NORD Study Shows That There Are No FDA-Approved Treatments For Over 90% of Rare Diseases

NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently available to patients.

Biosimilars and generic drugs are dissimilar. Generic drugs are developed using identical chemicals that are contained in the branded drugs. Generics have significantly lower price tags because there is less testing required.

Biosimilars are derived from living organisms. Their ingredients are not identical to the ingredients in the name-brand drugs. Therefore, some testing is involved.

NORD commissioned Avalere Health, a leading consulting firm, to conduct an analysis on regulations and existing laws. NORD’s intent was to determine whether these regulations and laws are causing new drugs for rare diseases to come to market.

The Orphan Drug Act

In 1983, when the Orphan Drug Act was passed, there were only 38 orphan drugs available. Between 1983 and July of 2020, 552 drugs were available to treat rare diseases. Specifically, by 2002 orphan designations numbered 1100 while approvals totaled 232 making treatment available to 11 million people.

To clarify, the approval allows marketing authorization while a designation is granted when a drug meets certain criteria and merits specific benefits.

The Orphan Drug Act has had a significant impact on treatments for rare diseases. The designation provides financial incentives such as market exclusivity for up to seven years and tax credits up to fifty percent for R&D expenses.

An amendment to the law set the definition of a rare disease as affecting less than 200,000 individuals. The FDA would consider a larger number of patients if the developer could prove that it could not recover its cost to develop the drug.

Avalere’s Investigation of Multi-Use Products

Avalere’s analysis found 154 orphan drugs that had been developed to treat one rare disease and had no other purpose. But when the drugs were submitted for reevaluation at a later date, they earned additional orphan status. Another 37 orphan drugs that had been FDA-approved for common medical conditions also earned additional orphan status.

Although these statistics are encouraging, the overall picture adds up to only 10% of all orphan drugs treating just a few diseases and even fewer rare disease patients.

For a better perspective, there are estimates of 400 million rare disease patients worldwide with 30 million in Europe. The U.S. has approximately 7,000 known rare diseases affecting 25 million Americans. Compare these numbers with 1,000 rare disease drugs currently being investigated in clinical trials.

20 Year Patent Protection

The NORD analysis included an examination of the 394 orphan drugs that did not qualify for biosimilar or generic competition. The researchers discovered that 80% of these products were still protected by patents and another 20% had protection due to orphan drug designation exclusivity. Most of the patents offered protection for over 20 years.

However, NORD’s CEO Peter Saltonstall, acknowledged that if it were not for the Orphan Drug Act, the majority of orphan drugs may not have been developed. He added that a framework is urgently needed to encourage the development of therapies for the majority of patients with rare diseases and also creates competition sooner.

The NORD report is available at

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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