Experts call for more guidance from FDA on registries in oncology

Officials from two global registries for rare pediatric cancers expressed a desire for more guidance from the US Food and Drug Administration (FDA) on its expectations for such registries.

The officials, who spoke during the second day of a two-day FDA workshop on the future of registries in oncology, acknowledged the challenges of managing registries for rare diseases but noted that the information collected by registries can provide valuable insights into disease patterns.

The second day of the workshop focused on ongoing efforts to build national and international registries for pediatric diffuse midline gliomas and diffuse intrinsic pontine gliomas (DMG/DIPG) and best practices in developing these registries. The first day of the workshop explored more general concerns in developing these issues. (RELATED: FDA officials explain how to ensure registries generate fit-for-purpose data, Regulatory Focus 28 August 2025)

Registries are a key source of real-world data to support drug approval in the rare disease space when it is not typically possible to collect data from large randomized controlled trials (RCTs).

Maryam Fouladi, a pediatric neuro-oncologist and co-director of the pediatric neuro-oncology program at Nationwide Children’s Hospital in Columbus, OH, and a professor of pediatrics at Ohio State University College of Medicine, stressed the need to find adequate treatments for these cancers. Fouladi is also the founder of the DIPG/DMG registry and its spinoff in Europe called the SIOPE DIPG/DMG registry.

Fouladi said that the survival rate for these patients with DIPG and DMG is “dismal” and most live an average of 8-11 months after diagnosis of the disease.

In August, the FDA granted accelerated approval for Dordaviprone (Modeyso) to treat DMG and DIPG, marking it as the first therapy approved for this previously untreated form of brain cancer.

Fouladi discussed some of the strengths and weaknesses of the registries. One of the strengths is that the data in these registries is of high quality. She also mentioned that the data is reliable and relevant with a clearly defined population.

Yet some of the weaknesses includes limited molecular information, as these tumors are often not biopsied due to the high risk involved in removing brain tumors for analysis.

Trent Hummel, an associate professor of pediatrics at Cincinnati Children’s Hospital and chair of the International DIPG/DMG Registry, reported there are currently 1,400 individuals in the registry, along with over 7,000 related studies focused on these patients. Established in 2012, the registry includes contributions from 12 countries. Its primary goal is to enhance the clinical understanding of DIPG and DMG.

He said that “we’ve have had multiple inquiries from industry on how they can use data from the industry to support their clinical trials. This is a timely two-day meeting for us.”

Hummel also discussed some strengths and limitations of registry data. Among the strengths are the linked data between MRI results and clinical features, as well as international collaboration between their registry and other organizations. Additionally, the registry has electronic consent options, allowing parents who wish to provide consent to do so electronically, which eliminates the need for faxing and paperwork. The registry also can elucidate patterns across large sample sizes.

There are a few limitations to consider, Hummel said. Firstly, there is a lack of profile data for some patients who have never undergone a biopsy. Additionally, differing legal issues in Canada prohibit the industry from using certain data in that country. For instance, while the use of data in industry-sponsored trials is permitted in the US, this is not the case in Canada.

Hummel said that “we will never be 100% perfect, we don’t have 175,000 patients like in the CIBMTR [Center for International Blood and Marrow Transplant Research] registry, we have 1,400 right now.”

Keith Desserich, the chairman and co-founder of The Cure Starts Now Foundation, and a steering committee member of the SIOPE/DIPG registry in Europe and the DIPG/DMG International Registry, said there are 14 EU countries participating in the registry, which has data for 1,290 patients.

Desserich noted that there is a significant labor component involved in data collection. Additionally, there are instances where language translation issues need to be corrected. In Europe, confidentiality concerns arise, along with restrictions on identifiable data, which hinder the immediate sharing of information.

FDA needs to address these registries

During the question-and-answer session, Hummel pressed the FDA to provide more details on what they want from these registries.

In response, FDA’s Nicole Drezner, the deputy director of the Division of Oncology 2 at the Center for Drug Evaluation and Research, said there are specific data elements that should be in the registry, such as patient-level data, eligibility criteria, prognostic factors, biomarkers, and treatment effects. “The more information we have, the better. For smaller treatment effects, we are particularly concerned about missing data.”

She added that there is a need for these registries to support drug development in the rare cancer space.

“Our thinking of the regulatory framework is that the gold standard of two adequate and well-controlled trials is not something we would ever require in oncology. It's not ethical to do a second clinical trial if the first trial is a winner. Utilizing an external controlled data source could potentially provide supportive or confirmatory evidence for a particular marketing application.”

Drezner also advised sponsors to request a pre-investigational new drug application (IND) meeting if they are interested in using a registry to support drug approvals in this area. She added that FDA plans to hold small meetings with “product-agnostic” registries as a productive first step for obtaining FDA input on them.

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Experts call for more guidance from FDA on registries in oncology

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