Medpace Voice

How Rare Diseases Are Showing the Way Toward Precision Medicine – Part 3

In our previous post, we discussed the approaches to help you identify, enroll and retain rare disease populations in clinical trials. Once the right patients have been enrolled in the trial, the challenge becomes measuring the efficacy of the experimental treatment and determining whether it’s producing the desired effect. However, invasive drug delivery methods, such as IV injection, can complicate this measurement and establishing whether the therapy actually reaches the target tissue can be difficult.

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How to Identify, Enroll, and Retain Rare Disease Populations in Clinical Trials

Enrolling enough subjects into a rare disease study can seem like an insurmountable challenge, especially when you’re looking at a subset of a rare disease, or an ultra-rare disease population. By mapping out your path, you can mitigate many potential issues. There are two key concepts: A patient-centric approach and referral networks.

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How Rare Diseases Are Showing the Way Toward Precision Medicine – Part 1

In Part 1 of a 3 part blog series, we will explore how the heritability of neurological diseases and psychiatric diseases is changing the clinical development landscape. From there, we will take a look at new genetic findings in a number of different diseases and how these findings are making us rethink our broad view of how we ought to approach these diseases. Finally, we will explore the history of treatment of rare diseases with CNS manifestations, touching on both successes and failures.

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How important is patient-centricity in rare disease studies?

Patient-centricity is becoming increasingly important in all areas, but particularly in rare diseases. In this post, Dr. Richard Scheyer discusses the importance of patient-centricity in rare disease studies.

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How to Leverage Research Networks to Accelerate Orphan Drug Development

In this post, Dr. Terence Eagleton highlights the growing interest in the development of products in the rare disease space, the unique challenges involved, and strategies for leveraging rare disease research networks to accelerate drug development.

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