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Challenges and Potential of Early Access Drug Programs

The demand for early access to drugs has grown as patients and doctors have access to information on clinical trials over the Internet and social media, according to the U.S. National Institute of Health.

Early access programs, also known as compassionate use, named patient, or special access programs, allow one patient or a group of patients the use of drugs that have not yet been authorized. These patients have serious and life-threatening diseases and no other treatment options. While specific programs are created by each nation’s regulatory authority, these programs typically include drugs that have undergone some clinical trials and may have entered the regulatory process, but lack complete information on safety and proper dosage. The logistics are typically handled through specialty pharmaceutical distributors who work with regulatory agencies on behalf of manufacturers to gain temporary authorization.

While early access programs allow individuals with unmet medical needs to be treated, they present some challenges for patients and drug manufacturers. One challenge is accurately identifying which patients will benefit from specific drugs and equitably connecting them with the drug. For example, in some cases, patients who had access to social media are able to find out about drugs and apply for them more quickly, yet patients who lack this access need the drugs, as well. Related to that is the challenge of ensuring physicians have adequate information so that they can recommend drugs in developmentto their patients and so that patients can make informed decisions about seeking access. Patients and doctors also sometimes face administrative hurdles that require considerable time to navigate. Another challenge is the tension between allocating resources for early access programs and continuing development of them for a general population. A final challenge is building consensus around exactly what conditions and situations constitute an unmet medical need for purposes of being included in the program.

For many patients who have no other treatment options, the benefit from the investigative drug outweighs any risks or challenges that may be associated with it. The administration of an investigative drug, a monoclonal antibody called ZMapp, to six patients suffering from the Ebola virus in West Africa, ultimately saved the lives of four of them. An open-label private trial of the drug Brincidofovir helped cure 7-year-old Josh Hardy of a virus he had contracted after a bone marrow transplant.

Pharmaceutical companies also can benefit from early access programs. For example, if the early access program is successful,physicians will become early believers in the drugs, leading to faster sales once it is commercially launched. Companies also will receive insights from real-world experiences which will guide development of their marketing, educational and support materials.

Through careful navigation of the system, both patients and pharmaceutical companies receive great benefit from these early access programs.