Rare Diseases Archives

Clinical Development

Patient-Focused Clinical Trial Design for Rare Diseases – Part 2

July 11, 2018

In our previous post, we discussed how patient-focused clinical trial design and collaborative approaches to site enrollment can accelerate rare disease clinical research. Yet, as our presenters emphasized in the presentation, the only way to ensure that trials are being conducted in a truly-patient focused way is to hear directly from rare disease patients and their families. In this post we summarize the insights from Beth Woelfel Harvey, a parent of a child with Duchenne muscular dystrophy (DMD).

Clinical Development

Patient-Focused Clinical Trial Design for Rare Diseases – Part 1

June 13, 2018

A few months ago, Medpace and the Cincinnati Children’s Hospital Medical Center presented a webinar on the importance of patient-centric clinical research. Over the next several weeks, we will build off each topic that was addressed during the presentation. In this post, we take a look at what patient-centric clinical trial design looks like for these studies.

Clinical Development

Q&A with Dr. Richard Scheyer: Novel Approaches to Confirming CNS Penetration and Target Engagement

May 29, 2018

Richard Scheyer, MD, Vice President, Medical Affairs at Medpace answers some questions about his upcoming presentation about CNS-targeted therapies at DIA 2018.

Clinical Development

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

April 18, 2018

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.

Clinical Development

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

February 20, 2018

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.

Clinical Development

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

December 19, 2017

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.

Clinical Development

How important is patient-centricity in rare disease studies?

September 6, 2017

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.

Clinical Development

How to Leverage Research Networks to Accelerate Orphan Drug Development

August 7, 2017

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.