Report from Brazil

Brazil’s Health Surveillance Agency (Anvisa) published a new regulation, Resolução da Diretoria Colegiada (RDC) 38/2013, on Aug. 14, 2013 that implements patient access to new drugs before they are officially approved and available for purchase. The new regulation offers drug options for patients with rare, severe, or debilitating illnesses for which there are no treatments available or where the existing medication is insufficient.

According to Anvisa, the resolution will provide promising access to new drugs without agency registration under a public health program that will be requested by the patient’s doctor. Patients who previously would not have access to treatments before drug registration will now be able to get their medicines through three programs: compassionate use, expanded access, and post-study program.

Medicines access programs
The compassionate use program will benefit patients with serious or rare diseases. These cases can involve importing new, unregistered drugs if needed. Patients with serious life threatening illnesses may participate through the expanded access program in cases that lack satisfactory treatments, Anvisa told BioPharm International. This program will offer new drugs that are not registered with Anvisa, but are undergoing, or have concluded, Phase III clinical trials. The post-study drug-supply program guarantees free drug supply to volunteers who benefited from the drug during clinical trials.

According to Anvisa, the authorization approval for the expanded access and compassionate use programs will be evaluated according to the severity of the illness. The current state as well as the absence of satisfactory treatment alternatives available for the patient’s condition in Brazil will also be assessed before issuing an authorization. Anvisa says RDC 38/2013 will benefit patients in Brazil because it guarantees innovative drug supplies to those whose illnesses are chronic or severe according to medical request.

Access to unregistered drugs was already available previously by RDC 26/1999 to patients in Brazil through the expanded access program, Anvisa told BioPharm International. The program, however, did not include compassionate use or post-study access and, therefore, had to be updated.

All three programs were included and are regulated by RDC 38, dated Aug. 12, 2013. “The process for obtaining the drug authorization will not be complex or bureaucratical,” says Anvisa. “The patient’s doctor will need to make a formal request to obtain the drug from the company funding the assistance program and the company will place a request with Anvisa, following RDC 38/2013 ruling.” Anvisa, however, points out that the negative point of the program from the industry’s perspective is the high cost of the assistance program. On a more positive note, data collected through the program may be used by the company as additional information for product registration.

“It is important to note that the data collected though the expanded access and the compassionate use programs will not substitute clinical trials for drug registration or delay the development of clinical trials for such drugs,” the agency adds.

Anvisa said the program would not pose any kind of risk or impact to the local pharmaceutical market and that patients will not have access to drugs without medical authorization and request. According to RDC 38/2013, chapter VIII section 18, the company funding the treatment will offer the complete treatment and medication at no cost for the patient under the expanded access, compassionate use, or post-study programs. The company will also be fully responsible for the products used in the program, including adequate stocking and distribution processes, according to the new rules.

Considerations for patients requesting access to new drugs
This improved medicines-access initiative has been positively viewed by many industry players and patients; however, patients should bear in mind some key considerations before requesting authorization to access new drugs. First, the efficacy and benefits of these drugs have not been proven considering that they have not been registered with Anvisa. Second, the risks and complete range of side effects of experimental drugs are unknown. Also, while physicians in Brazil tend to be open to patients’ requests, the doctor may not grant access to the new drug if he/she believes that the experimental drug could be dangerous for a specific condition, for example. It is always important to seek advice and ask for a second opinion on specific medical cases, if needed.

Hellen Berger is a business correspondent based in São Paulo, Brazil.