Improving study recruitment with patient-centric trial endpoints

Everyone wants to find a cure for a debilitating long-term condition, but what can often be overlooked is the value in being able to first make life more manageable, comfortable or enjoyable for the people living with such diseases.

As the patient voice gets louder, grows in confidence and becomes organised, however, that is starting to change. It’s a paradigm shift that holds potential for people living with chronic illnesses and industry alike.

Traditionally, the first input patients have with a clinical trial is when they sign up have an experimental drug tested on them. The product’s developers decide what need the new medicine should meet, and that is what success is measured by.

By turning that model on its head and asking people what their priorities are, patient-centricity is opening up trials to those who had previously felt ‘written off’ by the race to find cures.

It’s an approach, driven by the rise of patient centricity and the realisation that pharma has a lot to gain from listening, that is starting to yield results.

The power of the patient voice

In Duchenne muscular dystrophy (DMD), for example, the first study that was open to adult wheelchair users has recently taken place.

DMD, a progressive muscle wasting disease usually diagnosed in young boys, leaves most needing a wheelchair by the time they are teenagers. Cardiovascular and pulmonary complications can prove fatal, yet research was not addressing this.

Dr Oscar Henry Mayer, medical director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia, said: “A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly valued treatment options for pulmonary complications.

“Maintaining pulmonary function in patients with DMD has only recently become a prominent therapeutic objective in DMD, particularly in non-ambulatory patients.”

The expansion of industry focus to patient-centric trial endpoints such as these helps improve quality of life for patients while still working towards finding a cure. But there is still much to do.

Part of the problem is a disparity between the priorities of those who design trials and those who stand to benefit from them.

Written off by study design

In a survey by Barts MS blog, 88% of people with the progressive neurological condition rated hand and arm function as being more important to them than lower limb function. Yet trial after trial sets being in a wheelchair as an exclusion criterion.

The #ThinkHand campaign is calling on industry to rethink its approach in a therapy area that is already beset with inequity of treatments.

Advocate Trishna Bharadia, who was diagnosed with MS in 2008, said: “People with advanced MS who use wheelchairs are currently unable to participate in clinical trials of disease modifying therapies (DMTs), even though evidence suggests that the course of MS can be modified at nearly every stage of the disease, including in people who need to use mobility aids such as canes, crutches, or a wheelchair.

Trishna added: “Bart’s survey shows that people with MS put upper limb function above lower limb function, so why are we not using upper limb function as a measurement?”

Adding hand and arm function outcomes to trials, argue the campaign’s supporters, could lead to discoveries that would improve the quality of life in people who can often feel ‘written off’ – the NHS tends to withdraw access to DMTs if the disease moves into the progressive stage.

Speaking at last year’s MS Trust conference, Alison Thompson, research service design coordinator at the Blizzard Institute which has spearheaded the #ThinkHand campaign, said: “Although DMTs may not be able to preserve lower limb function in people with MS who already have walking problems, we think some might be able to preserve arm and hand function,” she said.

Using arms and hands meant being able to do the “things that make all of us human”, she added. Writing, typing or simply wiping after the toilet are all functions that are lost when upper limb function is allowed to deteriorate.

#ThinkHand wants the pharma industry, the NHS and the public about the importance of maintaining upper limb function – and the impact losing it can have on quality of life.

Win/win for industry

Trishna, who sat on the judging panel for last year’s Quality in the Delivery of Services (QuDoS) in MS recognition programme, added that focusing of patient centricity was in industry’s interest.

By listening to why people participate and what they want to get out of the study, industry can reduce the burden of participation making it easier to recruit, she said.

Working towards meeting the stated needs of a particular those with a certain condition by developing patient-centric trial endpoints holds great potential for industry – not least because it means developing drugs that doctors will want to prescribe and patients will want to take.

As Trishna explained: “There are so many ways pharma can make trials more patient focused at every stage of the drug development pathway: from research priorities to recruitment to the trial itself and the post-trial dissemination of results.

“The best way to meet patient needs is to involve them in the process.”

About the author: 

Amanda Barrell is a health and medical education journalist, editor and copywriter. She has worked on projects for pharma, charities and agencies, and has written extensively for patients, healthcare professionals and the general public.