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The Importance of Pediatric Drug Development: A Conversation with Pr. Gilles Vassal, Dr. Gregory Reaman, and Mr. Raymond Rodriguez-Torres

The Importance of Pediatric Drug Development: A Conversation with Pr. Gilles Vassal, Dr. Gregory Reaman, and Mr. Raymond Rodriguez-Torres


From L-R: Dr. Gregory Reaman, Pr. Gilles Vassal, Mr. Raymond Rodriguez-Torres


Oncology is a widely studied and researched field, and the advancements in scientific and clinical understanding of pediatric oncology provides an opportunity to better inform pediatric drug development. For a long time, there was an idea that children are “tiny adults” and that the treatments studied and approved for adult cancers would benefit and work in children with the same or similar type of cancer. This is typically not the case, and due to this there is a growing recognition within the oncology community to focus more on studying treatments for children with cancer.


Studying pediatric oncology has proven difficult since pediatric drug development can face unique challenges. Molecularly targeted agents and innovative clinical trial approaches, however, offer opportunities to address the shortfalls of new oncology drugs developed for pediatric cancer patients. Due to the need for studying pediatric cancers and developing treatments, Friends of Cancer Research (Friends) is holding a forum focusing on the acceleration of pediatric drug development.


In advance of the event, Friends reached out with some questions to a few panelists: Dr. Gregory Reaman with FDA and George Washington University School of Medicine and Health Sciences, Pr. Gilles Vassal of University Paris-Sud, France and Gustave Roussy, and Mr. Raymond Rodriguez-Torres of the Live Like Bella Foundation. Please find their responses below.

Please provide a short preview of what you will discuss at the Accelerating Pediatric Drug Development event on February 21.

Dr. Reaman: I will briefly describe the pediatric legislative initiatives that guide pediatric drug development in the U.S., the extensive limitations of PREA [Pediatric Research Equity Act] for pediatric cancer drugs and how we therefore attempt to maximize the regulatory authority provided by BPCA [Best Pharmaceuticals for Children Act] to expedite evaluation of relevant new drugs for children with cancer.


Pr. Vassal: First, let me thank you for inviting me. I will be traveling from Europe to share with you and discuss our strategy for accelerating oncology drug development for children and adolescents with cancer. One of the main goals of the European Society of Pediatric Oncology (SIOPE) in our strategic plan is to increase both the cure and quality of survivorship over the next 10 years. In addition, we created ACCELERATE, a multi-stakeholder platform with parents, regulators, pharmaceutical companies, and academic experts to build a new way of collaboration and speed up innovation for children with cancer.


Mr. Rodriguez-Torres: My goal is to provide a real-world perspective as to what clinicians and families who have children with cancer face each day relating to innovation in pediatric drug development.  As a former executive in the pharma/biotech industry and the current chairman of a global childhood cancer foundation, and a father who lost his daughter to cancer, I hope to offer a multifaceted perspective.  Ultimately, my desire is to offer my unique experience to help frame and continue the important efforts that are ongoing to accelerate innovative drug developments for children battling cancer. 

Do you think there should be more of an effort for global harmonization when it comes to developing and accelerating pediatric drugs?

Mr. Rodriguez-Torres: Yes. One of the greatest problems in advancing clinical research for children with cancer is proper patient recruitment to help power statistical outcomes.  By harmonizing efforts globally, we could accelerate enrollment in clinical trials, identify patients with certain genetic targets who would be better candidates, and increase education and awareness for parents, researchers, and clinicians. 


Dr. Reaman: The small study populations eligible for pediatric cancer clinical trials, especially those incorporating new drug and biologic products, increasingly mandate international collaboration. Additionally, given the limited number of patients and the magnitude of clinical research to be done, any possible duplication of effort must be avoided to preserve the scarce patient resources and answer the most meaningful scientific questions. Data sharing is critical and efforts to support and enhance international collaboration in study design and conduct are necessary.


Pr. Vassal: It is necessary! On one hand, each pediatric cancer is rare and even rarer and ultra-rare when molecular subtypes are considered. On the other hand, pharmaceutical companies are global and they must comply with pediatric regulations in the U.S. and Europe, and possibly other continents in the future. And you know, pediatric oncologists and hematologists have been working together across continents for the last 50 years implementing high quality academic clinical research to increase disease free survival at 5 years from less than 40% up to 80%, nowadays. In pediatric oncology, we think and we do global! So yes, global harmonization is needed to accelerate.

Please discuss the types of logistical issues or barriers you think exist that hinder these efforts in pediatric drug development.

Dr. Reaman: I find the logistical challenges to harmonization stem from the differences in process, procedures, and timelines dictated by different regulatory requirements in various parts of the world.


