The Future of Clinical Trials
Patient-centric trials offer huge opportunity but what are the barriers to implementation?
Patient recruitment and retention are two of the challenges currently facing the pharmaceutical industry’s approach to clinical trials.There is now a growing groundswell of opinion from within the industry which suggests that a more patient-centric approach is necessary.
“It’s not just about feeling sick; it’s also about feeling well.” That’s what one patient advised a researcher who was organizing a clinical trial of a treatment for depression. According to Sanofi’s Chief Patient Officer Dr Anne Beal, the researcher acted on the patient’s advice and “… found the treatment had little impact on the classical depression measures, but a huge impact on wellness and patient adherence ... Without this patient’s insight [the researcher] … would have completely missed the relevance of his intervention. Had he used just his own methods, he wouldn’t have found anything significant.”
This telling vignette - emphasizing the crucial importance to research of input from the patient - is recounted in eyeforpharma’s new whitepaper “Clinical Trials: How Bringing Patients to the Centre Can Cut Costs and Deliver Better Outcomes”.
The Challenge
The challenge to the industry is clear. For example, in 2003 IBM’s Institute for Business Value predicted: “Inefficient patient recruitment processes will increasingly become a formidable barrier to pharmaceutical companies’ success in launching new products.” This prediction was confirmed by recent research showing not only that a mere ten per cent of trials finish on time, but that 40 per cent of trial costs are spent on patient enrollment. And when eyeforpharma surveyed 427 representatives of pharma, biotech and contract research groups, the top two issues they identified were the speed of trial enrollment and the management of timelines.
One of the industry’s responses to these challenges has been an attempt to cultivate a more patient-centered approach to clinical trials; an idea that is now gaining traction. Thus, 75 per cent of those questioned in the eyeforpharma survey stated that one of their priorities for 2015 was “putting the patient at the heart of the clinical trial.”
What is “patient-centric”?
But how does intent translate into action? First, the terms must be defined; so what is a patient-centric clinical trial? Chris Frega, Head of Global Feasibility for Quintiles says it “… is one where there is an end-benefit to all patients from access to new therapies, while also making it as easy as possible for patients to participate in those trials.” And Tanja Keiper and Paulo Moreira – Associate Director, Global Clinical Operations, External Innovation at Merck Serono and Head of External Innovation, EMD Serono, respectively –define patient centricity in clinical development as “… any improvement in trial design, new technology or service that either directly or indirectly has a positive impact on the experience of the patient who has volunteered to participate.”
Quintiles Head of Patient Recruitment Programs, Bernadette Tosti makes the point that a patient-centric approach includes “… things like accommodating people on weekend hours or evenings to fit around patient schedules …” But when Tosti notes that “in the last few years what we’ve asked patients to submit to has become a lot more intensive and we need to balance that”, might one infer that the emphasis of clinical trials has not always been patient-centric? Sanofi’s Dr Anne Beal seems to think so: “The industry has often been a payer-centric and a physician-centric system, less than a patient-centric system.”
However, David Wright, Amgen’s Global Head of Trial Planning and Platform Services, disagrees: “I think patients have always been at the center of clinical trials. The patient-centric approach emphasizes exploring new techniques for designing and conducting trials with the aim of improving recruitment, retention and compliance.”
Barriers to a patient-centric approach
Having attempted to define a patient-centric approach, what are the barriers to its implementation? Anne Beal considers that the ethical and legal standards of what is a tightly regulated industry “… affect our ability to engage and talk with patients. And they vary by region and country. It’s a complicated maze that we need to navigate.”
She also highlights the challenge of dealing with a wide patient spectrum, at one end of which are patient advocacy groups with so-called “professional patients”. At the other end, Beal says, “… are single mothers with two kids and a full-time job – people who don’t have time to be involved with patient associations, for instance.” She points out, however, that “… [they] represent the lion’s share of the population we’re trying to serve, yet they may not have the time to be active in a patient group.”
For Chris Frega of Quintiles, “one of our biggest difficulties is time. You can never have enough time to plan studies.” A further difficulty Frega encounters “… is balancing the scientific and data needs versus the operational ability to conduct studies. Trials are very expensive and time-consuming, so you want to get as much out of the process as possible. That way, you have a higher likelihood of finding a benefit to somebody.”
Meanwhile, Amgen’s David Wright observes that “the biggest challenge is striking this balance between complexity and reducing trial burden. It’s particularly important with vulnerable or pediatric populations and trials with long follow-up and frequent site visits. Those are the types of trials that pose a high risk for recruitment and retention.”
