CAPT Conference Workshop: Defining “Decision Grade” Real World Evidence (RWE) and its Role in the Canadian Context: A Design Sprint
On Sunday October 21, 2018, the Canadian Association of Population Therapeutics (CAPT) hosted a full day workshop to discuss recent advances in the dialogue on RWE in Canada from a Regulatory, HTA and Reimbursement perspective. The day was attended by participants invited from various stakeholder groups including Health Canada, CADTH, Provincial Cancer Agencies, Provincial payer agencies, the pan-Canadian Pharmaceutical Alliance (pCPA), researchers/clinicians; patient stakeholder groups and industry.
The objectives were to identify the:
- value and applications of RWE in supporting pharmaceutical regulatory and reimbursement decision-making; and
- conditions upon which RWE will be considered of sufficient quality to inform decision-making.
The workshop began with 15-minute lightning presentations provided by Tammy Clifford (CADTH); Rhonda Kropp (Health Canada); Kelvin Chan (CCO) and Michael Duong (Hoffmann-La Roche), to set the context for the remainder of the workshops for the day. All these talks focused on the challenges that existing evidentiary requirements currently present for the introduction of new products specifically for the treatment of rare disease and certain cancers.
Summary of Key Points:
- Current evidentiary requirements are challenging and potentially not feasible for drugs used in the treatment of rare diseases and in oncology.
- Health Canada and CADTH are focused on partnership and alignment on approach for data requirements for regulatory as well as HTA assessment.
- Health Canada and CADTH will be prepared to offer advice to manufacturers pre- and post market for RWE initiatives as appropriate.
- If parallel product reviews are undertaken by Health Canada and CADTH with the overall goal of minimizing time to access, health care systems need to be “ready” simultaneously to track/study RWE associated with a product to avoid losing the time saving benefit of parallel product review. Being “ready” means that if RWE is part of a recommendation, the mechanism and process for data collection and evaluation needs to be in place.
- Health Canada and CADTH expressed commitment to partnering with all stakeholders across a product’s full life cycle and to ensure a consistent and transparent approach.
- This RWE approach requires a paradigm shift from current state of HTA to health technology management, ongoing collaboration, system infrastructure and capacity building, optimization of current databases and quality data capture, consistency in approach and transparency.
Two hypothetical case studies were reviewed as part of the workshop sessions, one in the oncology setting and one in the setting of a rare disease. Participants were divided equally into these 2 sessions with other attendees in observer roles.
In the oncology workshop there were 4 groups with stakeholders representing patient advocacy organizations, CADTH, Health Canada, clinician/researchers and industry (IMC and BioteCanada). The participants were first tasked with listing 8 areas of focus for RWE collection for the hypothetical case.
The second task was for the groups to discuss and identify where in the hypothetical product lifecycle (from Phase 1 through product maturity) the 8 areas of focus should be used/be of benefit. The facilitator collated and recorded all responses from the 4 groups and while there was some overlap, there were ~17 cases where RWE could be used.
In the third task, each group was challenged with aligning on a priority use case from the close to 17 cases that had been identified by all participants.
Finally, for their last task, groups discussed the data quality requirements for each use examples of RWE use included support for: label expansion; removing a condition associated with NOC/c; informing/refining HE modelling, re-pricing and reassessment for ongoing funding; gathering of patient reported outcome data, patient preferences and caregiver burden.
Identifying decision grade quality standards proved to be a challenging exercise with groups commenting on the difficulty with identifying standards common to RCT, observational, patient preference and anecdotal data. Ultimately, all groups recognized that the prioritization of use of RWE would be context specific and unique to each product’s data profile/ongoing data requirement to demonstrate benefit.
Implementation considerations were discussed at the end of the day and the following points were raised:
- review of international standards to inform Canadian approach: don’t reinvent the wheel;
- technical solutions are available, but the human element of transparent and collaborative dialogue must be considered;
- identification of practical considerations: who owns data, how is it collected, who pays;
- guidance from Health Canada and CADTH will be case specific but the focus for all is on collaboration and early dialogue with all stakeholders;
- initial guidance will be discussed but could evolve over time;
- clarity is required for an approach, but it cannot be prescriptive and should not include criteria but rather focus on factors to consider; and
- barriers discussed encompassed trust among parties, appropriate risk sharing (as opposed to one party assuming all risk), groups have previously operated in silos, data ownership, payment, consent, legal considerations.
The regulatory and HTA agencies in Canada are moving to adopt the inclusion of RWE in product evaluation across the product life cycle and key areas of focus are oncology and drugs for rare diseases. Critical to success is transparent and ongoing dialogue focused on factors to consider for each product’s unique situation, practical implementation approaches, proactive versus reactive stance and ensuring a healthcare system approach. It was discussed that patient support programs could be an excellent source of data and have the potential to be credible sources of rich data to inform payer decision making. This assumes patient support programs are properly initiated to ensure data is collected with a degree of rigour and quality that instills confidence for all stakeholders to inform decision making. Time should be spent evaluating the legal, financial and perception risk incurred with data collection to inform RWE but there is a very strong signal that there will be continued collaboration between Health Canada and CADTH to support RWE initiatives and this workshop will evolve to be a standing sustainable forum for ongoing dialogue.