By the Numbers: Managed Access and Outcomes-Based Agreements
October 31, 2023
The road to timely and equitable access to specialty medications in Canada is still under construction. Fortunately, new processes based on managed access models and corresponding market access agreements are smoothing some of the bumps. While managed access means different things to different people, some commonly used definitions can help ground the discourse and give context to the pertinent facts and figures.
Managed access1: Managed access schemes enable payers to fund innovative therapies while developing and gathering additional evidence, prior to finalizing the health technology assessment (HTA) and pricing negotiation processes. These schemes enable timely access for patients with a high unmet need for therapies that have evidence uncertainties.
Outcomes-based agreement (OBA)2: An OBA is an innovative market access agreement between a manufacturer and a payer in which the manufacturer will issue a refund or rebate to the payer based on how well the therapy performs in a real-world patient population, measured against an agreed-upon, predefined set of benchmarks.
Real-world evidence (RWE)3: Derived from real-world data (RWD) such as patient registries or claims data, RWE can be used to support managed access schemes and outcomes-based agreements. Implementing an RWE generation plan for new therapies with uncertain clinical trial evidence can help decision-makers to understand the value of the therapy.
Access innovation in Canada
2: managed access-like processes that may enable timelier access to some novel therapies with a high unmet need; one is a time-limited HTA recommendation by CADTH and the other is a proposed temporary access process (pTAP) advised by the pCPA. 4,5
1: calls for exploratory drug evaluation proposals from Quebec’s HTA body, INESSS.6
10-20: Estimated number of “simple” OBAs in Canada, with payment models tied to clinical outcomes. 7
6: Canadian public payers that have negotiated or implemented at least one OBA. 8
Ongoing access challenges
24.6%: Among cancer drugs that made it to Phase 2 clinical trials, proportion that move ahead to Phase 3; once at Phase 3, 47.7% proceed to filing an application for a new drug.11 Of all clinical phases, Phase 2 represents the largest hurdle in drug development.
19%: Proportion of submissions to CADTH’s pan-Canadian Oncology Drug Review’s Expert Review Committee supported by only Phase 2 evidence, as per a 2020 study. 10 Of these, 63% received a positive recommendation, as opposed to 82% for submissions with Phase 3 evidence.
> 1.5 years: Time to access for new drugs in Canadian public plans. The delay from regulatory Notice of Compliance (NOC) to first listing is about 580 days for oncology therapies and 670 days for orphan drugs.9
65%: Average proportion of drugs approved in Canada that receive public reimbursement within 2 years.9
Coming soon: New drugs that call for new thinking
> 9,000: Number of new medicines at various stages of development in the 2022 pipeline, up from 8,500 the previous year.12
11%: Average annual increase in the number of pipeline drugs since 2018.12
30%: Proportion of 2022 pipeline drugs devoted to oncology – the top therapeutic area in the pipeline.12
31%: Medicines in Phase 3 clinical trials or pre-registration given an early orphan designation through the FDA or the EMA, attesting to the rising trend of orphan-designated new medicines.12
Jurisdictions leading the way
31%: Difference between public coverage in top OECD countries (96%) vs Canada (65%) for drugs that treat unmet needs.9
1 or more: Early access pathways already established in many countries including UK, France, Germany, Italy, and Australia.13
86%: technologies recommended for routine use by the UK’s NHS following a period of managed access.14
0: days to access for 4 novel non-small cell lung cancer (NSCLC) therapies approved in the UK through Project Orbis and listed through early/managed access processes, enabling patients to receive the drugs an average of 7 months before routine listing.8
References
Cowling T et al. Early access for innovative oncology medicines: a different story in each nation. J Med Econ 2023;26:944.
Cheung WY et al. Building infrastructure for outcomes-based agreements in Canada: can administrative health data be used to support an outcomes-based agreement in oncology? Supportive Care in Cancer 2023;31. https://link.springer.com/article/10.1007/s00520-022-07486-5
The 20Sense Report. Issue 16, April 2021. https://static1.squarespace.com/static/61d708f9587415184afa9452/t/620684a04735485171ed5dac/1644594337712/Issue_16_EN_The_20Sense_Report.pdf
Proposed Process for Time-limited Reimbursement Recommendations. CADTH. March 2023. https://www.cadth.ca/sites/default/files/Drug_Review_Process/CADTH_TLR_Consultation.pdf
pCPA temporary access process (pTAP). pCPA 2023. https://www.pcpacanada.ca/pCPATemporaryAccessProcess
Avis aux fabricants de médicaments , de dispositifs médicaux liés à l ’administration de médicaments et de produits du système du sang. INESSS. June 12, 2023. https://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_fabricants/20230612_Avis_aux_fabricants_Invitations_ordonnancement_scenarios_economiques.pdf
Canadian Outcomes Based Agreements Experience and Perceptions: Survey Results, RWE & OBA Working Group, October 2021.
20Sense original research.
Hoskyn SL. Explaining public reimbursement delays for new medicines for Canadian patients. Innovative medicines Canada. http://innovativemedicines.ca/wp-content/uploads/2020/06/20200630-CADTH-TTL-Poster-FINAL.pdf
Li YYR et al. Reimbursement recommendations for cancer drugs supported by phase II evidence in Canada. Curr Oncol 2020;27:e495.
Clinical development success rates and contributing factors 2011-2020. Biotechnology Innovation Organization. https://go.bio.org/rs/490-EHZ-999/images/ClinicalDevelopmentSuccessRates2011_2020.pdf
Meds Pipeline Monitor 2022. Government of Canada. Last modified June 27, 2023. https://www.canada.ca/en/patented-medicine-prices-review/services/npduis/analytical-studies/meds-pipeline-monitor-2022.html
Early access to innovative medicines. Medlior Health Outcomes Research Inc. CORD conference presentation, March 2023.
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.]