Patient Support Program Data: A Uniquely Canadian Source of Evidence
April 18, 2023
Like everything else in healthcare, patient support programs (PSPs) are changing.
First introduced a couple of decades ago, these programs have become a mainstay for hundreds of thousands of Canadian patients taking specialty drugs.4 They help patients access financial support, learn about their medication, and navigate complex health systems to get the care they need. In the process, PSPs gather data – and this is where the most exciting changes are happening. PSP data collection, research, and evidence-generating capabilities continue to evolve. With over 400 PSPs in place in Canada,1 each with data collection infrastructure and capabilities, stakeholders across the specialty healthcare spectrum are taking notice.
What makes PSP data unique
Many patient support programs support the majority of – and sometimes all – patients who begin therapy on any given specialty drug. A large percentage of patients who are still on therapy after two years remain active in the associated PSP,7 and some programs are supporting patients beyond the five-year mark. As such, PSPs provide a major opportunity for longitudinal data collection.
PSPs have the added advantage of design flexibility, offering the opportunity to customize both patient services and data collection. Manufacturers can set up the PSP to collect data that will help fill evidence gaps and generate insights that can improve patient care. IQVIA Canada’s real-world evidence expert Brad Millson agrees: “patient support program data can enrich our understanding of product-specific health outcomes.”
Data collected through PSPs runs the gamut from patient demographics and prior therapies to lab results and treatment outcomes. Duration of treatment, dosing changes, reasons for discontinuation and quality of life: the PSP infrastructure can be employed to capture all these metrics and more. Data collection tools may include PSP enrolment forms, information captured in post-infusion reports and patient questionnaires, among others.
This breadth of data, coupled with the PSP infrastructure in place in Canada, has led Canada’s health care and research vanguards to recognize PSP data’s potential to not only enhance care for patients within a PSP, but to improve health care more broadly. After all, PSPs facilitate large real-world datasets of Canadian patients, presenting an excellent opportunity to fill evidence gaps and generate insights to support better health outcomes in this country.
THE CHANGING FACE OF PSP DATA
One criticism levied against PSP data, common to all observational databases, is that it lacks the rigour of controlled clinical trial data.13 Another is that an industry-funded data source may have a bias. Tackling these challenges head on, the research community has been working hard to elevate the quality of PSP data by engaging independent academic researchers to analyze and validate it. In some instances, these researchers are linking PSP data to other datasets, such as Health Canada’s SAP data14 and global safety databases.10 A few are even using advanced research methods, like predictive analytics, to arrive at key safety and efficacy outcomes – including overall survival.12 The data has been gaining further legitimacy as it finds its way into peer-reviewed journals.New paradigms for PSP data collection and usage
Leading the charge, manufacturers are investing more in PSP data collection capabilities, often bringing in data scientists and engineers to ensure their PSPs have the expertise to collect the best data. Recent years have also seen extensive efforts to increase PSP data quality, including focusing on privacy, ethics, compliance, audits, and data security practices.15 “Appropriate patient-centric data governance and consents, in addition to refined statistical models are increasing the ability to use PSP data,” notes Rana Qadeer, Senior Manager of the Patient Center of Excellence at AstraZeneca Canada, adding that “data quality initiatives like automated checks have made PSP data increasingly comprehensive. It’s an exciting time.”
“Appropriate patient-centric data governance and consents, and refined statistical models are increasing the ability to use PSP data.”
