Pre-Approval Access – the Theory and the Practicality

Whilst clearly-defined routes exist in most countries to allow pre-approval access to medicines under development, how these regulations play out in the real world is often very different from the written legislation.

With the raised profile of pre-approval access (thanks in part to Right-to-Try and the 21st Century Cures Act) there has been a noticeable increase in the number of companies and independent consultants seeking to become involved in this field. In and of itself this is no bad thing – increased choice for pharmaceutical and biotech companies is surely welcome.

However, the specificities of pre-approval access require more insight than theoretical consultancy can provide, and without direct experience of practical implementation of many pre-approval access programs any consultancy is by its very nature theoretical. Additionally, every single access program has a unique dynamic, specific to a wide range of factors which impact pre-approval access.

This presents a number of issues for pharma and biotech companies seeking expert guidance on an aspect of their operations which has a clear risk of impacting their reputation and their asset in the crucial period in the run up to launch, if it is not handled correctly.

Recently we were approached by a client who had taken advice from a consultancy which had recommended opening their pre-approval access program in a number of Latin American countries, but not France, Germany or Italy, on the rationale that they could capture data and charge for access more easily in Latin America. Whilst it is theoretically possible to charge for access and capture data in some Latin American countries, in practical terms it is almost impossible to recover payment, and it is unlikely that any European HTA body would use any data which could be captured in those countries,

We are seeing more and more of this sort of theoretical consultancy being provided to clients and it should be a concern to any company considering pre-approval access to any of their assets.

There is a place for theoretical consultancy, but at Inceptua we would argue that it is not within pre-approval access. Academic interpretation of regulations without the experience in the practicalities of how to provide access to patients with high levels of unmet need or ability to contextualize through direct experience of working through those regulations poses big risks.


Stuart Bell

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