eyeforpharma Philadelphia 2014

Apr 15, 2014 - Apr 16, 2014, Philadelphia

Make customer centricity work: smart pharma mindsets, models and technology that will seal commercial success

Qualified Sites the Biggest Issue for Rare Disease Trials

Setting up the right sites, in addition to recruitment and patient retention, makes trials of treatments for rare diseases challenging, a survey of clinical development decision-makers, conducted by Premier Research, has revealed.



Sixty-nine percent of respondents identified getting hold of or setting up qualified sites as the biggest challenge in starting trials of rare diseases and orphan drugs. Additional concerns were expressed over legislative complexity and constraints in access to appropriate patients.

“Much of the success in patient recruitment is based on finding the appropriate site for the small patient populations we have to work with,” Premier Research Executive Director, Clinical Trials Management, Angi Robinson said, noting that as the number of trials conducted outside of Western markets increases, the logistics of research becomes too complex to manage successfully.

The majority (88%) said the number of patients required to enroll in a rare disease and orphan drug study is reasonable, at the same time stressing the important role played by advocacy groups and medical networks in securing the right number of participants. The respondents pointed out that advocacy groups often needed more information about potential benefits to their organizations and the patients they represent before embarking upon cooperation with a trial recruiter.

Working with advocacy groups, which assist also in setting up appropriate sites, is more common in Europe than in North America, with 63% of Europe-based decision-makers interviewed for the purpose of this survey considering them ‘vital to their program’. Respective data for North America was not provided.

“Advocacy groups and medical networks have great potential to aid in patient recruitment and sponsors recognize the importance of working with contract research organizations that have those relationships,” Robinson said.

Nearly half (43%) of the respondents confessed finding approval for their study to be ‘difficult’ or ‘extremely challenging’, at the same time reporting that regulatory organizations have become more flexible in their advice during a post-Phase II meeting, while they remain rigid during data review and drug application process.

“There is a belief that regulators are fairly realistic in discussing clinical trial design when it comes to developing new treatments for rare diseases,” said Charli Sanders, Premier Research VP for Regulatory Affairs and Pediatric Consulting. “Experience in working with regulators can help to facilitate the process,” he added.

Premier Research, a contract research organization serving biotech, pharma and medical device corporations, interviewed fifty clinical development decision-makers in the US and Europe during February 2013.



eyeforpharma Philadelphia 2014

Apr 15, 2014 - Apr 16, 2014, Philadelphia

Make customer centricity work: smart pharma mindsets, models and technology that will seal commercial success