June 20, 2017. In summary, there are questions relating the LEADER trial that raises questions about its generalizability. With only one study, however large, we don’t even know if the results would be replicated. The LEADER study suggests that the drug does not significantly increase cardiovascular risk. However, the sponsor has not demonstrated that it reduces cardiovascular risk, especially not for U.S. patients whose BMI, diet, and exercise habits might be quite different from those in other countries.
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NCHR Testimony to FDA About L-Glutamine for Sickle Cell Crisis
May 24, 2017. Sickle cell disease and the crises that it causes are a serious problem. Patients deserve and need new treatments. This requires high-quality clinical studies to demonstrate whether new treatments are effective and safe. When many of the patients that start a study drop out before it is completed, it is impossible to accurately evaluate the benefits compared to the risks. … FDA should approve new treatments based on clearly demonstrated evidence of efficacy and safety. This requires high-quality clinical trials where post patients stay in the trial. FDA should not approve a drug with questionable benefit when the dropout rate is concerning.
Read More »NCHR Testimony to FDA About Neratinib to Prevent Breast Cancer Reccurance
May 24, 2017. The FDA should be sure that new treatments provide a real benefit to patients before they are approved. We recommend that the FDA not approve neratinib [Nerlynx] for breast cancer unless a clear benefit can be replicated, or a benefit for overall survival is demonstrated.
Read More »NCHR Testimony at FDA Meeting about Duchenne Muscular Dystrophy Clinical Trial
May 18, 2017. Patients with Duchenne muscular dystrophy and their loved ones deserve the benefits of the most rigorous research available. At the same time, investing in a clinical trial requires significant time and effort, and patients ALWAYS face risks without any guarantee of benefits. Patients and families who are willing to participate in a clinical trial that has the potential to benefit many should be fully informed about the potential risks and benefits of their participation in order to make a decision that is best for them.
Read More »NCHR Testimony at the FDA about a Vaccine for Respiratory Syncytial Virus (RSV)
May 17, 2017. We strongly urge this Advisory Committee to prioritize patient safety and urge the FDA to establish high standards for preclinical and clinical studies. Like any public health strategy, a vaccine’s risks must be weighed against its benefits. Given all the mistrust of vaccines in a substantial minority of Americans, it is especially important to determine how effective the vaccine is at preventing RSV and for how long. If the vaccine does not protect infants and young children through a vulnerable period of development or contributes to negative side effects, then the risks of this vaccine are too high. Given that there have been significant issues with past RSV vaccines, the FDA should ensure that they do not recommend this vaccine prematurely, especially for such a vulnerable population.
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