NCHR Testimony at the FDA Panel on Ataluren

September 28, 2017. We agree with FDA scientists that the data today do not indicate significant benefit in randomized, double-blind, placebo-controlled trials, such as Study 7. Only after making many post-hoc changes did ataluren show it was effective for ADP patients, but this was not replicated in Study 20. As you know, those post-hoc manipulations do not provide clear evidence of efficacy. For both studies, 90% and 79% of patients were White. But the CDC reports that Hispanic males are disproportionately likely to have these conditions. It is essential that an adequate number of Hispanic males be analyzed to determine if they can benefit from a treatment such as ataluren.

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NCHR Testimony at a Senate Panel on ECT

September 26, 2017. The new ECT devices are just as potentially dangerous as the old ones, and they should be required to be proven safe and effective. Everyone in this room has the power to do something about this, to convince FDA that ECT devices should remain Class III and should be required to be proven safe and effective before they can be sold.

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NCHR Testimony at the FDA on the Use of Abilify for Treating Irritability in Children with Autism

September 12, 2017. We are particularly concerned about Abilify because of its well established serious side effects of tardive dyskinesia, tremor, muscle stiffness, and sudden cardiac death.  Other risks include nausea, insomnia, anxiety, weight gain, high blood sugar, and high cholesterol. Moreover, the impact on a child’s developing brain is unknown because we lack high-quality, long-term research. The longer-term results indicated no significant difference between Abilify and placebo at week 16 in reducing the symptoms of irritability in pediatric patients who had already maintained a response for  the first 12 weeks of Abilify treatment. Clearly the evidence for the effectiveness and safety of Abilify for children with autism and symptoms of irritability is insufficient to outweigh the risks, especially for girls and nonwhite children. We urge the FDA to revise the labeling to state that Abilify has not been proven effective in the long-term for this indication.

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NCHR Testimony at the FDA on Shingles Vaccine, Shingrix

September 13, 2017. An effective shingles vaccine is important for public health. As patients get older, they are more likely to develop long-term pain, or post-herpetic neuralgia (PHN), as a complication of shingles. This pain can be severe and chronic. There is no cure, and treatments do not reliably relieve pain for all patients. The only way to reduce the risk of developing shingles and PHN is to get vaccinated. We urge this Advisory Committee to recommend that the FDA require critical post-approval long-term studies to further evaluate the efficacy and safety of Shingrix. We also strongly recommend that the company conduct subgroup analyses to ensure that the vaccine is safe and effective for both women and men and also people of color.

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