Amit Rakhit, MD, MBA

“Part of the research that we’ve done here is revealing that interviewing individual family members who take care of their loved one with Fragile X syndrome. What’s important for them?”

There are a number of rare genetic conditions faced by neurologists, clinicians, and health care practitioners which are severely limited in available treatments. Making this even more difficult is that they lack valuable and effective measurements to use in clinical trials.

This is the case in Fragile X syndrome, also known as Martin-Bell syndrome. To help address this, Ovid Therapeutics assessed what been done in previous trials of Fragile X syndrome to establish a Visual Analog Scale, incorporating behavior, cognition, speech and communication, sensory perception, emotions, social, activities of daily living (ADL), and notably, caregiver impacts.

To find out more about this scale and what prompted the incorporation of patient and caregiver centered facets into it, 

 spoke with Amit Rakhit, MD, MBA, chief medical officer, and head, Research & Development, Ovid Therapeutics, at the 2019 American Academy of Neurology (AAN) Annual Meeting in Philadelphia, Pennsylvania.

Rahkit additionally explored how this methodology could be adopted to develop similar scales in other conditions which lack an effective measurement tool.

Lee A, Cordeiro L, Marshall C, Wilson E, Visootsak J. Development of a Conceptual Model to Inform a Clinical Outcome Assessment Strategy in Adolescents and Young Adults with Fragile X Syndrome. Presented at: 2019 American Academy of Neurology Annual Meeting. May 4-10, 2019; Philadelphia, PA. Poster 067.

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The chief medical officer and head of Research & Development at Ovid Therapeutics spoke about what prompted the development of this scale to measure improvement for patients with Fragile X syndrome, and possibly other conditions.

Amit Rakhit, MD, MBA: Measuring Improvement in Fragile X Syndrome, Other Rare Conditions

“Part of the challenge in developing therapies for Fragile X syndrome is that there aren’t very standardized tools with which we can assess the impact a potential intervention can have.”

Fragile X syndrome, also known as Martin-Bell syndrome, is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment, which tends to impact boys more severely. Affected individuals usually have delayed development of speech and language by age 2.

A major obstacle in developing treatments for rare neurologic disorders and diseases like Fragile X is the lack of standardized measurements for these interventions, in part due to limited research. To help address this, Ovid Therapeutics assessed what been done in previous trials of Fragile X syndrome to establish a Visual Analog Scale, or VAS, to measure the impact of their candidate product. This conceptual model identified a number of symptom and impact concepts, categorized into 8 separate domains: behavior, cognition, speech and communication, sensory perception, emotions, social, activities of daily living (ADL), and caregiver impacts.

To find out more about this, how it incorporates the patient and physician perspectives, and how it can be used in the process of bringing an option to a condition with no approved therapies, 

The chief medical officer and head of Research & Development at Ovid Therapeutics spoke about the Visual Analog Scale that the company developed to help better understand the impact that potential treatments have on patients with Fragile X syndrome.

Amit Rakhit, MD, MBA

Articles

Amit Rakhit, MD, MBA: Measuring Improvement in Fragile X Syndrome, Other Rare Conditions

Amit Rakhit, MD, MBA: Developing Therapies for Fragile X Syndrome