Mr. Rodriguez-Torres: I believe there are regulatory differences that would need to be addressed particularly between the EU and U.S.  Additionally, there are efforts pharmaceutical companies and researchers could and should take for this. There is also the issue of increasing communication while protecting intellectual property, which is a delicate and important balance.  Lastly, and perhaps most significantly in my opinion, would be the creation of a global cloud based, HIPPA compliant genetic repository of data on children eligible for clinical trials.  


Pr. Vassal: The main challenge is a change of mindset. Pediatric Oncology drug development is by definition a non-competitive field, while pharmaceutical companies are in a frontal competition in the adult oncology field. Pediatric cancers are complex and there is a need for cooperation with academic experts early in development to best fit the needs of patients and embed new medicines in therapeutic strategies. In addition, there are many more oncology drugs in development in adults than we can explore in children. We need to be smart and to drive drug development through science. And there is a need for choosing and prioritizing the potentially best drugs to move forward. This is where I call for a change of mindset: working together with regulators, industry, patient advocates, and academic experts across company portfolios and to better inform and facilitate prioritization.

Pr. Vassal and Dr. Reaman, considering you work with regulatory bodies in different countries could you provide your thoughts on pediatric master protocols, and should these trials be international in scope?

Pr. Vassal: For us, master protocols exploring activity of several compounds, as single agents or in combination, are major tools to step up drug development. By avoiding opening a trial for each drug and by serving as a trial platform, they can easily accelerate and enlarge the number of therapeutic options we can propose to patients and parents in order to match molecular alterations found in the tumors of children with relapsed or refractory disease.  In Europe, we have set up an academic multi-arm multi-drug master protocol, the ESMART trial, which is running within the ITCC network, a network of 52 centers in 13 countries for early phase trials. I will present ESMART and our precision cancer medicine strategy in more detail at the Feb. 21 meeting.


Dr. Reaman: In my opinion, master protocols are one promising approach to the challenge of pediatric cancer drug development. Master protocols are especially pertinent to pediatric oncology given the existing clinical trial infrastructure which exists and the opportunity to evaluate multiple drugs across a variety of rare diseases in a single protocol open to a relatively small patient population.

Keeping in mind the regulatory differences in each country, how, in your opinion, can master protocols overcome challenges associated with these differences in regional regulatory requirements for pediatric drug development?

Mr. Rodriguez-Torres: I believe that master protocols particularly with international representation would be of great benefit to patient identification, enrollment, and powering trials for better and more meaningful statically analysis. 


Dr. Reaman: I think master protocols provide an opportunity for global collaboration in study design and conduct in order to evaluate multiple drugs in a single or across a variety of diseases.


Pr. Vassal: Here, the challenge is to make master protocols, run by either industry or academia, and the data they generate compatible with regulatory processes in an intent to file. To this extent, the new tools set up by the FDA and the EMA to accelerate evaluation of innovative compounds with outstanding activities demonstrate that things can be changed to adequately address the acceleration of drug development and speed up access of patients to new and effective therapies.

One final question: why do you think this event and topic are important to discuss?

Dr. Reaman: Anything and everything that facilitates or expedites timelines for new cancer drugs in children is an important topic to discuss.


Pr. Vassal: I think that this event will provide the forum for important discussions. We will discuss the different pediatric master protocols with new oncology drugs that are ongoing or planned and figure out their differences and their complementarity. We will put them in perspective and figure out how harmonization and cooperation between all stakeholders will accelerate innovation for children and adolescents with cancer. I very much look forward to participating in these nice discussions and interactions.


Mr. Rodriguez-Torres: Childhood cancer is the #1 cause of death by disease in children.  Ensuring that all stakeholders: industry, government, researchers, clinicians, patients, and families all have a unified and coordinated voice in beating childhood cancer is critical to changing the unfortunate reality we currently face with this disease. I’m confident that by working together, we will soon see the day when childhood cancer is a treatable and curable disease. 




The Accelerating Pediatric Drug Development Forum is taking place on February 21 from 8:30am – 1pm at the Washington Marriott Georgetown. For more information visit:


Dr. Gregory Reaman is the Associate Director for Oncology Sciences at the Office of Hematology and Oncology Products with the Food and Drug Administration and Professor of Pediatrics, George Washington University School of Medicine and Health Sciences.


Pr. Gilles Vassal is Professor of Oncology at the University Paris-Sud, France, and head of Clinical Research at Gustave Roussy, a large comprehensive cancer center taking care of children, adolescents, and adults with cancer.


Mr. Raymond Rodriguez-Torres is Chairman of the Board of the Live Like Bella Cancer Foundation and Bella’s dad.


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