Update on Managed Access and OBAs in Canada
October 31, 2023
Managed access and outcomes-based agreements have moved from the visioning stage to the drawing board, with new and exciting implementation processes under way.
Managed access has crossed a threshold in Canada: from interesting solution to item on stakeholders’ to-do lists. With drug innovation pouring in from all sides, and approval for promising medicines sometimes granted on the basis of incomplete but promising evidence, managed access has emerged as a fair and logical way to handle the deluge.
The surge of interest in managed access comes not a moment too soon, as many patients have been waiting – and waiting – for access to the treatments that could change their lives. Having travelled a long and hard road, often for years, they deserve to see change.
While managed access and outcomes-based agreements (OBAs) are hardly new ideas, Canadian policymakers have had understandable concerns about adapting them to the country’s unique healthcare architecture. How exactly would managed access work in our multiple payer environment? Could our infrastructure handle the added complexity?
In the interim, other countries have demonstrated that these concepts can work – and more importantly, figured out how to make them work. They have shown us that managed access can take many forms, with components mixed and matched depending on a jurisdiction’s needs and health system.
In Canada, it’s no longer a question of whether to implement these schemas, but of when and how – with some exciting new policies and initiatives coming to fruition. Here, we explore what stakeholders are doing to make timelier access to novel therapies a reality across the country, and where managed access and OBAs fit in.
THE ACCESS GAP
Specialty pharmaceutical products often face an awkward hurdle in their life cycle: as highly promising and disruptive technologies, they may receive relatively fast approval, but patient access lags far behind. For months or even years, patients come up against an impenetrable wall, consigned to “look at, but not touch” the treatments that could change their lives.
Approval of promising and complex treatments based on Phase 2 trials is becoming more common. These medications typically have narrow indications and serve limited populations, making it difficult to conduct a sufficiently powered Phase 3 trial15 – the standard evidence required by regulators. Patients with the serious conditions targeted by these drugs often have limited treatment options, with a rapidly closing window of time to change the course of their disease.
Decision makers are increasingly relying on Phase 2 data not just for drug approvals, but for determinations about public reimbursement. In such cases, health technology assessment (HTA) bodies may struggle to determine the value of the treatment, leading to greater reluctance to recommend it for listing. Indeed, oncology drug submissions based on Phase 2 trials are significantly less likely to receive a positive recommendation from CADTH, one of Canada’s HTA agencies, than those backed by Phase 3 data.10
Even if a drug does ultimately receive a listing, the delay between regulatory Notice of Compliance (NOC) and public reimbursement leaves many Canadian patients in limbo for long stretches of time: an average of 580 days for oncology therapies and 670 days for orphan drugs.14 These figures make Canada one of the slowest among OECD-20 countries to publicly reimburse innovative medicines, with a mean of 926 days from launch to listing, compared to the OECD-20 median of 519 days.14
Leading the charge
Why the gap? One reason: a lack of formalized early access pathways in Canada.1 The UK, France, Germany, Italy, and Australia have established formal pathways for publicly funded early access, enabling patients to receive promising new therapies while decision makers gather the evidence required to make a final recommendation.5
An analysis of managed access in England between 2016 and 2022 determined that, of the 22 treatments (of which 20 for cancer) reevaluated following a period of managed access, 19 were recommended for routine use.14 Most of the evidence submitted to resolve the uncertainty identified in the managed access agreement came from clinical trials, and some from real-world data (RWD). The researchers who conducted the analysis affirmed that “without managed access, these technologies would likely not have been recommended for routine use within the NHS in England.”
“Without managed access, most of these innovative technologies would likely not have been recommended for routine use.”
– Authors of an ISPOR paper on managed access in England
Going even further, with the political will and the right systems in place, access delays can quite literally disappear. Four cancer drugs approved via Project Orbis,16 a framework for concurrent regulatory review of oncology drugs developed by the FDA and adopted by partner countries, became available to UK patients on “day zero” – the very day the products were approved.8 The early access agreements that made this possible sped up patient access to these four drugs by an estimated 200 days each – a crucial benefit for patients with aggressive cancers whose treatment options narrow every day.
Learning from leaders: 3 other countries with unique managed access experience17
|
Country |
Experience with managed access agreements |
| Spain |
The Catalonia region has used results-linked reimbursement systems since 2011. In 2016, the region introduced a centralized system for implementing risk-sharing agreements between manufacturers and the CatSalut health service. |
|
Italy |
Managed access agreements (MAAs) are a standard feature of Italy’s drug regulatory process. To evaluate drugs covered by such agreements, the Italian Medicines Agency relies on comprehensive and continuous collection of data, including real-world data from patient monitoring registries. |
|
Israel |
A third party (the MAA team) identifies health technologies most suitable for MAAs and then mediates negotiations between manufacturers and payers. After an MAA expires, the treatment will not be removed from the Israeli formulary, though the price may change. |
National patchwork
The Canadian healthcare system, meanwhile, has no formal managed access policies or processes in place.1 No doubt, having multiple public payers presents a challenge in this regard, and Canada does not benefit from the lockstep stakeholder alignment found in single-payer systems with established managed access frameworks. Instead, to date Canadian stakeholders have been managing early access needs on an informal, case-by-case basis.
One approach taken by Canadian stakeholders has been to propose innovative market access agreements that support timely access, notably OBAs. Precision oncology and rare disease medications have emerged as a natural fit for OBAs, due to their high costs, the niche populations they serve, and the urgency of the clinical need they address. Absent a formalized framework, however, concerns about administrative burden, infrastructure, and evidence requirements have exerted a drag on implementation. At present, an estimated 10 to 20 “simple” OBAs exist in Canada, with payment models tied to outcomes such as disease progression scales used in clinical practice.7 Canadian payers have confirmed that OBAs exist in the areas of oncology, hepatitis C, HIV, multiple sclerosis, ophthalmology, and cardiology,8 but we’re still just scratching the surface of feasible pan-Canadian solutions.
SETTING THE STAGE
At the same time, signs of forward movement are all around us. By all accounts, Canadian policymakers recognize the need for a consistent and equitable approach to early access, which requires government oversight and coordination between HTA bodies, the pCPA and payers. The year 2023 saw major movements in this space, with initiatives that manifest an openness to new approaches to support timely access for patients – including the makings of the first managed access pathway in Canada.
Early access leadership: Three giant steps
To move the agenda forward, CADTH has implemented a new HTA process for time-limited recommendations (TLR) in September 2023, after stakeholder consultation earlier in the year.4,31 The process applies to drugs that receive NOC with conditions (NOC/c) and sets the deadline for bridging evidence gaps at 3 years, after which new evidence must be provided for reassessment within 270 days. This framework makes it possible to issue a time-limited recommendation for therapies with immature but promising evidence, while enabling additional evidence to be generated and considered in CADTH’s final recommendation. At present, CADTH regards only confirmatory Phase 3 clinical-trial data as suitable evidence to support reassessment and, while not accepting RWE as a matter of course, is open to considering it as supplementary.32
“We believe the introduction of time-limited recommendations is another way that health technology assessment can enable the timely and appropriate adoption of innovation and help governments expand access to treatments while managing uncertainty and risk.31
– Suzanne McGurn, CADTH President and CEO
Building on CADTH’s TLR process, the pan-Canadian Pharmaceutical Alliance (pCPA) has proposed a Temporary Access Process (pTAP) to inform listing agreements for medicines that follow CADTH’s time-limited recommendation pathway.5 pTAP lays the groundwork for manufacturers and public payers to establish an equitable, time-limited risk-sharing agreement to ensure the “sustainability and proper management of public funds in both the temporary funding period and beyond.” If public funding does not continue beyond the temporary access period, the proposed process specifies that manufacturers would have to provide coverage “for any patient started on medication during the temporary period where for any reason public funding is not continued beyond the temporary period.” A public engagement questionnaire on the process was open in August 2023, and updates are anticipated later this year.
In step with these efforts, in a June 2023 communiqué Quebec’s HTA body INESSS invited pharmaceutical manufacturers to amplify their drug submissions with exploratory economic scenarios, which INESSS will include in advisories to the provincial health ministry.6 Examples of such scenarios, which aim to optimize drug use and contain costs, include agreements to manage the budgetary impact of new medicines of uncertain value, agreements tied to performance, dose-ceiling agreements, and manufacturer-funded treatment initiation. “It’s great to see that public institutions are open to modernizing the approval and listing process,” says Eva Villalba, executive director of the Quebec Cancer Coalition, noting that “it will be important to include patients in these reforms to ensure they truly create value.”
