Extraordinary Measures: Biotech Meets Hollywood
19 CommentsBy Ed Silverman // January 20th, 2010 // 8:10 am
For the past few years, the John Crowley story has been widely circulated. A former financial consultant with two children suffering from Pompe disease, he borrowed $100,000 on his home and 401(k) plan to start a biotech company, raised $27 million in venture capital and later sold his small company to Genzyme. That’s where the drama really begins, as Crowley and Genzyme tussled over the trials and whether his children could participate (more here).
The saga is now a movie called ‘Extraordinary Measures,’ which stars Harrison Ford and Brendan Fraser (who plays Crowley), and opens this week. You can watch the trailer here and Crowley, who is now ceo at Amicus Therapeutics, was interviewed the other day by CNBC, which you can watch here.
pharmavet
I can’t think of a better example of why we need to preserve the R&D tax credit for Orphan Drugs. My concern is that under Obamacare we will adopt the principles of his medical consultant, Dr Ezekiel Emanuel, a staunch advocate for health care rationing. Based on Emanuel’s article last year in Lancet (”Principles for Allocation of Scarce Medical Interventions”,Persad, G et al, vol 373, p 423-431), Emanuel proposes that we allocate medical resources based on quality-adjusted-life years (QUALY’S). In this case, the elderly and the young would receive the fewest resources. In the case of the elderly, they don’t have enough QUALY’S remaining to justify much in the way of end of life care. In the case of sick children with terminal illnesses, (e.g. with Pompe’s Disease, the subject of the movie) they don’t have much in the way of QUALY’S to begin with. As far as I’m concerned, a victory for Scott Brown in Massachusetts was a victory for sick children everywhere if it moves us away from Obama/Ezekiel style health care rationing.
Dianne
Great argument. What is your proposal for individuals with pre-existing conditions and no health insurance?
pharmavet
Dianne, I don’t have a proposal, but I’m pretty confident that under the Obama/Ezekiel paradigm, with the reality of limited resources into the forseeable future, depending on the peron’s age and expected QUALY’S remaining, they may or may not get the health care they require, notwithstanding their pre-existing condition and lack of insurance. Don’t be sold on the idea of “Universal” health care. There may be greater availability under Obamacare, but Ezekiel is correct in saying that it will always be resource constrained, whether those resources come from the Federal Government, private insurance companies, or both.
Former Pharma Marketing Director
Hey Pharmavet,
We are not going to see health care rationing. Why? because it is unethical and WE THE PEOPLE know it, and so does Obama.
This story is about innovation, tenacity and self advocacy. It is also a story about keeping everyone focused on the goal of the project - get the drugs into the children.
We can do more and we can do better. But we have to stop the fear mongering, nothing good comes from it..
pharmavet
Dear Marketing Director. It costs approximately $300,000/year to treat one child with Pompe Disease, which insurance companies generally pay most of the cost of. Under Obamacare, with insurance companies profits squeezed to the max, do you really think that they will continue to pay to treat these rare diseases? I don’t think so. This is not fear mongering. This is the reality of the situation. You are right. The advocacy squeaky wheel gets the grease. But under Obamacare and rationing, there will be no grease (i.e. money) left to grease that wheel. Furthermore, if we go to a single payer system, how willing do you think that the Feds will be to fork over that $300K/year for each of theswe children? Again, not fear-mongering, just reality.
PharmaStudent
I like the story… and am a big advocate of caring for people - especially children - with such devastating diseases. My heart goes out to this family. I took a different approach however. I FIXED my children first, and now, am contemplating starting a pharma company. Advocacy is great; Business is great; the VC community is great; but honestly, if you’re in the game for your loved ones, then FIX THEM FIRST. Others can wait.
pharmavet
I applaud Pharma Student. Back in 2000 when I was involved with a start-up pharma company, most anyone with Powerpoint presentation, a Brooks Brothers suit and a decent business plan could get a few rounds of financing. We plugged at it, wound up with $150 million through five rounds of financing. Got three NDA’s approved in four years, sold the company off to Big Pharma in 2007 after a few failed IPO’s, and repaid our investors (and ourselves) quite handsomely. Definitely high risk, but high reward. Much tougher VC situation now. Get used to a few rejection letters, but if you have a dream and an entrepreneurial spirit, then by all means go for it.
