At the TELUS Health Annual Conference on May 3, 2023, a panel of industry experts shared their insights into how real-world evidence (RWE) can enhance reimbursement decision-making for private drug plans. Moderated by Daria O’Reilly, Lead Health Economist at TELUS Health, the discussion focused on the potential RWE has to help payors manage rising drug costs by providing a wealth of information about drug performance drawn from outside clinical trials.
The private payor perspective
Panelist Bobby Currie, Manager, Pharmaceutical Relations, at Canada Life, and a registered pharmacist, works with her team to evaluate drugs for drug plans based on clinical, economic, and stakeholder value and negotiate drug prices for product listing agreements. Clinical trial data, she said, often comes with a lot of uncertainty – for example, if only a small number of people were included in the trial or if the size of the patient population is unknown.
"When a drug is showing a lot of promise in a devastating or life-threatening condition, sometimes patients don’t really want to wait for every i to get dotted and every t to get crossed. As a payor, though, we really need to be thinking about [questions such as] is that drug going to live up to that promise [and] is there value for money?” she said. “Real-world evidence can help fill that gap. It can help us confirm effectiveness for those patients and support that assumption of value.”
Currie sees potential for RWE to improve access by enabling more effective risk-sharing, improve lifecycle management by providing an ongoing stream of additional information after the launch of a new drug, and improve health outcomes by supporting preventative interventions. Challenges include data silos that make it difficult to integrate data, diverse needs that require collaborative RWE study designs, and a need for robust, reliable data to make results actionable.
"We're just getting started with real-world evidence, [but] we need to make really good decisions, which means we need the best available evidence."
The public payor perspective
Panelist Nicole Mittmann, Chief Scientist and Vice- President, Scientific Evidence, Methodologies and Resources, at the Canadian Agency of Drugs and Technologies in Health (CADTH), defined RWE as “evidence about the use, safety and effectiveness of a medical product, technology or drug that is based on data from the real-world health care setting.”
Mittmann said RWE is playing an increasing role in health care decisions, but she emphasized that it doesn’t replace clinical trials and it doesn’t translate directly to greater access. While it can fill uncertainty gaps, it doesn’t provide causality and it cannot and should not be used for all drugs.
One of CADTH’s strategic goals in this area is to develop guidance for the use of RWE, including recommendations for quality and reporting standards for all RWE submissions intended for use in regulatory or health technology submissions in Canada.
“The work that we’re trying to do is expand the evidence base, and then [decide] how do we enhance our deliberations using that evidence at our organization [and] how do we actually start [to] build a common metric around [RWE] with respect to standards and outcomes,” Mittmann said.
The RWE producer perspective
Panelist Karine Grand’Maison, Vice-resident, Access and Government Relations at Pfizer, explained that RWE is best understood as a way to complement clinical data and demonstrate the full value of a therapeutic innovation beyond what is measured in clinical trials. For private payors, she said, it can provide an enhanced ability to assess value for plan sponsors and plan members, understand how a treatment meets the needs and preferences of plan members, enable innovative contracting, and support timely and appropriate drug access to the Canadian workforce.
Pfizer also recently ran a RWE master class that included global and Canadian private payors, public payors and patient association groups – all interested in advancing RWE decision-making.
“The workforce in Canada is counting on all of us to provide timely and appropriate access to drug treatments and vaccines. In some cases, we’ll need real world evidence to help facilitate the value assessment and the reimbursement decision,” Grand’Maison said. “The best way to make that happen is to collaborate as early as possible in the process so we can make sure that all the perspectives are taken into account.”
The consultant’s perspective
Panelist Catherine Beauchemin is a partner at PeriPharm, which works with pharmaceutical companies to develop key components of the submission dossier that payors use to make reimbursement recommendations. Before approving a drug, Health Canada asks two critical questions: is it safe and does it work? Payors have a range of other questions, which often include, how does it compare with existing treatments, is it good value, is it needed and is it affordable? These are often best answered with RWE.
Through three case studies, Beauchemin explained how a gap in RWE can impact decision-making:
- An oral treatment where the standard of care was injectable was expected to improve patient convenience, satisfaction and adherence – but there was no evidence to support this, so payrs didn’t recognize the therapeutic value of the innovation
- Some RWE was available to support the effect of a new mental health drug on work productivity and caregiver burden – but only from other countries, not Canada, leading to increased uncertainty in economic evaluations and pay
- There was limited Canadian evidence of a rare disease drug’s impact on patient quality of life, health state utility values and healthcare resource utilization – but having to look at data from another disease again increased uncertainty in economic evaluations and pay
To provide new sources of RWE, PeriPharm has launched the Proxy Network. Its goal is to help generate patient-centred evidence to inform decision-making, and it has already completed its first study of a migraine treatment with 100 participants.
“Canadian payors need comprehensive evidence on the therapeutic and economic value of a new drug,” Beauchemin emphasized. “Although real-world evidence will never replace the randomized controlled trial, it undeniably brings value to drug assessments and helps better inform decisions. Considering the increasing cost of specialty drugs and drugs for rare diseases, real-world evidence will certainly become a pillar in health care decision-making.”
Consensus that RWE can help protect drug plan sustainability
Though each of the four panelists examined RWE from a different perspective, they agreed this type of information can be extremely valuable in helping private drug plans navigate an environment of rising drug costs. There are obstacles to overcome, but there is also tremendous potential to improve decision-making.
As O’Reilly explained, “Some pharmaceuticals may enter the market with limited evidence [and] there’s pressure for the payors to make pricing and reimbursement decisions about these drugs at the point of market launch. To further complicate things, many of these drugs have a very high price tag, and thus decisions about their value are challenging – which can result in delaying or even denying listing of drugs on plan formularies.”
Part of the solution going forward, the panelists said, will be RWE. Given that the top 10 most expensive drugs from the TELUS book of business in 2022 showed the average cost per claimant ranged from $400,000 to $1,800,000 a year, plan sponsors need all the information they can get to make the best decisions to protect the long-term sustainability of their drug plans.
View the full presentation:
