The review, ‘Adaptation through Collaboration: Developing Novel Platforms to Advance the Delivery of Advanced Therapies to Patients’, is the first step towards a roadmap to advance the development, approval and adoption of these innovative medicines.
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.
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:
“By looking at the growing number of public-private partnerships and open innovation projects, as well as emerging accelerated development schemes, this review attempts to define the new business and organisational standards needed to ensure patients get new innovative treatments faster.
“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.”
Frontiers in Medicine is a peer-reviewed Open Access journal focusing on the latest advances in key areas of medicine. The article, published in the Regulatory Science section, outlines the novel organisational and business strategies needed for the delivery of the growing wave of cell and gene therapies to health systems and the processed needed to establish a sustainable industry.