Patient Support Programs (PSPs) are complicating drug safety surveillance

In today's pharmaceutical and biotech landscape, Patient Support Programs (PSPs) have become an indispensable tool. These programs aim to improve patients' access to innovative therapies and their medication adherence, ultimately enhancing clinical outcomes and driving commercial success. For many companies, a robust PSP is no longer a nice-to-have but a strategic necessity, especially in complex, high-cost rare disease and oncology treatment. Yet this critical function brings a new and often overlooked challenge: it is making the very drug safety surveillance system it is meant to support more complicated.

From a regulatory standpoint, the core issue is how to classify adverse event (AE) reports collected through PSPs. The traditional pharmacovigilance (PV) framework rests on two main report types: "spontaneous (or unsolicited)" reports, which are typically unexpected and presumed to have a causal relationship to the product; and "solicited" reports, collected through organized systems such as clinical trials, patient registries, or post-marketing studies. The former carry a heavy regulatory burden, often requiring expedited submissions to authorities, while the latter — though also strictly regulated — are treated differently because the collection method is known and systematic.

PSPs, however, blur that line. They are not clinical trials, yet their data collection is not entirely "spontaneous" either. When patients and caregivers interact with program staff — whether seeking financial assistance, medication reminders, or logistical support — they often volunteer mentions of side effects or health problems. This can inadvertently trigger a cascade of regulatory obligations. The problem is that many PSPs are run by teams outside the dedicated pharmacovigilance function, such as medical affairs, marketing, or outsourced vendors. These teams may be experts in patient relations, but they can lack the specialized training and procedural rigor needed to collect and report adverse events compliantly.

This creates significant compliance risk. If regulators treat AE reports from PSPs as spontaneous, companies must respond rapidly with limited information and may have to file large volumes of expedited reports. That not only consumes substantial resources but also generates "noise" that clutters regulatory filings without yielding meaningful new safety signals. Conversely, if a company treats this data as solicited without a rigorous, auditable collection system in place, it may face regulatory non-compliance and scrutiny. For example, while the European Medicines Agency's (EMA) GVP Module VI acknowledges that data from well-managed PSPs can be treated as solicited, it offers no concrete definition of "well-managed," leaving companies caught between differing regulatory interpretations around the world.

The need for new thinking: risk management and special-purpose PSPs

As PSPs proliferate — particularly for high-risk or novel therapies — we need a new paradigm that goes beyond traditional pharmacovigilance. For products with a Risk Evaluation and Mitigation Strategy (REMS) in the US or comparable risk minimization measures in the EU, PSPs are no longer just market access tools; they are an integral part of the product's safety management plan. This is especially true for advanced therapies such as gene and cell therapies, which require mandatory long-term follow-up and specific safety monitoring. In these cases, the PSP effectively becomes a mandatory, managed patient population — a de facto registry — that is critical to the product's lifecycle management.

So how should the industry respond? As PSPs become ubiquitous, we need a new paradigm. The traditional siloed approach that treats pharmacovigilance as a standalone, downstream function is no longer viable. Instead, PV teams must be integrated into PSP design from the outset. That requires a shift in both mindset and investment.

First, companies need to embed pharmacovigilance expertise directly into the design and operation of PSPs. That means PV professionals working with commercial and medical teams to co-write patient-facing scripts, train support staff on what constitutes an adverse event, and establish clear, unambiguous reporting pathways. Contract terms with third-party vendors are equally critical, spelling out their roles and responsibilities in AE collection and holding them to the same rigorous standards as internal teams.

Second, companies must harness technology to streamline the process. The rise of digital health tools and patient-centric platforms offers an opportunity to build more systematic, more compliant data collection. For example, a digital platform that guides patients through structured AE reporting — with clear disclaimers and established reporting protocols — can move these reports out of the "spontaneous" gray zone into the more predictable "solicited" category. This not only improves compliance but also produces higher-quality, more valuable real-world data (RWD) for benefit-risk assessments and post-marketing studies.

Finally, and perhaps most importantly, regulatory thinking needs to evolve. Authorities could provide clearer, more harmonized global guidance defining what constitutes a compliant PSP data collection system. That would relieve the burden companies currently bear in navigating a patchwork of local rules, letting them focus on what truly matters: generating high-quality safety data to protect patients. The goal should move beyond rigid interpretations of "solicited" versus "spontaneous" toward a focus on the quality and integrity of the data collected, whatever its source. Only through joint effort by industry and regulators can PSPs continue to play their vital role in patient care while strengthening — rather than weakening — the foundations of drug safety.