- More pediatric oncologists need to work in pharmaceutical companies to help bridge the gap between academia and industry.
- The cost of cancer drugs could be lowered several ways, such as through academic-industry collaborations and reducing the regulatory burden associated with clinical trials.
- Parent and advocacy groups can help accelerate drug development through fundraising, forming working groups, and pushing authorities to make regulatory improvements.
The panel discussion brought together speakers from academia, industry, and parent groups to explore ways to accelerate drug development for pediatric cancer. Kathy Pritchard Jones, of the University College London, opened the discussion by noting how the pediatric drug development landscape used to be a desert, in which biological insights into childhood cancers, access to new drugs, and interest among pharmaceutical companies were all lacking. Now there is a wealth of data about disease mechanisms, but as Jones said, “how do we translate that information into knowledge…and ultimately therapies?”
Panel speaker Paul Workman, of the Institute of Cancer Research, London, stressed the importance of academic and industry groups, healthcare systems, and patient and other funding groups, working together to bring compounds to clinical trials. Academia plays a key role in drug discovery because there are no shareholders and venture capitalists in this setting. “We can take out much of the risk that way,” Workman said. However, he noted that early data can have reproducibility and robustness problems; academic labs need to be rigorous about validating targets, such as through CRISPR, RNAi, and addiction screens. During the question and answer period, John Maris, of the Children’s Hospital of Philadelphia, pointed out that the issue with data reproducibility occurs in both academia and industry.
Maris also commented that there are important differences between big pharmaceutical companies and start-up companies. Big pharmaceutical companies are generally interested in hearing from academic researchers about a target compound’s mechanism of action and mechanism of resistance. “We can think with them about combinatorial approaches and what they have in the pipeline,” Maris said. “Those collaborations have been very exciting.” On the other hand, a small biotech company with one drug in their portfolio may be more interested in model systems that academic labs develop to demonstrate proof of concept.
Louis Chesler, of the Institute of Cancer Research, posed a question to the panel about how academic labs can streamline the process of getting access to companies’ target compounds and mouse model systems to validate compounds. Panelist Raphaël Rousseau, of Gritstone Oncology, Inc., recommended academic labs establish a master agreement, where they have access to the company’s portfolio, and master collaboration. Beyond providing the drug, Rousseau said, industry insiders would also like to “provide you with additional expertise so that we jointly work on a project and we jointly publish.”
Facilitating those collaborations is one of many reasons why it is important to bring more pediatric oncologists into industry, Rousseau said. He also noted that numerous companies organize fellowships so pediatricians can spend one to two years learning about drug development. During the question and answer period, Mignon Lee-Cheun Loh, of the University of California, San Francisco, discussed the resources that her institution puts into training their faculty.
An audience member challenged the panel to consider ways to lower the cost of drugs. Panelist Stefan Burdach, of the Technical University of Munich, argued that it’s possible to reduce the cost of clinical trials by reducing unnecessary paperwork. “We all want safe drugs, but I want to posit that the equipoise between safety gain and regulatory volume has been lost,” Burdach said. He suggested that researchers talk with regulatory authorities about reducing drug development costs, and encouraged parent and advocacy groups to push politicians who fund regulatory authorities to ease the regulatory burden.
The fourth panelist, Patricia Blanc, of Imagine for Margo and Unite2Cure, represented the parent perspective. Blanc talked about creating Imagine for Margo, a French foundation, six years ago after her daughter Margaux died of an aggressive brain tumor for which there were no innovative treatments or even clinical trials. She proposed three areas where parents can help improve access to innovative drugs for children: fundraising so researchers can start trials earlier and enroll more children; cooperating with other shareholders to form working groups that tackle barriers to drug access; and advocating on a national and European level for regulatory improvements, which she does with Unite2Cure, a European network of parents and patients organizations.