In the complex landscape of modern medicine, access to unapproved drugs can be a critical lifeline for patients facing severe or life-threatening conditions when conventional treatments fall short. Globally, regulatory pathways and special access programs are in place to facilitate compassionate use of these potentially life-saving treatments, ensuring that patients and healthcare providers have the support and resources needed support to explore every available option.

Unapproved Medicines: Their Role in Clinical Trials & Patient Access
Unapproved medicines – also known as unapproved products – are treatments that have not yet received formal approval by regulatory bodies such as the FDA, EMA, or local health authorities. These drugs are either still in clinical trials or awaiting approval after trials have been completed. Their primary purpose is to offer potentially life-saving treatments when no other options are available, with patient access facilitated through clinical trials or special access programs.

In clinical trials, unapproved medicines encompass a wide range of investigational therapies:

• Investigational Medicinal Products (IMPs): Drugs still undergoing investigation.
• Biologics and Biosimilars: Monoclonal antibodies, gene therapies, and other complex treatments requiring extensive clinical testing and regulatory authorization.
• New Medical Devices: Innovative devices that must meet specific regulatory standards before they can be marketed.

These products are essential for advancing medical research and developing new treatments, but must undergo rigorous evaluation to ensure their safety, efficacy and regulatory compliance.

How to Access Unapproved Drugs
Access to unapproved drugs is facilitated through specialized programs designed to address specific patient needs while adhering to country-specific regulatory frameworks. Common programs include:

• Compassionate Use (CUP)
• Early Access Program (EAP) or Expanded Access Program (EAP)
• Named Patient Use (NPU)
• Special Access Routes (SAR) and Emergency Use
• Personal Use (PU)

Availability and eligibility criteria vary by country, and some nations may not offer these programs at all.

Approval Process
The approval process for accessing unapproved drugs involves healthcare providers, regulatory authorities, and ethics committees, with specific requirements varying by country and program. Healthcare providers can request CUP or PU for patients with serious conditions. National regulatory bodies, such as the FDA or EMA, review and approve these requests, especially for EAPs. Additionally, ethics committees or Institutional Review Boards (IRBs) assess the ethical aspects and approve the use of unapproved drugs in clinical trials or compassionate use situations.

Conclusion
Access to unapproved drugs can be a lifeline for patients facing limited treatment options. Understanding the available programs and approval processes helps patients and healthcare providers navigate the complexities of securing these potentially life-saving treatments.

Marken stays committed to supporting clients in navigating the regulatory and trade landscape of the global clinical supply chain. For more information or if you have any questions, contact our team at TradeCompliance@marken.com.