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  • Home > Blog > Mesothelioma > NORD Responds to JAMA Article Claiming Drugs for Rare Diseases Such as Mesothelioma Could Have Safety Issues
Posted on Thursday, Jun 30, 2011

NORD Responds to JAMA Article Claiming Drugs for Rare Diseases Such as Mesothelioma Could Have Safety Issues

The National Organization for Rare Disorders (NORD) responded to the editors of the Journal of the American Medical Association (JAMA) regarding an article they recently published claiming that orphan disease clinical trials may not be managed as rigorously as those for other illnesses.  There are close to 7,000 rare diseases in the United States, and many patients rely on these clinical trials for breakthrough treatments that would not otherwise be available.   Mesothelioma, an asbestos-related cancer, is one of the rare diseases for which patients and researchers closely follow clinical trials in hopes of finding a new, effective treatment.

NORD is dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them.  It is with this commitment that NORD President and CEO Peter L. Saltonstall said “We felt it important to share our thoughts in response to the article,” and to clarify the role of the Food and Drug Administration (FDA) in the regulatory process for orphan products. 

NORD specifically responded to a statement in the JAMA article that said “Orphan products constitute most new drug approvals in oncology, but safety and efficacy questions have emerged about some of these agents.” NORD stated in their letter that they are not “aware of a higher rate of safety and efficacy questions for orphan products” and that they did not believe the article supported the claim.

Saltonstall supported the efforts of the FDA stating in the letter that the FDA has shown flexibility in working with companies on “pivotal trials of orphan drugs,” yet they continue to ensure that the high standards required of all American drugs are met.

The NORD letter concluded by stating, “more resources are needed to support innovative research,” and that it is important to understand that there are special challenges inherent in developing a treatment for rare diseases.  In addition, NORD believes that patients with rare disorders are “willing to accept reasonable risk in return for hope of effective treatment.”

This belief by NORD is supported by a study conducted by researchers at Mayo Clinic, Scottsdale Healthcare and the Translational Genomics Research Institute where they found that cancer patients are willing to undergo additional tests and procedures so they can receive advanced experimental treatment in clinical trials.  Mesothelioma patients and others with rare diseases continue to hold out hope that they will find the one clinical trial that can extend their lives and help find a cure for others.

NORD Responds to JAMA Article