217 Medicines and Vaccines Are in Development To Meet Special Needs of Children

A new survey by the Pharmaceutical Research and Manufacturers of America (PhRMA) found 217 medicines and vaccines in development to help and heal children. The survey also found that 25 medicines for children had been approved by the Food and Drug Administration in the past year and that pharmaceutical companies will soon begin clinical trials on an additional 52 potential medicines for children.

Medicines in Development for Children*

*Some medicines are listed in more than one catagory

The progress made in improving the health of children through use of innovative pharmaceuticals during this century has been phenomenal. At the beginning of the 20th century, death claimed 4 out of 10 American children before they were old enough to leave home. But vaccines have virtually eradicated the diseases that used to break up families and break the hearts of parents: diphtheria, whooping cough, polio, measles and others. And medicines are saving even the youngest patients. U.S. infant mortality sank in 1996, in large part because of medicines that accelerate lung maturity in premature babies. Thanks to innovative new medicines, children who once would have died young of leukemia, cystic fibrosis and other diseases are now growing into healthy adults. And medicines are raising the quality of life for children with asthma, diabetes and other diseases, enabling them to be kids *#151; not just patients.

Among the new medicines are:

• 47 to treat cancer, which remains the leading disease killer of children under 15, despite the fact that death rates for childhood cancer have declined 57 percent since the early 1970s.
• 15 to treat asthma, which has seen both rising death rates and incidence among children in the past decade. Some 4.8 million American children suffer from asthma, and about 400 children die of the disease each year. The annual cost of asthma exceeds $6 billion, including $2.5 billion in costs associated with emergency care, hospitalization and death.
• 13 to treat bacterial infections. These include a new medicine to prevent ear infections, which cause permanent hearing damage to 13,000 children a year. The medicine is a synthetic version of a complex carbohydrate that occurs naturally in human breast milk.
• 14 for AIDS and related disorders, including protease inhibitors, a key part of the combination drug therapy that helped cut AIDS deaths nearly in half in 1997.
• 11 for cystic fibrosis, which affects 30,000 patients and is the most common fatal genetic disease in the United States. In the past two decades, medicines have helped double the life expectancy of children with cystic fibrosis.
• 10 for genetic disorders such as hemophilia, sickle cell disease, and Duchenne's muscular dystrophy.
• 10 for psychiatric disorders, including depression, schizophrenia and attention deficit hyperactivity disorder.

In addition, companies are working on new medicines for diabetes, epilepsy, gastrointestinal disorders, growth disorders, viral and fungal infections, neurologic disorders, respiratory disorders, rheumatoid arthritis, skin disorders, medicines to help make transplants effective and vaccines to prevent a variety of diseases.

The survey shows that medicines for children are a very active area of pharmaceutical research and development, despite the practical, legal and ethical difficulties of testing medicines in children.

In 1997, Congress established a program to provide incentives for studying medicines in children. This ground breaking program will, over the next five years, encourage research on even more medicines for children and on new formulations and treatment information for existing medicines used by children. This incentive program promises to make still more medicines available to help children run, jump, dance, learn and grow into healthy adults.

Alan F. Holmer
President
PhRMA

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