March 2nd, 2021

Why involving the patient voice in clinical trial design is so important

The importance of including qualitative measures in clinical trials


Quantitative measures in randomised clinical trials are the undisputed gold standard for evaluating the safety and efficacy of therapeutic interventions. These are determined by using laboratory tests and physical or physiological examinations. However, quantitative measures do not fully and exactly reflect the impact on a patient’s quality of life. This is what qualitative measures are for.

There are different methods used to record qualitative evidence of the impact of a therapeutic intervention on a trial participant. These include entry and exit interviews, quality-adjusted life-year (QALY) measurements, or questionnaires.

Unfortunately, if a new therapy’s QALYs fail to impress the team reviewing the dossier, registration of the new therapy could be delayed or jeopardised. Patients undergoing the trial and accessing this drug could therefore need to change their treatment plan after the trial is completed, which can impact their health and quality of life.


How to better reflect a therapy’s impact on quality of life


We spoke to Jessica Bean, Chairperson at Patient Voice Initiative, an organisation that works to make sure that decisions about healthcare in Australia are being better informed by the knowledge of patients, health consumers, carers and patient groups. Ms Bean is living with cystic fibrosis and was herself a participant in a clinical trial. This inspired her work at Patient Voice Initiative, as she saw gaps in how the value of the therapy was being reflected in trial data.

“The therapy was a completely new ballgame. And very early on, I realised that this drug was doing things that I thought were never imaginable at my point of disease progression. Within the first five years of treatment, I went two years without a hospital admission at all. My husband was able to return to work.

“When I became aware of my clinical numbers, I realised that the significance of the treatment wasn’t reflected in those numbers. The dramatic improvement in my quality of life wasn’t reflected in the data. If you looked at the data, you would be really underwhelmed with what it had been able to achieve.

“At that point, I realised that there would be a problem in how patients were going to access this high-cost treatment, because how do you determine value if you can’t see the full picture and if you’re not actually asking patients, ‘What matters to you? How did this make an impact in your life?’

“And I think that knowing what I learned now, had there been more patient input at the start of that process when determining the trial design, some of significant quality of life improvements that I experienced could have been captured in the study.”

The qualitative measures used in Ms Bean’s clinical trial (surveys) were developed some time ago for older cystic fibrosis treatments. Therefore, these quality-of-life assessments did not give trial participants a chance to express these significant improvements accurately.

“They {assessments} didn’t go far enough, they didn’t look at the type of endpoint that these new therapies could achieve,” said Ms Bean.

How can clinical trial design improve?


Ms Bean said: “We have to get patients involved right at the beginning. When you get to the point of doing surveys, there’s already been assumptions made about the patient experience, and about what matters to patients.

“It does get tricky and it has to be a large multi-stakeholder approach. There has to be a collaboration, not just between patients and industry, but there also needs to be decision-makers brought in at that point, people who are going to be regulating and reimbursing these therapies.”

Speaking of her personal clinical trial experience, Ms Bean said: “Maybe they {the trial sponsor} didn’t expect their treatment to be so successful in the ways that they were. I don’t know how you preempt that other than really understanding the details of patient’s lives.

“There are things that patients tell their doctors about their experience that are common knowledge in the patient community, but a revelation to their doctors. The little hacks, the little day-by-day moments that patients know happen and that we learn to navigate. If we can have better opportunities to build relationships with all stakeholders in a clinical trial, there are better processes to design the clinical trial.”

An untapped goldmine to learn more about the patient experience is social media: “There is so much information in places like Facebook communities, they’re really data rich. And we can find really amazing leaders in those communities that sometimes have amazing insights about what patients need and expect and want in their treatment. And at the moment, there’s not a lot of recognition about those changing and evolving types of patient communities,” said Ms Bean.


In Australia: still work to do


A literature review compared different countries for the number of clinical trials between 1999 and 2016 which mention ‘qualitative’ measures. The review found that the UK led the rest of all the countries (570 trials, 38.2%), followed by the US (425 trials, 28.5%), Canada (71 trials, 4.6%), France (67 trials, 4.5%), Australia (43 trials, 2.9%), Germany (37 trials, 2.5%) and Denmark (34 trials, 2.3%). None of the remaining 45 countries accounted for more than 2% of all confirmed qualitative trials.

Although some improvements to existing qualitative measures could be achieved by involving the patient in the clinical trial design process, these are essential to include in any trial to obtain information on the value of the treatment. Australia lagging behind other comparable countries in this field demonstrates a lack of emphasis placed on the importance of reflecting quality of life progress.

Read more about the Patient Voice Initiative here. 

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