New doses, devices are eyed for children

Doctors have long struggled with how best to treat small children with drugs and medical devices that are mostly designed, tested, and approved for use by adults.

A Cambridge-based nonprofit that is officially being launched today hopes to change that. The Institute for Pediatric Innovation says it will work with three major children's hospitals in California, Kansas, and Ohio to redesign drugs and devices to better fit children.

Such efforts may get a boost from the US government. Legislation approved Thursday by a committee of the US House of Representatives would provide such organizations with $6 million annually for the next five years and has incentives to encourage manufacturers to introduce products for the pediatric market.

Currently, physicians treating the smallest patients often have to improvise. About 70 percent of pharmaceuticals are used "off label" in children, which means they are adult drugs whose doses are customized to a child's height, weight, and age.

For conditions that require medical devices, doctors are often forced to be more creative. For instance, to treat sleep apnea, a condition that interrupts breathing, adults can be outfitted with a rubber mask that guarantees an uninterrupted supply of air. Because such masks are too large for infants, Dr. Ann Halbower, medical director of the Johns Hopkins University's pediatric sleep disorders program, once modified one to treat a baby. That solution, she said, got her reported to the hospital's risk management staff.

There are limitations on what doctors treating children can do with many adult products, especially such devices as stents, wire mesh tubes used to prop open arteries, because they do not change as a child grows.

The institute has raised the first $1 million of the $10 million it seeks to raise over the next five years from two corporations and the Ewing Marion Kauffman Foundation, which funds innovation.

Its first steps will be developing a list of the most promising drugs and devices to be reformulated, beginning with a step the institute calls product imagination. Doctors and nurses would meet with engineers to market to brainstorm solutions based on the priority needs of the children's hospitals in the coalition.

In its first year, the nonprofit wants to reformulate a generic drug and work with clinicians to pick a medical device to reengineer for use in children.

Donald Lombardi, the institute's chief executive, left Children's Hospital Boston last year and has since built partnerships and raised money to achieve the nonprofit's mission. He highlighted the institute when he testified at a congressional hearing last month . He said he understands the value of commercializing ideas that are born in doctors' offices.

During his 15-year tenure at Children's Hospital, establishing and heading its Intellectual Property Office, six medical products invented by doctors were licensed, generating tens of millions of dollars in revenue for the hospital. A dozen more ideas are in clinical development. But most of the products have been designed, at least initially, for use by adults, Lombardi said.

In the past, Congress has attempted to encourage pharmaceutical companies to test their products in children by offering to extend the period during which they can profit from a product without competition from generic versions. Food and Drug Administration reform bills considered by the House last week would extend similar incentives to medical device manufacturers.

In addition to speeding the process for manufacturers to bring a pediatric device to market, Congress is poised to permit them to set prices for those devices sufficiently high enough to make a profit, provided no more than 4,000 are distributed annually.

"The profit will be there," said Representative Edward J. Markey, Democrat of Malden and cosponsor of the legislation. "Otherwise, there just would not be a large enough market for them to ever manufacture a device at all, and the medical community would wind up just continuing to jerry-rig adult devices."

When a company decides to produce a drug or device for pediatric treatment, the firm faces staggering challenges, some of them financial. Pediatric uses account for between 5 and 10 percent of drug and device sales, Lombardi said. "But pediatrics isn't one market ," he added. " It's at least six submarkets, from the preterm baby to the teenage mom carrying it."

Manufacturers interested in targeting a younger market must contend with especially stringent oversight of clinical trials involving children. Signing an informed consent document to participate in a trial, routine for an adult, is "not a meaningful concept" for children, Lombardi said. As a result, review boards force researchers to clear significant hurdles before allowing a clinical trial that involves children.

In some cases, one of the institute's corporate partners -- Children's Medical Ventures, based in Norwell -- might take the next step of pushing a new product to the hospital market. In other cases, the institute says, it would leverage government funding or advocacy group funding to cover development expenses. The institute itself would absorb the expense of the earliest part of the development process and would hand off a partially or fully developed product to a company that would make and sell the product.

Reformulating a single drug for pediatric use, such as a treatment for lead poisoning, could open a market valued at $25 million to $50 million per year, Lombardi said, small in comparison with a major new adult drug, but still a moneymaker. "We believe that there is a significant portfolio of such drugs that can be developed at a cost of between $2 million and $7 million," he said.

Diedtra Henderson can be reached at dhenderson@globe.com.

(Correction: Because of a reporting error, a Page One story Monday gave the wrong location for a children's hospital that is helping a Cambridge nonprofit adapt drugs and devices for pediatric use. The hospital is in Kansas City, Mo.)