Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA), speaks with Chris Houchin, Partner at Guidehouse, in a video interview about the challenges and opportunities of using data sets for rare disease treatment, using decentralized trials, the possibility of leveraging real-world evidence for cell and gene therapies, and partnering with the FDA.
Speaking about the challenges and opportunities related to data sets for treatments affecting small population sizes, Marks says that small populations come with unique challenges. Traditional randomized trials are not acceptable here for a variety of reasons, often because of the lack of a large enough patient number or the small number of patients that exist would not accept traditional randomization.
Marks explains that such situations require devising novel trial designs and extracting as much information as possible. It means that the data from each individual has to be leveraged as much as possible to understand the effectiveness of a product.
Next, Marks goes on to speak about how decentralized trials (trials outside traditional clinical sites) can assist in rare disease investigation by getting additional individuals enrolled. He points towards the balance between special assessments and the ability to attract more individuals to the community. In some cases, this can be balanced by having initial visits at an academic center and then follow-up visits in local communities, Marks adds.
Sharing his views on how real-world world evidence (RWE) can aid the improvement of cell and gene therapies, Marks says that while it has gathered a fair amount of experience in the world of oncology and vaccines for diseases like Covid 19 and influenza, it is yet to play a major role for cell and gene therapies.
Marks however maintains that real-world evidence is helpful once a product is approved. It helps in trying to continue to gather data on the product in its use. For gene therapy, the follow-up data can include durability, cost and convenience for the patient, and standard of care data.
On a similar note, Marks mentions that while being introduced, the regenerative medicine advanced therapy (RMAT) designation anticipated the potential use of real-world evidence for selling gene therapies because it's one of the ways that post-approval commitments can be fulfilled for accelerated approval.
He adds that real-world evidence around a class of products may help get a better sense of what is required for future product development in the gene therapy area.
When asked about how the industry can make better use of data to help inform FDA and or support the regulatory approval process, Marks says that the industry can gather baseline data on conditions before they start development programs or trials. This is because it is a challenge in rare diseases and the more data, the better.
The other aspect that Marks highlights is the need for industry stakeholders to engage in a robust dialogue with the agency (FDA) as they are planning to gather data both early on and later in the development programs. He explains that gathering certain data that might seem reasonable may not necessarily be the right thing from the FDA’s perspective. That dialogue is a good thing to have early on, Marks concludes.
CDO Magazine appreciates Dr. Peter Marks for sharing his insights and data success stories with our global community.