We’re living in a time of rapid pharmaceutical innovation, with new therapies emerging faster than ever. For many patients with no other options, compassionate use programs provide access to experimental treatments that could save lives. But managing these programs is no small task, especially when it comes to pharmacovigilance.
Compassionate use programs require balancing patient safety, regulatory compliance, and data collection in a setting that’s less controlled than clinical trials. It’s challenging, but with the right strategies, it can be done effectively. Here’s what you need to know about managing pharmacovigilance effectively in compassionate use programs.
What Is compassionate use, and why Is it important?
Compassionate use programs allow patients access to experimental drugs before they receive full regulatory approval. These programs are designed for individuals who face serious illnesses and have exhausted all other treatment options. Compassionate use isn’t just a lifeline for patients—it also offers pharmaceutical companies an opportunity to gather real-world safety and efficacy data earlier in the drug development process.
However, this early access comes with responsibilities. Ensuring patient safety through robust pharmacovigilance is critical, as these programs often operate outside the controlled environments of clinical trials.
Regulatory variability in compassionate use programs
Compassionate use programs vary significantly across key regulatory agencies, reflecting different frameworks and priorities.
Key differences lie in scope (individual vs. group access), regulatory processes (centralized vs. decentralized), and implementation (EU variability vs. national schemes like EAMS). Pharmaceutical companies must adapt their strategies to navigate these differences effectively.
Challenges in Pharmacovigilance for compassionate use programs
Managing pharmacovigilance in compassionate use programs comes with several unique hurdles that require strategic approaches.
One of the biggest challenges is the variability in regulations across regions. In the EU, compassionate use programs are subject to different requirements in each Member State, while in the U.S., they operate under investigational new drug (IND) protocols. These differences demand meticulous planning and localized expertise to ensure compliance across jurisdictions.
Another issue is inconsistent data collection. Unlike the controlled environment of clinical trials, compassionate use relies heavily on physicians to voluntarily report adverse events (AEs). This can result in delays, incomplete information, and inconsistencies in the data, making it harder to maintain robust safety monitoring.
Global coordination adds another layer of complexity. Compassionate use programs often span multiple countries, each with its own healthcare systems, languages, and cultural practices. Ensuring consistent and accurate AE reporting across these diverse settings requires a tailored approach that accounts for local differences while maintaining global standards.
Finally, engaging healthcare providers remains a persistent challenge. Physicians are integral to pharmacovigilance in compassionate use programs, but their time and resources are often stretched thin. Encouraging them to actively participate in AE reporting calls for strategies that make the process simple, efficient, and minimally disruptive to their workload. Addressing these challenges is key to ensuring the success and safety of compassionate use programs.
How to manage pharmacovigilance in compassionate use programs
Successfully managing pharmacovigilance in compassionate use programs requires a strategic framework that integrates patient safety, regulatory compliance, and operational efficiency. By combining streamlined systems, collaborative efforts, and proactive training, pharmaceutical companies can navigate these challenges effectively.
Here’s how:
Engage National Competent Authorities for program alignment
Compassionate use programs are often managed and classified differently across countries, even within the same region, such as the EU. These variations depend on factors such as country-specific regulations, the development stage of the medicine, available treatment alternatives, and other local considerations. While a unified approach to compassionate use management across all countries is an ideal goal, discrepancies in local requirements may arise. In such cases, consulting with the relevant National Competent Authorities is essential to provide justifications for program classification and work towards greater harmonization.
Leverage local expertise for compliance
Collaborating with local experts or vendors who possess regional knowledge and experience can greatly assist in navigating the complexities of diverse regulatory landscapes. These partnerships ensure adherence to compliance requirements while streamlining program execution.
Simplify AE reporting for healthcare providers
Efficient pharmacovigilance in compassionate use programs relies on simplifying the reporting process for healthcare providers. Providing clear guidelines, intuitive tools, and accessible support can encourage timely and accurate adverse event (AE) reporting. Streamlined systems not only reduce the risk of errors but also enhance compliance and data quality.
Collection of safety information is a key.
