Rare Diseases 2021: maintaining the human element in decentralised trials
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Rare Diseases 2021: maintaining the human element in decentralised trials

By Kezia Parkins 22 Sep 2021

AGTC’s director of patient enrollment discusses the importance of the human touch in decentralised rare disease clinical trials.

Rare Diseases 2021: maintaining the human element in decentralised trials
AGTC’s partnership with 2020 Onsite allows the company to drive around the US to screen and assess patients, reducing the burden on them to travel. Image credit: 2020 Onsite

Finding the right patients to recruit into clinical trials is one of the most challenging aspects of clinical research. With rare diseases, this is greatly exacerbated due to patient populations that are few and far between, as well as gaps in diagnosis and a lack of experts in the disease areas.

The move to decentralisation in recent years has been a boon in many areas of clinical research, including in the rare disease space, but as Florida-headquartered biotech AGTC’s director of patient enrollment, Halley Losecamp outlined in her presentation at last week’s Clinical Trials in Rare Diseases Conference 2021, in rare diseases the human element in clinical research should not be lost.

The firm’s current clinical trials are testing treatments for patients with rare inherited retinal diseases (IRDs). Because their patients have issues with their vision ranging from achromatopsia (a condition that means patients cannot see colour and have poor visual acuity) to X-Linked Retinitis Pigmentosa (XLRP – where patients progressively lose vision or see the world through a pinhole), with Covid-19 AGTC had to get creative with its patient recruitment, enrolment and engagement as travelling for these patients can already be incredibly hard.

Clinical Trials Arena sat down with Losecamp to discuss how to conduct optimal patient enrolment in rare disease trials, the importance of patient engagement and how AGTC is decentralising studies while maintaining all-important human contact with its trial participants.

 

Kezia Parkins: How can sponsors reduce the burden on rare disease patients participating in a trial?

Halley Losekamp, Patient Enrollment, AGTC
Halley Losekamp, Director, Patient Enrollment, AGTC

Halley Losecamp: It is a really heavy burden. Our patients typically have some type of vision disability. They don’t want to go to the airport because they are not set up for people with vision impairments. It’s loud, it’s chaotic, it’s bright, just not a good environment for them to move around in. But, patients often have to travel to get to study sites.

At AGTC, we’ve tried to make sure that our concierge services are set up to help them pick the days and times best for them to travel when the airport isn’t as busy. We also try to not be as stringent on the costs or budget needed for these patients to move around.

We have a lot of patients from the Mennonite community and they don’t use planes, go on trains or even have cars. We are really flexible with their needs and ability to travel. They choose their own days and times to travel and can choose their own person to bring them into the study site.

Covid-19 added a whole other layer for them because they already have a lot of anxiety to travel. Now, restrictions make it even worse so we’re trying to help support their travel and send them Covid kits ahead of time.

One of the biggest things that we’re doing, though, is the mobile vision clinic, a partnership with 2020 On-site.

KP: Tell us more about the 2020 On-site partnership.

HL: When Covid-19 hit, everybody was scrambling to keep their patient visits within window. Someone at AGTC had the idea to put a bus on the road. Everybody thought that was crazy and wouldn’t work. But, that one person insisted. He got on to 2020 On-site, a company that drives around to businesses to provide routine optometry to employees. We asked them if they would be interested in working with us and they agreed, which actually kickstarted their entire service offering for clinical research.

We set the bus up and we have been driving around the United States, visiting our patients with achromatopsia to ensure they are seen within the window required. The study is from ages four up to late adulthood, so it just simply allows patients to get on with normal life. We pull up in front of their house, or oftentimes, to malls near them.

It’s not easy but it works really well, there are no issues with the data and the patients really like it.

Now we’ve launched the pre-screening programme, which basically tells patients if they should fly or not for screening. We go to their homes and tell them whether they will likely qualify for this study. If so we get them on a plane, if not we have gladly saved them the trip.

The partnership with 2020 On-site continues to grow and we are getting more buses on the road. That hybrid/decentralised version of a study is how AGTC is trying to address the burden for patients.

KP: How do you think the industry handled the move to decentralisation during the pandemic?

