The Future of Care for Future Generations

You may have noticed that children are not little adults. It’s not a surprise, but it does present a unique challenge. Children deserve safe and effective medicines that are developed specifically for them.

Here’s something you may not already know: cancer is one of the leading causes of death in children, second only to accidents. And yet, despite the startling statistics, clinical studies of cancer medicines in children have historically been scarce. Due to unique challenges with studying cancer in children, these studies are often not considered until a medicine reaches Phase III or later in adults.

As ASCO marks its 50th anniversary this year, we’re looking ahead to pediatric oncology as an area in cancer care that will require significant change.

One of the biggest mistakes we can make would be to assume that study results in adults can be easily applied to children. Rather, cancers in children – with a few exceptions – are distinct diseases compared to those observed in adults. Because children are still growing, how their cancer grows and how their bodies react to treatments may be markedly different than in adults.

It’s an overlooked reality that the conventional, adult-centric approach to the development and approval of cancer medicines may not always be appropriate for children.

Pediatric oncology is an area that still needs major reform across the research, development and commercialization spectrum.

Today, 70 percent of children with cancer are likely to be cured, but many of them will suffer long-term side effects, some due to their treatments. For the remaining 30 percent, new and different medicines are urgently needed.

At Genentech, we’re focusing our efforts and taking action. These are early days, but I’m hopeful about what we can accomplish.


One of the difficulties related to childhood cancers is that children, as a population, are exceptionally diverse.

For example, did you know that the term “pediatrics” covers everyone from newborn babies to teenagers who have essentially reached physical maturity? This means that in addition to possible differences in the underlying biology of cancers, variations in factors like body weight and metabolism can make it incredibly challenging to determine appropriate dosing of investigational medicines over such a broad age range.

Development of new pediatric oncology medicines is further complicated by the rarity of childhood cancers. Pivotal studies in adults usually require hundreds of participants in order to get statistically powerful results – numbers that could never be reached in many pediatric cancers. To truly target pediatric cancers, we may need to rethink our current clinical trial model.

Perhaps the greatest challenge is an ethical one. Even if an investigational medicine may be considered safe to study in pediatric clinical trials, the risk of long-term side effects may remain unknown for many years, possibly not manifesting until well into adulthood. “Long-term” takes on new meaning when we’re talking about children who may still have years of growth, brain development, puberty and fertility ahead of them. This can make families and doctors think twice about enrolling children in randomized trials, where they may be taking an essentially unproven medicine.


Here’s the good news: we’re ready to take this on. We have recently created a special team across multiple disciplines that will be devoted to addressing childhood cancers. The innovative Pediatric Oncology Drug Development team is dedicated to improving the lives of children with cancer, no matter where they fall on the pediatric scale.

Changing outcomes for children with cancer is everyone’s responsibility. Those in industry, regulators, doctors, academics, families and advocates need to take action. Together.

And we’re going to follow the science.

We will not assume all medicines developed for adult tumors will be appropriate for children. Similarly, we should not restrict our studies by assuming medicines for specific cancers in adults won’t help children just because they don’t develop those types of tumors.

Bottom line? To develop the right treatments for children, we need to understand the biology of how childhood cancers are unique. Once we identify the key genetic and molecular cancer growth drivers, we can select from our portfolio of investigational medicines the ones that best target the specific biology of these pediatric tumors.

We need to specifically focus our efforts on children at a much earlier stage of drug development, even if the investigational medicine will ultimately not be approved for adults.

Let’s move away from the model where we wait for adult clinical trial results before we invest resources to collect safety information in pediatric models, or to develop age-appropriate formulations (liquid or smaller pills). If we do, we may be able to start studying investigational medicines in children soon after Phase I study results are available in adults, instead of during or even after Phase III.

We can’t rely solely on changing our own processes, though. Regulatory reform is needed as well. Currently, global authorities have differing recommendations and timelines for how to approach childhood cancers. Lengthy administrative processes to obtain approval of pediatric clinical development plans further impact timely initiation of clinical trials in children.

15 Years of Focus

Raphaël Rousseau, M.D., Ph.D. discusses development of therapies in pediatric oncology.

Improving outcomes for children with cancer is a goal that has guided my career. I’ve worked in pediatric oncology for 15 years, with a focus on developing innovative therapies for hard-to-treat pediatric cancers, especially immunotherapeutics.

At Genentech, I lead the development of the innovative Pediatric Oncology Drug Development (iPODD) team, a group of 25 people dedicated to evaluating investigational medicines for pediatric indications. We are implementing some important changes as part of our increased focus on childhood cancers. I’m proud of these early steps we’re taking to turn ideas into action.

Working with academic thought leaders, regulators and parents, we should establish a clear consensus that prioritizes the most promising options for children early on. Additionally, collaboration between industry and academic networks can help us identify appropriate investigational medicines and test them in rare patient populations.

More than anything, we need commitment from everyone involved.

We will need to create a framework for tracking young patients many years after treatment ends – possibly for life – to determine if there are long-term side effects on their developing bodies. Here as well, we are making proposals that could be extended across the industry.

There’s no debate – these solutions aren’t easily implemented. They require new attitudes and new ways of thinking. It’s time to combine and organize our efforts. Our children deserve it.