New Canadian processes to enable timely access
1. CADTH’s Procedures for Time-Limited Reimbursement Recommendation
2. pCPA’s Temporary Access Process (pTAP)
Getting real with real-world evidence
In fact, the whole managed access/OBA paradigm depends on the judicious use of RWE. As noted by Brad Millson, IQVIA Canada’s General Manager, Real World Solutions, “RWE can play a pivotal role in informing managed access agreements by answering questions that cannot be done through traditional clinical trials in appropriate timeframes.”
“RWE can play a pivotal role in managed access by answering questions that cannot be done through traditional clinical trials in appropriate timeframes.”
– Brad Millson, General Manager, Real World Solutions, IQVIA Canada
While 20Sense has already reported on the progress of RWE earlier this year, some developments that could impact the managed access/OBA paradigm are worth noting here. In May 2023, CADTH published a Guidance for Reporting Real-World Evidence, laying a foundation for the use of RWE in regulatory approval and HTA in Canada.18 The document includes a detailed checklist to guide the submission of acceptable RWE. After the Guidance underwent a consultation process that drew 54 responses, CADTH published a feedback-response document described as “a crucial first step toward the end goal of integrating RWE into decision-making in Canada.”19 Next on the agenda: implementation procedures specifying how RWE will be appraised and used in decision making.
Quebec has also had its eye on RWE for many years now. As early as 2017, the 10-year Quebec Life Sciences Strategy proposed to establish an innovation fund to accelerate the adoption of new technologies and the use of RWE in research and elsewhere.20 As a case in point, an INESSS lung cancer study used administrative clinical data to estimate the real-world outcomes of patients on a novel therapy called EGFR-TKI and compare the results to those reported in published studies.21
MAKING IT HAPPEN
More than anything else, the managed access model requires buy-in from all stakeholders. The good news: if current activity is any indication, stakeholders are as eager as policymakers to get the ball rolling. The initiatives described below are paving the way to implementation.
Big-picture HTA
Managed access is making strides in British Columbia. An interactive session in the May 2023 CADTH Symposium, called Lifecycle Health Technology Assessment for Precision Oncology, reported on the progress toward a managed access infrastructure in the region, from data collection and use of RWE (including patient-reported outcomes) to managed access agreements.22 The research challenged the evaluative process that constrains timely access and proposed a life-cycle approach to HTA as an alternative.23 This big-picture model considers the value of a drug holistically, across all its phases. According to Dr. Tania Bubela, dean of Simon Fraser University’s Faculty of Health Science, “implementation of life-cycle HTA has the potential to accelerate patient access to cost-effective new therapies.”30 The caveat: “The approach depends on ongoing monitoring of patient outcomes, which will require close coordination between regulators, payers, and systems.”30
In line with this thinking, BC Cancer is piloting the life-cycle HTA framework within a program called PREDiCT [Precision Evidence Development in Cancer Treatment], co-funded by Roche Canada and the Canadian Personalized Healthcare Innovation Network. 22,29 The pilot’s goal has been to apply the framework to a drug that has not undergone a complete HTA review and to use health-system data to generate RWE, while facilitating early access for patients. If the pilot phase proves successful, the PREDiCT framework will help shape new reimbursement pathways for personalized cancer treatments.29
Better access on the Atlantic horizon
In addition to delivering value, new access pathways must save time. With this vision in mind, New Brunswick’s Horizon Health Network has partnered with Amgen Canada to develop a new access pathway for innovative treatments that target a specific type of lung cancer.24 The pathway runs parallel to the existing process for introducing new medications to market. Crucial to the scheme is that the province’s Regional Health Authority will have direct access to therapy. Margaret Melanson, Horizon’s interim president and CEO, notes that “the traditional pathway for bringing innovative medical solutions to market in Canada is very linear and can take two to three years from start to finish.” The new access approach “is providing an express lane for patients to have access to new treatments.”
