Does going ‘social’ help terminally-ill patients gain expanded access?

BMC Medicine recently published an article investigating whether the use of online petitions and social media actually helps patients gain access to experimental drugs. Co-author of the work, Timothy Mackey, explains more.

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As a father of two young children, I can only hope to never experience the devastating news that a child of mine is suffering from a terminal illness. Yet, this is exactly what thousands of families have to come to grips with everyday, when they discover that their loved ones are suffering from an incurable disease and that there is no effective treatment.

Such is the tragic dilemma of patients and families who seek access to experimental drugs through expanded access programs (also known as ‘compassionate use’).

Patients do this out of desperation, as experimental drugs are just that, ‘experimental’, offering no guarantee of effectiveness or even safety, but may nevertheless offer the only hope for a potentially life-saving treatment.

Expanded access programs

As explained by the U.S. Food and Drug Administration (FDA), expanded access “provides a pathway for patients to gain access to investigational drugs…for serious disease or conditions”.

However, this ‘pathway’ is fraught with arduous hurdles, chief among them getting a manufacturer to agree to provide you an untested drug when they have little incentive to do so.

Though the FDA approves the vast majority (over 99%) of expanded access requests it receives, this high approval rate is a misnomer, as patients often never get to the point where they can send in a request.

This has given rise to ‘right-to-try’ laws in several U.S. states, which are equally challenged and untested in their ability to enable access to experimental drugs.

What we set out to investigate

It is no surprise that terminally-ill patients and their families resort to the use of online petitions.

Given these challenges, it is no surprise that terminally-ill patients and their families resort to the use of online petitions (websites such as Change.org that allow anyone to create a petition that can then be signed/supported online) and social media platforms (Facebook, Twitter, YouTube) to champion their cause. Our study sought to better understand this phenomenon and also assess whether it actually helps patients or not.

What we found was disappointingly inconclusive. Out of the 23 expanded access online petition and social media campaigns we reviewed (39% of which were children); we observed a huge range of variation.

Some garnered as much as half a million signatures, while others only a few hundred. However, regardless of how ‘successful’ a campaign was, we couldn’t find any pattern that these campaigns actually ‘worked’ and led to better access to experimental drugs.

This leads to a difficult question. Is it worth it for patients and their families to spend so much time and energy on online petitions and social media, even though it may represent a very slim chance to compel access to an experimental drug?

The answer communicated in these campaigns seems clear: yes, because there is no other treatment alternative, and experimental access likely represents the last and best hope for survival.

What we hope will be achieved in the future

As researchers we strive to understand, educate, inform, and improve society through scientific inquiry. However, there are no easy answers or solutions when it comes to expanded access.

As researchers we strive to understand, educate, inform, and improve society through scientific inquiry. However, there are no easy answers or solutions when it comes to expanded access.

Some federal legislative efforts to improve the expanded access process were included in the CURE Act Provisions of the 21st Century Cures Act, but the future of this legislation is now in limbo.

Despite this fact, we sincerely hope our study will have a positive impact on the lives of terminally-ill patients by providing better information on the challenges they will face on their expanded access journeys.

We also strongly believe that these patients and their families deserve better, and that there should be a centralized resource that identifies expanded access pathways in a simple and clear way.

Patients should also have better information to determine if they are the right candidate for an experimental drug, help from patient advocacy groups in getting through the process, and most importantly, be afforded the dignity of knowing the reason why their request has been approved or denied.

With the rise of everything ‘digital’ in health here to stay, there is little question that use of online petitions and social media in an attempt to advocate for expanded access will continue.

Our job now is to improve expanded access policies so that terminally-ill patients can go back to using these platforms to interact with their loved ones and celebrate their lives.

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