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.

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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.

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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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NCHR Comments to FDA Advisory Committee on Organ Care System-Lung

May 17, 2017. There is tremendous pressure to have more treatment options, but if the FDA approves OCS despite the lack of unbiased evidence of non-inferiority, many lives will be put at risk. Improving the studies – including following the clear advice of the FDA – will answer all the essential questions about whether OCS is non-inferior to cold storage, and for whom.

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