The project will identify a group of eligible patients in the first year, with the potential to expand in the future. John Snowden, Executive Director at Amgen Canada, applauds “the original thinkers at Horizon Health Network in New Brunswick who took on the challenge of navigating a complex system to put patients first. Echoing Ms. Melanson’s comments, Mr. Snowden notes that “Canada’s reimbursement review system is slow – delaying access by about 22 months after a medicine is approved by Health Canada. This new framework in New Brunswick will identify patients for treatment with a prescription medicine that targets a specific type of lung cancer that is metastatic or not amenable to curative therapy.”
Other initiatives on the must-watch list
Here are four initiatives that promise to yield both information and action on the managed access and OBA front.
1. The RWE and OBA Working Group, a collective devoted to researching RWE and OBAs and removing implementation barriers for Canada, recently conducted research with public payers and drug manufacturers to gain insight into which OBA financial models are appropriate and preferred by Canadian stakeholders.8 Stay tuned for the publication of the results.
2. In June 2023, CADTH announced upcoming changes to its reimbursement review process.25 Intended to tighten all stages of the review process, these changes include streamlining review requirements for certain drug products, exploring how to incorporate new evidence midstream in a review, exploring innovative models such as OBAs, among others. The announcement also pledges to “bring greater transparency and clarity to our processes” and to “better engage with industry, clinicians, and patients with a view toward improvement.” What’s not to like?
3. Montréal InVivo, a nonprofit organization dedicated to fostering life science and health innovation in Quebec, has established a committee to explore value-based reimbursement agreements in the province.26 The initiative will go through several phases, including identification of suitable therapeutic indications and uncertainties, characterization of the reimbursement agreements proposed for the selected treatments, RWE collection and analysis, and recommendations for guidelines and best practices. To Robin Durand, Montréal InVivo’s Director of Innovations in Life Sciences, the project’s greatest value lies in “the framework that we will develop and put into play to manage therapeutic uncertainty, using RWE to leverage better access to new science.”
4. The Government of Canada has recently pledged to invest $1.5 billion over 3 years to implement its Rare Disease Drug Strategy, endorsed in 2019 to accelerate the diagnosis and treatment of rare diseases.27 The vast majority of these funds ($1.4B) will be distributed to provinces and territories to improve access to new and emerging treatments, with the rest allocated to advancing research, evidence collection, and governance structures for rare diseases. Best case, the investment will lead to routine collection of RWE for rare-disease drugs and support eventual OBAs or other forms of managed access for these treatments. The devil will lie in the details, but the funds are an excellent start.
WHAT NEXT?
As these initiatives exemplify, managed access and OBAs have moved up the priority list for Canadian stakeholders. Other countries have shared their experiences with us, on the podium and in publications, and home-grown research has helped us understand how these tools might support innovation and best practices in this country.
Helen Stevenson, founder and CEO of the Reformulary Group and a leader in the specialty pharmaceutical space, frames the opportunity succinctly: “Every new specialty drug confronts us with three key questions,” she says. “Does it work better than standard treatment? Does it not work as well as expected? Does it cost more?” Canadians need “good information so they understand that some drugs work better than others and there is usually more than one option to treat a condition.” 28 Managed access and OBAs can give us the means to support access to the right treatments, collect data in the real world, and optimize outcomes for each patient.
While a step in the right direction, the initiatives described here will not necessarily streamline access to the extent patients require and deserve. We need not just progress, but commitment to setting and reaching access goals. As we move forward in the space, we must keep our focus on the central question: How can managed access approaches and OBAs be implemented to enable timely access for patients to novel therapies and ensure value for payers?
That’s the crux of the matter: access and value. If a medication shows promise of value, patients should have access to it while we fill in the evidence gaps. Managed access schemes and innovative market access agreements such as OBAs make this possible. Let’s keep breaking down the barriers.
References
1. Cowling T et al. Early access for innovative oncology medicines: a different story in each nation. J Med Econ 2023;26:944.
