It has now been almost four years since the FDA introduced its Breakthrough Therapy Designation, and yet a certain air of mystery still surrounds it. Indeed, just this week, the FDA itself released a Q&A to “help readers understand not only the lingo, but also the implications of expedited reviews and approvals”.
At BioProcess International European Summit 2016, Merck Executive Director of Biologics Process Development and Commercialisation Gargi Maheshwari told of her experience of working on one of the biggest successes of the accelerated approval programme; KEYTRUDA.
The drug gained market approval a little over a year after being named a breakthrough therapy, and Dr Maheshwari highlighted how “the timelines that we had to work with were very different to the traditional pharma timelines of up to 10 years”, which inevitably created a lot of challenges.
On top of such time pressures, the strict regulations you’d expect still need to be met – “You need to find a balance between being as fast as possible to get the product to patients, whilst still ensuring consistency and quality.”
Dr Maheshwari regretfully revealed that there is no secret to meeting these challenges, and that quite simply “there is no substitute for strong science and a really sound understanding of the process and analytics”.
Additionally, communication – both internal and external – has to be spot on; there simply isn’t the time for unnecessary delays through misunderstandings. “I cannot over emphasise the importance of close collaboration between the early development, late development and operations teams” Dr Maheshwari explained, before adding: “Frequent and data-driven interactions with FDA were very, very critical to the success of KEYTRUDA.”
Though such advice may sound obvious, when under the everyday time pressures of developing a breakthrough therapy they can often be forgotten and the benefits of receiving a designation can all too easily be lost.