Can true value-based pricing ever be a reality in Europe? If the answer is no, we may well be headed towards a price/access “crisis” as rising oncology costs continue to outstrip country healthcare budgets. If the answer is yes, it certainly seems like there’s still a long way to go.
A key theme at this year’s European Society for Medical Oncology (ESMO) conference was, once again, how to ensure access to valuable new medicines. And as usual, this discussion took place against the now well-established backdrop of increasing pricing pressure and disparities in access and outcomes throughout Europe. Across all of these presentations, one theme stood out: The current approach to European pricing and access may have a missing link in the process for connecting the value offered by an innovative new medicine with a fair price for which payers should be willing to grant access.
It’s always easier to identify problems than it is to propose solutions, and this was indeed the approach taken by many at the conference. Manufacturers were frequently criticized for inappropriate trial design or altogether excessive pricing, while the inconsistent approaches to health technology assessment (HTA) between EU nations also came under fire. However, amidst the finger-pointing, we identified a few topics that seem to be gaining significant momentum. Could one of these topics be that missing link?
Broader Application of ESMO’s Magnitude of Clinical Benefit Scale
Like their colleagues at ASCO, an ESMO working group launched a value framework in May 2015 that’s intended to act as a validated tool for the consistent appraisal of the benefit offered by new cancer therapies. The framework has been updated based on feedback from the medical community, and the group presented “version 1.1” at this year’s conference.
To date, the framework has primarily been studied from an academic perspective, but the authors clearly have ambitions to see the framework assisting in European HTA. Analysis presented at this year’s conference suggested that trials aren’t being designed with the ESMO criteria in mind (only 38% of 109 recent randomized controlled trials met the scale’s threshold for clinical benefit), and the team also claimed that there’s currently no relationship between cost and benefit. In fact, the group observed a negative correlation between cost and the score awarded.
As part of these presentations, speakers made a direct appeal to HTA bodies to enhance the assessment of value within their processes for drug review and price negotiation. Whether or not manufacturers think that the scale is a fair approach to the assessment of value, and whether or not they agree with the feasibility of a consistent assessment of value given differing academic and political perspectives between countries, they should remain aware of the moves that the group is taking to strengthen the scale’s applicability. These include:
- Integration of the scale within ESMO clinical guidelines (accelerating the scale’s exposure across the medical community)
- Coordination between the American Society of Clinical Oncology and ESMO with an eye to the potential convergence of the two organizations’ value frameworks (adding additional “weight” behind the framework)
- Collaboration between ESMO and Germany’s Institute for Quality and Economic Efficiency in Healthcare (potentially paving the way to proof of concept for the scale’s applicability as part of European HTA)
Linking Value-Based Payment Models With the Increased Application of Adaptive Pathways
While still in their infancy, adaptive pathways appear primed to become an established route to market for novel oncology medicines. The objective is to achieve a prospectively planned adaptive approach to drug licensing that balances timely access with the need to ensure adequate insight into a new drug’s benefits and risks. This requires:
- A prospectively designed iterative development plan
- Availability of real-world data to supplement clinical trials
- Adequate input of all stakeholders, but particularly HTAs, to ensure that manufacturers are appropriately incentivized to pursue this route to market
As part of the European Medicines Agency’s priority medicines program, known as PRIME, a well-defined process is emerging for the design of an adaptive development plan, supported greatly by the offer from regulators for enhanced scientific advice and early dialogue.
However, the latter two requirements remain harder to come by. To borrow a phrase from the conference, “Real-world data is easy to say but difficult to capture.” Particularly in Europe, the landscape for real-world data is fragmented and heterogeneous. Manufacturers that wish to pursue adaptive pathways will need to carefully evaluate the multitude of real-world data options available to them (such as electronic health records, disease and drug registries, early access programs, third-party vendors, etc.), and ensure that these can provide a necessary level of accuracy and depth of information to support their development plan.
But the final and most challenging piece of the puzzle remains the payer. While forums such as HTA Network and the European Network for Health Technology Assessment continue to cooperate on the issue, and recommendations recently have been published for potential synergies between regulatory HTA interactions, the process for ensuring the appropriate reimbursement of medicines approved through adaptive pathways remains unstructured and fluid. One key (and likely required) change will be more innovative payer reimbursement models that allow manufacturers to be paid different amounts depending on the specific value offered by their medicines in different treatment settings, accompanied by increased flexibility that changes to reimbursement as new evidence is gathered.
In short, manufacturers embracing adaptive pathways will need to be open to broad collaboration across regulatory and HTA agencies, and should be ready to exert a point of view when required.
Expanding the Application of Patient Outcome Measures
Part of the difficulty in defining value lies within the different ways that drug outcomes may be measured. Not only are these sometimes intangible in nature, but the outcomes that are most important to a patient may not be the same as those that are important to a physician, and these, in turn, may not be the same outcomes that are important to a payer. Common types of outcomes include:
- Clinical (quantifiable measures of disease signs and symptoms)
- Patient (physician- or patient-reported measures reflecting experience on the drug)
- Health system (direct economic benefits of a drug’s impact on healthcare costs or resources)
- Societal (indirect economic benefits resulting from the drug’s patient benefits)
Patient outcomes have found recent favor with payers and manufacturers alike as an additional reason to believe in a drug’s value. Along with the increased use of traditional patient outcomes such as quality of life, manufacturers also should be aware of emerging measures such as the benefits/risk assessment, which reflects a patient’s own preferences for or against treatment given her unique point of view on a product’s potential benefits and risks.
Such measures could allow companies to demonstrate their value propositions in a way that’s relevant to all stakeholders at the famous “dinner for three,” providing patients greater insight to inform their decision-making process, physicians better understanding of the patient considerations to accommodate when making a recommendation, and payers better insight into the real-life value offered. Such insights may be particularly useful to payers as a modifier of assessment results in cases where the clinical benefit is marginal.
At what rate payer systems will adopt these novel measures into their traditionally rigid assessment procedures remains to be seen. However, some agencies have already started to discuss them as part of their scientific exchange.
With the tagline for ESMO 2018 already set as “Securing access to optimal cancer care,” manufacturers can expect an increasing need to express their positions on the solution to the challenges facing European pricing and access.
Those manufacturers that are willing to evaluate emerging opportunities, embrace external partnerships and invest in bold new solutions may stand to profit from being the first to fix the “missing link.”