Expanded access programs, compassionate and post-study medicine supply: review of regulations

On October 27, 2023, the Brazilian Health Regulatory Agency (Anvisa) published Public Consultation 1,210/2023, which aims to revise RDC 38/2013, which regulates expanded access programs, compassionate use and post-study medicine supply. The Public Consultation will be open from November 3, 2023 to January 2, 2024 for contributions.

During the 10 years that the standard has been in force, ANVISA has identified points that needed improvement when compared with the experiences of other countries. In addition, ANVISA believes it is important to simplify the analyses, without jeopardizing patient safety.

According to the opinion of ANVISA Director Meiruze de Freitas, there is a need to reduce the analysis time and insert a critical evaluation for the approval of expanded access programs for medicines.

In summary, the proposed revision aims to incorporate risk-based and more simplified analysis, ensuring greater agility, with the use of a notification instrument in cases where the risks are lower.

Points of attention

In order to achieve the proposed objectives, Anvisa is proposing the following new features:

• Insertion of risk criteria: the compassionate use assistance program will be categorized and analyzed based on risk. When considered low risk, it should only be notified to Anvisa. In the case of high risk, it must be applied for and await the agency’s approval.
  • A medicine registered for the intended indication, even if by a foreign health authority, has a lower risk compared to an unregistered medicine, for example.
• Indicated Patient Access Program (API): a new category of assistance program proposed by the Public Consultation, which will allow the incorporation of the risk-based analysis rationale through the notification of medicines already registered by foreign ICH member authorities or by Anvisa, but not available in Brazil. It is intended for individual use by patients suffering from a serious debilitating and/or life-threatening illness and with no satisfactory therapeutic alternatives available in Brazil. Responsibility is shared between the doctor in charge and the sponsor.
• Post-study medicine program maintained, but under notification: considering that the post-study supply program is based on ethical aspects and its regulation is the responsibility of the CONEP/CNS system, the program will be maintained, but under notification. Questions regarding the length of time the sponsor must provide the medicine to the participant after they leave the clinical study will be regulated by the ethics body.
• Expanded access program: in the case of a registered but unavailable medicine, API would be applied; however, expanded access is intended for a group of patients, while API is only for one. In the proposed standard, the criteria for the expanded access program have not been adjusted, but a generic article has been inserted which provides for Anvisa to readjust the standard in the event of a public health emergency.
• Risk management of changes to assistance programs: lower-risk or non-substantial changes to assistance programs can only be notified to Anvisa, while higher-risk changes must be applied for with the Agency’s approval, with criteria to be published in the guidance manual. 
• Assistance program follow-up reports: sponsors will be able to report suspected, unexpected, and serious adverse effects through VigiMed, as is already the case for clinical trials. These events will no longer be presented in the annual reports.

The Public Consultation proposes maintaining the obligation to guarantee the supply of medicines by the sponsor or PRO in expanded access, compassionate use or API programs as long as there is a benefit to the patient, at the discretion of the attending physician and/or the physician in charge.

Among the other general changes proposed are: (i) reduction of some articles; (ii) inclusion of the definition of API; (iii) consolidation into a single annex; (iv) exclusion of the requirement for the doctor’s Lattes Curriculum, with the requirement for other relevant documents.

Next steps

The Public Consultation will remain open for 60 days, from 03/11/2023 to 02/01/2024, without prejudice to any extensions. For contributions, simply access the Anvisa website.

After the end of the public consultation, the proposal will return to Anvisa’s Board of Directors for consideration. To find out more about the subject, contact Souto Correa’s Life Sciences & Healthcare team via e-mail at lifesciences@soutocorrea.com.br