Research & DevelopmentPediatrics

America’s biopharmaceutical research companies are leading and committed to advancing innovative treatment options for children. In recent years there has been tremendous growth in pediatric clinical research, resulting in critical advances in our understanding of pediatric illnesses and how to best treat them. The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) have transformed the way medicines are developed and labeled for pediatric populations. Together, these laws spur pediatric research and development, through a careful balance of incentives and obligations, and have greatly advanced children’s medical care in the United States. While we celebrate the continued success that biopharmaceutical companies have made in pediatric research, more work remains to be done to develop treatments and cures for patients with unmet medical needs. New knowledge and new medicines can offer hope that children today will recover from illnesses and grow into healthy adults.

Industry Commitment to Pediatric Research

The innovative biopharmaceutical industry is firmly committed to conducting pediatric research, and is making great strides against pediatric illnesses. Recent research has provided important new dosing, safety and efficacy information in pediatric populations that is changing the treatment landscape across a range of serious and life-threatening diseases, including:

  • Asthma: pediatric patients with allergic asthma now have important treatment options that address the underlying biological processes that cause the disease, compared to other medicines available at the time, which only addressed the symptoms. It is estimated that in children, at least 80% of asthma is allergic.
  • Acute Lymphoblastic Leukemia: a first-in-class medicine offers pediatric patients with a specific form of this rare blood cancer the first new treatment option in 10 years.
  • Hepatitis C: the first direct-acting antiviral treatments are now available for children and adolescents patients with this devastating virus.
  • Rare Genetic Brain Tumor: children with a rare genetic disorder, tuberous sclerosis complex, now have a treatment option for tumors that occur in the brain. Affecting about 1 in 6,000 newborns, this disease primarily occurs in childhood and this new approval offers a dissolvable dosage form that is easier to take for pediatric patients.
  • Chronic Myelogeneous Leukemia: additional research revealed that a breakthrough targeted therapy for this rare blood cancer is safe and effective in very young children, offering an important treatment option for physicians to use in treating this rare blood cancer.
  • HIV/AIDS: new combination therapies are now available for pediatric patients, reducing the complexity of treatment for this devastating disease by offering options that can be taken once a day in a fixed-dose pill.

A robust drug development pipeline offers tremendous promise for the future. Currently there are over 1,300 industry-sponsored pediatric clinical trials underway across a variety of therapeutic areas, including diseases where there is significant unmet need, such infectious diseases, neurologic conditions, genetic disorders, and several forms of cancer. 

Policies to Foster Pediatric Drug Development

Prior to the passage of the Food and Drug Administration Modernization Act (FDAMA) in 1997, there was growing concern that many FDA-approved drugs had not been clinically tested in children, resulting in inadequate or unavailable information on dosing, safety, efficacy and side effects. Because medicines that may work one way in adults may not work the same way in children, it is important that medicines intended for children are studied in children.

The need for pediatric-specific information in drug labeling prompted action, leading to the passage of two laws that address the study of drugs in pediatric populations: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). PREA and BPCA work together to foster pediatric drug development, providing previously unavailable information on dosing, safety, efficacy, and side effects.

  • PREA gives the FDA the authority to require pediatric studies requires pediatric studies for certain new medicines approved for use in adults where the indication for use in children would be comparable to that for adults, and produce formulations appropriate for children (e.g., liquid or chewable tablets).
  • BPCA complements PREA requirements by providing sponsors with an incentive (6 months additional marketing exclusivity) to undertake voluntary pediatric studies at the request of FDA, many of which may not be required under PREA. BPCA has proven to be an effective incentive for stimulating pediatric drug development, especially in cases where PREA requirements do not apply (for example, for orphan diseases, including rare cancers).

Made permanent with large bipartisan support in 2012, PREA and BPCA have been key drivers of pediatric research, generating important safety and efficacy information on the use of medicines in children. This balanced approach has driven research on innovative medicines in pediatric patients, resulted in more treatment options for children, and has greatly advanced children’s medical care. Learn more here.

 

"Before BPCA and PREA became law, more than 80% of the drugs approved for adult use were being used in children, even though the safety and effectiveness had not been established in children. Today that number has been reduced to about 50%.” – Dr. Lynne Yao, FDA

Unique Research Challenges

Developing medicines for pediatric diseases poses unique scientific and operational challenges. Biopharmaceutical companies are committed to working with all stakeholders to combat the issues that prevent further research and results. A recent multi-stakeholder workshop highlighted the range of challenges facing pediatric researchers and explored ways to overcome obstacles, particularly in the oncology space. Learn more here.

 

Advancing Solutions Together

America’s biopharmaceutical companies are committed to advancing innovative treatment options for pediatric patients. Collaboration will be instrumental in harnessing innovative research approaches and addressing the scientific and operational challenges to conducting pediatric research.

Provisions within the 21st Century Cures Act, as well as the PDUFA VI agreement, will help accelerate pediatric medicine development. Learn more here.

Collaborative, pre-competitive initiatives can help address some of the identified scientific and operational challenges in pediatric oncology medicine development.

  • In October 2016, Children’s Cause for Cancer Advocacy (CCCA), PhRMA, and BIO convened a workshop that brought together, for the first time, the full spectrum of key pediatric oncology stakeholders to address some of the identified scientific and operational challenges in pediatric oncology medicine development. (These included 15 biopharmaceutical companies, patient and provider advocacy groups, FDA, NIH, and some of the country’s leading pediatric oncologists). The workshop participants resolved to work together to pursue the creation of a pre-competitive public-private partnership to explore potential approaches to solving identified challenges.
  • A newly announced pediatric clinical trial network, the Institute for Advanced Clinical Trials for Children (I-ACT), seeks to improve the quality and timely completion of global pediatric studies to address the pediatric evidence gap.

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