Anonymous Patient
Dear Pharmavet,
I side with Former marketing - I am on a life saving drug in a country that covers it…..No rationing, stop hypothesizing…..
Former Pharma Marketing Director
Hey Pharmavet,
I can’t find the link on this website, but not too long ago Ed presented a story about a patient on a drug for a rare disease, PNH - treatment cost approximately $500 per year, insurance companies are covering it. It strikes relatively young adults. They lead a normal life….
That being said, I think a few things are likely to happen, one of them is negotiation, which may sound like a bad word to some people, but really it is time. The cost of some of these drugs have gone beyond what is needed to pay back investments and profits. You and I both know that.
I am not worried about rationing…Pharma is going to have to sit down to the table and sharpen their pencils….
pharmavet
Marketing Director: There are several things to consider 1) high fixed manufacturing costs. I worked with Genzyme and know something about manufacturing these proteins. Because of dangers of cross-contamination they have to be made in dedicated bioreactors, which cost $75-100 million each, 2) the R&D tax credit for orphan drugs has an expiration date of seven years post-approval,3) given the rarity of these disease, there is essentially a fixed market which can’t be grown by Genzyme or any other company that makes these drugs, 4) generic biosimilars will eventually lower the price of the drugs by competition.
Genzyme and perhaps other biotechs have had much lower cost technology for making recombinant proteins for a number of years but have hit many regulatory roadblocks from FDA. Rrecently the first drug, antithrombin III, made in transgenic goats has been FDA-approved after many regulatory hurdles. As one Genzyme colleague told me, instead of spending $75 million on a bioreactor, mostly what is needed is a breeding farm and lots of grass for grazing. Eventually more products like Myozyme will be made in transgenic animals, which should translate into lower drug costs.
harpy
There are kids dying now from common, treatable illnesses because they don’t have health insurance. What are they worth?
Former Pharma Marketing Director
Pharmavet,
Then we need to get out of the business, there is no sense trying to make these drugs if the prices we have to charge make them inaccessible.
In fact I think it is time for us to stop fooling ourselves and just pull away from this. Our greed have has surpassed our ability to innovate and find ways to bring these important discoveries to market.
Maybe it is time we just stopped trying to figure out how to save humanity and cure and treat diseases, who are we kidding anyway?
Once we find treatments we stick a gun in the patients backs and say “your money or your life” I’d rather die rich and leave the money to the kids and hope they don’t ever get sick.
As far as lower drug costs, malarkey….No one will lower their costs, why should they? the market bears the outrageous prices we charge.
I have been involved in many new drug innovations. At the end of the day we way over price it, take huge profits and spin stories to convince ourselves that what we are doing is right - it isn’t it never was and it never will be.
I sleep better at night speaking the truth and unlike you I do not have to worry about the lies I tell myself and anyone who will listen. There is no way to justify the wrong that we have done - no way…..
Keep telling yourself your B.S. It is going to change, it will have to. Sick people will realize that we are playing them for fools and they will just stop worrying about it…Most of them will o what they should do, walk away from the treatment…Walk away from the doctors it is all a game of grabbing money - when it comes down to it, we don’t really care, just give us your money….There is nothing any different from this and snake oil sellers, the only difference is that we have bought off our government and tricked them into believing that what we do is right. More often than not, it isn’t….
pharmavet
Marketing Director, in case I wasn’t clear, Genzyme and other companies have developed excellent programs to help insurance largely defray the cost of these orphan drugs to patients for as long as they need them. Patients pay only a small fraction of the cost. Actually I sleep better at night knowing that these sick children with inborn errors of metabolism such as Gaucher’s Disease, Fabry’s Disease, etc have life-saving drugs to prolong their lives. Shire Pharmaceuticals has a BLA approval pending for a Replagen for Fabry’s disease to compete with Genzyme’s Fabrazyme, and has a drug in development for Gaucher’s Disease to compete with Genzyme’s Cerazyme. These generic biosimilars will lead to more choices and lower costs.
