They gave a joint presentation about implementing the right COAs to support the patient experience in clinical trials. This covered how getting it right could reduce the burden and improve data quality and strategies behind COAs to provide a rigorous data-driven approach to meet regulatory experiences.
Although from different sides of the business and coming at this approach from two different angles, both say they work really well together.
Elan said: ‘We look at the timeline of regulatory acknowledgments and how things are moving towards patient-focused drug development. Meaningful change can also mean something to patients in terms of how the treatment is going to improve their lives.
‘In the next few years we are going to see an increase in the amount of patient listening and that is happening in clinical trials in many different ways – not just because it is a good idea but because in 2016, the FDA had the 21st Century Cures Act which spawned the patient-focused drug-development activities and from that, there have been four new guidance in six years, which is different from the typical glacial speed for this industry.
He went on to explain that the term ‘meaningful change’ is not just the way instruments and measurements are selected to support endpoints but also to understand how they are communicating to and supporting patients.
‘None of these things are new, we have been doing this for decades.
‘What’s really changed is where the 21st century Cures Act comes in. Now when they're making regulatory decisions on products, patients are involved in those decisions, and they are not just going to be looking at the safety and efficacy.
He said the expectation is that you're going to be safe and effective, that the biology and chemistry and the work that they're doing has to have, bar none, the patients as the primary focus to make sure the treatment makes a positive difference in their lives.
‘We all heard the joke years ago about chemotherapy sometimes being worse than the disease. And that has a severe impact on the patient. You can't always rely on the patient saying, ‘I'm going to go through six months of difficult times, to be able to get through to that other side.’
Elan and Anthony both believe ways must be found to minimize exteriorization in patients’ for their health-related quality of life but that the reason they should do this is was so patients could spend time with family when they are most vulnerable – and get the support they need.
Anthony adds ‘incorporating that patient voice in the decision-making process means that there needed to be a fundamental change in how we run clinical trials.
‘Sometimes they surprise us. Sometimes they're willing to tolerate certain discomforts in exchange for something else. That's really key, just because we've established or identified that a certain disease has a symptom, then alleviating that symptom is a good thing. But there might still be something better that is more meaningful or impactful for that patient.’
He also said there is an emphasis on the importance of capturing the voice of the patients, which he points out was done using outdated methods including pen and paper up until the industry started leveraging lots of different types of technology and making advancements.
Patients as the primary focus
He said: ‘I think we're getting to a point now where we have a lot of technology advancements, where we can capture that voice and where the activity of the capture isn't yet another burden on the patient.
‘Another aspect of capturing their voice is actually sharing that information back and that's it's a discipline called patient engagement. And that's becoming more and more prevalent and more popular, a lot of sponsors are asking how you manage patient engagement, and it’s from the standpoint of not only keeping the patient engaged in the study, but how to keep them engaged, or keep them informed of a study.
‘You can't always pare it back, what are the results you want to log or whether to unblind a patient depending on what you're tracking and tracing etc., but there are times and we've actually heard several times that patients just like to know where they stand. Where are they compared to the rest of their cohort and we have all the information, it is just a matter of sharing it.’
Elan said there is a sea of change happening on the regulatory side of clinical trials, but that ‘it's just a matter of time before that starts being pushed out to therapeutic care where patients get that insight and are in control.
‘We are on that solid road and it's important to remember that while we're creating these tools to measure these different things, it's another thing to put them in the hands of patients where they're going to be executed in their bedrooms, in their kitchens in with their family. Making that convenient while patients are giving us a gift of their time.
‘This is time that they're taking away from their families from their own time and from their personal efforts. To some extent, but also for posterity and for other patients in making a difference. Enabling that, not squandering that, gifting and not burdening that patient is one of the things that this team really does fantastically well - from the scientific, to the research and implementation point of view.’
He said these things are important and to make sure that there is that hand-holding relationship between what they're trying to achieve from a scientific perspective, but also making sure that the quality of life of the patient is not overly impacted just by the mere process of trying to gather that insight.
‘There’s a myriad of ways we can engage with the patient. You look at a protocol and your protocols say that you need me to administer certain types of instruments at this type of cadence - there could be 100 different ways that you can do that.
‘I've seen a lot of bad designs too, but you can ease the subject through the lifecycle of the of the study, so it doesn't feel like a tremendous burden because some people shudder at the thought of say, doing a daily food diary every day for a year - just the thought of that sounds bad. So, instead of doing that, what about just taking a picture of your dinner every night? And not only that but I will prompt you, and you just take the picture, and then that's it.
‘Those are the types of things that we're going more towards.'
Investigational new drug application
On whether the guidance and regulations are encouraging people to be reactive rather than proactive, Anthony said he has not come across a single sponsor who focuses on one area at a time with a thought to giving other areas attention at a later date.
‘When the guidance comes out, it's important guidance. Everybody wants to get to the point of doing their investigational new drug application and to getting their drug approved for marketing.
‘They want to show that they have adhered to the guidance as closely as possible. Regulators rarely come out with guidance because we are all doing things right. They come out with guidance because they think we can do better but the expectation is we continue to do so and don’t stop improving.’
On diversity, Anthony went on to explain that while there are around eight billion devices around the world, not everyone has one and that there were socio-economic concerns there, which he said can all lead to bias.
He said on the diversity front IQVIA are definitely in the process of working on their presence in a number of countries in the different areas of the world they want to attempt studies.
‘It is absolutely increasing including the diversity part of it. We all have to figure out how do we help facilitate all the topics. It's easy in Boston, everybody is going to have a cell phone but how are you going to do that in South Africa, the Congo, parts of Central America to make sure you can capture the voice of those patients as well.’
Both agreed that at the beginning of the pandemic DCT was not only the big buzzword, but it’s also what a lot of companies were doing. Everybody thought it was going to be the future, and that nobody would want to go into clinic.
‘And what we're seeing now is we have sites that want to see patients, we have patients that want to see clinicians, and what we're really seeing is a hybrid approach to be able to capture all the data as accurately as possible.’
