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The Importance of Ensuring Quality in a New Class of Medicines
October 1, 2019
Alnylam Pharmaceuticals
Imagine mapping out your daily schedule down to the very minute…for years in advance! Sounds impossible, right? But that’s exactly what we had to do on the Alnylam Product Quality team to ensure we could launch ONPATTRO (patisiran), the first-ever RNAi therapeutic and our first commercial medicine, within 48 hours of its regulatory approval in August 2018.
You might ask why that was necessary? And what’s so important about 48 hours? The simple answer is that we make medicines for people who have serious diseases, and who have no or inadequate treatment options. For them and for their loved ones, every day matters, and we take that personally. Of course, there’s more to it than that, but helping people, and doing the very best we can, is what drives us to come to work every day – even navigating Boston traffic (by bicycle!).
So how did our team do it, and why was it a critical piece in the successful launch of ONPATTRO in 2018?
Well, we have to go back to 2016, when I joined Alnylam and understand where the company was back then…two years out from our first commercial launch. Back then, Alnylam didn’t have a product quality team because we didn’t need one. As a clinical stage development company, our focus was the lab and the clinic. Product quality was still critically important, as we needed to make sure that the medicines we were using in clinical trials were as safe and effective as possible. Commercial companies need a distinct Product Quality function, and I was hired to build it.
Looking back, I think my years of experience prepared me well for those very important minutes. I started my career in research, working in the laboratory. I later got involved with chemistry manufacturing controls (CMC), and started to see how all the pieces of delivering medicines to patients fit together. By working at the intersection of the manufacturing, supply chain, quality and regulatory teams, I was able to help bring molecules from early clinical studies through the commercial stages.
In Product Quality, we have a truly big picture view of the medicines we’re making to ensure they are compliant with good manufacturing practices and local regulations and that they are safe for patients to use. Our job begins the moment raw materials arrive at our doorstep – before they become the medicine that reaches the patient. We test at every step to ensure every lot of drug substance and drug product meets rigorous standards as we need to intimately understand how the commercial product that we're delivering to patients will behave. And we take great care to understand the unique regulatory requirements of each country where our medicines will be available commercially.
Which brings me back to mapping minutes. On the Product Quality team, we were all on the edge of our seats waiting for regulatory approval of Alnylam’s first medicine. We had planned everything down to the minute – from verifying the specifications of the medicine, to batch disposition, to getting shipment documentation ready – all so that our supply chain colleagues could get medicine out the door and to patients quickly. We had conducted countless practice runs to ensure everyone knew their part, and it was due to that preparation that everything went seamlessly. I am so proud of the job that we accomplished, and the systems we established that have now paved the way for our future medicines.
As I think ahead, I’m excited about how far we’ve come so quickly and just how much more we have yet to deliver to patients with rare diseases. Our work never stops but being a part of the birth and now the ongoing growth of this new class of medicines has been nothing short of extraordinary.
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