Press Release

PhRMA Statement on Tremendous Success of BPCA and PREA

PhRMA April 26, 2013

Washington, D.C. (April 28, 2011) — Pharmaceutical Research and Manufacturers of America’s (PhRMA) Sascha Haverfield, Vice President of Scientific & Regulatory Affairs, issued the following statement today regarding the need for permanent reauthorization of these two important pieces of legislation:

“The combination of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) has driven research to create innovative medicines for children younger than 18 and has greatly advanced American children’s medical care.

“As Sen. Christopher Dodd, former Senate Health, Education, Labor and Pensions Committee Chairman noted, ‘The story of the Best Pharmaceuticals for Children Act is one of huge success for children and their families.’

“PhRMA urges Congress to permanently reauthorize these two important Acts to allow pediatric research to continue to thrive and to create more therapeutic options for children and healthcare providers.

“Already, 181 medicines to treat a broad range of diseases have completed the program established under BPCA. This has armed pediatricians with vital information about which medicines and which doses are safe and effective for pediatric patients. And, since 2007, 324 pediatric studies have been completed, involving more than 125,000 pediatric patient volunteers.

“The beneficiaries of this resurgence in innovative research are children, teenagers and their families. A child born today can expect to live 30 years longer than a child born a century ago, according to the National Center for Health Statistics. Because of major treatment advances, 80 percent of children diagnosed with cancer in 2009 survived five years or longer, according to the American Cancer Society.

“Today, more than 230 medicines are in development to meet the special needs of children. While PhRMA celebrates the continued success that our member companies have had in pediatric research, more work remains to be done.

“As PhRMA points out in today’s testimony during the Institute of Medicine’s public meeting regarding pediatric studies under BPCA and PREA, to help to ensure that pediatric research continues at today’s impressive pace, it is imperative for Congress to provide greater certainty to innovator research companies and to allow for more predictable regulations. Both actions would help to spur increased medical research into today’s treatments and tomorrow’s cures for pediatric ailments.

“Pediatric product development would benefit from updated regulatory guidance from the Food and Drug Administration (FDA) to help both the innovator industry and the Agency’s reviewers achieve a mutual understanding of the requirements under the law. Because of the current five-year sunset and reauthorization cycle for BPCA and PREA, no such current guidance exists. Each authorization changes the law. If Congress were to permanently reauthorize both Acts, the FDA would be in a better position to publish and maintain up-to-date guidance.

“As evidenced by our participation in today’s public session, PhRMA and its member companies are committed to continued dialogue about these important issues.”

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives. PhRMA companies are leading the way in the search for new cures. PhRMA members alone invested an estimated $49.4 billion in 2010 in discovering and developing new medicines. Industry-wide research and investment reached a record $67.4 billion in 2010.

Find PhRMA Online:

For information on how innovative medicines save lives, visit:

For information on the Partnership for Prescription Assistance, visit:

For information on ensuring the flow of medicines during public health emergencies, visit