The advent of cell and gene therapies brings the rare disease community hope of powerful treatments and potential cures. Still, this emerging area is also creating challenges for various rare disease stakeholders centered on collecting, storing, and sharing data.
Data is critical for enabling the clinical development for cell and gene therapies in rare diseases in part because of the compressed development timeline for these treatments relative to other drugs and biologics. Having adequate data is essential for gaining insight into designing a clinical trial for therapy and identifying potential endpoints. Because trials for these therapies often rely on small patient populations, they may be performed without a comparator arm, with natural history studies serving as an alternative to using a placebo control.
The emergence of cell and gene therapies, though, adds to the data demands on drug developers as pressure from regulators and payers adds to the data burden they face. As data requirements have grown, cell, and gene therapy developers wonder how to meet their data demands best.
“It’s a challenge for drug developers to maintain registries,” said RARE-X board member Betsy Bogard, who is a program leader in early development at the gene therapy developer AVROBIO, and chair of the research committee of the rare disease organization International Fibrodysplasia Ossificans Progressiva Association. “There’s a need to collect data over the long-term. Doing it in a shared platform might be more sustainable than trying to have one single drug developer support a single registry.”
A McKinsey & Company report from 2019 on the opportunities and challenges for gene therapy notes that clinical development organizations will need to make “significant” data collection investments beyond launch. These investments include patient registries, confirmatory trials, and innovative usage of real-world data to monitor safety and efficacy over extended periods, in addition to continuing to build the value story.
The U.S. Food and Drug Administration requires gene therapy producers to provide up to 15 years of follow-up data on their products. The agency wants to understand not only how long the benefits they provide last, but whether there are safety issues that become apparent over time, such as whether the therapy can increase the risk of developing cancer.
For payers, the concern is figuring out the value of these high-priced therapies. A gene therapy that cures a rare disease and may prevent the need for a costly enzyme replacement therapy could justify a heftier price tag than one that has its benefits slowly fade over time.
“Payers need assurance that there’s value for money,” said Bogard. “They also need a data source to demonstrate that the gene therapy that they’ve paid for is safe and durable, and provides a meaningful clinical benefit. And the payers typically don’t really want the data coming from the drug developer themselves. They either want data from their own claims data sets or potentially from a third party.”
That creates an opportunity for the rare disease patient community to serve as stewards of their data. Doing so can ensure data gets to all of the groups that need it to accelerate the development of new therapies and make sure the ones that are approved are as safe and effective as they are believed to be.
“Right now, there is no uniform platform for doing this and there is not a playbook in place yet for doing it in a way that’s going to meet the requirements for all the regulators and payers. It has to be created,” said RARE-X board member Morrie Ruffin, who is managing partner for the consulting firm Adjuvant Consulting and executive director of the Alliance for Regenerative Medicine Foundation for Cell and Gene Medicine. “These types of registries do not exist. There are a few companies, including some of the larger pharma companies, that have started to invest in this, but it’s not trivial. It’s pretty daunting for a lot of the small- and medium-sized companies.”
Patient organizations may be well-positioned to oversee patient data, ensure access, and eliminate demands on patients and clinicians having to provide data anew to different entities each time a new treatment emerges from a different drug company. It can also provide therapy developers with critical data sets that are ready to use and immediately accessible, an alternative to the time consuming and expensive process of collecting data during the development cycle.
The problem is that most patient organizations lack the financial muscle and technical expertise to build the infrastructure necessary to take on such a role. That’s where RARE-X has an opportunity to have an impact by providing a platform to enable patient organizations to build data sets robust enough to meet the expectations of regulators, drug developers, and payers.
Ruffin believes the next wave of gene and cell therapy programs will be an early test for the industry as a number of significant product approvals are anticipated in the next two to three years. With those approvals will come significant outcomes data reporting and long-term follow-up requirements. He said the question would be, how will they satisfy the regulators and satisfy the payers?
“They’re going to need to do it, and they’re going to have to do it quickly,” he said. “There’s some urgency attached to this.”