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Jan. 5, 2016

TOPIC: Advocacy

Wall Street Journal Publishes NORD Letter to the Editor

Posted by Jennifer Huron

wsjThe following Letter to the Editor by NORD President & CEO Peter L. Saltonstall appeared in the Wall Street Journal (January 2, 2016).  You read the letter online on the Wall Street Journal website.

Wall Street Journal
January 2, 2016

Dr. Makary urges fundamental changes in the Orphan Drug Act, alleging that some drugs developed for rare diseases in fact have become blockbusters, treating other diseases.

We within the rare disease patient community regard this 1983 legislation as one of the most successful laws ever enacted to support our community. It provides incentives for the development of new drugs for patients with rare diseases. And it has been successful.

The commentary surprisingly does not even use the word “patient” until the very last sentence. Our view is that the focus of any discussion about drug development should be on patients—in the case of rare diseases, the one in 10 Americans who has or will develop a rare disease, and then encounter a 95% likelihood of zero treatment. This is the public health issue that the Orphan Drug Act addresses.

The author cites his own study that relied on sales figures to indicate how widely a drug product is used. The more appropriate measure of drug use is how many patients receive it. Many recently approved drugs are expensive, especially biologics, and so the sales figures for them can be high. Thus sales figures are not an indicator of actual product use or value.

We all would like drug prices to be lower. But it takes an average of 12.5 years and $1.5 billion (in 2014 dollars) to bring a new drug from the preclinical stage through FDA approval. And each drug development project is highly risky. Most fail. The costs and risks increase when you factor in the challenges of working with small, dispersed patient populations in the rare-disease community.

It should be seen as welcome news that therapies developed for a limited patient population can and do provide value to others. As drug developers invest in possible new uses of their products, they seek FDA approval when the data support it. We need to continue to trust that the FDA will implement the Orphan Drug Act with discretion and make sound judgments in determining which drugs qualify for the incentives provided for in the law.

Peter L. Saltonstall

President and CEO

National Organization for Rare Disorders (NORD)

Danbury, Conn.

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