Early access

Early Access

Background

The PHC Catalyst Alliance has been exploring the potential of early access (EA) to medicines in improving access to personalised care.

For patients who have exhausted the available registered medicines or have a condition for which no registered medicines exist (yet), early access can offer a solution. Early access is an arrangement whereby a patient can be treated with an unregistered (personalised) medicine if there are no proven effective drugs and it is impossible to treat the patient in an investigational setting.

The PHC Catalyst has prepared a position paper on this issue. This report examines the opportunities that early access offers to improve access to personalised medicines and is summarised in the question: How can early access contribute to improving timely and sustainable access to value- and data-driven personalised medicines?

Current status

On 24 November, the PHC Catalyst organised a round table session in response to the position paper. All perspectives were present (patient organisations, doctors, health insurer, scientists, industry), except for one crucial perspective: that of the government.

The Care Institute, the Ministry of Health, Welfare and Sport and the Medicines Evaluation Board, among others, took note of the position paper, but due to the current coronahectic, were unable to free up time to attend the session.

How can early access contribute to improving timely and sustainable access to value driven and data driven personalised medicine?

Next steps

Based on the dialogue in several break-out sessions, the following follow-up steps for the PHC Catalyst have been defined:

The PHC Catalyst Alliance should focus on the pilot-plus level. It is important to create a learning movement involving all stakeholders, rather than getting bogged down in pilots that are disconnected.
Define the horizon/concrete vision and closely involve oncology.
Then describe a step-by-step plan, taking oncology as an example to further professionalise and then extend to other diseases.
The approach must be presented as 'the only way' that no one can escape -> sense of urgency and clearly state what is needed and how to achieve it. Part of the approach will be EA.
Make an inventory of the legal basis for AE. What can be done without adaptation? What is currently stated the law that keeps us from moving forward? Good legislation would also make it possible for smaller firms to start an early access procedure. A sound legal basis would also make it possible to shorten the so-called 'lock-in period'.
What is needed to create a central service point to streamline the EAP and provide all stakeholders with the right information?

Funding and promoters are currently being sought for the follow-up. Are you interested?

For more information about this initiative, please contact us. This can be done via our general address or directly with Bas Ruyssenaars, the project leader of this initiative.