Newsletter - October 20, 2016
Developing Rare Disease Products: Lessons from Serepta and Biomarin
Last month’s approval of Sarepta’s drug for Duchenne Muscular Dystrophy (DMD) by FDA shook the regulated industry due to the extremely arbitrary nature of the FDA decision. The review of the summary basis of approval, which lists the data FDA relied on to approve this drug, would not help. So, should all developers of products for rare diseases employ Sarepta’s strategy of extreme public campaign to pressure FDA? Read More
Does Screening Tests Save Life? Ben Stiller Creates Prostate News
Earlier this month, actor Ben Stiller announced that he was free of prostate cancer thanks to a routine PSA test he got. His essay encouraged all men to get PSA tests. However, the U.S. Preventive Services Task Force (USPSTF), a voluntary non-profit that makes recommendation for screening tests, does not recommend PSA tests due to very high false positive of such tests (almost 80% false positive) leading to unnecessary biopsies and risk of surgery. Read More
Developing Rare Disease Products: Lessons from Serepta and Biomarin
Last month’s approval of Sarepta’s drug for Duchenne Muscular Dystrophy (DMD) by FDA shook the regulated industry due to the extremely arbitrary nature of the FDA decision. The review of the summary basis of approval, which lists the data FDA relied on to approve this drug, would not help. So, should all developers of products for rare diseases employ Sarepta’s strategy of extreme public campaign to pressure FDA? Read More
Does Screening Tests Save Life? Ben Stiller Creates Prostate News
Earlier this month, actor Ben Stiller announced that he was free of prostate cancer thanks to a routine PSA test he got. His essay encouraged all men to get PSA tests. However, the U.S. Preventive Services Task Force (USPSTF), a voluntary non-profit that makes recommendation for screening tests, does not recommend PSA tests due to very high false positive of such tests (almost 80% false positive) leading to unnecessary biopsies and risk of surgery. Read More
