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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 age 6 to 11 now have a therapy option that addresses severe eosinophilic asthma, a complex and challenging condition to treat, which was previously only approved for use in children age 12 and older. This breakthrough is a significant step forward, as asthma is the third-ranking cause of hospitalization among children younger than 15.

  • Type 2 Diabetes: The first non-insulin drug approved to treat type 2 diabetes in pediatric patients is now available. The medicine enhances the incretin system, a natural body system that helps to regulate glucose, and improves blood sugar control.

  • Peanut Allergy: A new oral immunotherapy designed to reduce the incidence and severity of allergic reactions due to accidental peanut exposure is now available for children with peanut allergy. The therapy delivers a controlled daily dose of peanut protein that is gradually increased over months to build tolerance in the immune system’s overreaction to peanuts.

  • 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 2,100 industry-sponsored pediatric clinical trials underway, involving 1.2 million pediatric patients 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.

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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 the 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

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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.

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Advancing Solutions Together

America’s biopharmaceutical companies are committed to advancing innovative treatment options for pediatric patients. Collaboration is instrumental to harness innovative research approaches and address the scientific and operational challenges to conduct pediatric research.

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

  • In October 2016, the Children’s Cause for Cancer Advocacy (CCCA), PhRMA, and Biotechnology Innovation Organization (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. Participants 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.
  • Additionally, in 2017, the Institute for Advanced Clinical Trials for Children (I-ACT for Children) was launched to address challenges in bringing new therapies to children. I-ACT for Children, an independent nonprofit, seeks to foster public private collaboration in finding solutions to barriers to pediatric medicine development, as well as improving the pediatric clinical trial process.