eClinical and regulatory services specialist Calyx reports that its medical imaging services and other eClinical solutions were used in approximately half of the oncology treatments approved by the US Food and Drug Administration (FDA) as Breakthrough Therapies over the past two years. This designation enables the agency to grant priority review to drug candidates if preliminary clinical trials indicate the therapy has potential to offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases.
Outsourcing-Pharma (OSP) recently spoke with Calyx CEO Gavin Nichols (CN) about the significance of the Breakthrough Therapies designation, and what it might mean for pharma firms, development partners and other stakeholders.
OSP: Could you tell us what the Breakthrough Therapies designation means to your company, and what it says to clients/potential customers?
GN: Playing a role in new product approvals – especially breakthrough therapy approvals – is our raison d’etre and the motivation of our teams. Calyx exists to solve the complex clinical development challenges our customers face so that they can bring new medical treatments to patients who need them, faster; we understand the urgency of the drug development process and we’re proud of every drug approval we’ve helped our customers bring to market – over 250 of them.
Breakthrough therapy studies evaluate the effects of therapies on the most severe diseases, where patients have irreversible morbidity or mortality (IMM) or symptoms that represent serious outcomes of their sickness. When a sponsor has a potential breakthrough drug or has already received the designation, the pressure to deliver quality data is immense because the cost of not delivering can result in patient deaths that may have been prevented.
Every aspect of a trial is heightened when it is an accelerated or breakthrough designation. Sponsors are under tremendous pressure to conduct their trials efficiently and submit reliable data that meets the rigor of the FDA’s accelerated review process.
Having supported so many accelerated approvals, we have translated that experience into proven methods to support our customers facing the specific challenges of breakthrough therapy trials. For example, when medical imaging is required, trial sponsors need to quickly collect patient images, onboard & train expert readers, and be prepared for the FDA’s rolling review process – as well as have the flexibility to include both known and exploratory biomarkers.
We’ve seen breakthrough therapy trials require multiple, separate criteria per patient. Data can be required in less than one month, so having a solid process in place to ensure rapid delivery of high-quality imaging data is critical; therefore, part of our approach is to truly partner in support of the sponsor, from providing consulting on imaging charters and advising on optimal modalities to suggesting experienced and high-quality sites, readers, and principal investigators.
The breakthrough therapy approvals we’ve supported demonstrate Calyx’s ability to successfully navigate these and other challenges our customers face. We have proven imaging processes that act as the engine in helping sponsors meet each data cut requested by regulatory authorities. We’ve also been involved early, helping our customers provide preclinical evidence as they sought and ultimately received breakthrough therapy designation.
Our customers know they can rely on Calyx’s operational, regulatory, medical, and scientific expertise to help them meet regulatory scrutiny as they seek breakthrough therapy approval and bring these life-saving compounds to patients sooner.
OSP: Could you please share how long it takes for a treatment to receive designation after first applying?
GN: Requests for breakthrough therapy designation are reviewed within 60 days of receipt, and the FDA will either grant or deny the request. Once a drug is designated as a breakthrough therapy, the FDA will expedite its development and review. This is where the ability to respond quickly, and confidently, becomes so important. Rapid delivery is everything in this trial incubation phase.
OSP: What if anything can you tell us about the breakthrough therapies-designated oncology treatments Calyx has supported?
GN: To protect our clients’ confidentially, we’re unable to name the breakthrough therapy approvals we recently supported. I can tell you that our work supported the approval of treatments for a host of oncology indications – including prostate cancer, breast cancer, bladder cancer, and leukemia (to name a few) and approvals outside of oncology – in Idiopathic Pulmonary Fibrosis (IPF), neurological disorders, etc. – that are now offering advantages over the options that previously existed for patients with these life-threatening diseases.
OSP: Is there anything else you’d like to tell us, about Calyx, your eClinical solutions, interesting projects/partnerships, etc?
GN: We’ll soon be announcing new programs, offerings, and collaborations that enable us to further improve clinical trial and regulatory efficiencies, ultimately accelerating clinical development for our global biopharmaceutical customers and helping them deliver new treatments to patients sooner. Stay tuned!