Unlocking a New Paradigm of Healthcare through Advanced Therapies

In today’s complex healthcare landscape, creativity has become a necessary ingredient for sustained success. We are undoubtedly entering a new paradigm of healthcare thanks to these novel and innovative technologies.

This edition of Arar Notes is a great reference point to discuss the area of Advanced therapy medicinal products (ATMPs) that have already played a transformational role in the lives of patients with rare diseases.

What we first thought of as novel, innovative treatments now seem relatively standard in comparison to today’s advanced therapy offerings, and the landscape of the future looks set to change even more.

New categories of advanced therapy medicines are emerging, most notably gene editing technologies, which offer the potential to cure disease and provide real hope to patients suffering from life-threatening or life-altering diseases.

Although there is much excitement, investment, and promise with these novel technologies we must proceed with caution. Balancing the inevitable concerns over demonstrating safety without creating unnecessary barriers to patient access is not a new challenge, but it remains a significant one for developers and regulators alike.

Gene-editing technologies add further complexities to this dilemma due to the potential for permanent changes to a patient’s DNA, either deliberately or as a potentially catastrophic side effect. On one hand, gene editing could offer life-saving treatment that has never previously been possible, but on the other hand, it could potentially induce unintended and possibly even life-threatening consequences.

What a challenge then for the regulator who has the responsibility of giving the green light to these treatments. There is an even greater emphasis on collaboration, information sharing, and keeping abreast of industry developments.

It is fair to say that 2023 was another landmark year for ATMPs with so many updates that it is difficult to keep up with, highlighting the historic approval of the first gene-editing technology, Casgevy “Exagamglogene Autotemcel” It is a CRISPR/Cas9-based technology from CRISPR Therapeutics and Vertex Pharmaceuticals, designed to treat the rare diseases ?- thalassemia and sickle cell anemia.

First approved by the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) in November 2023 and closely followed by the European Medicines Agency (EMA) and FDA approvals in early 2024. Not only is this an interesting case study as the first-ever gene-editing approval, but it could also provide insight into agency thinking for future approvals of similar technologies.

A continuing theme with all medicines, but especially advanced therapies, is needed to identify and mitigate potential adverse effects. This can be significant in the case of gene therapies where secondary malignancies and other off-target effects may be experienced.

It is therefore essential for regulators & developers to continue monitoring the use of these therapies in the post-licensing phase and introduce any changes to labels as required to keep patients safe. This year has already seen a high-profile example of such a post-licensing change, with a ‘black box’ class warning applied to all B and T-cell immunotherapies in the US and a Pharmacovigilance Risk Assessment Committee review enacted in the EU.

It’s also important to consider Regulations, Pricing & Reimbursement as some of the most oft-forgotten parts of medicine development among the initial excitement of approvals and the promise that new medicines bring even with high upfront costs followed by how conventional reimbursement schemes affect the ability to pay.

Questions do remain concerning the longevity of medicines, the scalability, and potential future supply to patients since all are critical elements. There is a need to focus on solutions to these challenges, through ongoing studies, new gene therapy platforms, technology advances to support ATMPs manufacturing at scale, as well as alternative drug pricing models.

At the end of the day, ATMPs have saved and changed lives. Taking positive steps must remain a priority so that greater numbers of innovative products can reach more patients.

See you in the next edition!

Arar Notes is Bayan A. Arar newsletter issued monthly with the latest updates across HealthCare & Life Sciences. Read about this month’s insights and subscribe to get monthly updates on the latest in global health, Life Sciences, wellness, and more.