HL: We have been so heavily reliant on this brick-and-mortar idea of a study site and put the burden on the patient, so I think it was necessary.

However, I felt like there was such a rush in the industry to move to decentralisation that the waters got muddied. I think we did ourselves a bit of a disservice because nobody really understood what ‘decentralised’ was and how all those components fit together. We’re starting to pull that apart and really understand the best tactics and use cases for these patients, but personally, I think there needs to be some element of human involvement. Patients crave that, especially patients with rare diseases. They want to feel that there’s hope, they want to feel important.

The biggest thing I think people should focus on is home health – allowing nurses to go to the patient, allowing patients to see their physician for some of their assessments or check-ins, giving them that flexibility to maintain humanistic contact in a space that makes them feel a little more comfortable.

I hope that as an industry, we continue to decentralise in a way that is benefiting the patient and keeping them centred. I don’t want to swing the pendulum so much that we’re reducing the human part of the interactions and the relationships between the physician, the study coordinator and patients. You see over and over research papers coming out that find people enjoy being in clinical trials – they’re more engaged with their own healthcare, it saves them money, they feel better about themselves and would do it again.

I don’t think we do a very good job of getting eye-level with the patients and actually asking them what they would like to do – giving them options on whether they come to a study site, understanding their preferences.

KP: What should sites and sponsors consider when recruiting and advertising is mostly online?

HL: Consent starts the minute a patient sees your ad. You need to do a lot of work at the beginning, such as creating an experience pathway, creating some insights, and then letting those funnel down and tell you what kind of experience your patients, providers and caregivers should have to engage them.

It’s also about what your stuff [online] looks like. Does it look nice? Would you click it? Then, make sure you do your homework to put the ad where the patients are actually spending their time.

I think study teams, in general, don’t do a very good job of that, where they strip the patient recruitment budget, throw some ads up on Facebook and assume patients are going to find it. It doesn’t work that way; you have to find the patient.

You can also hire a vendor to do all kinds of insights for your social listing. They can tell you what patients are talking about and where they are talking to each other. Are they talking about therapies or clinical trials? That will help tell you what your strategy should be for online engagement.

Lastly, make sure that engagement and experience is continuous. From the moment they click your ad you have to keep them engaged. This also starts your retention. The more engaged they are, the more trustworthy they think you are. Then, that retention is going to be there throughout the study.

KP: How do you design an experience pathway for a patient recruitment campaign? Why is that so important?

HL: Creating an experience pathway is an easy exercise. You create one for your patient, one for your provider and one for your caregiver and you learn everything about them that you possibly can.

I have seen many times that this exercise has a direct impact on protocol development. For example, we were doing protocol development and we had to get the patient at the point of diagnosis. We were realising in several countries outside the US that they weren’t actually diagnosing their patients in the pathway that we thought so we had to go back and amend the protocol to allow for a different diagnosis pathway. This can have an impact upstream and downstream. Just do your homework. It will have a big impact at the end of the game for you.

KP: How can social media be harnessed for patient recruitment in rare disease trials?

HL: In rare disease recruitment, I just don’t see often that social media is successful. But, don’t not have it. You have a kind of surround sound when you’re doing outreach and social media adds to that. I would never suggest having social media as your only tactic when you’re recruiting patients in the rare disease space, though.

KP: Tell us about some of AGTC’s recruitment successes, failures and what was learned.

HL: Aside from the bus, the biggest successful technique we have used is physician engagement and referrals. That’s because patients that are seeing their physicians are just healthier patients and you need healthy patients for clinical trials. Patients feel valued when their physician refers them so they’re more engaged, or willing to stay or enrol in the study. It starts to create that connection before they even get to the study site for screening. Also outreach for the genetic testing labs has been pretty successful for us.

At AGTC, if a patient tells us they don’t like an experience, we would immediately go back and figure what went wrong and how to fix it. There have been things that didn’t work but in rare diseases, sometimes that’s the best thing that can happen because then you can start finding the direction that does work. I don’t look at patient recruitment in terms of success or failure. It’s all building together to create a plan, and we learn. As long as you have that mindset, you’re going to be successful. You have to be willing to accept the highs and lows in patient recruitment.

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