n Canada, more than 60% of pharmaceutical companies offer a variety of Patient Support Programs (PSPs) to help patients receive timely access to medications and provide support during their treatment. Approximately 10% of prescription drugs in Canada are supported by a PSP. The most common therapeutic areas with PSPs in Canada are oncology, multiple sclerosis, rheumatology, and rare disease therapies. These programs can generate a high volume of adverse events (AEs) and therefore often make PSPs a key focus during regulatory inspections.

A PSP is also an organized patient data collection system that supports the use of the Marketing Authorization Holder’s (MAH) drug, and manages the patient’s medical condition through its three-way mechanism of communication between the MAH, patients, and healthcare professionals (HCPs).

The combined key objectives of PSPs are to:

• Enhance patient understanding of their disease and/or treatment
• Improve patient adherence to treatment
• Lead to better patient health outcomes.

Many PSPs also provide services that include education, financial assistance, or reimbursement support, often referred to as Patient Assistance Programs (PAPs).

Regulatory Requirements

From a regulatory perspective, it is important that all AEs arising from PSPs are reported in accordance with applicable regulatory reporting requirements, which include Health Canada, the UK Medicines & Healthcare products Regulatory Agency (MHRA), the US Food and Drug Administration (FDA), and the European Medicines Agency (EMA).

Due to the complexity and severity of conditions treated in PSPs (particularly in therapeutic areas such as oncology and rare diseases), these programs can generate a high volume of adverse events (AEs).

There are many different sources of AE reports during a PSP, including patients, caregivers, family members, physicians, and pharmacies. PSP staff working on behalf of MAHs often receive AE reports from field nurses who administer the product to patients, or from other program or administrative staff.

This high volume of AEs and other information regarding the patient’s use of the drug, generated by interactions with the patient from initial visit through product administration, often makes PSPs a key focus during regulatory inspections.

Risk-Specific Regulatory Reporting Requirements in PSPs

Depending on the program or product, important reporting requirements include:

• Risk minimization measures: A Risk Management Plan (RMP) is one of the most important pharmacovigilance (PV) tools, designed to manage and mitigate risks associated with a product following market authorization. Regulatory authorities such as Health Canada, the EMA, and MHRA require pharmaceutical companies to implement RMPs to protect public health by proactively identifying and addressing potential risks for the product. In the US, Risk Evaluation and Mitigation Strategies (REMS) are enforced by the US Food and Drug Administration (FDA). REMS are designed as a regulatory enforcement tool and are required only when FDA deems them necessary for a marketed drug with serious safety concerns and describes measures to monitor and minimize those risks.

  A key element of RMPs are risk minimization measures, which specify the measures needed to prevent or reduce the probability of an undesirable outcome or reduce its severity should it occur. Examples of these additional measures include providing educational materials such as HCP guides or checklists, and/or patient guides and patient cards distributed to the patients, caregivers, pharmacists, and physicians participating in the PSP. The MAH must ensure that the most current version of the educational materials is provided in a timely manner so that all stakeholders are kept informed of the risks for the product.

• Follow-up questionnaires based on product safety concerns: Also known as “Targeted Follow-up Questionnaires,” the purpose is to collect specific information on AEs or safety concerns to better characterize safety concerns that may impact the product’s benefit-risk profile. Their distribution may be requested by the regulatory authority to address important product risks or missing product information.
• Commitment to submit certain types of safety information: For example, when an MAH’s product might be confused with a product with a similar name or spelling used for a different disease or patient population, the regulatory authority may request a commitment from the MAH to report all serious and nonserious medication errors in the country/region where this confusion exists for a defined period (such as three years post-approval). Regulatory authorities such as Health Canada and EMA have recommendations regarding the potential for a proposed name to be misleading or confused with another product authorized for use, with the aim of reducing medication errors.
• Product-specific training and reporting requirements: A product label is a scientific document which provides factual information about a drug product required for its optimal, safe, and effective use. The product label is referred to as the product monograph in Canada, the summary of product characteristics (SmPC) by the EMA, UK SmPCs, and the US Prescribing Information (USPI) in the United States. A product label that does not provide clear details about that product’s use may also create confusion about AE reporting. MAHs must ensure that product labels provide specific and clear guidance on how to report AEs and any other potential safety information for that product. Ensuring that the correct information is reported according to regulatory requirements reduces the risk of under-reporting or over-reporting AEs and ensures PV reporting compliance.

Best Practices Moving Forward

• MAHs and PSP vendors must establish robust PV systems and processes: Contractual agreements must reflect current regulations and practices, effective safety data exchange processes, good quality system and documentation procedures, and staff training on AE reporting processes, deviations, and record retention and archiving as described by Health Canada, EMA, and MHRA.
• MAHs must work with their PSP vendors to ensure inspection readiness in accordance with Good Pharmacovigilance Practices (GVP) as defined by Health Canada, EMA, MHRA, and FDA, and to ensure that safety reporting during the PSP maintains the same standards as those applied by the MAH. (FDA has not issued guidance on this specific topic.)
• Regulatory authorities must continue to review PSP pharmacovigilance processes in accordance with Good Pharmacovigilance Practices (GVP) to monitor and assess the safety and effectiveness of the MAH and PSP safety systems (including AE and PV reporting). They must also ensure ongoing communication of any potential safety issues identified during the market authorization process to support effective post-market monitoring and reporting of AE and safety information.

Conclusions and Collaborations

• PSPs not only increase patient access to a company’s products; they also provide an opportunity to generate important data on product safety, efficacy, utilization, and adherence in actual practice, ultimately leading to enhanced patient care and better health outcomes.
• Ensuring patient safety throughout this process is a shared responsibility between companies, regulatory authorities, and healthcare professionals, a collaborative approach that helps patients receive the highest standard of care through PSPs.