A compelling model for small and medium-sized organizations in the current EU regulatory landscape
Regulatory dynamics in the EU continue to evolve, with higher expectations for pharmacovigilance (PV) as each nation has rigorous and complex patient safety regulations. The shifting landscape represents a unique challenge for small- and medium-sized enterprises operating in this strictly regulated marketplace.
We often talk about achieving PV excellence via people, processes and technology but not in the European context. Our role is to ensure patient safety while optimizing a drug’s benefit-risk profile, and today we will consider an EU-specific PV model that builds excellence with people, quality and compliance.
People: developing organizational excellence
Smaller companies have limited or no in-house PV capabilities, technology and resources. As a result, it is typically outsourced to multiple CROs, along with clinical development. Therefore, no central, single PV database exists, as data are fragmented across multiple CROs. Companies reaching late Phase III need to consolidate this data, either by building PV expertise in-house or by outsourcing to a company like us.
Additional resource challenges include the EU requirement for a responsible person (RP) during the clinical phase and a qualified person responsible for PV (QPPV) during market launch. Small- and medium-sized organizations need to ensure effective communication and awareness by allowing the RP/QPPV to influence processes across the organization with the support of a market authorization holder (MAH), which applies for registration in the country. Managing resources and costs of the complex process and requirements, along with having a dedicated QPPV, is a task in itself for the MAH. However, as the MAH expands, a dedicated QPPV office can support the process and manage deliverables.
Growing organizations need expertise and tools that enhance PV throughout the patient journey. Examples include safety evaluation, risk management plans (RMPs) mandated by European regulations and European-regulation PV system master files (PSMFs). Staff members need the capability to work with tools for signal detection and tracking.
Sponsors also face the complexity of establishing standard operating procedures (SOPs) and processes. Growing companies that attempt to build these capabilities in-house will inevitably look at outsourcing due to the realities of scale and finances.
Quality: building excellence into the QMS
The need to develop a robust quality management system (QMS) is driven by inspections as much as legislation; therefore, the focus is reactive rather than proactive. PV professionals become corrective and preventive action (CAPA) experts, as they respond to non-compliant procedures. For any noncompliance, they file a CAPA, but these often patch the problem rather than address the root cause, which may require additional time and expertise.
Agencies have a perception that some manufacturers produce poor quality reports, as seen in ISMP, NCBI, Expert Opinion on Drug Safety and inspection outcome data from the MHRA and the FDA. The quality of data processed and submitted to the agencies needs considerable improvement, and the value of the data being processed is questionable for the manufacturer. Small- and medium-sized organizations run a higher risk of noncompliance and its consequences, therefore turning to companies like us to manage the quality process and systems that support it.
Manufacturers understand the limitations of data from certain sources and their patient safety values. However, they are often focused on meeting regulatory obligations, even though “more data” rarely means “better-quality data.” Safety organizations cannot build an effective QMS in isolation; safety needs a voice at the corporate level to influence the organization’s quality efforts and activities.
Compliance: driving excellence in meeting regulatory requirements
Individual case study report (ICSR) compliance for patient adverse events (AEs) has been a key PV driver for WHO members. The standard has been 98% but hitting that target does not grant immunity from noncompliance, as specific PV aspects may not be under control. From an agency perspective, compliance is closer to 90%, according to a Reuters Health report. We must, therefore, focus not just on quantity, but on quality of data sources.
Increased scrutiny of PV and the QPPV role has created the need for resource-intensive monitoring. But what should companies monitor, and where should they allocate resources? Reconciliation among business partners is time-consuming, with little ROI. Small- and medium-sized organizations may not have adequate resources to build a PV department capable of handling risk management profiles (RMPs), signal detection and related functions. As companies expand, they need to ask whether a one-size-fits-all approach is appropriate, or if they should be taking a more pragmatic, risk-based approach. For example, they can develop simpler processes and focus fewer resources on managing mature products compared with those in the pre-approval period, while utilizing external expertise to fill operational gaps.
Advances and best practices in PV technology have often lagged requirements. Safety databases were built as transactional AE repositories and can barely keep up with reporting requirements. Signal detection tools have limited ability to integrate external data sources. Risk and benefit are often separately assessed by different resources and then cobbled together for a periodic report. Leveraging electronic health reports (EHRs) has yet to come to fruition from a risk-benefit perspective. As an industry, we need to effectively harness technology to gain efficiencies and manage resources more effectively.
Today’s heightened level of operational excellence can only be achieved if we remember that the role of PV is to ensure patient safety while optimizing a drug’s benefit-risk profile. As small- and medium-sized organizations expand their global reach by introducing products to the EU market, building PV excellence in terms of people, quality and compliance is critical. A provider like us offers flexible staffing, quality systems and regulatory expertise to help these organizations succeed in the highly regulated EU landscape.