3. The 20Sense Report. Issue 16, April 2021. https://static1.squarespace.com/static/61d708f9587415184afa9452/t/620684a04735485171ed5dac/1644594337712/Issue_16_EN_The_20Sense_Report.pdf
4. Proposed Process for Time-limited Reimbursement Recommendations. CADTH. March 2023. https://www.cadth.ca/sites/default/files/Drug_Review_Process/CADTH_TLR_Consultation.pdf
5. pCPA temporary access process (pTAP). pCPA 2023. https://www.pcpacanada.ca/pCPATemporaryAccessProcess
7. Canadian Outcomes Based Agreements Experience and Perceptions: Survey Results, RWE & OBA Working Group, October 2021.
8. 20Sense original research.
10. Li YYR et al. Reimbursement recommendations for cancer drugs supported by phase II evidence in Canada. Curr Oncol 2020;27:e495.
14. Bee C et al. What goes in must come out: an analysis of NICE recommendations for drugs exiting managed (early) access in England. NICE poster. [Full article in Value in Health 2022;25:S478.]
15. Dubuc A. Cancer treatment: Canadians don’t have access they expect and deserve, Oncology Medicine Access Roundtable. April 2022.
16. Project Orbis. FDA Oncology Center of Excellence. https://www.fda.gov/about-fda/oncology-center-excellence/project-orbis
17. McPhail M et al. Should Canada adopt managed access agreements in Canada for expensive drugs? J Law Biosci 2023;10:1.
18. Guidance for reporting real-world evidence. CADTH. May 2023. https://www.cadth.ca/sites/default/files/RWE/MG0020/MG0020-RWE-Guidance-Report.pdf
19. Guidance for reporting real-world evidence: response to stakeholder feedback. CADTH. May 2023. https://www.cadth.ca/sites/default/files/RWE/MG0020/MG0020-Response-Document.pdf
20. 2017-2027 Quebec Life Sciences Strategy. Government of Quebec. 2017. https://www.economie.gouv.qc.ca/fileadmin/contenu/publications/administratives/strategies/sciences_vie/strategie_sciences_vie_ang.pdf
22. CADTH 2023 symposium. Preliminary program. https://symposium.cadth.ca/event/c35022f2-d7f1-4500-bb79-e823ee12d7fc/websitePage:7b14e083-a2ac-4a29-8145-6f4f3da026af
24. Innovative partnership will deliver expedited access to cancer treatment for New Brunswick patients. Horizon Health Network. June 6, 2023. https://horizonnb.ca/news-releases/innovative-partnership-will-deliver-expedited-access-to-cancer-treatment-for-new-brunswick-patients/
25. Upcoming improvements to the CADTH reimbursement review process. CADTH. June 5, 2023. https://www.cadth.ca/news/upcoming-improvements-cadth-reimbursement-review-process
26. Projet sur les ententes basées sur la valeur. Montreal INVIVO presentation. July 6, 2023.
27. Government of Canada improves access to affordable and effective drugs for rare diseases. Health Canada. March 22, 2023. https://www.canada.ca/en/health-canada/news/2023/03/government-of-canada-improves-access-to-affordable-and-effective-drugs-for-rare-diseases.html
28. Technology and healthcare walk hand-in-hand to provide accessibility, with Helen Stevenson. The Brand Is Female podcast. August 22, 2023. https://www.thebrandisfemale.com/podcast/season6ep32
29. Roche Canada Announces Collaboration to Improve Access to Personalized Healthcare with Real World Evidence. BC Cancer. May 28, 2021. https://www.bccrc.ca/dept/ccr/news/roche-canada-announces-collaboration-improve-access-personalized-healthcare-real-world
30. Life cycle health technology assessment: real-world evidence for precision oncology. Genome BC. November 23, 2022. https://www.genomebc.ca/blog/life-cycle-health-technology-assessment-real-world-evidence-for-precision-oncology
31. CADTH’s Time-Limited Recommendation Category Aims to Support Earlier Access to Promising Drugs. CADTH. September 2023. https://www.cadth.ca/news/cadths-time-limited-recommendation-category-aims-support-earlier-access-promising-drugs
32. CADTH Procedures for Time-Limited Reimbursement Recommendations. CADTH. September 2023. https://www.cadth.ca/sites/default/files/Drug_Review_Process/CADTH_Time_Limited_Procedures.pdf