• Manoj Swaminathan

The 'Four Facets' of Unlicensed Medicines, and Pharmacovigilance Impact

Updated: Jul 26

On 05-Jun-2020, the Indian Ministry of Health and Family Welfare (MoHFW) laid out the draft of specific rules to amend the New Drugs and Clinical Trials Rules, 2019. The selection allows medical institutions and hospitals to use drugs undergoing phase III trials in India or any other country "for treatment of patients suffering from a life-threatening disease or disease-causing serious permanent disability or disease requiring therapy for unmet medical need." The draft also allows for the compassionate use import of a new drug undergoing clinical trials.

The COVID-19 situation made it possible for regulatory agencies globally to explore all the four facets of unlicensed medicines, namely:

  1. Emergency Use Authorization

  2. Compassionate Use

  3. Named Patient Basis

  4. Expanded Access Program


The first and foremost thing to understand is that the medicine in question can fall into any of the following categories: (a) licensed for a particular indication in a specific country, or (b) approved for altogether another indication, (c) still undergoing clinical trials, and not authorized in any country. 1. Emergency Use Authorization (EUA) General recommendations and procedures applicable to the authorization of the emergency use of certain medical products, to sustain and strengthen national preparedness for public health, military, and domestic emergencies involving chemical, biological, radiological, and nuclear (CBRN) agents, including emerging infectious disease threats such as pandemic influenza, or other pandemics. 2. Compassionate Use Compassionate use is a treatment option that allows the use of unauthorized medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorized therapies and who cannot enter clinical trials. In the European Union, these programmes are only put in place if the medicine is expected to help patients with life-threatening, long-lasting or seriously debilitating illnesses, which cannot be treated satisfactorily with any currently authorized medicine 3. Named Patient Basis In some cases medical fraternity or even the regulatory agency may approach a manufacturer directly to request the supply of a new medicine that does not have a product license, to be used for a patient under their direct responsibility. 4. Expanded Access Program Some manufacturers may run "expanded access programs". A company may choose to run an expanded access program to allow early access to their medicine, for example, for patients who have been treated with the medication during a clinical trial and wish to continue treatment. In an expanded access program, patients are usually followed up in the same way as patients in a clinical trial. Pharmacovigilance Impact

  1. Literature Monitoring: This is especially relevant when the company does not hold authorization/ license in the country where its product is being supplied. If it can be tracked that the complete supply has been consumed, then one may not consider monitoring until the shelf life of the provided product.

  2. Signal Management: Robust signal management process is necessary primarily when significant quantities are being supplied, and/or when the drug toxicity is already known. In the case of Pandemic supply, where there is no known therapy, the regulatory agency may also volunteer to undertake signal management.

  3. Safety Data Exchange Agreements and Reconciliation: This can be challenging at times, but indeed required. Challenges are mainly when the supplies are made to the developing countries.

  4. Reporting Adverse Events to Original 'MAH': It is the moral responsibility of the importer to report adverse events (if any) associated with unlicensed medicines.




Given the desperation, with rising cases and non-availability of definitive treatment, one cannot rule out that the four facets may be extended to vaccines as well as biological products. The natural course of disease progression will continue to remain a confounding factor. Despite these factors, Pharmacovigilance continues to remain a crucial aspect, that needs to be considered.


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