All through the year, we talked innovations here on the blog. We gave you all the relevant information about the ins and outs of clinical trials, practical tips on how to deal with everyday life with a chronic disease, looked into the new treatments being developed for various conditions… And we’d like to conclude the year with some more updates from the world of medical research.

At the beginning of 2018, the FDA issued the ‘Payment and Reimbursement’ Guidance for Institutional Review Boards and Clinical Investigators and we at FindMeCure find it appropriate to say farewell to this year by discussing what this means for the future of clinical trials.

So let’s make the meaning of some terms clear before we dissect what the FDA Guidance actually tells us and what it means in a wider context.      

Reimbursement refers to the trial sponsor(s) covering expenses related to the participation in the trial. Such expenses may include the cost of traveling to and from the trial site, accommodation if the stay requires it, loss of income due to unpaid leave for the purposes of participation and the list can go on to also include a paid caretaker for the participant’s children and so on.   

Compensation can mean one of two things: either the payment participants receive for being part of the study, or a kind of insurance that covers any additional health risks or harm posed by participating in the clinical trial in case there is such. In some cases, it’s recognized that a trial is not associated with any additional risks and so no compensation is needed.   

Undue influence is a term we’re going to be using a lot in this post and it refers to the persuasion of patients to take part in a clinical trial in cases when such persuasion diminishes the value and significance of their informed consent.   

Okay, now that you know exactly what these terms mean and how they’re used, let’s take a look at what the FDA Guidance is actually telling us (and, well, Institutional Review Boards, IRBs, and clinical investigators to which it’s addressed).

The Guidance makes an important distinction between reimbursement and compensation (in the extent to which it refers to payment for participation) and for the first time makes it plainly clear which is what and what kinds of implications it gives rise to. The FDA states that reimbursement of travel expenses and lodging, unlike payment for participation, is free of ethical concerns as it does not raise any undue influence questions. So, in a way, the FDA endorses the practice – an important step towards a more patient-centered approach to clinical research.

Payment for participation, on the other hand, is addressed with more caution. The FDA remarks that it presents some issues regarding undue influence – if paying patients for taking part in the clinical trial persuades them to underestimate the risks associated with participation, the consent they give may be questioned and the integrity of the trial compromised.

But ethical concerns are not the only thing the FDA wants IRBs and investigators to consider. Though the Guidance states that paying for participation is a generally ‘acceptable practice’ and it’s a ‘recruitment incentive’, it also poses some questions.

What should participants be paid for? Their time, the inconvenience, the discomfort or something else? And how can the cost of their being inconvenienced be estimated? Additionally, such compensation should be just and fair with every participant receiving the same amount of money. However, it’s likely that not every participant suffers the same discomfort.

Then, what principle should guide the investigators in establishing the payment plans – that of equality or that of equity? According to the FDA, “The amount and schedule of all payments should be presented to the IRB at the time of initial review.”, meaning that what patients receive for their participation should be clear before the trial proposition gets the green light.

But still, why are we sharing this information with you? What does the FDA stance on reimbursement has to do with you? Well, we believe that the FDA Guidance marks a shift in the clinical trials industry and its approach to trial participants.

To understand why we’re so passionate about clinical trials, you have to picture a world without them. Without clinical trials, no new treatments will be possible, no innovations, no new understanding of the human body and the way disease affects us, no new technology and no promise of a cure of the illnesses that plague modern-day humans. But what makes clinical trials possible in the first place, is you. No trial and by extension, no drug can exist without the patients that take part in research.

So, the patient-centered shift that we witness in recent years paves the path for a change in the industry that is going to benefit all of us. It used to be the case that participants only had to pay a small parking fee or the cost of a taxi ride to get to the trial site that was situated not too far from their home.

But now, with the expanding scale of clinical trials and especially in the case of developing rare disease treatments, participants travel all across the country to take part in clinical research. Plane tickets and overnight accommodation in a foreign city pose a financial barrier for patients that would otherwise consider enrolling. It’s not only unfair to make participants pay for such things out-of-pocket but it’s also inefficient research-wise. Making participation as affordable as it can be, increases the chances of full and diverse enrollment – a hugely important ingredient in guaranteeing scientifically sound data.

Reimbursement is not (yet) mandatory, as you may remember from our post on clinical trial costs. But it would appear that the FDA endorsement of the practice is a step forward in the right direction. Since covering participation-related expenses not only benefits participants but also the study itself and by extension – everyone who could potentially need the new treatment if it receives FDA approval, investigators will only win by making participation affordable for a wider population. It appears, yet again, that prioritizing patients’ access benefits everybody involved.

Legislators also seem to realize this. In October Pennsylvania became the second state after California to legislate reimbursement of participation-related expenses. The bill clearly states that reimbursement is not coercion and does not pose undue influence issues, and it also recognizes the impact reimbursement could have on enrollment numbers.

It’s still a slow process but change is surely on its way and it’s going to benefit clinical trial participants as well as patients outside of research studies who could nevertheless significantly improve on the treatments in development, once they get FDA approval.   

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