Partners in Progress: A Recap of the FDA/OSI Workshop

The workshop opened new possibilities for smarter trial designs to advance development of innovative treatments, and forge stronger partnerships between regulators and industry.

The Osteosarcoma Institute (OSI) is focused on improving outcomes for patients with osteosarcoma. However, because osteosarcoma is both rare and different from many other cancers, researching and developing new drugs is challenging, and treatments have not improved much in decades.

In October 2025, OSI and the U.S. Food and Drug Administration (FDA) held a workshop to discuss strategies that could improve osteosarcoma drug development. The workshop brought together more than 370 leading researchers, biopharma industry representatives, regulators, and patient advocates to discuss the major roadblocks in the clinical trial and regulatory landscape and to establish future action steps.

These are some of the workshop’s key takeaways with insights from doctors and professors who participated in the discussions.

Connecting Stakeholders: An Essential First Step

“The best and most important part of the workshop was getting many of the leading experts in one space to start addressing some of the major challenges,” says Lara E. Davis, MD, professor of medicine and head of the sarcoma program at Fred Hutchinson Cancer Center and a member of OSI’s Strategic Advisory Board.

When pharmaceutical developers and the FDA work together early in the drug development process, odds are better that they can avoid expensive delays and get new drugs to patients faster.

“Osteosarcoma creates real challenges for industry partners in terms of seeing a viable path forward for trials,” says Katherine Janeway, MD, MMSc. Dr. Janeway is a senior physician at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pediatric Oncology Section Chief at Dana-Farber Cancer Institute. “Each party who is involved with building a clinical trials portfolio in osteosarcoma was able to present their perspective and be heard. There was good listening between the different parties. That’s an essential first step.”

The workshop created the opportunity to dive into the issues and identify action items to address them. “We need all of these people working together to do that,” Dr. Davis says.

One action item that came out of the workshop: ongoing working relationships with the FDA. The FDA is committed to keeping lines of communication open so experts can find ways to effectively measure success in osteosarcoma treatment and create clinical trials that target these measures and move drugs forward faster.

Finding New Measures of Success

Osteosarcoma presents unique challenges that set it apart from many other cancer types. As such, FDA, researchers, and industry attendees agree that developing clinical trial endpoints that can accurately measure whether a treatment is effective is a critical priority.

For example, with many cancers, tumors shrink when treatment is working – this is reported as a “response rate.” But osteosarcoma tumors often create dense, bone-like structures that remain behind and show up on imaging studies, even after cancer cells have been destroyed. Therefore, measuring shrinkage isn’t the most reliable way to determine how well a treatment is working in osteosarcoma.

“Response rate is a good measure of effectiveness for most cancers and is what the FDA and pharma are comfortable with and repeatedly design trials around. But it is not a good measure in osteosarcoma,” Dr. Davis says. “The FDA representatives in the room were open to thinking about new approaches and new designs.”

For example, the FDA speakers pointed to other cancers that share osteosarcoma’s problem. “It was very helpful for us to hear that from the FDA,” Dr. Janeway says.

Osteosarcoma treatments will need to find new strategies beyond gold standard large, randomized phase III trials, which are unfortunately challenging to perform in rare cancers.

Working together will help identify potential strategies, such as credentialing new biomarkers to assess response in osteosarcoma that can supplement or replace the usual endpoints.

“When we’re creative but also practical, it’s a win for the patients, a win for the investors, and a win for the whole drug development process.” —OSI Strategic Advisory Board Member Melinda Merchant, MD

Designing Drug Trials for Osteosarcoma

Because osteosarcoma is rare and clinical trials are expensive, pharmaceutical companies don’t often focus on this type of cancer.

“Comparing two different treatments in a rare cancer is quite difficult. It requires a lot of study sites and a lot of patients, and that can be prohibitive for our industry partners who are working with limited resources and strategizing about where to use those resources to help the largest number of people,” Dr. Janeway says.

What may happen instead is, after a drug trial is designed and endpoints are set through a long and resource-intense process, they might ask how it could work for osteosarcoma. Workshop discussions sought to flip this approach.

“It makes more sense to have these conversations early on to see if we can design something for osteosarcoma that can move forward quickly and meaningfully,” Dr. Davis says. “And it seems like everybody, from academia to industry to patients and advocates, is ready and willing to make that happen. People with osteosarcoma have a lot of potential years left to live. We need to find ways to get exciting, brand-new therapeutics to them.”

Centering Patients and Families

Patient and family voices drove home the urgency behind osteosarcoma drug development. “My industry friends who haven’t been in the clinic spoke about the impact afterwards,” says Melinda Merchant, MD, PhD, chief medical officer at Normunity and a member of OSI’s Strategic Advisory Board.

OSI, researchers, and the FDA are already having follow-up meetings to help outline a path forward and springboard development. They are leveraging relationships and finding ways for stakeholders to connect and interact. Long-term, the goal is to improve treatment with:

More, and more efficient, clinical trials
Stronger connections with drug developers
New fit-for-purpose and feasible approaches to drug development for osteosarcoma.

“When we’re creative but also practical, it’s a win for the patients, a win for the investors, and a win for the whole drug development process,” Dr. Merchant says. “We get something that is more effective for these young adults and teens who need something new. The willingness of the FDA to work together on challenges and potential ways to address them is a really important ray of hope.”