5 ways to carry out a patient-centric clinical trial

Researchers rely on a steady stream of volunteers to test and validate their products – yet clinical trial recruitment and retention are significant barriers to success.

Fewer than 5% of all those eligible to take part sign up, and the global average dropout rate is around 30%. Faced with such stark figures, sponsors must do everything they can to attract, and hold onto, willing participants.

We look at five ways the industry is embracing patient centric clinical trials in a bid to secure the future of their study programmes and develop drugs that make a real difference to people’s lives.

1. Patient-reported outcomes

While healthcare professionals often measure impact through disease scores, these rarely translate into what actually matters to patients. A lessening of inflammation, for example, is a less tangible benefit than an increase in energy or a reduction in fatigue.

Patient-reported outcomes (PROs) are increasingly being used in clinical trials to assess the impact of a medical treatment or intervention, according to an article published in the BMC journal last year.

In fact, 27 per cent of the 26,337 studies registered on ClinicalTrials.gov between 2007 and 2013 included PROs, which look at functional health, well-being and psychological issues from the patients’ perspective, without interpretation by a clinician.

Understanding what patients want from new medicines is the first step in developing products that make a difference. So, how do you know that the product you are testing tackles an unmet need? It’s simple – ask them.

Many companies work with patient organisations or focus groups early on to identify priorities and ensure pipeline products measure up.

2. Patient-centred study design

Sometimes, protocol design can alienate participants before the study has already started. Logistical issues that might not even occur to sponsors when designing a trial can have a huge impact on the programme’s ability to recruit and retain participants.

People with fatigue-inducing long-term health conditions, for instance, don’t have the energy to travel at certain points of the day. And those with dexterity problems may find it difficult to open a multitude of blister packs.

The only way to ensure the protocol is feasible is to ask potential patients to review proposed study designs early on. Sponsors often hold workshops that bring researchers, patients and advocates together to work on a solution that works for everyone involved.

“It’s very easy to make assumptions about what will work for patients, but no one can truly know without asking them”, Parkinson UK’s Research Involvement Manager, Natasha Ratcliffe, told us last year.

“Sometimes people do not see the value of working with patients. They think it is just common sense, but until you sit down and ask them you don’t know what you don’t know,” she said.

3. Remote reporting

In recent years, the number and frequency of data points being collected during studies has increased massively. At the same time, the advent of more personalised medicines has led to the emergence of a “needle in a haystack” model of clinical trial recruitment.

It means participants already coping with complex health conditions are being asked to travel further and attend more.

But in a world of instant messaging, video calls and wearables, there is little need. Regulatory bodies have accepted this and no longer insist on 100% on-site monitoring, which can significantly impact a sponsor’s ability to recruit and retain enough people to adequately test their product.

Of course, monitoring needs to be accurate and robust. But today’s range of available, validated research technologies, combined with careful forethought and planning, can drastically reduce the frequency of site visits.

4. Rapid reimbursement

People face all manner of logistical barriers when taking part in a clinical trial, but many of these can be overcome quite easily. Reimbursement of expenses, for example, is an issue that regularly comes top of the list of participant concerns.

While it is quite normal in pharma circles for payment terms to number 60 or even 90 days, such a wait is not practical for most study participants.

Many companies have either rewritten their processes or are working with partners to ensure out-of-pocket expenses are paid before they can impact on patients’ personal finances.

Speaking to pharmaphorum last year, CEO of Greenphire, Jim Murphy, explained that removing such logistical barriers were an easy win when it came to recruitment and retention.

“We can’t control protocol – protocol is what it is – but if you can take care of all those other elements of patient convenience in a way that actually reduces the burden on the investigative site, you’ve added connectivity and allowed more time for the site to focus on the patient,” he said.

5. Lay summaries

Providing lay summaries, as well as clearly communicating with participants throughout the trial, is fundamental to engagement – yet many people say they are never even told the results of the work they participated in.

The European Medical Association, the Association of the British Pharmaceutical Industry and the US’ National Institutes for Health (NIH) all encourage sponsors to ensure participants have easy-to-access information.

Said the NIH: “It is critical that scientists become knowledgeable about how to reach and communicate in their communities before, during and after a trial.

“Key to encouraging public support for, and participation in, clinical research is trust, and researchers must take the time and make the effort to build [it]. Communicating directly with participants as they join a study — and throughout the process — is critical to earning and maintaining trust.”