Guidelines on Submission of Documentation for Emergency Use & Compassionate Use Authorization (Eua/Cua) of Health Products and Technologies

The Pharmacy and Poisons Board (hereinafter referred to as “the Board”) is mandated under the Pharmacy and Poisons Act Cap 244 Laws of Kenya (hereinafter referred to as “the Act”) to regulate Health Products and Technologies.

Ordinarily, prior to any consideration for Marketing Authorization of a product, sufficient scientific and clinical evidence must be collected to demonstrate that it is safe, efficacious and of suitable quality. The scientific evidence includes quality data, safety and efficacy results from human clinical trials or non-clinical studies; it should be evident that the benefits of the product outweigh risks associated. However, less sufficient information on quality, safety, efficacy/immunogenicity/performance may be accepted in times of public health emergencies where there is no other available treatment/intervention. In such circumstances, additional mechanisms like; risk-management plan, pharmacovigilance, and post-market surveillance for compliance verifications as well as investigations of potential health hazards should be implemented.

Furthermore, where necessary, inspections of manufacturers, packagers/labelers, testing laboratories, importers, distributors, and wholesalers of the product may be conducted to ensure that they comply with Good Practices (GXPs). Alternatively, available, and reliable evidence of compliance or non- compliance with good practice (GXPs) requirements can be leveraged as part of the risk-based inspection planning process as prescribed in the Board’s guideline on desk review assessment and WHO guidance on good practices for desk assessment and Guidelines on Reliance for Regulatory Decision Making in Kenya. Therefore, PPB, whenever applicable, shall rely on inspections already performed by a reference regulatory Authority or institution, such as, SRA, the WHO, Notified Bodies, MDSAP, or other recognized institutions in order to demonstrate compliance to GXP’s. If PPB deems an inspection is needed it will leverage a remote or desk audit of which the scope will focus on those elements not already inspected by another reliable source such as the ones listed above. These guidelines prescribe data, which is required to be submitted to the Board to demonstrate the safety, efficacy and quality of the product being applied for market authorization.

Emergency use Authorization shall be applied during a potential or actual public health emergency i.e., the use of a medicine (therapeutic), vaccine, or in vitro diagnostic or medical device) on patients in a life-threatening situation or condition, including chemical, biological, radiological, or nuclear attack, in which no standard treatment or diagnostic is available, and in which there is no sufficient time to obtain product registration. Emergency use authorization procedure may also be applied in extreme situations such as during war.

It is noted that for emergency use products, there may exist limited data, including clinical data hence the Board may accept reduced data requirements with commitment by the manufacturer/applicant to submit more data once available. Applications for emergency use (EUA) or compassionate use Authorization (CUA) shall follow product specific guidelines and the general guidance included in this guideline.

Compassionate use Authorization shall be applied for use in isolated cases of individual (s) or subpopulations where an investigational medicinal product is made available to “patients with a chronically or seriously debilitating disease, or a life- threatening disease or condition, including chemical, biological or radiological attack and who cannot be treated satisfactorily using a registered product. Notably, this does not require a declaration of public health emergency to apply rather, it can be implemented whenever a situation occurs.

Products for compassionate use may be allowed to be used outside of clinical trials when: the disease is serious and life threatening, no alternative treatment for the disease, the individual is not part of or eligible for the clinical trial, the doctor justifies that there are no other options, and the experimental treatment may be of help. The Board may grant compassionate use authorization, based on evidence of such approval by an SRA or WHO whenever the Board may not be in possession of the data on the proposed product. In case the investigational medicinal product is undergoing clinical trial studies within the country and where there has been no authorization by SRA or acceptance by WHO, the Board may approve for compassionate use upon advice from the mandated advisory scientific committees and or WHO.

This guideline shall be read with international guidelines on quality, safety and efficacy, such as international council on Harmonization of requirements of Pharmaceuticals for Human Use (ICH) guidelines and as cited in this guideline namely the World Health Organization (WHO), US FDA and European Medicines Agency (EMA) on emergency Health products and Technologies and PPB’s product specific guidelines.