Has the pharma industry been looking at rare disease recruitment wrong all this time? Has the rise of the internet provided a solution the industry hasn’t yet fully considered?

Imagine this: You’re a researcher looking to run a clinical study on a subset of an ultra-rare disease.

That disease has a prevalence rate as low as 1 in 17,000.

After months of hard work (and a lot of money), you manage to find some patients, but you realize that many of them don’t live near any of your trial sites.  

Plus, many of the patients who live near trial sites can’t participate in your study because of outside factors like work-life disruptions. Or they don’t like certain aspects of the study design, such as taking an IV.

There’s a massive disconnect between you and the patients you’re trying to help.

If only you could understand more about these patients before planning your study.

Easier said than done in the rare disease space, and until now, that’s been a massive problem.

Rare disease recruitment is complex

Recruiting rare disease patients for trials is often an uphill battle for sponsors.

An analysis of 736 trials by Global Data found that 25% of rare disease trials were terminated between 2016 and 2020 because of low patient numbers. Another study by ISR Reports conducted in 2020 found that the most challenging issues reported by people running rare disease studies were around patient access.

Finding patients to take part in rare disease clinical and real-world studies can be incredibly challenging. Patient populations are small and spread out all over the world. When you factor in the strict eligibility criteria of the study, that patient number gets even smaller.

Rare disease studies can feel like a shot in the dark

With more common diseases, researchers can quickly run feasibility studies or look at the natural history of the disease to understand factors like where patients are based, what study design will work best, study relevance, and so on. In the rare disease space,  we don’t have that luxury.

There are over 7,000 known rare diseases, and medical literature about each of those diseases and their subtype isn’t always available, limiting the amount of information available to the researchers when making study decisions.

So how can researchers find the missing (and very crucial) pieces of information needed to run effective studies?

Patients and caregivers are the experts in rare

It’s often the patient and caregiver that knows more about their rare disease than anyone else. Patients typically struggle for years to get an accurate rare disease diagnosis, perhaps because healthcare providers might not have encountered the rare disease before, or they might want to rule out more common ailments first.  When patients finally get an accurate diagnosis, there will be lots of unanswered questions. And so, they end up doing a majority of the fact-finding themselves, which inevitably involves the internet.

The internet is bringing highly dispersed patients closer together

The number of people using the internet has been rising exponentially - 4.66 billion people worldwide use the internet as of January 2021, up by 316 million (7.3 percent) since January 2020. According to Google’s VP of health, Dr. David Feinberg, around 70,000 searches are made about health every second. People are looking for information on topics ranging from symptoms, medications, and insurance to directions to the nearest clinic.

People affected by a rare disease are connecting with others in a similar situation. Smartphones have granted internet access to many computer-cautious or economically deprived individuals. They can share experiences, support one another, and keep each other updated on the latest news and developments - all from the palm of their hand.

Harnessing patient insights through digital channels

Digital channels are already helping solve some of the challenges in rare disease study recruitment. Through precise audience targeting and online community outreach, these digital channels are helping find and inform the targeted patient populations that researchers seek.

Suppose digital channels provide us with ways to engage with people that have traditionally been out of reach.  Could we use these techniques to inform study planning? For example, if researchers know in advance, which rare disease studies in their pipeline will be the most difficult, they can factor that in when they're planning their research. Perhaps they’ll need to extend the recruitment timeline or take another look at the eligibility criteria or revise their site location plan.

When researchers know the potential barriers that will prevent patients from taking part in a study, they can create mitigation plans in advance.

Engaging with patients before study planning

We’ve identified several ways to use digital channels and broad stroke campaigns to gain early insights from patients.

At its most basic, a properly structured campaign can assess how many people we will reach through online means and what channels will work best. We can analyze the digital behavior of the patient population being investigated. Doing so will help us learn the demographics of our digital audience, the kinds of information they're regularly trying to find answers for, and any key themes that may emerge.

An expanded campaign will invite people to answer initial survey questions around study relevance and factors that may influence study design, like finding out if patients would be open to decentralized approaches.

With the proper expertise and understanding of rare diseases and key digital channels’ inner workings, early insights campaigns like these can be delivered quickly. Since they are not promoting specific studies, they don’t need IRB approval, giving us the freedom to design them as simple or complex as required.

They can help bridge the gap between researchers and the patients removing that disconnect, and keep studies patient-centric from the outset.

We can help you collect early insights to inform and strengthen your studies. If you have rare disease programs in your pipeline, get in touch today.

By Jeremy Edwards, CEO of Xperiome

Sources:

Global Data Report: https://www.globaldata.com/25-rare-disease-trials-terminated-due-low-patient-accrual-rates-says-globaldata/  

ISR Report: https://isrreports.com/reports/rare-disease-study-design

Read next:

Patient Insights Report: Survey on drivers and barriers to clinical trial participation in rare disease

Increasing trial retention: How best to keep people with rare diseases engaged in clinical trials

Early insights study example: Preferences for injectable drug-delivery options in sickle cell patients