How Pharma Can Be a Good Collaborator in Pediatric Cancer Research

How did you develop your approach to patient advocacy in pediatric oncology? 

Several of Day One’s leaders had already been long-time advocates for drug development in pediatric cancer. I was new to the community, so we fielded a survey of the pediatric oncology advocacy community to better understand what they wanted from pharma and how we could be a good partner to them. The key takeaways were centered on principles of engagement, which directly influence how we collaborate.

What were the principles of engagement that emerged from the research? 

The first principle of engagement is to communicate early and often. We learned that it’s critical to have an ongoing, two-way dialogue based on authentic and transparent communication and to invest the time needed to build relationships. 

The second principle is around participation. Historically, pharma has not invested in pediatric cancer drug development, so a critical part of demonstrating our commitment has been getting involved with the community in meetings, conferences, and events where we listen, learn, and take part in the discussion. 

The third principle is about sharing our strengths with the community. As pharma, our unique expertise is in drug development. Sharing our insights and the considerations pharma brings to drug development is important for engaging advocates, patients, and families and enabling their insights to inform our work. 

And related to that, the fourth principle is about being inclusive: being supportive and creating  opportunities for other pharmaceutical companies to come to the table with the community, and collaborating with each other so that we can all benefit from our collective efforts and experience. 

"Historically, pharma has not invested in pediatric cancer drug development, so a critical part of demonstrating our commitment has been getting involved with the community."

Can you tell me about the tovorafenib journey, and how it was shaped by multistakeholder collaboration?  

This journey started about 20 years ago, when a small group of families whose children had been diagnosed with pediatric low-grade glioma (pLGG) – the most common type of pediatric brain tumor – provided seed funding for a dedicated research program at Dana Farber. That program led to the discovery of preclinical activity of tovorafenib (formerly known as MLN2480, TAK-580, and DAY101), which was eventually acquired and developed by Day One.

Collaboration between parent advocates, academia, and industry was critical to the advancement of tovorafenib. Without minimizing the knowledge and expertise of the team of researchers who steered this drug in the right direction, it was the collaboration among these groups of people that brought tovorafenib to where it is today. 

To explore this dynamic, we put together a workshop that deconstructed the tovorafenib journey as a case study and identified the components of multistakeholder collaboration that could be applied more broadly in pediatric cancer, as well as in other rare diseases. 

What were the components of collaboration identified in the workshop? 

The first component we identified is a core group of individuals dedicated to a specific therapeutic area. You need the right people from different stakeholder groups coming together and saying, “We are going to invest our time and resources on this disease.” 

The second component is strategic, long-term, sustained funding of a focused research program. Research and drug development are expensive – millions of dollars over a decade or more – in even the best-case scenarios. It’s critical to secure diverse and significant funding sources to both establish and sustain a research program over time. 

Third, you need an industry champion – at least one – committed to doing the work within their organization and securing support and resources for developing the drug, bringing it to market, and ensuring broad access.

The fourth component is a multidisciplinary academic research program, with a solid infrastructure and institutional support to lay the foundation for longer term efforts leading to potential drug approval by health authorities.

Finally, patient nonprofit organization involvement throughout the journey is essential. These organizations and the patients they represent are the experts in unmet needs and priorities of their community. In addition to raising awareness and understanding of unmet needs, they can rally support from potential donors and funding sources and help engage the broader community of patients and families that the drug is intended to treat. 

"As pharma, our unique expertise is in drug development. Sharing our insights and the considerations pharma brings to drug development is important for engaging advocates, patients, and families."

How translatable is this model to adult cancers and rare diseases?

When we set out to learn from the tovorafenib example, it was with pediatric cancer in mind exclusively. What we soon realized is that our learnings were applicable in any cancer or rare disease where there is limited funding for research, lack of disease awareness, and significant unmet need. 

What specifically does pharma bring to this model of multistakeholder collaboration? 

It’s important to recognize that, historically, academia has played the most significant role in conducting clinical research in pediatric cancer, while nonprofits, philanthropic organizations, government bodies, and pharma have provided funding to support that research. Where pharma and academia can be more collaborative is in the design of academic clinical trials.

Typically, academic research is undertaken with a strong incentive for publication. The criteria for an exceptional study for publication are not the same as those required by the FDA to support a potential new drug approval. So, where pharma can play an important role is in advising academic researchers on “fit-for-filing” academic studies, so that their study results can more effectively lay the foundation for development of a new treatment. 

Pharma partners brought in early can evaluate clinical study designs and identify the gaps that could prevent an otherwise promising investigational drug from being picked up by a pharmaceutical company for development into a new medicine.

"There has been a paradigm shift in terms of patient advocate/pharma relationships and appreciation for what’s possible when we work together."

How is patient advocacy enabling deeper inter-stakeholder collaboration in the R&D landscape? 

I’m seeing more and more startups hiring for patient advocacy earlier in the process than they have in years past. I’m also seeing industry advocates collaborating with each other to determine how best to navigate their organizations and ensure patient advocacy is part of the process. 

It differs across organizations, but the more we can demonstrate the impact of our work and share those experiences, the better. There is tremendous upside for pharma companies when they have strong patient advocacy functions. 

Patient advocacy has evolved so much. There has been a paradigm shift in terms of patient advocate/pharma relationships and appreciation for what’s possible when we work together. Everyone understands that drug development is risky, there are a lot of unknowns, and that it will take all stakeholders working together to advance the field and make meaningful change for patients and families.

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