I really didn’t completely understand your post. Are you suggesting that we become some sort of neo-agrarian pre-Industrial Revolution society where disease were treated with herbs and potions? Please explain.
Former Pharma Marketing Director
Pharmavet,
We don’t treat people we find ways to get at their money.
Pharmaceutical companies need to stay out of drug dispensing programs. People who qualify for them have to declare that they are essentially bankrupt, they are not allowed to own anything, other than a car in most cases. Other patients turn to charitable organizations.
What I am saying is we should stop the chain of blame. We say we charge so much because, because so and so charges so much for clinical trials, or another company charges so much for a specific technology, all to work hard to “hide” the price. People charge what they do because they have seen us get away with, well robbery and murder…
How long will these children be left on these drugs, how bankrupt will their parents become. I have worked with AIDS families (AIDS received courtesy of blood transfusions) who have children and have to decide which one walks to school and which one can take the bus. Or what kind of food they can afford to eat. We force these people to come begging to the very companies who charge the prices for the drugs.
Sure people are happy to be alive, but what good is it when we are making them so broke they cannot afford to actually live…
pharmavet
Just a few facts:
1)Cerezyme is covered under most private insurance plans
2) For uninsured patients the income declaration requirements for Cerezyme are no different from any other patient assistance program.
3) in many countries outside the US, Cerezyme is provided free of charge. I’ve attached the link below.
http://www.genzyme.com/commitment/patients/free_programs.asp
I saw the movie last evening and was disappointed in its factual inaccuracy. If I spoil the plot for anyone wanting to see the movie, don’t read further. I’ve read every book written on clinical drug trial methodology, and never have I come across sibling studies as a valid model. When Fabrazyme was initially approved for infantile Pompe Disease, it was done so on the basis of historical control. The ongoing trials in older kids (i.e. the Crowley children) is a more conventional placebo-controlled trial. My guess is if the placebo-controlled nature of the study were told in the movie, it might expose potential ethical concerns that might interfere with the happy ending of the movie.
Save your money on this one. Brendan Fraser should stick to action movies but should lose about 30 lbs first. Harrison Ford just plays a grumpy old man. Kerri Russel should have stuck to modeling. Roger Ebert gave the movie 2/4 stars. To me that was generous.
laura
Pharmavet,
Since you have worked with Genzyme and seem to know a great deal about the clinical trial process I’m wondering… how is it possible that a clinical trial for Genzyme’s Seprafilm disqualifies participants with known allergies to hyaluronic acid when there is no such warning in Seprafim’s labeling? Is this an FDA issue, a Genzyme issue, or a little of both? I don’t want to interrupt the discussion here, but I am interested to know your opinion on this.
Former Pharma Marketing Director
Pharmavet,
I am not just pointing a finger at Cerezyme.
Mostly all the patient assistance programs require that patients be on welfare. That is a problem…
On the other hand, I am not saying that companies need to give away their drug. I think a certain amount should be put aside and handled by the government directly.
These diseases do not only strike people with money and great insurance programs, we know that. I have worked with many patients, as a whole we are way off the mark.
Any company that brings a life saving drug to the market that requires hundreds of thousands of dollars a year, hasn’t done anything, except show these people once again that they are third and fourth class citizens in their own country…
We have to do better…
pharmavet
In general it is standard language of a clinical trial protocol to exclude patients who may be allergic to the active ingredient in a drug. I don’t know why such language is not in the labeling for the approved product. As for the inherent allergenicity of the product, i’ve referred your question to a board-certified allergist. I’ll let you know what I find.
laura
Thank you, Pharmavet. I’ll be interested to hear what the allergist has to say.