Continuing progress and promise in pediatric drug development

Guest Contributor
Guest Contributor November 15, 2016

Continuing progress and promise in pediatric drug development.

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health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view. Views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today.

We’re pleased to host a guest blog from Dr. Christopher-Paul Milne, Director of Research at the Tufts Center for the Study of Drug Development and Research Associate Professor at Tufts University.

It has been almost two decades since the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were passed in order to increase the number of medicines appropriately labeled for safe and effective use in children. BPCA and PREA have led to significant improvements within the field of pediatric drug development, according to a recent survey conducted by the Tufts Center for the Study of Drug Development that assessed the changes in pediatric drug development since 2008. The survey involved 11 companies, including 7 of the top 10 pharmaceutical companies by market valuation.

According to respondents, overall progress toward the goals of BPCA and PREA has been “good” and is “moving in the right direction,” with some participants seeing “a great deal” of progress, especially in key areas, such as increasing pediatric dosing information in labeling and increasing pediatric expertise within both the U.S. Food and Drug Administration (FDA) and biopharmaceutical companies. Importantly, respondents agree that BPCA and PREA have helped make pediatric studies a routine part of the drug development process.


However, this progress has come with new challenges. The survey also found that pediatric drug development costs have increased by at least 25 percent for a majority of the companies, with close to half of the companies reporting an increase of more than 50 percent. These increases may reflect the growing complexity of drug development in this space: Two-thirds of the surveyed companies reported a greater than 50 percent increase in study complexity, with medical-scientific and regulatory factors being the most significant contributors to the increase.

One particular challenge in conducting pediatric research is the very limited number of patients, which creates intense competition among research organizations. This competition is exceptionally impactful in cancer research, where the use of biomarkers further segments the patient population into smaller and smaller subsets eligible for recruitment into targeted drug trials. Neonatal studies also present a special challenge, with only one company reporting “good” improvement in this area.

The improvements made within pediatric drug development are a result of multiple factors. Several responders indicated that, since 2008, better communication between all stakeholders involved in drug development – researchers, investors, and regulatory officials – has created a more productive environment for pediatric drug development. Advances in technology and experience companies have gained from working on rare diseases (which often have similar challenges to pediatric research) have also helped enhance the pediatric drug development environment.

Looking forward, several key trends hold promise for future pediatric research and development. Nearly all respondents indicated that regulatory science, defined by the FDA as “the science of developing new tools, standards, and approaches to assess the safety of efficacy, quality, and performance of all FDA-regulated products,” is driving tremendous innovation in pediatric research. These tools include the qualification of clinically meaningful biomarkers, the increased use of patient-focused endpoints, and the development of more accurate drug dosage models. Most respondents also felt there is promise in pediatric clinical trial networks, which could increase access to patient populations, reduce costs, and shorten drug development timelines. Additionally, more than half of the respondents think that continuing advances in developmental sciences will also play a role, just as the BPCA and PREA programs continue to spur the development of safe and effective medicines for children.

The survey results can be found in the Nov/Dec 2016 Impact Report, available here.

Topics: Research and Development