Conducting clinical trials and registering an innovative drug takes decades. Patients can get access to treatment only after the state registration of the drug is complete. However, in the situation of critically ill patients, there are controversial issues. For example, a patient may simply not wait for the registration of a drug that has already undergone phase I-III clinical trials. It organized special programs of early access for patients to unregistered medications for such cases. For patients to take part in early access programs, the attending physician must identify the patient’s medical need for unregistered therapy, apply for patient participation in the program, and, if approved, the patient will receive the drug at the manufacturer’s expense until registration approval is obtained.
How do patients get access to non-registered drugs?
Gaining access to non-registered medicines is quite a challenge for patients. Regulatory agencies in some countries have taken the plight of seriously ill patients into account and have enacted the documents to allow the use of new drugs on humanitarian grounds until the registration process is complete.
Early access programs are operating in the United States, Canada, Great Britain, Belgium, Germany, France, Italy, Spain, Portugal, Australia, and Japan, as well as in Russia. The mechanism of such programs is: after the completion of the second phase of clinical trials, the manufacturer here and the “early access” (aka “extended”, “temporary”, “individual”) works.
In Russia, it stated the conditions of receipt by a patient of unregistered medicines in Order of the Ministry of Health and Social Development of the Russian Federation from 09.08.2005 № 494 “On the order of use of medicines in patients on vital signs”. According to the Order, the decision to prescribe the medication shall be made by a Concilium of the federal specialized medical organization, documented in a protocol, and signed by the chief physician or director of the federal specialized medical organization. Patients receive the drug after the decision of the medical board, registration in the program, drawing up a patient file, and signing an informed consent to take the unregistered drug.
On March 1, 2020 amendments to FZ-61 came into effect, allowing the importation of a specific batch of unregistered drugs into the Russian Federation. Besides the import of a batch of medicinal drugs for clinical trials or expert examination for the state registration of medicinal drugs, there is an opportunity to import to provide medical care for life indications of a particular patient or group of patients if there is a corresponding decision of the medical commission about ineffectiveness or inability to use other registered medicinal drugs in a particular patient.
Implementing early access programs is of great importance both for individual patients and for society.