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Published On: Fri, Apr 26th, 2013

Rand Paul calls for foreign drug studies to be accepted in US for orphan diseases

US Senator and physician, Rand Paul of Kentucky took to the Senate floor Thursday with a very personal message. During his 4:30 minute talk on the floor, Dr. Paul appealed to the fellow Senators about clearing regulatory obstacles by accepting foreign drug studies as our own, and allowing these drugs to get to the people that need them.

Senator Rand Paul uses American sign language in the beginning of his speech on orphan diseases Image/Video Screen Shot

Senator Rand Paul uses American sign language in the beginning of his speech on orphan diseases
Image/Video Screen Shot

The personalized presentation included a story of his nephew, Mark Pyeatt, who is stricken with the rare disorder, neurofibromatosis 2.

Neurofibromatosis 2 is characterized by recurrent neurologic tumors and its signature tumor is one of the auditory nerve.  It’s relentless course ultimately destroys the hearing, thus the use of sign language in the speech, both by the Senator and by an interpreter.

Paul said, “Neurofibromatosis 2 is a rare disease.  Some call it an orphan disease.  Orphan diseases face certain obstacles that other diseases do not.  Money is allocated typically for research based on how prevalent the disease is.  For rare diseases the resources are likewise rare.

“In order for investors to invest in a cure for neurofibromatosis 2, regulatory obstacles need to be cleared. We need to allow foreign drug studies to be accepted and not repeated in the US.  We need to have speedy approval for drugs that are already being used by the general populace in foreign countries.”

In the speech, he also talked of his chief of staff’s sister, who is also stricken with an orphan disease, pulmonary fibrosis, and her situation.

“She takes a medication that is part of an experimental trial in the US but has been on the general market for years in Japan.  If she did not live near a center of research it would be illegal for her to take the drug.  If her family did not pay the 1500 per month out of pocket, she could not receive it.  This drug should clearly have been approved already.  It went through trials here, it is already approved in Europe and Japan.  200,000 Americans are denied it today.

“We all want safety in drugs and cures.  We all acknowledge that it is a balancing act.  We should all acknowledge that the regulatory obstacles and burdens new drugs face in our country are oppressive and counterproductive.

“My hope is by putting a face on two orphan diseases, that are close to my family and friends, others will come to realize we must do something to get rid of government obstacles to cure.”

Orphan drugs are those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

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About the Author

- Writer, Co-Founder and Executive Editor of The Global Dispatch. Robert has been covering news in the areas of health, world news and politics for a variety of online news sources. He is also the Editor-in-Chief of the website, Outbreak News Today and hosts the podcast, Outbreak News Interviews on iTunes, Stitcher and Spotify Robert is politically Independent and a born again Christian Follow @bactiman63

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  1. Rand Paul calls for foreign drug studies to be ... says:

    […] Rand Paul calls for foreign drug studies to be accepted in US for orphan diseases The Global Dispatch Paul said, “Neurofibromatosis 2 is a rare disease. Some call it an orphan disease.  […]

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