Manufacturers are also collaborating with PSP vendors to set data collection goals “way before setting up the PSP,” says Kelly Isaacs, Vice President of Patient Programs at BioScript’s NavieGo. The conversation “should be based on what clinical trials are doing now and will be doing in the future,” she says. Dr. Winson Cheung, a professor of medicine at the University of Calgary and principal director of the Oncology Outcomes (O2) research program, has experienced this approach first-hand, with “more frequent data-focused meetings and earlier coordination between the manufacturer, PSP vendors, and data experts.” These extra investments have yielded a big dividend: higher-quality data. For example, one PSP service provider moved from recording blood pressure as “normal, high and low” to tracking of the actual numbers, recognizing that more granular data would mitigate bias and categorization errors. With data a priority from the get-go, PSP enrolment forms are being harnessed to gather comprehensive baseline data that can inform both current and future research activities. To ensure cleaner and more complete datasets, many PSPs have moved to fillable enrolment forms, including tick boxes and drop-down menus, which also take less time to fill out.15
In tandem with these structural changes, many research organizations are developing expertise in analyzing PSP data, as they increasingly conduct PSP data studies. They may start small – for example, using deidentified patient data to gain insight into how calls from nurses impact patient adherence.16 Building from there, they may gather data that could be helpful to inform discussions between physicians and patients, such as quality-of-life data.17
Engaging patients in PSP data
It goes without saying that PSP data collection and research depends on patient consent, and manufacturers have been working diligently with their service providers and patients to improve the consent process. As noted by Isaacs at NavieGo, PSPs should ideally have “a single-consent process for research for every patient from the beginning of the program.” In fact, such one-stop consent processes are gaining ground as more PSPs are being set up to enable data collection for research. To reassure patients that the research won’t compromise their privacy, consent forms specify that the data will be deidentified, anonymized, or aggregated.15 PSP service providers and manufacturers are also working to involve patients in decisions around data collection, governance, and use.15
Patient representatives, for their part, largely support the use of PSP data for research with the right protections in place. “The patient voice is integral to improving patient care,” says Ursula Mann, Principal and Chief Patient Officer of Patient Voice Partners, which helps organizations work with patients. “Patient experts value sharing their insights while having their privacy maintained, providing PSPs the opportunity to better understand the patient experience.”
“The patient voice is integral to improving patient care.”
Lisa Machado, founder of the Canadian CML Network, which represents Canadian chronic myelogenous leukemia patients and their healthcare providers, sees PSPs as “a natural vehicle for data collection,” with an important caveat: “Industry needs to work with patients to ensure the outcomes collected matter to them.”
Some companies are doing just that. As Anne Marie Hayes, Director of Patient Experience at Hoffmann-La Roche, explains, the company’s Patient Co-creation Councils help them design patient support programs “through the lens of the experts – those living with the condition.” Involving patients “not only unlocks more value from research, but also builds credibility as real-world data plays an increasingly important role in informing access decisions.”
PSP research is getting published and talked about
With all this energy poured into PSP data research, it’s hardly surprising that we’re seeing such data appear in more and more published studies.15 8 peer-reviewed published studies have used Canadian PSP data and infrastructure as part of the research,15 and the number increases further if we include posters and abstracts presented at conferences.
The fact is, PSP-driven studies can generate real-world evidence (RWE) at a relatively low cost – an opportunity that forward-thinking stakeholders don’t want to miss. These studies, which go through the rigour of ethics research board approvals,12 can provide important RWE that help deliver insights to physicians. In a study published earlier this year, for example, Taiho Canada used its PSP to collect baseline and treatment data. By tracking start dates to treatment discontinuation due to death or disease progression, study investigators were able to estimate progression-free survival in over 100 patients taking LONSURF for metastatic gastric or gastroesophageal cancer.12
In another recent study, IQVIA Solutions used PSP data to gain insights on the benefits of lorlatinib therapy in a group of patients with non-small cell lung cancer – specifically, to understand time to discontinuation and quality of life at various time points during treatment.11 In a poster presentation about these study findings, attendees had the opportunity to weigh in on what might lead health technology assessors (HTAs) to accept this RWE in their decisions about the therapy’s efficacy and value.11 Such information gathering goes beyond a thought experiment: it’s only by sharing and critiquing PSP data that its quality can continue its rise to the level of informing broader healthcare decision making.
“By sharing and critiquing PSP data, its quality can continue its rise to the level of informing broader healthcare decision making.”
WHAT’S NEXT FOR PSP DATA?
If current trends are any indication, future PSPs will align more closely with value-based healthcare principles: tracking and sharing the outcomes that matter most to patients, using data to drive efficiencies in their programs, and customizing patients’ experiences. PSP service provider Sentrex Health Solutions is already working in this direction. In collaboration with experts at an academic institution, the group is building algorithms using retrospective ophthalmology data from their PSPs, with the goal of generating insights that will allow PSP interventions to be fine-tuned accordingly, so the right services can reach the right patients at the right time, and helping them to optimize treatment.15The top rung for PSP data
In the next logical step, PSP data would join the fold of datasets that inform healthcare decision making about a drug’s efficacy and value. As Canada continues to adopt value-based healthcare principles and innovative market access approaches, PSP data could even help expedite patient access to medications. With CADTH hard at work on a framework for the use of RWE to support decision making, we can expect to see some exciting movement in this regard.18 Dr. Nicole Mittmann, Vice-President, Scientific Evidence, Methodologies, and Resources at CADTH, confirms that “PSPs are definitely on our radar. We’re having a lot of discussion around them.”19 While alert to the “potential bias in using a data source developed and funded by industry,” she notes that “independent analyses to ensure the reliability of the data can help overcome the issue.”
