There’s a growing trend whereby patients have to pay to be in a clinical trial, which are called pay-to-play clinical trials.
Proponents of this idea point toward potential benefits in the form of patients gaining access to innovative therapies. They also argue that this approach provides a much-needed funding mechanism for the trials and that it could lead to more insurance coverage of therapies in development.
Opponents of pay-play-play argue it is unethical to require a patient to pay (sometimes large sums of money) to be a guinea pig to get a still experimental therapy that could do no good or even do harm. They also point to the risk that pay-to-play encourages more fly-by-night operations such as the dubious clinics in the stem cell field.
Is play-to-play worth the potential risks? Is it a trap? Is it instead liberating and helpful to patients?
What do you think? Take our poll and weigh in via the comments section.
Really good article on medium.com from Alexander Masters about this issue and his attempts to get funding for a oncolytic virus trial in Neuroendocrine cancer:
https://medium.com/mosaic-science/a-plutocratic-proposal-deba1f979bff#.qvedskg0j
“My idea was this: there are over 100,000 people in the world worth more than £20 million. According to medical statistics, between three and five people in every 100,000 get neuroendocrine cancer every year. So, three to five supremely wealthy people will have neuroendocrine cancer.
For £1 million, I was going to sell one or two of these plutocrats a place on Professor Essand’s bug trial.”
My assumption is that companies intending to gain FDA approval to market their products would follow FDA regulations for clinical trials so as not to risk rejection of data in marketing applications. FDA marketing approval is the first hurdle on the path to insurance reimbursement etc.
Bill Jones notes that ” Perhaps a smaller company doesn’t have enough money to both self-fund a clinical trial and stay in business? Maybe they can afford to pay some, but not all of the costs so they have to pass the rest through to the subjects in the trial. Otherwise, there will be no trial.”
Most if not all major contract research organizations (Quintiles, PPD, Parexel, etc.) will absorb costs for trials in return for licensing agreements. While it’s true owners of a small company would share any eventual profits with the CRO, these joint ventures surely beat no profit.
FDA cost recovery, i.e. allowing sponsors to charge for investigational drugs in trials, may be very helpful to small companies. Such companies should investigate the option because the worst FDA can say is no.
Check out FDA’s Small Business and Industry Assistance Program: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/SmallBusinessAssistance/default.htm
Why are we assuming that just because a company charges patients to cover the cost of a trial, that it’s profiting from it? Perhaps a smaller company doesn’t have enough money to both self-fund a clinical trial and stay in business? Maybe they can afford to pay some, but not all of the costs so they have to pass the rest through to the subjects in the trial. Otherwise, there will be no trial. Or perhaps they are all evil and “dubious”, preying on the uniformed, defenseless rich folk they’ve duped in order to reap obscene profits with no intention of actually helping them.
I’m sure this is discussed elsewhere on the blog but it bears repeating here.
Sponsors may not charge for investigational drugs without written authorization from FDA.
21 CFR 312.8 Charging for investigational drugs under an IND.
http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfCFR/CFRSearch.cfm?CFRPart=312&showFR=1
Costs associated with the trial must be disclosed in the consent form. No competent IRB would approve a trial where subjects are required to pay thousands of $ to enroll because one of the requirements for IRB approval of proposed research is that selection of subjects is equitable. In trials where costs of routine medical care will be charged to subjects’ insurance (in oncology trials, for example) IRBs routinely require sponsors to provide subsidies to cover costs to subjects without insurance. The requirement for equitable selection of subjects applies to IRB review of clinical investigations of drugs (including biologics) under IND or IND exemption and of devices under IDE or abbreviated IDE
Paul, we already have a model for “drugs” found to not work or to do harm…Settle a class action suit, rename the drug and get approval for another disease even when it does harm… Does that sound familiar?
You’re absolutely correct about controls and blinded study omission. If the treatments are already established as safe and efficacious in other countries already having done research, then open label treatment seems reasonable, yes?
You’re also correct on elite access and lack of justice. That problem is squeezingly obvious with drug availability and costs.
Justice must work both ways. It’s immoral and unjust to not have a pathway for people without money to pay for trials that have a reasonable chance of helping. Especially for compassionate care.
That’s justice. Not narrowing access, but broadening it.
I thin all cellular therapy studies need to be regulated, but some of these treatments are working, so a trial sponsor’s confidence in the treatment is justified.
@Brian, My issues with trials that require payment have nothing to do with the patients’ choices themselves, but with those running the trials.
What happened to the tradition of the trial paying the patients, who are the heroes and the ones taking risks?
My sense is that the trial folks even with good intentions who charge patients believe that their experimental treatments work even though they don’t have the data yet. That’s not how biomedical science is supposed to work.
And some others doing pseudo-trials are just in it for the money.
“Profit” is not inherently problematic in the broadest sense, but it is in certain contexts such as when patients are used as a source of profit when there is no good evidence that a treatment works or is safe.
Expensive trials also raise real issue of access and justice too as they have an unwritten exclusion criteria (i.e. $) that bars those from participating who don’t have the money or can’t raise it from family, friends, etc.
