Navigating Alternative Market Entry Pathways: Ensuring Pharmacovigilance for Orphan Drugs, MAPs, and Donations in Africa

The typical pathway for medicinal products to enter the market is that manufacturers apply to the Health Authorities (HA), which in turn conduct scientific evaluations of the applications before approving them. This process is very lengthy, sometimes taking up to three years.

Alternative market entry pathways can be used when the product is needed for emergency use, such as in the current case of Mpox vaccines. Alternative pathways can be donations, Managed Access Programs (MAPs), and orphan drugs.

Before we proceed, let's understand what they are:

Managed Access Programs (MAPs) include:

• Compassionate use: This allows access to products undergoing clinical trials or one whose marketing authorisation application is still being evaluated for patients suffering from life-threatening, long or earnestly debilitating diseases.?
• Named patient programs: A doctor requests an unauthorised product directly from the manufacturer to treat an individual patient. In Nigeria, the products are called service drugs.

Orphan drugs are used to treat rare diseases, disease rarity hereby determined by the number of patients affected. The figure varies from country to country, for example, less than 25,000 in Tanzania and less than 200,000 in Nigeria.

Donated medicines are to meet the specific needs of the receiving entity which cannot otherwise afford such products.

This month, I supported a client in setting up a local pharmacovigilance (PV) system for products entering the market through the alternative pathways. During the exercise I became curious about regulations in other African countries covering such categories of products in their scope.?

What I learned.

Whereas countries like Uganda and Tanzania have guidelines for the donation of drugs, focus is on importation requirements. The guidelines are silent on the responsibilities of the donor and those of the receiving entity, especially concerning monitoring the safety of the donated drugs. Although not clearly stated, the receiving entity would be expected to undertake the roles of health facilities stated in the respective national PV guidelines. Is the donor expected to follow the same guidelines as the Marketing Authorization Holder? That’s a question for the health authorities to address. I will surely get back to you when I get an answer.?

Ghana, Nigeria, and Tanzania have guidelines on orphan drugs. These are unfortunately also limited in the steps for registering the drugs and do not address the PV responsibility of the entity introducing the orphan drugs to the market. The Tanzania regulations are broader compared to the rest and state that orphan drug applicants should read the regulations together with other rules under the act that established the HA. This means most pharmacovigilance regulations in the country apply to orphan drugs as well.

The Gambia has a guideline on Emergency Use Authorisation (EUA) of medicines, biologicals, and in-vitro diagnostics during declared public health emergencies. The guideline provides for setting up the PV system during EUA, including appointing a focal person and reporting serious adverse events.

South Africa has a guideline on access to unregistered medicines. Clause 40 of the guideline requires the healthcare worker or any other person to report suspected adverse drug reactions to the HA. It further stipulates the importer's vigilance responsibility to inform the HA of adverse drug reactions and risk management activities.???

In conclusion

Health Authorities need to clearly state PV expectations during donations, MAPs, and the introduction of orphan drugs into their respective markets. MAHs should read guidelines on such products in conjunction with existing PV regulations. Healthcare professionals and health facilities should continue to meet their responsibilities as stated in their countries’ PV guidelines when they receive medicines through such programs.?

Does your country have regulations that cover vigilance for orphan drugs, MAPs and donations? Are the regulations adequate? What gaps exist? Are such products adequately monitored for safety? Let’s discuss this in the comments section.?

Helpful reads

1. Navigating Pharmacovigilance in Africa: Strategies for Marketing Authorization Holders in the Absence of Regulatory Guidelines
2. Safety monitoring of orphan drugs: The SFDA experience