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Projects: FDA Meeting

PEM Friends was invited to join the IPPF in a conversation with the US Food and Drugs Administration (FDA), the regulator and approver for new drugs in the USA. This was an event called an Externally Led Patient Focussed Drug Development meeting, known as an EL-PFDD. The intent of these sessions is to be able to explain to as many members of the FDA as possible about our diseases, how they affect us and the implications of current treatments for pemphigus and pemphigoid. We also talked about our hopes for future medications. The outcome is that any new treatments will be proposed to a regulatory audience who understand the context.

The FDA is recognised as a world respected regulator of new drugs and the fact that this matters in the UK was confirmed in the 2023 UK Spring Budget when the Chancellor said that the approval of new medicines will be expedited, and he confirmed that the Medicines and Healthcare products Regulatory Agency (MHRA) is "exploring partnerships with trusted international agencies, such as in the US, Europe and Japan to provide simple, rapid approvals for medicines and technologies that have received their approval from 2024". The chancellor stated that the MHRA would move to a new model to allow "often near automatic sign-off for medicines and technologies" that have already been approved by regulators in jurisdictions such as the US, Europe and Japan.

Preparation for the EL-PFDD, which occurred on 25th January 2023, took a lot of time. The organisers (the IPPF) were supported by a representative from the FDA and had input from the APPF (our French friends), the Japanese PEM patient organisation and ourselves. A survey was also conducted to which many PEM Friends contributed.

The meeting had inputs from many patients about their disease and the treatments they received. Our own PEM Friends, Andy, Isobel and Mark contributed short videos and Ingrid and Trina presented live. In the audience were patients, FDA representatives, drugs companies, doctors and anyone with an active interest in pemphigus, pemphigoid and treatments now and in the future.

It was a very full day: uplifting, distressing, sad and joyful and very worthwhile. We left knowing that the FDA had heard everything we can say about the traumas of pemphigus and pemphigoid. Additionally, there were presentations by four eminent experts in pemphigus and pemphigoid from around the world: Aimee Payne MD PhD and Victoria Werth MS MD are from the USA, Professor Pascal Joly from France and Professor Dedee Murell from Australia all spoke about the diseases and the present and future medications for pemphigus and pemphigoid.

Following the meeting, these four specialists participated in a paper, drafted by Aimee Payne, and subsequently published in the Journal of Investigative Dermatology. PEM Friends was named as a contributor.

You can see a recording of the meeting, divided into four instalments. In addition, a comprehensive document was produced which is held on file by the FDA pending new drugs applications for our diseases and providing the FDA with a record of all the matters raised in the EL-PFDD meeting.

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