The promise of real-world evidence with patient support program data

For the past two decades, patient support programs (PSPs) have played a critical role in helping patients in Canada navigate the complexities of accessing specialty[WC1.1] medications. These programs have enabled Canadian patients to gain the reimbursement, therapeutic, and educational support they need to initiate and adhere to their treatment regimens.

Female patient using her phone while receiving an infusion.

While the primary purpose of PSPs is patient service delivery, their high level of touchpoints with patients creates valuable opportunities for comprehensive and robust data collection. Increasingly, manufacturers and other healthcare stakeholders are recognizing that PSP-generated data may offer value well beyond the provision of services alone. Broadening the use of this data may unlock new opportunities to generate real-world evidence (RWE) that strengthens decision-making around patient access and reimbursement in Canada. This growing awareness brings with it a timely opportunity to explore RWE needs earlier in PSP design to ensure data collection is purposeful, thorough and reliable – all while maintaining data security, quality and trust in the evidence generated.

The unique opportunities of PSPs

PSPs represent a rich source of real-world data (RWD) spanning patient populations receiving a particular therapy across all provinces and territories. The data can potentially be used to enhance clinical and payer decision-making and ultimately improve patient outcomes by leveraging RWE to fill evidence gaps. This is particularly relevant in therapeutic areas where randomised controlled trials are either not feasible or fall short, for example, in rare diseases and specific forms of cancers where only phase 2 data may be available.

Additionally, PSPs can provide insights into a product and its patient population across different stages of the product lifecycle. In one recent PSP example, a retrospective cohort study was conducted at two and four years post-launch of the PSP to assess real-world outcomes of patients treated for plaque psoriasis. Notably, of the nearly 2,500 patients enrolled, treatment with the newer biologic produced rapid and sustained skin clearance, and the majority of patients reported meaningful improvements in quality of life, highlighting the real-world value that PSP data can deliver long after a product's initial launch.

A close up of a patient's arm with a nurse in the background.

Beyond PSP data alone, pharmaceutical manufacturers are increasingly pursuing PSPs as a strategic component of broader RWE generation by combining PSP data with external data sources and leveraging digital technologies to expand data access. For example, integrating data with patient registries can yield a more comprehensive picture of real-world patient outcomes, encompassing biomarker testing, health outcomes, and patient reported outcomes. Emerging digital tools are also being explored to enable the use of technology to extend data capture beyond the PSP itself, including the mining of unstructured data from physician charts, which can be linked with PSP data to support a more connected and holistic understanding of the patient journey.

These developments underscore the growing value of PSP data as a source of RWE, particularly in therapeutic areas where clinical trial data is limited or not fully reflective of routine practice. Whether utilized independently or linked with registries and broader other health system data, PSP data can help fill evidence gaps and support long-term value for a product in the real world.

The need for high-quality PSP RWD to support decision-making

As the expansion of data use occurs, particularly to support decision-making, data quality, transparency and methodological rigor in PSP data is imperative.

Health authorities across Canada have sought to strengthen the evidentiary standards around the use of RWE in regulatory and health technology assessment (HTA) decision-making. In 2023, Canada’s Drug Agency (CDA), Health Canada, and the Institut national d’excellence en santé et en services sociaux (INESSS) jointly issued the Guidance for Reporting Real-World Evidence . The guidance laid the foundation for reporting RWE in Canada and for leveraging that evidence to support decision-making. Central to this guidance is an emphasis on transparency and standardisation as prerequisites for trust in RWD.

Building on this foundation, the Canadian Personalized Healthcare Innovation Network (CPHIN) led an initiative with several industry partners, including Innomar[BY6.1][OA6.2], to focus on strengthening PSP data quality by building greater rigor and reliability into PSPs so that the data collected can be trusted to generate meaningful insights. To operationalize this, CPHIN developed the PSP Real-World Readiness Framework . The framework uses a stepwise approach to identify priority use cases, define the variables needed to address them, and evaluate the relevance and reliability of the available data. A number of existing PSP datasets were assessed against this framework to understand whether the conditions necessary to support decision-making – including governance, traceability, documentation, provenance, and quality practices – were in place. While the framework outlines five priority use cases, early findings from this assessment suggest that current PSP data are most well-suited to reimbursement and regulatory-related applications.

