Posted 09 March 2015
By Michael Mezher
When a public health crisis like Ebola emerges, public health officials need the flexibility to respond quickly and effectively. But as a new paper in the journal Public Library of Science - Medicine (PLoS Medicine) argues, many countries currently have in place regulatory hurdles which would delay access to experimental new emergency treatments during such crises.
Many global healthcare product regulators have in place provisions designed to speed access to healthcare products, including accelerated pathways for products that treat or diagnose rare diseases. These so-called orphan products are often approved based on less clinical data than a typical product would require.
Some regulators also provide measures for the “ compassionate use” of unapproved products in cases where a patient lacks any alternatives. This type of use is usually applied in situations where a patient faces at serious or life-threatening risks without treatment.
The US Food and Drug Administration (FDA), European Medicines Agency (EMA) and World Health Organization (WHO) each have procedures that allow for the emergency use of medicines.
FDA’s emergency use authorization (EUA) allows for unapproved products to be used in situations where “there are no adequate, approved, and available alternatives.” Similarly, EMA allows for the conditional approval of products based on limited data in emergency situations. EMA also has provisions for providing “rapid scientific advice” in the context of an emergency.
WHO’s emergency use assessment and listing (EUAL) procedure allows for the procurement and use of unapproved product in an emergency context based on three review components:
- ”Review of technical documentation relating to safety and performance (e.g. analytical and clinical evidence, stability data);
- Review of the documentation relating to the manufacture of the product and the manufacturer’s quality management system (QMS);
- An independent laboratory evaluation coordinated by WHO of the product’s performance and operational characteristics.”
Recently, these procedures have been used to fast-track the availability of treatments and diagnostics used in the fight against Ebola.
In his essay in PLoS Medicine, Jerome Amir Singh argues that the lack of emergency use procedures in many countries can “stymie time-sensitive efforts to contain public health threats.” Singh finds that in cases where there are no effective alternative treatments, it is an ethical responsibility for authorities to provide access to experimental treatments.
Singh suggests regional communities such as the African Union could develop emergency frameworks that would facilitate access in emergency situations.
Providing access is not the only thing regulators need to be concerned about. Even though treatment options may be limited, regulators must still make ethical considerations and weigh the risks of using products that have not been fully vetted.
In the face of international health crises like the Ebola outbreak where there is a high mortality rate, it can be necessary to accept greater risks in developing treatments. In his essay, Singh argues that this risk can be mitigated through the development of “robust monitoring and evaluation component that will inform product development and licensure.”
Singh says that transparency is critical to ensure that ethical practices are adhered to when unapproved experimental interventions are used. Other ethical considerations, such as obtaining proper informed consent from patients, are vital to protect patients’ rights.
Humanitarian Access to Unapproved Interventions in Public Health Emergencies of International Concern