Data for Outcomes-Based Agreements
April 22, 2021
The OBA value proposition rests on the collection and evaluation of real-world data (RWD) and real-world evidence (RWE).
In theory, the data can spring from virtually any source as long as the manufacturer and payer agree on its validity—and keep administrative complexity to a minimum. As a leading expert advised in a presentation on managed entry agreements in the EU, “keep criteria for analysis as simple as possible to avoid paralysis.”29
Overall survival, arguably the simplest metric of all, brought clarity to the Australian data collection program for Xalkori, a treatment for patients with a subset of non-small-cell lung cancer. The program supports the managed entry scheme set out for the drug, which requires the manufacturer to rebate a pre-specified percentage depending on 12-month survival rate.32
The UK’s NICE also includes survival, along with ventilation and respiratory events, motor function, and scoliosis surgery, in its data requirements for the spinal muscular atrophy (SMA) drug Spinraza.45 In the interest of preserving data integrity, physicians are required to enter specified clinical data into the SMA REACH registry. The agreement also mandates the collection of patient-reported outcome measures.
A hidden Canadian data gem: the patient support program
Recent months have seen patient support programs (PSPs) emerge as a prime candidate for OBA data collection in Canada. At Market Access Canada’s 2020 summit, AstraZeneca Canada Vice-President Mo Amin highlighted the opportunity for PSP patient data to support access.46. Similarly, AstraZeneca’s Dr. Michael Seewald flagged PSPs as a fitting source of Canadian OBA data and a vehicle for understanding the patient experience in a recent conference on RWE methodologies.47 Building on the theme, a Pfizer presentation at the 2020 CAPT conference positioned PSPs as a potent source of RWE and a bridge between the clinical assessment and lifecycle management of a drug.48
In fact, PSP-based data collection is well underway in Canada. All PSP vendors who responded to a 2020 Canadian PSP data capabilities survey build data collection into their infrastructure, and all but one surveyed manufacturer receive data from their PSP vendors.49 Most importantly, close to half (47%) of manufacturers have used the data for HTA analysis. By all indications, Canadian stakeholders are gaining experience in harnessing PSP data to support market access and reimbursement—and plan to continue to invest in this mechanism.
PSP data stands to benefit all stakeholders. While even basic PSPs help patients gain access to medications, building real-world and patient-reported data collection into a PSP can yield insights that improve outcomes and support OBAs. The insights can feed into the PSP itself in a cycle of continuous improvement, culminating in a program that offers the services that matter most to patients and the best value for all parties.
Figure 1: PSP Levels in Canada: Operational, Strategic and Patient Value
Putting PSPs to work
There is no formula for creating a PSP. Designed to meet specific clinical, market and patient needs, they differ widely in the patient services and data capabilities they offer. From the OBA perspective, this flexibility has a big upside: manufacturers and PSP service providers can plan for the data-collecting mechanisms needed to support a particular OBA. At the same time, some payers may have concerns about data collected within a manufacturer-sponsored program.
Does this concern have merit? And why turn to PSPs for data in the first place? Below, leading Canadian data and PSP experts weigh in.
Tara Cowling, Director and Managing Principal, Medlior Health Outcomes Research, on the benefits of using PSP data over other administrative datasets: “PSP data is typically national-level, longitudinal, with clinical confirmation of included patient populations, which is why they are suited to examining specialty drugs. Some of the data collected from PSPs—patient-reported outcomes, caregiver-reported outcomes, and measures of disease progression, for example—are not routinely captured by health system datasets, which can also help evaluate the cost-effectiveness of a treatment.”
Kimberly Fougere, Associate Director, Offerings Management, STI, on the power of PSP data: “It allows us to provide insights to the manufacturer on patient adherence, payer landscape, and other parameters. Having these discussions before the PSP is designed ensures we can build in the desired data requirements. We take data privacy seriously and follow confidentiality guidelines sourced from Canadian privacy laws.”
Kelly Isaacs, Vice President, NavieGo Patient Programs, BioScript Solutions, on the efficiency and cost-effectiveness of PSP data: “Data from PSPs leverages the existing infrastructure. The ability to collect information directly from patients and physicians is invaluable. When designing a PSP, we work with clients to decide on what data to collect and how to do it. And the earlier it happens in the planning process the better the data can meet the needs of all parties.”
Taflyn Hornibrook, Co-CEO and Head of Patient Programs and Stakeholder Relations, Sentrex Health Solutions, on the power of PSP data to support OBAs and help all stakeholders: “It seems natural to take PSPs a step further and collect data for an outcomes-based agreement. There is an opportunity to define upfront what success looks like for all parties involved in a PSP—patients, physicians, manufacturers, and payers.”
Remi Menes, Vice President, Specialty Patient Programs, McKesson Canada, on the value of PSP data to manufacturers and payers: “PSPs are designed to gather the data points needed to answer key questions about the patient journey, which is very helpful to the manufacturer. PSP data could allow the payer to see how the drug is used and what value it offers. Ideally, to support OBAs, payers would be engaged in the upfront PSP data design process.”
Jodi Adams, Director, Business Intelligence and Transformation, SDM Specialty Health Network, on the opportunity for PSP data in the digital world: “The increasing role of digital channels to connect with patients and caregivers represents a chance for PSPs to collect more targeted information. By working within a PSP framework, payers can gain better access to patient-reported outcomes and real-world data to support coverage decisions.”
Sean McBride, Director of Commercial Operations, Bayshore Specialty Rx, on the challenges and opportunities for real-world data collection: “Real-world data can help answer clinical questions that the trial setting cannot. At the same time, the integrity of the data depends on how it is sourced, collected, and validated. To get the most out of the data, it is important to identify and address inherent gaps or biases that may impact the outcome, and ensure consent and privacy are addressed.”
In brief, the power of data lies in its reliability, which in turn depends on training the right people to do things the right way. Diamonds in, diamonds out.
Whether generated from PSPs or another source, a strong data program builds trust between manufacturers and payers. And trust is the commodity OBAs depend on more than anything else.
References:
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
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
45. Managed access agreement for Spinraza. NICE. https://www.nice.org.uk/guidance/ta588/resources/managed-access-agreement-july-2019-pdf-6842812573
46. The Patient perspective – Instill the Patients’ Voice into Your Market Access Strategy to Enhance Your Product’s Success, Mo Amin, AstraZeneca. Presented at the Market Access Summit, Oct. 7, 2020.
47. Using RWE to Inform Opportunity for VBAs: European Experience and Opportunities for Canada. Dr. Michael Seewald, AstraZeneca. Presented at the RWE Methodologies Conference, Oct. 21, 2020.
48. Modern methods of generating Real World Evidence to demonstrate value. Pfizer. CAPT Conference, Oct. 20, 2020. https://www.capt-actp.ca/wp-content/uploads/2020/10/session-3-IQVIA-CAPT-2020-Conference-Panel-Discussion-Master-Deck_FINAL.pdf
49. Can Canada’s patient support program infrastructure support the collection of real-world data for use in outcomes-based agreements? The RWE and OBA Working Group.