Establishing a comprehensive system for collecting and managing safety information is fundamental to an effective pharmacovigilance framework. This includes creating clear reporting protocols and centralized repositories to meet diverse regulatory requirements. Preparation and foresight are key—implementing tools and workflows that facilitate a streamlined process can significantly improve operational efficiency and data reliability.
Foster cross-functional collaboration
Compassionate use programs involve various functions, including pharmacovigilance, regulatory affairs, and medical affairs. Clear workflows and well-defined communication channels are essential to ensure consistency and reduce redundancy. The pharmacovigilance team, in particular, must maintain oversight of all available safety data sources, monitor supply status, and establish direct communication with participating healthcare providers. Effective collaboration across teams enhances responsiveness, minimizes errors, and ensures seamless integration of program activities.
Prioritize training & support
Educate all stakeholders, including physicians, on the importance of pharmacovigilance in compassionate use programs. Provide ongoing training and resources to ensure they feel confident in their role, including instructions and best practices of use, potential risks and possibility to consult. Transparent and direct communication ensure higher engagement of healthcare providers, trust on company and willingness to report.
Continuous risk assessment
Regardless of the medicine’s development stage, companies bear the responsibility of establishing effective systems for collecting and managing safety information. These systems must support ongoing risk assessment by integrating data from diverse sources, including clinical trials, compassionate use programs, observational studies, and literature monitoring. Routine signal detection across these data streams is essential to identify emerging safety risks early and take appropriate action.
Maintaining an appropriate frequency for signal detection and promptly addressing triggers is key to mitigating potential risks. Additionally, timely and transparent communication about medicine-related risks empowers healthcare providers to make informed decisions for their patients. A reliable and well-communicated risk management system not only safeguards patient safety but also builds confidence among stakeholders in the program’s integrity.
Periodic reporting
Periodic reporting is an essential component of pharmacovigilance, with formats and requirements varying based on agreements with regulatory authorities and the medicine’s development stage. These reports provide an opportunity to consolidate data from all available sources, enabling comprehensive benefit-risk assessments. By maintaining regular and transparent updates on the medicine’s evolving safety profile, companies can foster trust with regulators, healthcare providers, and patients.
Oversight
Effective management of compassionate use programs requires centralized coordination across territories to ensure consistency and regulatory compliance. Engaging subject-matter experts in risk assessments ensures evidence-based decisions and timely actions to address safety concerns, reinforcing program integrity and patient safety.
Routine pharmacovigilance activities
If your company holds the status of a Marketing Authorisation Applicant (MAA), it is essential to comply with all routine pharmacovigilance requirements, including:
- Qualified Person for Pharmacovigilance (QPPV):
Each MAA must appoint a QPPV who serves as the main contact for competent authorities and is available on a 24/7 basis.
- Management of the Pharmacovigilance System Master File (PSMF):
Each MAA must maintain a comprehensive description of their pharmacovigilance system in the PSMF. This document must be routinely updated and made available to the authorities within seven calendar days upon request.
- Global Literature Monitoring:
MAAs are required to perform weekly monitoring of global literature sources within recognized databases to identify any potentially relevant articles related to their medicinal product or product class.
- Continuous Risk Assessment:
MAAs must ensure ongoing safety monitoring of their medicinal products through activities such as signal detection, monitoring of regulatory authorities, and other relevant sources.
By adopting these best practices, pharmaceutical companies can ensure compassionate use programs achieve their dual goals: providing life-saving access to experimental treatments while maintaining the highest standards of patient safety and regulatory compliance. This streamlined approach not only safeguards patients but also strengthens the foundation for future innovation and trust within the healthcare ecosystem.
The importance of compassionate use programs today
Compassionate use programs are about more than just accessing experimental drugs—they’re a way to demonstrate a commitment to patients and public health. These programs provide pharmaceutical companies with valuable insights into real-world drug performance while helping patients in urgent need.
In today’s environment, where patient-centered care is paramount, compassionate use programs highlight the role of the pharmaceutical industry in addressing unmet medical needs. By managing pharmacovigilance effectively, companies can ensure these programs are safe, ethical, and beneficial for everyone involved.
