2025-10-21

The Patient Support Program (PSP) is complicating drug safety monitoring.

In today's pharmaceutical and biotechnology landscape, Patient Support Programs (PSPs) have become an indispensable tool. These programs are designed to improve patient accessibility to innovative therapies and medication adherence, ultimately enhancing clinical outcomes and driving market success. For many companies, a robust PSP is no longer a "nice-to-have" but a strategic necessity, particularly in the complex and costly therapeutic areas of rare diseases and oncology. However, this critical function also introduces a new, often overlooked challenge: it is making the pharmacovigilance system it is meant to support more complex.

From a regulatory perspective, the core issue lies in how to classify Adverse Event (AE) reports collected through PSPs. The traditional pharmacovigilance (PV) framework is built upon two main types of reports: one is "spontaneous (or non-solicited)" reports, which are typically unexpected and presumed to be causally related to the drug; the other is "solicited" reports, collected through an organized system such as clinical trials, patient registries, or post-marketing studies. The former carries a heavy regulatory burden, often requiring expedited submission to regulatory authorities, while the latter, although also strictly regulated, is treated differently because the method of collection is known and systematic.

However, PSPs blur this boundary. They are not clinical trials, yet their data collection is not entirely "spontaneous." When patients and caregivers interact with program personnel—whether seeking financial assistance, medication reminders, or logistical support—they often volunteer mention of side effects or health issues. This can inadvertently trigger a cascade of regulatory obligations. The issue is that many PSPs are managed by units outside of dedicated pharmacovigilance, such as Medical Affairs, Marketing, or third-party vendors. While these teams are experts in patient relations, they may lack the specialized training and procedural rigor necessary to compliantly collect and report adverse reactions.

This introduces significant compliance risks. If regulatory authorities classify adverse reaction reports from PSPs as spontaneous reports, companies must respond quickly with limited information and may need to submit a large volume of expedited reports. This not only consumes substantial resources but can also generate a lot of "noise," cluttering regulatory submissions without providing meaningful new safety signals. Conversely, if a company treats this data as solicited without establishing a rigorous, auditable collection system, it may face regulatory non-compliance and scrutiny. For example, while the European Medicines Agency's (EMA) GVP Module VI acknowledges that data generated by well-managed PSPs can be considered solicited, it lacks specific guidance on the definition of "well-managed," leaving companies in a difficult position amidst differing regulatory interpretations worldwide.

A Need for New Thinking: Risk Management and Special Purpose PSPs

As PSPs become increasingly common, particularly for high-risk or novel therapies, we need a new paradigm that moves beyond traditional pharmacovigilance. For products with Risk Evaluation and Mitigation Strategies (REMS) in the US or similar risk minimization measures in the EU, PSPs are no longer just a market access tool; they are an integral part of the product safety management plan. This is especially true for Advanced Therapy Medicinal Products (ATMPs), such as gene and cell therapies, which require mandatory long-term follow-up and specific safety monitoring. In these situations, the PSP effectively becomes a mandatory, managed patient cohort—a de facto registry—which is critical for the product's lifecycle management.

So, how should the industry respond? As PSPs become increasingly widespread, we need a new paradigm. The traditional siloed approach, viewing pharmacovigilance as a separate, downstream function, is no longer viable. Instead, PV teams must be integrated into the design of PSPs from the very beginning. This requires a shift in mindset and investment.

Firstly, companies need to embed pharmacovigilance expertise directly into the design and operation of PSPs. This means having PV professionals collaborate with commercial and medical teams to co-author patient-facing scripts, train support personnel on what constitutes an adverse reaction, and establish clear and unambiguous reporting pathways. Contractual terms with third-party vendors are also critical, clearly defining their roles and responsibilities in adverse reaction collection and ensuring they meet the same stringent standards as internal teams.

Secondly, companies must leverage technology to streamline processes. The rise of digital health tools and patient-centric platforms offers an opportunity to establish more systematic and compliant data collection. For example, a digital platform that guides patients to report adverse reactions in a structured manner—complete with clear disclaimers and established reporting protocols—can shift these reports from the "spontaneous" grey zone toward the more predictable "solicited" category. This not only enhances compliance but also yields higher quality, more valuable Real-World Data (RWD) for use in benefit-risk assessments and post-marketing studies.

Finally, and perhaps most importantly, regulatory thinking also needs to change. Regulatory authorities could provide clearer, more harmonized global guidance that explicitly defines what constitutes a compliant PSP data collection system. This would alleviate the current burden on companies of navigating patchwork regulations across different jurisdictions, allowing them to focus on what truly matters: generating high-quality safety data to protect patients. Our goal should move beyond a rigid interpretation of "solicited" versus "spontaneous" and instead focus on the quality and completeness of the data collected, regardless of its source. Only through concerted efforts by both industry and regulators can we ensure that PSPs continue to fulfill their critical role in patient care while strengthening, not undermining, the foundation of pharmacovigilance.

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The Patient Support Program (PSP) is complicating drug safety monitoring.

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