The role of technology in patient-centricity
When eyeforpharma’s whitepaper considered which technologies are most vital in enabling patient-centricity, Sanofi’s Anne Beal noted that there are “tremendous opportunities” as far as patient engagement is concerned: “For example, if you have panels for patient surveys, you can send out questions via text message. That way, you can tap into millions of patients for real-time data very quickly.” Beal also sees opportunities for patient self-management, “… for instance allowing patients to monitor their own weight, blood pressure, glucose levels …”
Dr Beal, however, also highlights the risk of technology being seen as a “shiny new toy”: “It should never be a substitute for something that is well thought out. Technology should always be a means-to-an-end that leads to patient-centricity.”
Both Paulo Moreira of EMD Serono and Merck Serono’s Tanja Keiper identify communication technologies and tools as “… the most vital technology components for enabling patient-centricity. A few buzz words in this context are: crowd-sourcing; the use of big data; communication flow to and from patients; patient-reported outcomes and assessments; [and] de-centralized clinical trials.” As with Beal, Moreira and Keiper sound a note of caution, pointing out that technology is not a solution for everyone: “We must keep in mind that there are areas of the globe that are not yet networked or where patients may not have access to technology themselves or be technologically savvy.”
The future
eyeforpharma’s whitepaper asks what the priorities will be over the next two years if we are to get closer to a process of patient-centric drug development.
Moreira and Keiper identify three key priorities: sticking to the patient-centric vision; thinking globally; and following through on commitments made to patients and patient advocacy groups. They also note that “EMD/Merck Serono is now engaged in assembling Patient Advisory Boards for all of our trials. The first one of its kind was held recently and proved to be a success story … What we heard from [patients] loud and clear was how important it was to have their voice heard and to make a difference for fellow patients undergoing clinical trials.”
Dr Beal also identified three priorities, “… the first being that we need to change our mind set. We need to engage patients as partners and it’s really incumbent upon us to open up.” Second, and having “opened up”, Beal is concerned “… to think about whether patients are ready to step in. The question is which outside groups we should be working with, whether it’s patient advocacy groups, people who come and volunteer or online communities.” She is emphatic that “[w]hat we need to avoid is having the first three patients represent all the patients who are out there.” Beal’s third priority concerns regulatory and legal issues: “It would be great if some of the regulatory requirements move beyond safety and efficacy to include patient-centricity or relevancy to patients.”
Bernadette Tosti of Quintiles is clear that “… we’re moving towards more meaningful engagement with patients. With the emergence of so much health information on social media and other digital channels, we’ll be able to get a better grasp of what patients are saying, and we’ll be able to engage with them actively around specific protocol design issues and understand attitudes towards treating their disease.”
Re-emphasizing the need to define one’s terms, Tosti’s colleague at Quintiles Chris Frega foresees the evolution of a “… stronger, more unified definition of what patient-centricity is. Right now, it means so many different things to many different people. The way organizations are implementing the concept is different.”
And Amgen’s David Wright anticipates a need “… to better understand the value of different techniques and approaches for enhancing patient engagement. Then, based on that, the question is how can we best embed those practices in the organization in the way we design and conduct trials.”
Patient-centricity is both a defensive response to these problems and a more positive embracing of an opportunity.” The whitepaper considers that the patient-centric approach can cut costs; provide greater convenience to patients; improve clinical results by making research relevant to people’s lives; and “… capturing the totality of latent insight in the patient population and fusing that with the best professional know-how and technology available.
Conclusion
eyeforpharma’s whitepaper collates the thoughts and observations of leading players in the pharma industry. Having noted the challenges involved in the recruitment and retention of patients for clinical trials – quite apart from the “… complexity of research, the growing legal and regulatory burdens, and some unhelpful reputational issues …” – it states: “Patient-centricity is both a defensive response to these problems and a more positive embracing of an opportunity.” The whitepaper considers that the patient-centric approach can cut costs; provide greater convenience to patients; improve clinical results by making research relevant to people’s lives; and “… capturing the totality of latent insight in the patient population and fusing that with the best professional know-how and technology available.”
Given the probability that at some time or another in our lives we will all be patients, a patient-centric approach to clinical trials should not only be encouraged within the pharmaceutical industry; some might argue that it should be demanded.
To download a copy of the eyeforpharma whitepaper "The Future of Clinical Trials: How Bringing Patients to the Centre Can Cut Costs & Deliver Better Outcomes", click here.