A Fragmented Regulatory Landscape
Each country has its own set of rules governing these access programmes, leading to inconsistencies in access, approval processes, safety requirements, and timelines. The differences in nomenclature alone can cause confusion, as terms such as “Expanded Access,” “Compassionate Use,” “Named Patient Programs,” and “Early Access Programs” may have different meanings and regulatory implications depending on the jurisdiction.
Variations in Approval Processes and Timelines
The approval process for obtaining access to investigational therapies varies widely. Some countries require extensive documentation and regulatory reviews, while others have more streamlined processes. For example:
- In the United States, the Food and Drug Administration (FDA) offers an Expanded Access process with clear guidance allowing passive approvals within 30 days or emergency approvals granted within 24 hours.
- The European Medicines Agency (EMA) provides legislative basis (e.g. Article 83 of Regulation (EC) No 726/2004), but individual member states regulate their own Compassionate Use programs with differing interpretations, leading to variability within Europe. For example, Germany requires approval from BfArM/ PEI for a CUP while Ireland requires import notification only.
- Emerging markets often have complex and lengthy approval requirements, which can delay access for patients in urgent need. E.g. Turkey’s Compassionate Use Programme can take up to 3 months for approval and is a 3-layered application process.
Safety and Reporting Requirements
Another critical challenge is the variation in safety and pharmacovigilance requirements. Some regulatory agencies mandate rigorous monitoring and reporting of adverse events, similar to clinical trials, while others have more relaxed requirements.
These discrepancies can create administrative burdens for pharmaceutical companies and healthcare providers seeking to provide investigational treatments on a global scale. While safety must be at the centre of any programme involving humans, striking the right balance between ensuring safety while not over-burdening industry/ healthcare professionals serves as a key incentive.
Challenges for Cross-Border Access
The lack of harmonization creates significant barriers to cross-border access to investigational drugs. For example:
- Varying labelling requirements makes it difficult to establish standardized manufacturing and supply planning.
- Some countries do not allow globally unlicenced drugs at all, making patients in these countries unfairly placed to not have the opportunity to access drug in clinical stages under appropriate access programmes outside of clinical studies.
- Localized mindset, e.g. requirement for in-country presence that some authorities enforce, may be seen as deterrents for industry to operate access programmes in some markets.
Call to Action
The absence of harmonized regulations in the Expanded Access and Compassionate Use space remains a significant hurdle to global implementation. These variations lead to inefficiencies, delays, and can result in unequal access to potentially life-saving therapies for patients who need them urgently but live in different regulatory environments. So what can be done?
- International Regulatory Collaboration: Increased cooperation among regulatory bodies, such as through frameworks like the International Council for Harmonisation (ICH), can help align standards and streamline approval processes.
- Mutual Recognition Agreements (MRAs): Regulatory bodies could recognize each other’s EA and CU protocols, allowing for a more seamless, unified approach.
- Clearer Global Guidelines: Developing standardized guidelines for EA programmes, with flexible protocols adaptable across regions, would simplify applications and approvals, benefiting patients, providers, and companies alike.
- Industry collaboration: As a niche sector, it is critical for each player involved in this space to share knowledge and collaborate in bringing harmonization initiatives. Afterall, this space is not a competitive space given the compassionate basis upon which drugs are provided ultimately for the purposes of saving patients lives who otherwise would have no other hope.
Conclusion
Let’s stay committed to advocating for regulatory harmonization, transparency, patient safety, and compassionate care—because every effort counts in offering new hope and potential healing to those who need it most.
You can access an example regulatory landscape assessment showcasing the varied landscape of EA, CU and NPU by clicking the button below:
