Register now for the Rare Disease Day webinar to join the discussion with Medpace experts on successful approaches to achieve cellular and gene therapy approvals in rare diseases.
Read More
In this on-demand webinar, Medpace experts share the operational and regulatory challenges associated with pediatric rare disease clinical trials.
Read More
Medpace gathered experts in all areas of rare disease clinical research and created a three part webinar series. All three parts are now on-demand and available to watch.
Read More
In this three-part series of discussions with top experts in clinical development for rare disease and orphan indications, Medpace, along with industry partners, will address some of the key challenges, opportunities, and considerations for conducting research in this dynamic and complex area of research.
Read More
We are delighted to announce our participation in Rare Disease Day for another consecutive year. In addition to our internal activities, Medpace will be hosting a webinar on how advanced therapies are dramatically changing the way patients are treated for a wide variety of diseases – particularly for patients with rare diseases and orphan indications.
Read More
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).
Read More
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.
Read More
Richard Scheyer, MD, Vice President, Medical Affairs at Medpace answers some questions about his upcoming presentation about CNS-targeted therapies at DIA 2018.
Read More
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.
Read More
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.
Read More