Drugs for orphan diseases seeing more FDA approvals


In the past decade, federal regulatory agencies have signed off on more new product approvals for treatment of so-called neglected diseases.

Federal regulatory agencies are signing off on more new product approvals for treatment of so-called neglected diseases in recent years. One reason for the rise in approvals may be tied to increased research and development (R&D) funding available, report researchers at the Tufts Center for the Study of Drug Development (CSDD) in the November/December Tufts CSDD Impact Report.

It was found that more than 160 new medicines for neglected diseases had been approved in the decade between 1995 and 2005, compared with 108 such approvals in the decade prior, and fewer than 10 in the 1970s. The neglected disease category includes malaria, diarrheal diseases, helminth infections, meningitis, bacterial pneumonia, and typhoid.

“Orphan drugs have made a significant impact on patient health since 1983 following the passage of the Orphan Drug Act,” said Formulary Editorial Advisor Michele B. Kaufman, PharmD, RPH, clinical pharmacist and president, PRN Communications Inc., of New York City, and former project leader/clinical pharmacist, Emblem Health. “In the past 10 years, there have been more agents that have garnered FDA approval, and the Europeans have even modeled a similar program from the United States.

“Biotech companies and specialty pharmacy companies—since many orphan drugs are specialty drugs—like this because it increases their worth. Managed health plans see orphan drugs as a benefit or a detriment. Either way, the costs of managing these patients on the medical and pharmacy sides can be exorbitant. These drugs cannot be denied to patients in our health systems. Yet having the right [orphan] drug for managing many of these pediatric illnesses that never before had treatments, improves patient care,” Dr Kaufman adds.

She continues, “Who pays for these orphan drugs is often debated. The payor side is often neglected in decisions about these drugs. Yet they are life-saving and important agents for many…and need to keep being studied and approved to better patient care. In keeping up with these agents and learning more about them in the world of drug information, there is at least one new peer-reviewed journal, which began in the mid-2000s. It is indexed in PubMed and devoted to orphan diseases. The Orphanet Journal of Rare Diseases [www.ojrd.com] makes orphan drugs and information about them more mainstream.”

Increased R&D funding has resulted in approvals for 26 drugs and vaccines, according to Tufts CSDD study author Joshua Cohen. Study authors reviewed findings from a 2002 study that noted just 16 of 1,393 drug entities promoted from 1975 to 1999 focused on tropical diseases and tuberculosis. The actual number was 33, according to study authors. The study’s results led to a boost in R&D spending from approximately $100 million per year about 10 years ago to more than $2.5 billion annually now.

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