Making the Patient-Centric Trial a Reality

Learn what a panel of experts had to say on how we can create value for patients in trials, keep them engaged, and treat them like gold.



While the concept of patient centricity has become part of the industry vernacular, its effective implementation on the ground has continued to bamboozle many for whom how to optimize patient advocacy and patient centricity remains an unanswered question.

A recent webinar provided an insight on metrics, culture change and the patient perspective when it comes to patient centricity, and sought to tease out exactly how valuable partnerships between patient advocacy groups and industry can be built and delivered. Aspects such as measuring patient perceived costs to create effective trials, better understanding of the patient perspective to increase study reach, and how to design trials in order to find the sweet spot between patient benefit, risk and time, were all covered in depth.

Partaking in the webinar were three people who know more about true adoption of patient-centricity than most. Jamie Roberts, Senior Clinical Project Manager, Clinical Trials Transformation Initiative (CTTI), Victoria Dibiaso, Global Head Clinical Operations Strategy & Collaboration, Sanofi, and Deborah Collyar, President, Patient Advocates in Research (PAIR), all gave their views on what defines patient centricity, its impact on the clinical development process, and how true engagement between the various stakeholders can deliver value and positive outcomes for all parties. They participated in an interview following the webinar, addressing a number of key questions that the discussion elicited from participants.

Not just a buzzword

When it comes down to it, is patient centricity simply a buzzword? While it can be an awkward term, the speakers agree that it has come to represent the wider shift in the clinical development process where the focus is more on patients instead of regulation and products.

According to industry insider Dibiaso, patient centricity is the “essence of what we’re trying to achieve”.

“Anything that we do comes from the perspective of patients, she says, adding that early engagement is critical.

“We work with patient organizations early on in the study development stage – it’s not just tactical outreach, it’s done with the goal of advancing drug programs. It is a natural progression, as the natural next step is to have a design that is appropriate – we need their support,” she says; time spent building collaboration and partnership with patient groups means that effective outreach is easy, with no requirement for a third party.

Roberts echoes this, stating that while patient centricity is a philosophy, patient engagement is the operationalization of that philosophy.

“We have made the moral argument for patient centricity and patient engagement is what we need to do and we need to figure out next just how we do that.”

The need for guidelines

But how exactly should industry engage with patient advocacy groups in order to achieve the best possible outcomes? The speakers agree that there is possibly a need for a universal framework, with guidelines and regulations to frame all. In the absence of this, the CTTI has recommendations for specific types of trials, as well as a broad range of implementation tools.

Trust and transparency are two non-negotiable elements of any partnership between industry and patient advocacy groups. Building trust must be the first step, while transparency ensures all stakeholders are on the same page.

Roberts asserts that transparency is key – the industry organization and the patient advocacy group must be clear on the specific goals of the research project.

“The CTTI recommendations or clinical trials addresses that very question – in terms of considerations that are important to research sponsors and patient advocacy groups. Transparency is a major one and being well-aligned on the goals of the research project itself.”

According to Roberts, diversity is another keyword, and she believes industry should strive to engage with a wide variety of patient advocacy groups and organizations – and vice versa.

“You need to be diverse in who you engage with, for instance, encouraging patient groups not to engage with just one industry organization but to identify any industries that might be working in their space and make sure they are identifying what the landscape look like. The same thing goes for sponsors, they need to understand which patient advocacy organizations exist where their interests lie and also where their expertise and assets lie, so making sure they are aligning their programs with the right patient advocacy groups.”

Collyar agrees wholeheartedly: “All of those points are incredibly important. We discussed in the webinar that there are different types of advocacy group and different levels of input that you need at different times during the development process.”

According to Dibiaso, communication and dialog with the patient group or groups in question should not end after the initial early engagement. She believes that it needs to have “full loop closure”.

If we want to be true partners, we need to have the discussion as part of a development partnership that we are mutually vested in moving forward the clinical research in the best manner possible to suit the needs of patients… rather we have to come up with a collective approach.

“In our experience, we need to keep those groups that we have engaged with in the loop as to what came of that engagement – often times in development, we get mandates from health authorities requiring certain things to be built into a clinical trial but we have to be very clear and transparent about things that we cannot change, but also when we do get feedback, we should really be taking that seriously and making the adaptations where possible. I want to make sure the industry understands that collaboration means acting on advice that we have received.”

Diabiaso believes it is important that clear guidelines on collaborations between industry and patient organizations are established, so that “true partnership is demonstrated”; “It is very important that companies do not use patients and patient advocacy groups.”

True partnership

Indeed, some companies may be of the opinion that adopting a guise of patient centricity is merely a fast route to enhancing the notoriously difficult process of patient recruitment and retention.

Harking back to her earlier point on early engagement, Dibiaso states firmly that industry should not begin a discussion with patient groups “when recruitment is in trouble”.

“If we want to be true partners, we need to have the discussion as part of a development partnership that we are mutually vested in moving forward the clinical research in the best manner possible to suit the needs of patients… rather we have to come up with a collective approach.”

Collyar echoes this, adding that one should naturally follow the other.

“I see recruitment as being a by-product of good patient engagement because if we have engaged the community and created a study that is centric to their needs from an emotional, physical and medical perspective, that we have communicated in a manner that they feel is important, made that information available to them, and they can actually find us, patients should become part of the process and that translates into them signing up for the project and being included… It can’t just be tokenism.”

