White Paper

A Novel Approach To Early Phase Studies For Advanced Therapeutics

Source: Worldwide Clinical Trials
10 Steps To Better Alzheimer’s Disease Research

A paradox inherent in any effort to develop a therapy targeting a rare disease—defined in the US as one afflicting fewer than 200,000 people and in the EU as one afflicting fewer than 5 in 10,000 people1,2—is that testing the efficacy of the therapy requires identifying and then engaging individuals with the disease in clinical trials. When a disease afflicts so few people, it can be very challenging to find and recruit afflicted individuals for a clinical trial— particularly when the disease is complex to manage. Sites that have an established pedigree in interventional clinical research may not have a sufficiency of individuals with the condition within their catchment area. Centers and large academic institutions—where rare disease trials are traditionally conducted and which have expertise in the clinical management of patients with rare disorders—may have neither the staffing nor the infrastructure appropriate for interventional clinical trials administered according to current good clinical practice (GCP) protocols.

While a Phase I clinical trial for any investigative product (IP) involving prototypical small molecules typically recruits healthy volunteers, trials involving advanced therapy medicinal products (ATMPs) generally cannot be evaluated in that population. The question of whether an ATMP targeting a rare disease will be safe and potentially effective for the population afflicted by the disease becomes the focus in clinical management. Thus, the ability to conduct studies with sufficient rigor, at centers with appropriate expertise, and with patients afflicted with the rare disease becomes a key operational challenge and a precursor to successful program completion.

This unique aspect of clinical research involving rare disease patients poses a number of novel questions about patient contact, pre-screening and screening, management, and logistics. This paper explores those questions, the challenges they create, and how they can be addressed. 

To learn more about Worldwide Clinical Trials, visit their Clinical Leader page.

VIEW THE WHITE PAPER!

Get unlimited access to:

Trend and Thought Leadership Articles
Case Studies & White Papers
Extensive Product Database
Members-Only Premium Content
Welcome Back! Please Log In to Continue. X

Enter your credentials below to log in. Not yet a member of Clinical Leader? Subscribe today.

Subscribe to Clinical Leader X

Please enter your email address and create a password to access the full content, Or log in to your account to continue.

or

Subscribe to Clinical Leader