Frameworks for Outcomes-Based Agreements
April 22, 2021
OBAs need to offer a degree of certainty in a landscape riddled with unknowns. They also need to integrate with HTA, payer processes, and commercial strategies—to “speak the same language,” as it were.
List now, revisit later
In the EU, countries with well-developed managed access pathways, such as France, Germany, Italy, and the UK, manage uncertainty by requesting RWE and using it to reassess a drug’s value.20 The model follows more or less this sequence:
- HTA assessment
- Creation of an OBA to enable quick listing, with built-in requirement for RWE generation
- Agreement to the commercial terms and pricing of the drug within the OBA
- Collection of RWE
- Analysis of RWE to reassess the drug’s health-economic value
- Price adjustment as necessary, and closing the OBA
Such a model can only work if the health-economic evaluation complements the initial clinical assessment of the medication, rather than competing with it—one of the principles outlined in the UK’s Commercial Framework for New Medicines.27
National and provincial leadership
Though not quite as far along the path as Europe, Canada is taking steps to align its HTA process with commercial realities—what Suzanne McGurn, president and CEO of CADTH, refers to as “health technology management over the lifecycle.”33 In a recent podcast, McGurn noted the “opportunity to bring [this life-cycle approach] forward as part of what CADTH does.” This will involve “figuring out which are the right things to go back and look at.”
The province of Quebec has embraced a similar vision in its 2017-2027 Life Sciences Strategy, which counts faster access to promising drugs and support for RWE projects among its objectives.34 Recent HTA recommendations by INESSS [Institut national d'excellence en santé et services sociaux] confirm that the province is walking the talk. These recommendations generally follow the model previously described —accelerated recommendation, followed by RWE generation and reassessment—as exemplified by Galafold, a drug for Fabry Disease. Assessed in late 2018, Galafold represents the first instance in which INESSS evaluated a drug based on a promise, rather than definitive proof, of clinical value. 35 Just a month later, INESSS recommended listing the spinal muscular atrophy (SMA) drug Spinraza on the condition of RWE generation and clinical follow-up, with the possibility of delisting at a future date if the data fails to demonstrate clinical value.36
INESSS took a similar route with another game-changing SMA therapy, Zolgensma, in recommending an initial listing followed by reevaluation of the drug within 3 years based on real-world data collected by the manufacturer. 37 CADTH followed suit with a positive recommendation in March 2021, though with more “classic” eligibility criteria and a request for a price reduction.38 Zolgensma manufacturer Novartis had evidently hoped for more, as reflected by its community statement expressing both satisfaction with the positive recommendation and disappointment that it is “limited by age, without a mechanism for case-by-case review.” 39 While the provinces typically wait for HTA recommendations and pCPA negotiations to be complete before proceeding to drug listing, Alberta decided Zolgensma called for a new approach: the province listed the drug on a case-by-case basis just six weeks after Health Canada approval.40
“There is no budget cap—we are focused on kids not falling through the cracks while the regular review processes are ongoing,” 41 said Alberta Minister of Health Tyler Shandro in a Jan. 2021 announcement, giving families affected by SMA new reason to rejoice. As SMA parent Lana Bernadin put it, “we feel a great sense of joy that no other family will be faced with raising money for the world’s most expensive medication.”11 While Ontario has yet to announce a formal program to cover Zolgensma, the province stated it would authorize it on a case-by-case basis after a family launched a GoFundMe page to help pay for the drug. 42
RECORD TIME
The province of Alberta raised the access bar to new heights when it listed Zolgensma, the world’s most expensive drug and a game-changer for patients with spinal muscular atrophy, just 6 weeks after Health Canada approval.
This approach lines up with the European access program for Zolgensma, Day One, which runs ahead of the HTA process to speed up access and gives payers outcomes-based rebates along with the option to defer payments to manage budget impact.43 From the manufacturer’s perspective, the collection of RWE before pricing negotiations can bolster the value story and alleviate payer concerns about cost-benefit.
Done right, OBAs promise wins for both manufacturers and payers—and above all, for patients.
What Canada can learn from the UK
A peek at the UK’s drug appraisal and commercialization strategy reveals some forward-thinking developments. In the oncology realm, the Cancer Drugs Fund (CDF) marries the complementary objectives of speeding up access to cancer medications and giving pharma companies a fast track to NHS funding.44 Reformed in 2016 to curb the approval of cost-ineffective treatments, which had increased by £185 million in the previous year, the CDF focuses on drugs for which ongoing data collection can resolve clinical uncertainty.26
More recently, in its 2021 Commercial Framework for New Medicines, the NHS set out broad principles to bring clinical assessments and commercial negotiations into alignment, including “avoiding burdensome data collection and disproportionate additional cost” in OBA-type arrangements.27 The managed access agreements (MAAs) described in the document give pharma companies a mechanism to “offer a value proposition at or below the lower end of the standard NICE cost-effectiveness threshold range.”27 Key components of these MAAs include a data collection agreement to mitigate clinical uncertainty and a commercial access agreement. This high-level coverage of commercial options for complex drugs makes the document a unique source of learning.
References:
11. Families of Alberta children suffering from spinal muscular atrophy (SMA) may now be eligible to receive funding for gene replacement therapy treatment. Cure SMA news release. https://curesma.ca/2021/01/28/zolgensma/
20. Drawing the blueprint for Canada’s rare drug program 2022. CORD webinar. January 29, 2021. https://www.youtube.com/watch?v=cBD5hsD7q8Y&feature=emb_logo
26. Walton MJ et al. A review of issues affecting the efficiency of decision making in the NICE single technology appraisal process. Pharmacoeconom 2019;3:403.
27. NHS commercial framework for new medicines. https://www.england.nhs.uk/wp-content/uploads/2021/02/B0255-nhs-commercial-framework-for-new-medicines.pdf
28. Real-world evidence and outcomes-based agreements working group. 2019 research & outputs. https://static1.squarespace.com/static/58fd16af1b631b1afffae9e0/t/5f8e437dd9a86a5bd6b09076/1603158910148/2019_Nov_RWE_OBA_WorkingGroup_ExecSummary.pdf
29. Spearpoint P. Implementing a managed entry agreement within the EU. NextLevel Pharma presentation. Oct. 13, 2015. https://www.youtube.com/watch?v=wSEDxa6MOBc&feature=youtu.be
30. Health Technology Innovation Platform. Institute of Health Economics. https://www.ihe.ca/research-programs/innovation/htip
31. CanREValue: value-based decisions from real-world evidence. Canadian Centre for Applied Research in Cancer Control. https://cc-arcc.ca/canrevalue/
32. Public summary document. March 2017 PBAC meeting. Section 6.09, crizotinib. https://www.pbs.gov.au/industry/listing/elements/pbac-meetings/psd/2017-03/files/crizotinib-psd-march-2017.pdf
33. NPC Healthbiz weekly podcast. Feb. 16, 2021. https://healthbiz.substack.com/p/s03-e03-keeping-evidence-current?r=5jlzh&utm_campaign=post&utm_medium=web&utm_source=linkedin
34. 2017-2027 Québec Life Sciences Strategy. Gouvernement du Québec. https://bit.ly/2ZqCCku
35. Galafold. Inscription—avec conditions. INESSS. Oct. 2018. https://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_au_ministre/Novembre_2018/Galafold_2018_10.pdf
36. Spinraza. Inscription—avec conditions. INESSS. Dec. 2018. https://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_au_ministre/Janvier_2019/Spinraza_2018_12.pdf
37. Zolgensma. Inscription—avec conditions. INESS. Dec. 2020. https://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_au_ministre/Janvier_2021/Zolgensma_2020_12.pdf
38. Zolgensma. CADTH final recommendation. March 26, 2021. https://cadth.ca/sites/default/files/cdr/complete/SG0649%20Zolgensma%20-%20CDEC%20Final%20Recommendation%20March%2026%2C%202021%20for%20posting.pdf
39. Novartis community statement—CADTH recommendation and access to Zolgensma. March 30, 2021.
40. Jury still out on whether the targeted negotiations process (TNP) impacts pCPA metrics. Morse Consulting. March 17, 2021. https://morseconsulting.ca/jury-still-out-on-whether-the-targeted-negotiations-process-tnp-impacts-pcpa-metrics/?utm_source=wysija&utm_medium=email&utm_campaign=pCPA-review-feb-12
41. Announcement about decision to list Zolgensma. Province of Alberta. January 21, 2017. https://www.youtube.com/watch?v=BuHRfRZo9Wo&feature=emb_logo
42. Laucius J. Ontario to cover cost of drug for rare neuromuscular disease on a ‘case by case’ basis. Ottawa Citizen, Jan. 8, 2021. https://ottawacitizen.com/news/local-news/ontario-to-cover-cost-of-drug-for-rare-neuromuscular-disease-on-a-case-by-case-basis
43. Grubert N. Zolgensma’s “Day One” programme—an intriguing new venture for managed entry in Europe. https://www.linkedin.com/pulse/zolgensmas-day-one-programmean-intriguing-new-venture-neil-grubert/
44. Cancer Drugs Fund. NICE. https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/cancer-drugs-fund