What Mitochondrial Disease Patients Say Matters About Clinical Trials

A new clinical trial aims to be the first treatment for mitochondrial disease to be granted FDA approval. Whether or not the trial results in a landmark treatment, it is already establishing a milestone for the structure of future research. It appears to be the first clinical trial investigating and reporting what patients say draws them to or pushes them away from a clinical trial. Keep reading to learn more, or follow the original story at newswise.com.

Mitochondrial disease is the name given to a group of inherited conditions which all affect the energy production center of the cell. Because all human cells contain mitochondria, the people with mitochondrial disease may be affected in almost any body system. The most commonly affected parts of the body include the brain, muscles, heart, liver, nerves, eyes, ears, and kidneys. Symptoms and severity may be very different depending on the affected system. Common symptoms include poor growth, weakness and loss of muscle control, and developmental delays. Click here to learn more about mitochondrial disease.

The diversity of both conditions and symptoms under the umbrella of mitochondrial disease was one of the major factors that encouraged researchers to better understand patients. The study’s leader, Dr. Marni Falk said that the complexity of mitochondrial disease made it crucial to understand what patient’s identified as their most “prevalent and disabling symptoms.”

With this kind of information it will be more possible to design trials and conduct research that better serve patients and their concerns.

Researchers accomplished this by conducting a series of online surveys. An independent group supported by the NIH also conducted a similar survey.

Factors that made patients feel more comfortable with a study included drugs that they could administer to themselves, and daily treatment.

Patients also reported that they preferred to be guaranteed access to treatment and be included in the late-stage (phase 3 clinical trials) as well. Shorter travel distances were also preferred.

Researchers also found that if the drug was previously untested in human trials, patients were less likely to participate.

Another big factor was whether or not a patient would be required to cease their current treatment. Trials that required patients to progress through their disease were also voted against. Other unfavorable conditions included those which required daily, and invasive or uncomfortable medical procedures.

While the FDA certainly has strict requirements on what kind of results are necessary to approve a drug, not all those measurements have been fully explored by trial designers and researchers. Patient survival, of course, important to the FDA, says Dr. Falk, but so is the patient’s sense of well-being. How a patient feels can be equally important as how they function.

Dr. Falk continued to explain that clinical trials are often new experiences for patients. Having a study produce robust measures and be well designed are all important. That data is extremely valuable. Patient priorities, feelings, and needs, however, should be considered valuable as well. Dr. Falk views the ability for groups to form “strong partnerships among researchers, patients, and advocacy organizations” as the way forward.


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