For-profit trials also undermine science as they cannot be truly controlled or blinded. How many patients would pay if there was a chance they’d be given a placebo?
Overall, I wonder how much valuable data will really come from any given open-label stem cell trial study of this kind that charges patients say $10K or $100K up front.
And what if the overly optimistic trial folks are wrong and the treatment actually doesn’t work and/or isn’t safe?
“Pay for play” is condescending to those of us who invest serious intellectual capital before putting our money down for “experimental” treatment.
The notion that “government knows best” is equally condescending and, in my experience, patently wrong. I have frequently observed governments behave in the most moronic fashion when it comes to making decisions in areas which have expertise in. Now, I’m being condescending — but I know that I can no more hurt the feelings of a government than I can hurt the feelings of brick wall.
For which diseases medical research money gets directed and towards which therapies it gets directed is an issue that you have not considered. The notion that governments should have a monopoly on such decisions may fit comfortably with the values system of people in Europe but not with my values. (With respect to Europe: Derelictus sum ego vidi.)
To my eyes, governments (and regulators) act as though some types of disease and some types of people are more fashionable, more deserving, more urgent, more important, than others. There are those who say that everyone should comply with a common values system — I’m not one of those.
This is the essential point, Paul. If I am inclined to invest more money in medical research than that which is extracted by taxation, then I am inclined to invest it as I see fit. Furthermore, I shall not feel well disposed to a government (or any other outfit/person) that prohibits me from investing.
On the other side argument, there is always risk to any medical procedure and likely higher risk for some types of experimental procedure. (Experimental or not, a treatment might be high risk.) The important thing here is being highly informed of what the level of risk really is. It is at this point where every effort must be made to ensure openness to scrutiny and freedom to express intelligent analysis.
Finally, there is the issue of “profit”. So far as I can tell, the last person who provided his/her services for an extended time without being paid ended up dead from exhaustion and starvation. Animal Farm had boxer the horse. Scott’s expedition to the South Pole had Lawrence Oates; the biggest and hardest working man who was only given the same ration as the rest of the “team” and was, therefore, the first to die.
I cannot decide if I am more shocked by the idea itself or by some of the comments. It might be because I am used to European free health care. I think it is self-explanatory that in a capitalistic society all clinical trials must be tightly regulated by the government. As bad as the public officials can be sometimes, it is still better than giving total freedom to profit-driven companies. I think that “pay to play” system would lead to two outcomes:
1. The rich having an even easier access to healthcare than the poor.
2. Families involved in desperate crowd-funding campaigns to pay for the treatment of their loved-ones.
And yes, the costs of clinical trials, and healthcare in general, are huge. That is why you need to properly tax corporations and the rich.
The question is loaded, and more derogatory than objective.
Given the changes and privatization of funding patient expenses for clinical research over the last 25 years, it’s a false consciousness (societal myth) that it’s somehow inappropriate or unfair for patients to pay.
Why? Because someone ALWAYS pays for patient participation.
Patient costs are HUGE for a clinical trial. If larger research projects published their per-patient participant costs for a given trial, I think the 10’s and 100’s of thousands of dollars, huge to an individual, would be put in context. Not to mention it’s actually not easy for researchers who are actually conducting trials (not ‘trials’) to recruit subjects for participation.
I think there are several better ways thinking people, including researchers, describe the situation.
Companies carrying out clinical trials should get more financial support… but certainly not like this.
Patients and their families are already in a situation that is bad physically, psychologically, and likely financially. Demanding this extra sacrifice from them in order to take place in a trial is taking advantage of desperate people, I feel.
On top of that, this is not expanding access, but on the contrary, restricting clinical trial avaiability, as it would seriously hamper the possibilities of getting in for those less well off.
This blog post by Chis Ceteno on amniotic stem cells would be funny if it wasn’t so pathetic: http://www.regenexx.com/amniotic-stem-cell-treatment/ it demonstrates why the FDA needs to play a role. Just saw an add on TV for a skin product made of stem cells from a rare apple!!! Money drives it all.
How many that take the poll are patients or caregivers? There is no way to tell, so frankly, I don’t see much point to it. As for those referring to the courage and time it takes to participate in clinical trials as “pay to play”, those words are not well chosen. I don’t offend easily, but I’m sure that there are those who find that term very offensive.
@Barbara,
I see your point on “pay to play” and in part that’s why I put it in quotation marks. At the same time it is becoming a widely accepted terminology and I can’t think of a better way to describe the situation. “Fee for enrollment” trials? I also believe that in some cases where patients have to pay, sometimes 10s or 100s of thousands of dollars, those running the “trial” are taking advantage of the patients and “pay to play” invokes that kind of dynamic. The patients themselves are heroes, but in some cases those running the trials and demanding payment, maybe not so much. Paul
The stakes are so high in the fight for cure, that even asking people to help with the testing financial should be considered. This is life and death stuff, and I know I would help pay if someone I loved had a chance to get better.
@Don, Thanks for the comment. I totally get where you are coming from, but I’m worried that patients having to pay could lead to abusive situations in some cases (maybe already happening) and moral/ethical traps.
Paul