Collaboration is ‘game changer’ in delivering cell and gene therapies

Collaboration between the pharmaceutical industry, patients and health regulators will be a ‘game changer’ in delivering exciting new cell and gene therapies to treat conditions like cancer and vision loss, says a new review article written by Association of the British Pharmaceutical Industry (ABPI)’s Head of Manufacturing Innovation, Dr Magda Papadaki.

The review is the first step towards a roadmap to advance the development, approval and adoption of these innovative medicines.

Access to treatment

Despite the emergence of new cell and gene therapies for long-term management or even cures for some of the world’s rarest diseases and immuno-oncology, patients still face delays in accessing these new treatments. Dr Papadaki argues that there needs to be a shift towards continual, long term monitoring of the benefits and risks of medicines instead of the current assessments made on limited, up-front evidence, to make sure therapies live up to their potential. If this does not happen, patients might miss out or face significant delays in getting access to this new wave of medicines.

Continuous collaboration

The paradigm shift in the way therapeutic products are developed and assessed will rest on an unparalleled level of early and continuous collaboration between unfamiliar bedfellows; from industry and regulators, to payers and patients, says Papadaki.

Dr Magda Papadaki is the ABPI Director for the Medicines Manufacturing Industry Partnership (MMIP) and supports the UK’s industry-government ATMP Manufacturing Taskforce: examining the UK’s position in medicines manufacturing and identifying opportunities for improvement in the manufacturing and development of small molecules, biologics, vaccines and cell & gene therapies. Speaking about the review paper, she said:

"As decision-making from lab bench to the clinic becomes more coordinated, it is imperative that ATMP developers redefine how they operate in this uncharted territory and build new ‘fit-for-purpose’ business models that capitalise on the evolving partnerships between the industry and the NHS – including with shared clinical and data infrastructures – for the benefit of patients."

Review

The paper was reviewed by Professor Andrew Webster, Director of the Science and Technol​ogy Studies at the University of York, and Professor Michael Barrett, Director of Studies in Management at Hughes Hall, University of Cambridge.