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Key Questions to Ask When Considering Clinical Trials

We live in exciting times. It seems like almost every day, breakthrough scientific discoveries provide novel insights that could result in new and better ways to treat your children. These advances are clear signs of progress and reasons for optimism. Companies are leveraging these scientific insights to initiate clinical trials with novel therapeutics.

Families recognize the need to participate in these trials and are eager to do so. Many have reached out to ask for advice and what factors to consider when making this decision. Although study participation is a highly personal decision, there are basic factors that families should consider.

This blog will focus on one of the first questions. Specifically, what is the quality of the research that suggests the treatment will be safe and effective for individuals with Rett Syndrome?

The good news is that a top priority of the FDA is to assure safety of study participants. And safety is an even higher priority when studies enroll individuals who are typically too young and/or incapable of legally providing their own informed consent. Although there are always risks associated with participation in clinical trials, families can rest assured that there has been a thorough and independent assessment for safety if the trial has received FDA approval.

Assessing the quality of the efficacy data is more challenging. FDA approval does not provide assurance that the drug is likely to provide benefit. In fact, we do not yet know exactly what type of efficacy in animals is most important for predicting efficacy in humans with Rett Syndrome. This should not come as a surprise as even drug development professionals find this challenging – only 10% of all drugs that are advanced into FDA -approved clinical trials by pharmaceutical and biotechnology companies actually demonstrate benefit sufficient to be approved by FDA.

The recognition that promising results in animals were rarely translating into comparable benefits in humans prompted in depth reviews by top scientists and organizations such as the National Institute of Health and the Institute of Medicine. These reviews provided recommendations for how to improve quality and reproducibility of animal research, and to increase the ‘rigor and quality’ of the animal studies.

An important finding was that inadequate reporting of animal study results correlated with overstated reports of efficacy. As a consequence, detailed guidelines were developed to improve transparency and reliability of research in animals, and it was recommended that journals only publish research that complied with these guidelines.

Although it is difficult for non-scientists to assess quality, a simple question to ask is whether the efficacy results have been fully reported in a credible scientific journal. If so, this provides assurance that the results have been reviewed and approved by independent scientists.

Another important finding was that many failed clinical studies were based upon a single animal study in a single animal model. A second simple question to ask is whether the efficacy results have been replicated in an independent laboratory and in more than one animal model? While not a guarantee of scientific integrity, confidence is increased if the answer to both questions is “yes.”

What can families do when considering trial participation? Ask the sponsors and clinicians conducting the trial:

  1. If the animal research for the drug they are now testing in people has been published in a reputable peer-reviewed scientific journal
  2. Whether the study has been independently replicated
  3. Whether the study has been conducted in multiple animal models


Note that company press releases are not a substitute for scientific articles.  Press releases should be viewed as advertising and a means of increasing company valuation, boosting stock prices, and/or attracting investors.

Human trials based on a strong scientific foundation have the greatest potential to move the field forward. It’s critical for researchers – as well as families – to make sure that valuable resources and energy are utilized in the most productive way possible.

Future blogs will focus on issues families should consider when evaluating the clinical trial design.