Diabiaso agrees that principles such as those from CTTI are important because they give a framework around which to think about this, but notes the difficulty in developing guidelines to cover all types of engagement with diverse patient groups, each with different goals.

“To actually operationalize it, I think it is important to understand the motivation of each group and what I mean by that is, what is their main focus for the patient world of advocacy? Some groups focus on fundraising, watchdog advocacy, or research, among other aspects. Depending on what the questions are at the different points in the development process, it will depend on what information comes through, and that comes back to Jamie’s point about speaking to different groups. There are lots of different voices.”

Patients as value-drivers

Industry has finally woken up to the fact that there is a cacophony of patient voices who may be able to help with multiple aspects of the drug development process. Dibiaso explains that Sanofi and others are now working on a model that would help companies and sponsors identify the best groups to speak to at different times, adding that this is at the very early stages.

“It is a project based on the whole idea of creating a framework towards working with advocacy groups and it is very much built around what guidelines do we use to identify these groups, what approach do we use to connect with them.”

Collyar adds that PAIR has also done work in this area, coming up with recommendations for best practice for engagement between research sponsors and academia and the patient advocacy communities. This included helping patient groups to identify what exactly they can offer to trial sponsors; for example, what value-drivers they can offer industry.

“We spent two years working on this, and created some tools which allow patient advocacy groups to realize where their assets are, and create an ‘inventory’ of these assets, so they can really begin to promote those assets to the industry organizations that come to it asking for partnership.”

Identifying where a patient advocacy group’s assets can fit along the research and development continuum and knowing what they can bring to the table, allows these groups to highlight the importance of these assets to key value-drivers for industry. Collyar adds that industry must be able to articulate to patient advocacy organizations what their shared goals are but also listen to their needs and respond appropriately. She says that guiding principles should include transparency, respect for privacy, and continued communication throughout the process so that the loop is circular; “It is about valuing the voice of the patient advocate as part of the philosophy of becoming patient-centered.”

Diabiaso notes a key problem is that many patient advocacy organizations lack knowledge of the clinical research process, as it may not have been their focus.

“They are supportive but they may not understand the implications, especially when we start talking about study design and things like that. On my wish list would be to have a consortium pool of training tools available to use with advocacy groups. That probably needs to come more from us in the advocacy community but building those tools could be a collaboration between industry and advocacy.”

We do tend to get greedy once we begin designing a study about how much we can learn, without thinking about the burden not only on the patients but also on the site.

Roberts strongly agrees; she says that patient organizations are keen to engage in “building the architecture”.

While previous pilot schemes have been moderately successful, inherent challenges remain and there is a need for a bigger effort, she feels.

“If each has mutual objectives and a desire to work together, you can solve these problems. In terms of study design, it is not the science but more about the patient centricity – how many procedures, the demand on patients.”

Indeed, the true burden of a clinical trial on each patient must be determined – balancing what is necessary versus what is possible.

Collyar says PAIR often asks how many additional procedures and tests are truly necessary for the trial – or are merely those that the researchers are curious to know about.

“A lot of times, the input that we give is ‘do you really have to have those monthly procedures, couldn’t they be quarterly instead?’. What is going to answer the scientific question here rather than what you are simply curious about?”

Roberts says this very issue is addressed comprehensively in the CTTI quality recommendations by CTTI, noting that researchers tend to “become greedy” when they are faced with a patient population ripe with hitherto unknown data.

“We ask, can you tie every procedure that you are putting into that study into the primary endpoint or a necessary secondary endpoint – if not, just get rid of it. That may sound like an over-simplification, but we do tend to get greedy once we begin designing a study about how much we can learn, without thinking about the burden not only on the patients but also on the site.”

It is Collyar’s belief, however, that trial participants should be treated “like gold”.

“What most of them want is how much information can you find out about me, so rather than wasting their contribution, we do want to find out as much as possible and not just answer a single question. But that’s still got to be within reason, and it can’t cause someone undue inconvenience or even harm. There is a balance to be struck.”

Roberts adds that the CTTI quality by design recommendations suggest that if an important opportunity to learn more arises, carrying it out on a smaller, representative subset is preferable, “rather than having a massive loss due to follow-up because the patients can’t deal with the burden”.

The tipping point

The speakers agree that the concept of patient centricity is now at a tipping point. It has developed from an idea into an ethos widely adopted and endorsed by industry, academia, and the entire clinical trials space. Yet its implementation remains patchy, and best practice examples should serve to inform the wider research community how true patient engagement should be achieved.

According to Roberts, as the phrase enters the collective consciousness, she believes that the role of society in clinical trials could expand significantly.

“I am hoping that one of the by-products from this is that society begins to understand more how therapies and treatments ae made and an invigoration around how everybody can play a role in this. You don’t have to have a disease to be a health control, for example. If everybody did something just once, at one point in their lives, we would be able to accelerate the rate of medical advancement at an unprecedented speed.”

Dibiaso says she has observed first-hand the shift in industry groupthink and the natural integration of patient centricity into industry norms.

“We went from, is this just the latest buzz word and then to cautious optimism, and finally to a true realization that this is actually changing things. I attend more and more conferences where I see not just what we did to work with patient groups recruit to our trial, but how we are changing the culture of our organization to make this an important component of how we do business.

“It is as simple as letting our clinical development teams engage with the patient – understanding the embodiment of living with the disease and what we are here to do. It brings things from a very scientific mode into a human into reality. We bring society and the exam room into our office. That has happened by pure natural progression.”


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