) as a sort of “best we could do at the time” solution, and we have never tried anything else. They really aren’t that great. In fact, it is very crude, scientifically weak, limited method that doesn’t tell us very much of what we need to know. They can ask only very narrow questions that are unrepresentative of the patient popultaion that will be treated after approval, and they give us answers only about populations and average reposnses within the population. but populations don’t walk in to doctors offices complaining of anything. how is this going to work for disease we now know are genetically diverse and individualized? It isn’t working because it can’t. it is like trying to drive a nail with a kitchen sponge? It is simply the wrong tool.
After almost 50 years of using a system that has barely worked for the diseases that are still killing us, one would think we would have reciognized that we need new methods and come up with more scientific, more effective, less expensive, and less barbaric ways to determine whether drugs work. One might wish we had also come up with new approaches that allowed our progress delivery system to accomodate the needs of real patients, who do not fit into the FDA’s one-size-fits-all approaches.
Fred Baron is one of those people who doesn’t fit, and all he wanted was to try an approved drug off label - something he could have done without even a phone call to the FDA or the drug company with hundreds of other approved drugs that weren’t, in the judgement of his highly-qualified physicians, the one he needed.
When a federal regulatory system, and the massively powerful financial and professional special interests of the clinical trialing industry, staunchly resist change of any kind (as they have for decades and are furiously doing now) to the way we test and approve drugs, and that resistance stalls progress and holds promising and even solidly proven new drugs away from hundreds of thousands of Americans for many months and even years, and those patients die prematurely waiting for progress to reach them (believe me - this is happening), isn’t it time to start changing things?
We have a big, big problem here. We have lost sight of why we started out looking for cures in the first place. Was it to perpetuate obsolete science and a stagant regulatory system? Was it to preserve stagnant regulation supported by professional and financial special ineterests? Or was it to make progress in the only place that really matters - in people who actually have these diseases?
So what should happen? Should all those people simply continue to wait and die prematurely, or should we consider this to be the major regulatory failure it actually is, and start implementing solutions? That is what we, our dying and late constituents and their families, and our advocate allies have been trying to do, and we could use your help.
Steve Walker
Abigail Alliance
paul_1149
Former Pharma Exec, I don’t have any inside info on the trial. I was speaking generally, that if people can get experimental drugs outside of trials, it would be hard to enroll placebo-controlled trials.
Someone
Dear Steve (from Abigail Alliance)
Thanks for your message.
You are right, it is already affecting each and everyone of us, whether we realize it or not.
We have lost sight of the reason we all exist in pharma, health care, etc. It seems the only reason we exist is to make money.
You said:
“When a federal regulatory system, and the massively powerful financial and professional special interests of the clinical trialing industry, staunchly resist change of any kind (as they have for decades and are furiously doing now) to the way we test and approve drugs, and that resistance stalls progress and holds promising and even solidly proven new drugs away from hundreds of thousands of Americans for many months and even years, and those patients die prematurely waiting for progress to reach them (believe me - this is happening), isn’t it time to start changing things?”
I just lost a very good friend, quite young, to this very situation last week. The drug he was denied was proven to work in patients but the drug company has chosen not to pursue that indication anymore.
“Mistakes” can be buried, dead people do not speak…
Mary Wehmeier
Ed,
An excellent interview which shines the light of day on both medical ethics and regulatory issues. While Nancy’s answers lead to more questions than answers, it proves we still will never have clear cut answers in dire situations.
The reality check I continue to remind everyone in healthcare is that “The patients are the reason we are all here. Without them and their needs, we could close up shop and go home.”
And this includes pharma and regulatory.