Clinical studies of breakthrough therapy-designated drugs present multiple clinical supply management issues, such as the need to adapt to rolling FDA recommendations regarding clinical study design, endpoints, and study size, even once the study is underway. On top of this, sponsor companies must be able to change their clinical supply management in a timely manner. How can clinical supply professionals plan for clinical studies of breakthrough therapy-designated drugs with so many changing variables? Later we will discuss how Portola Pharmaceuticals and Sherpa navigated the clinical supply challenges presented by the breakthrough-therapy designation (Or download it now).
First of all, let's define it...what is Breakthrough Therapy Designation?
Section 506(a) of the FD&C Act states that breakthrough designation is given to a drug “. . . if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”1
Why does Breakthrough Designation present unique clinical supply challenges?
The Breakthrough Designation is intended to ramp up development and approval programs. The ultimate goal is to move it rapidly to market and significantly improve patient care. This means sponsors’ clinical studies are under pressure to move quickly, and clinical supply chain strategy and execution must be airtight, responsive and flexible.
Under the Breakthrough Designation, the FDA will give a rolling review on data as it becomes available to give the drug the best chance of receiving an expeditious FDA approval. The agency will also assemble multidisciplinary teams to advise sponsor companies on how to move forward with clinical trials. These two factors significantly increase the probability of changing parameters and specifications during clinical studies. Having a clinical packaging partner that can be trusted to nimbly adjust to these ever-changing parameters is crucial to the study’s success.
So how can clinical supply professionals overcome this?In the case of Portola's Andexanet Alfa, the breakthrough designation added significant pressures to its planned clinical studies, accelerating their pace and putting added stresses on the clinical supply chain. This necessitated speedy changes in logistics and clinical supply. Running multiple trials in several countries, with finite drug supplies, complicated matters even further.Portola selected a clinical packager that was fast, flexible and reliable to support the clinical supply management of Andexanet Alfa. Sherpa Clinical Packaging’s built-in flexibility, creative problem-solving, and speed were instrumental in keeping Portola’s multiple clinical studies on track. Sherpa was responsive in the face of ever-changing clinical supply requirements, a common issue with trials for Breakthrough drugs.For an in-depth look into how to navigate clinical supply needs for breakthrough-designated drugs, download our in-depth look at how Portola Pharmaceuticals worked with Sherpa to navigate this set of challenges.
1Guidance for Industry. Expedited Programs for Serious Conditions. Accessed on August 30, 2017.