Access to unauthorised medicinal products turns out to have limited clinical added value

Expanded access is an access route to unauthorised medicinal products in situations where there are no medications that have been proven effective, and where it is impossible to treat patients in the context of a clinical trial. Expanded access may be useful to patients who urgently need treatment and cannot afford to wait for the lengthy development process to be completed, and for patients who may benefit from a medicinal product that is not (or no longer) authorised where they live. Unauthorised medicinal products can be applied for for individual patients (to be delivered upon presentation of a doctor’s note) or for groups of patients (compassionate use programmes). On 10 October, Tobias Polak was awarded a cum laude doctorate for his research on expanded access. 

In his PhD dissertation, entitled Unlocking the Value of Expanded Access: Ethical, Statistical, and Policy Considerations, Tobias Polak outlines how expanded access came into being and how it can be facilitated in real life. In his research project, which was part of a public-private partnership between the Erasmus School of Health Policy & Management, Erasmus MC and myTomorrows, Tobias analysed problems encountered on the work floor in a scientific manner. He demonstrated that expanded access was increasingly used as a research method by regulatory bodies, researchers and insurance companies alike. In order to be better able to use these data, he developed a new statistical method, allowing him to integrate expanded access data into clinical trial analyses.

Expanded access provides doctors with an important method to meet healthcare needs that are currently unmet, but Tobias’s research results show that the clinical added value of access to unauthorised medicinal products is limited. Therefore, the choice to grant expanded access must be carefully weighed against the risks associated with therapies involving experimental drugs, the possibility of quitting further treatment and individual patients’ wishes. 

In his thesis, Tobias shed a light on several ethical aspects of expanded access, such as affordability and equal access. At the same time, he proposed several ways to streamline and improve policymaking with regard to expanded access. You can download and read Tobias Polak’s dissertation here.

More information

Tobias received a Prins Bernhard Cultuurfonds grant allowing him to spend several months at New York University to conduct some of his doctoral research. The United States is the birthplace of expanded access, and the CUPA (Compassionate Use and Pre-Approval Access) working group of the Division of Medical Ethics exclusively conducts research on expanded access.

Articles on Tobias’s research project were published in prominent journals such as Lancet Oncology and JAMA. In addition, several videos and animations were created to make the research conducted easier to understand for the public at large. All the publications and code were published open access.

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