Preexisting social, financial, political, organizational, and ethical barriers prevent expanded access to unapproved investigational drugs, according to a study in Health Policy.

Because of the nature of expanded access programs, all parties — the treating physician, the pharmaceutical company providing the drug, and the hospital institutional review board — must consent to the treatment taking place. According to researchers, “any one of these entities can unilaterally prevent expanded access from happening.” Recent years have found both public and private organizations acting to remove these barriers.

Researchers evaluated the medical literature using terms including early access, compassionate use, pre-approval access, and named-patient programs. Additionally, the researchers performed both semistructured and unstructured interviews with a total of 35 stakeholders residing in The Netherlands. Specifically, interviews were performed among experts from academia (n=8), patient organizations (n=4), biotechnology and pharmaceutical companies (n=4), physicians’ associations (n=3), health insurers (n=3), hospital-based pharmacists (n=3), the Dutch Medicines Evaluation Board (n=3), policy-makers at the Ministry of Health, Welfare and Sport (n=3), the Dutch Health Inspectorate (n=2), and the Dutch HTA agency (n=2).

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According to findings from the literature review, “by way of exemption” has been the traditional route to grant expanded access, but the expanded access has only been allowed when no other options are available or when patients are not deemed appropriate for clinical trials. In the United States and many jurisdictions in the European Union, there are few regulations for improving access to investigational drugs for patients. In many countries, expanded access is not part of routine clinical practice, despite many authorities rarely rejecting patient requests.

Social inequities may exist that contribute to the variability in the chances of receiving expanded access. The investigators suggest that initiatives for making expanded access available to all may “counter the social inequalities that are prevalent in current systems, as they aim to ’empower’ all patients, thus also the less privileged, to get access.” Respondents to the semistructured and unstructured interviews felt that streamlining request reviews and reimbursement arrangements may be appropriate if expanded access becomes more widely available for patients with unmet needs.

Additionally, there is a debate as to whether publicity surrounding expanded access will create real change or will simply result in fostering false hope among terminally ill patients. Additionally, many companies have little incentive for investing in expanded access. Variability also exists among countries with regard to the reimbursement systems for expanded access. In some countries, the financial burden may fall on the healthcare system whereas in other countries, the costs may fall on the patient.

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Expanded access programs, if implemented in a responsible manner, may “be used to improve and accelerate the processes of drug development, marketing authorisation and health technology assessment, and thus contribute to the development of safe and effective medicines for future generations of patients.”


Bunnik EM, Aarts N, van de Vathorst S. Little to lose and no other options: Ethical issues in efforts to facilitate expanded access to investigational drugs [published online June 18, 2018]. Health Policy. doi:10.1016/j.healthpol.2018.06.005.