Words by James Coker
“Please sir, I want some more.” That immortal line from Charles Dickens’ classic novel Oliver Twist is just as relevant for considering the unprecedented amount of evidence being demanded within the healthcare industry. Driven by a shift towards personalised therapy, early detection, and outcomes-based pricing, just like young Oliver with his request for extra food, a much larger portion of evidence is required to feed modern clinical development.
Shane Woods, VP, Life Sciences, Flatiron Health, says: “This demand for evidence is putting additional pressures on traditional approaches to evidence generation. So the clinical trial is seeing more and more pressure.”
It is in this context that the potential of real-world evidence (RWE) has come to the fore, with methods such as electronic health records, patient registries, and observational studies providing an efficient way of measuring endpoints over a long period. It is certainly true to say that the gates standing in front of the riches RWE has to offer are beginning to swing open to the pharma industry. “There have been recent advances in policy that are changing what’s possible and we’re seeing RWE actually elevated over the last 3 or 4 years as a legitimate evidence-generation avenue”, comments Woods.
What we’re trying to find out is how we generate precision evidence that can actually be used for regulatory and reimbursement purposes
Yet there is currently a distinct lack of unity regarding its practical use in clinical development. A major issue is that different stakeholders tend to attach varying degrees of value to the forms of RWE collection. Dirk Vander Mijnsbrugge, VP, Medical Affairs Lead International Developed Markets, Pfizer, explains: “Patients and patient organisations definitely attach a lot of value on patient-reported outcomes (PRO). Other groups pay relatively less attention, certainly not to the same extent as they do for comparative effectiveness or safety when it comes to treatment patterns, adherence, and compliance with RWE. And then when it comes to cost-comparison or cost-effectiveness, we typically see higher value being attributed by institutions, payers, and also health technology assessment (HTA) bodies, but to a lesser extent by regulatory authorities and patients.”
It is now incumbent upon medical affairs, with their honed communication and information sharing practices, to become the vehicle for finding a coherent approach to implementing widespread use of RWE in clinical development.
The first step is establishing the precise role of RWE as a partner to clinical data. “The development of the RWE strategy is complementary to a clinical development programme”, says Mijnsbrugge. “There’s no point in establishing a RWE plan or strategy where your clinical development plan is finished or close to termination: you need to do it right from the very beginning and hand in hand with your clinical development programme.”
This requires the type of close collaborations with relevant parties that has become the hallmark of MA. While talking to patients and patient organisations to help establish PRO measurements in clinical trials is now standard practice in pharma, the same must now be replicated when deciding upon a RWE strategy.
Additionally, it is vital that consideration is given to bringing the product to market and establishing a value-based pricing method early. “By bringing together RWE and seeing its application in new ways, what we’re trying to find out is how we generate precision evidence that can actually be used for regulatory and reimbursement purposes”, outlines Jeff Elton, CEO, Concerto Health AI.
We really need to work with the regulators on the acceptability of RWE and MA definitely have a role to play in that
To not also closely consult with regulatory authorities, HTA bodies, and payers before completing a RWE strategy would be like setting up a football club but failing to register for a league to play in. Parallel advice meetings have been shown to be effective in enabling pharma to get a clear understanding of the views of regulators and payers, and this could be particularly fruitful when creating a RWE blueprint. It is also a two-way street, in which MA can explain to and persuade stakeholders using their knowledge of RWE, ultimately designing the most effective approaches. “We really need to work with the regulators on the acceptability of RWE and MA definitely have a role to play in that. And I think it’s important for us to participate in those multidisciplinary groups where regulators, pharma, and academia come together”, comments Pol Vandenbroucke, Chief Medical Officer, Pfizer Hospital Business Unit.
In a broader context, MA also have the opportunity to become the catalyst in establishing a much higher degree of coherence on the use of RWE in the future. “When we see guidelines in the UK, Germany, France, and other countries in Europe, they’re not necessarily aligned, and the expectation is that they will be very different from one issue to another. It may be difficult to address different needs of different HTA bodies going forward so I think it’s a call for action together”, states Mijnsbrugge.
With practices and processes still a work in progress for RWE in clinical development, MA should cast themselves in a lead role in establishing strategies that revolve around the interests and insights of various stakeholders. With RWE set to form part of the transformative new age of healthcare, MA has the chance to ensure it can work for the benefit of patients in the shortest possible time.