PharmaBoardroom - Introducing Cell & Gene Therapy

Cell & Gene Therapy

Introducing Cell & Gene Therapy

Once considered almost science fiction in medicine, personalised medicine is increasingly becoming a day-to-day reality for many patients.

Written by Louis Haynes on 14.12.2020

As innovative pharma continues to invest heavily in high-risk frontier research involving stem cells and the harnessing of a patient’s own immune system to attack the onset of a disease, a brave new world of potentially curative treatment possibilities is unfolding. This has become most evident in fields like oncology where expensive, yet thoroughly transformative CAR-T therapies are displacing classic treatments and offering newfound hope to patients where previously there was none.

Yet, although CGT has already become a powerful new engine of value creation for patients and heralds a paradigm shift in the treatment of some of the world’s most devastating and intractable illnesses, widespread uptake of these game changing technologies remains, thus far, elusive. This featured section strives to look in detail at some of the outstanding bottlenecks and challenges to adoption, while considering potential solutions.

We will explore efforts to standardize and mature the manufacturing process so as to de-escalate costs and improve safety profile. We will consider the fundamental importance of capturing, processing and harmonizing real-word data (RWD) so as to be able to perform health technology assessments and devise workable outcome-based, risk-sharing agreements for reimbursement.

Finally, we will consider the need for a fresh spirit of collaboration with all actors pulling together along the length and breadth of the value-chain to speedily get these unorthodox therapies through the many hurdles of development and commercialisation and into the awaiting hands of the patients that will benefit from them most.