The CPHIN initiative continues to advance, with notable milestones already achieved – including the publication of the framework white paper and a subsequent paper characterizing the foundational readiness of Canadian PSP datasets for secondary use and identifying common strengths and gaps that could influence future RWE applications.

These findings point to early and promising potential for Canadian PSP datasets to support RWE generation. By embedding greater rigor and reliability into program design and data collection practices, PSP data can be positioned as a trusted source of meaningful, actionable insights. Those insights can, in turn, inform the design of more effective PSPs from the outset and increase the likelihood that the resulting data are robust enough to support downstream decision-making.

Future potential and next steps for PSP RWE

Initial progress has been made in strengthening PSP data quality and recognizing the need to consider RWE requirements early in PSP design. Looking ahead, expanding data collection, increasing digital tool use, and connecting PSP data with public health system and registry data will be important next steps toward generating more trusted, decision-ready evidence. Together, these advances could enable a more complete view of the patient journey, generate broader clinical insight and support comparative analysis across a therapeutic area – moving beyond single product perspectives to a more comprehensive understanding of treatment value. While coordinating data across multiple public and private datasets in Canada presents real challenges, it also creates an opportunity to build a more integrated evidence base that can better inform decisions over time.

Future use of RWD gathered from PSPs also holds significant promise for informing outcomes-based agreements or access arrangements. It can provide payers with patient-specific evidence on adherence, patient-reported outcomes, and longer-term measures such as reductions in hospitalizations or overall health system spending, thereby demonstrating continued treatment value over time. This is especially relevant in rare diseases, where small patient populations and high upfront drug costs make it particularly important to generate strong evidence for payers. Focusing on rare diseases also offers a compelling and practical starting point for demonstrating how PSP-based RWE can inform reimbursement discussions, support value-based funding approaches, and build confidence in broader use cases over time.

Conclusion: Demonstrating value, building trust in PSP data for RWE

The potential to harness RWE from PSPs to support decision-making is significant — and considerable work is already underway to address barriers and build trust in the data. The CPHIN initiative is a step in the right direction, working to build consensus on the evidentiary standards payers and regulators expect. It is also focused on establishing fit-for-purpose data and data quality with regards to completeness, accuracy, transparency, and provenance. With growing emphasis on data and greater use of digital technology to turn complex, unstructured data into meaningful insights, the opportunity to tap into the full value of PSP data will only expand.

To realize this potential, manufacturers must take a proactive and deliberate approach by engaging in early PSP planning to support purposeful, sustainable data collection, defining from the outset how the data will be used to demonstrate value, and ensuring it addresses payer needs. With the right steps in place to improve quality and trust of the data, PSPs can serve as a trusted source of evidence to support decision-making. This has the potential to not only facilitate early patient access to medicines through PSPs but also generate evidence to support long-term reimbursement of life-changing drugs.

About the authors

Belinda Yap

Belinda Yap, MPH, PhD, is Director, HEOR, at Innomar Strategies, where she leads a team of HEOR specialists focused on evidence generation, outcomes research, and complex modelling to address reimbursement challenges for new therapies. She brings extensive experience delivering innovative HEOR and real-world evidence solutions and provides strategic, technical and leadership guidance across RWE initiatives and HTA submissions.

Shazia Hassan

Shazia Hassan is a Senior Manager of Market Access at Innomar Strategies, specialising in reimbursement strategy and payer submissions for clients. She has an interest in real-world evidence and innovative data generation, and how to use it to strengthen value demonstrations.

Disclaimer:

The information provided in this article does not constitute legal advice. Cencora, Inc., strongly encourages readers to review available information related to the topics discussed and to rely on their own experience and expertise in making decisions related thereto.