Ann: Percheron launches post-trial access program, page-68

We are continuing on with the discussion of post trial access not because it has academic interest but because it is telling us something profound about what is happening in the Phase IIb clinical trial.

First of all, lets turn to the World Medical Association Conference on the Declaration of Helsinki held in Bangalore in January 2024. Lets start with this observation;

• trial participants are not mere means for research
• humanity precedes science, research and norms

The conference presentation discusses the principle of non-maleficence i.e. the obligation of a physician not to harm the patient. Zooming out we can now more fully understand the criteria that the request for PTA must be made via the trial investigator. We are told that the principle of non-maleficence demands continued access if withdrawal of an investigational medicine after the end of the trial would cause harm. Specific reference is made to chronic diseases where the trial participants who are benefiting from the investigational intervention will be harmed if they are taken off treatment. Zooming out again, the trial investigators for the Phase IIb could reference the history of the original nine patients in the Phase IIa together with natural history in DMD.

We are turning now to a point canvassed in the presentation that is particularly salient in reference to the Phase IIb trial of avicursen in DMD. It is noted that while drug trials have different phases, PTA generally refers to Phase III drug trials.

Phase III or in other words a pivotal registration trial.

Remember how we previously talked in the PTA setting about Percheron providing avicursen for a period of time. Can we provide more clarity about how long is that particular piece of string? According to a leading global healthcare company, the goal of PTA is to ensure that eligible patients are given the opportunity to have access to the investigational drug until the point at which it is reasonably available in the local market.

The challenge is to connect the dots, analyse the reason for the placement, dwell upon the significance of the Declaration of Helsinki and understand the gravity of the responsibility placed upon trial investigators in caring for their patients. The issue for Percheron management is whether PTA is for a period of time or for as long as it takes.