Three big ideas to push PSP data forward
So where can we expect to go from here? Stakeholders looking to take PSP data to the next level can set their sights on three tangible goals:
1. Continue to improve data quality. Manufacturers, PSP service providers, and data experts can work together to increase data quality, which will generate trust that the datasets reflect real-world scenarios. The “practice makes perfect” adage applies here: the more stakeholders use PSP data to generate insights, the better they can identify and fix data quality issues that arise.
2. Continue to use PSP data for academic research and publications. This will increase both the industry and research communities’ expertise in using PSP data, as well as HTAs’ and payers’ acceptance of the data as a legitimate source of RWE. Manufacturers are already investing in the PSP data capabilities needed to generate publishable data; it’s now a matter of keeping up these efforts.
3. Prepare PSP data for value-based healthcare. Determine how to make PSP data robust enough to meet HTAs’ and payers’ standards. Once the data rises to the required standard, it also has the potential to support innovative market access agreements such as outcomes-based agreements. This vision feeds into a larger movement toward value-based healthcare – a philosophy that prioritizes health outcomes that matter to patients and judicious use of resources to allocate healthcare services humanely and equitably. To start the conversation: Could a registry of real-world evidence from PSPs raise the profile of PSP research and inspire conversations about standards and best practices? Would more widespread use of third-party analyses of PSP data allay stakeholders’ concerns about bias? To be continued.
The Canadian way
Multifunctional, comprehensive PSPs are a uniquely Canadian phenomenon, and PSP data offers a Canadian-made opportunity to generate evidence to further improve patient outcomes. The immense opportunity for PSP data – specifically, its potential to support real-world research, clinical decisions, more responsive healthcare services, and earlier access to life-changing medications – gives us all the motivation we need to capitalize on this unique data source. Patients, who have the most to gain from RWE generation, are counting on us.
Five Key Advantages of Collecting Data through a PSP
1. The data comes from Canadian patients in the real-world setting.
2. Can tailor data collection to fill evidence gaps.
3. Can capture a large percentage of patients within PSP datasets.
4. Can collect and analyze data in subpopulations for whom clinical studies may not be feasible.
5. Can add data collection at minimal cost compared to other forms of evidence generation.
References
1. Waldron & Associates original research.
4. Innovative Medicines Canada and Ernst & Young LLP. Innovative Medicines Canada Data Analytics and Members’ Economic Footprint and Impact in Canada. October 2017. https://bit.ly/3Z7xxKd
7. RWE OBA Working Group. PSP Data Survey Results. Sept. 2020.
10. Gitman V et al. Pregnancy outcomes of women with multiple sclerosis treated with ocrelizumab in Canada: A descriptive analysis of real-world data. Mult Scler Relat Disord 2022; 62:103792.
11. Vinh On P. Leveraging patient support program infrastructure to gather data to supplement HTA for rare tumors: What constitutes quality real-world evidence? Canadian Association for Population Therapeutics 2022. https://bit.ly/42Cbuyp
12. Ding P et al. Trifluridine/tipiracil in the real-world management of metastatic gastric and gastroesophageal junction cancers in Canada. Curr Oncol 2022;30:130. https://bit.ly/40OJE03
13. Lubleck DP. Use of observational databases (registries) in research. Chapter 6 from: Clinical Research for Surgeons (Humana Press). http://eknygos.lsmuni.lt/springer/587/95-104.pdf
15. 20Sense original research.
16. Marshall JK et al. Canada's Study of Adherence Outcomes in Patients Receiving Adalimumab: 3-year Results From the COMPANION Study. Clin Ther 2018;40:1024.
17. Cheung W et al. Quality of life in a real-world study of patients with metastatic colorectal cancer treated with trifluridine/tipiracil. Curr Oncol 2020;27:e451.
18. CADTH. Real-world evidence for decision making. March 30, 2023. http://bit.ly/3Kd1rIR
19. Bringing real-world evidence to the decision table. 20Sense. Jan. 30, 2023. https://www.20sense.ca/articles/23-03