Patient and Stakeholder Engagement

Premier Research’s New Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges

Rise of Patient Networks and Advocacy Groups Helps in Design of Clinical Trials As Well As Patient Recruitment

PHILADELPHIA, April 23, 2015 — A new survey of clinical trial decision makers commissioned by Premier Research reports that nearly two-thirds (64%) of respondents said that among the most difficult factors in recruiting for a rare disease or orphan drug clinical trial was not only finding patients to join and remain in trials, but identifying and setting up investigative sites for studies.

The difficulty in recruitment reported by Premier Research in its benchmark 2013 survey on rare diseases continues to be an issue for the majority of respondents.

For this survey, 50 clinical development decision makers in the U.S. and Europe were interviewed by an independent third-party research firm during March 2015.

Regulatory Approval More Difficult

Approximately three-quarters (74%) of respondents said that securing regulatory approval for study protocol and design of their most recent rare disease or orphan drug clinical trial was “extremely challenging,” “difficult,” or “somewhat difficult.” This is an increase in difficulty from two years ago, when the figure was 67%.

“The regulatory pathway has become more complex because so many more companies are entering the rare disease space and presenting data on certain diseases for the first time,” Premier Research’s Director, Regulatory Affairs, Nachiket Dave observed. “The sheer volume of applications is a challenge to regulators. In fact,” he added, “the number of orphan drug designation requests that the FDA reported receiving last year increased by 35 percent.”

Dave noted that there have not been a lot of data available on many of the rare diseases. So the EMA in Europe and FDA in the U.S. are adapting to this increased interest in trials for rare diseases and the development of orphan drugs.

Increase in Startups Slowing Regulatory Process

Premier Research Executive Director, Pediatrics and Rare Diseases, Angi Robinson said that although respondents are saying that the regulatory approval process has gotten more difficult in the past two years, she believes that regulators in the U.S. and Europe actually are seeking ways to be more supportive to sponsor companies.

Dave noted that regulators are bringing more experts on staff and permitting a smaller sample size for rare/orphan diseases trials. “The real challenge is that many of the sponsor companies that have entered the rare disease space are high-tech startups which lack expertise and internal resources in the regulatory process.”

Dave added: “Everything starts and ends with regulatory.”

“The regulatory bodies, including the clinical regulatory reviewers, recognize the urgency of getting the treatment out into the marketplace,” Dave explained. “I believe the ‘old school’ view of doing large trials is changing so the process can be expedited.” he said. “The clinicians at the regulatory agencies are weighing in more and looking to bring therapies to the patients in an efficient manner.”

Nine of ten (90%) respondents said the number of patients they are required to enroll in a rare disease or orphan drug study is reasonable. Dave noted that this finding is surprising given the inherently smaller universe of patients available to enter such a study — as well as the respondents’ perception of the difficulty in finding qualified sites and in recruiting patients.

Role of Advocacy Groups Grows

Survey respondents also indicated that advocacy groups and medical networks play a substantial role in the clinical development of rare disease and orphan drug compounds. In fact, 74% respondents said that access to patients via advocacy groups was either “priceless” or “valuable,” which compares to 68% in the 2013 benchmark survey.

More than half (56%) said that the use of advocacy groups was “successful” or “vital to the program” of rare disease and orphan drug trials.

“Advocacy groups are where the patients are,” Robinson pointed out. “The groups are key to understanding and identifying how the patients’ experience plays out and where patients are getting their information.

She noted that Premier Research also uses social media in its patient recruitment efforts. Social media generates awareness of studies and the need for patients, then helps drive patients to try specific websites where they can read more about a study, where it is being conducted, and determine if they are eligible to participate. This information is also posted on advocacy groups’ sites, where patients can then gain access to the data.

Robinson emphasized the importance of thoroughly grasping how patients matriculate through the healthcare system. “Our experience in this space is what gives us an advantage. We really analyze all of the different ways in which to identify patients, including understanding the patient pathway.”

Advocacy groups are used by half (50%) of respondents to assist with their site or patient recruitment, while medical networks are used by 58% of respondents.

Robinson note that advocacy groups are essential in building awareness of rare disease and orphan drug trials. In fact, respondents cited “awareness” as the most beneficial attribute an advocacy group can convey to a trial sponsor.

“Advocacy groups have become more willing in the past couple of years to work with sponsor companies in clinical trials. The level of rare disease research has grown tremendously in the past few years,” she pointed out.

“The advocacy groups’ sophistication has also grown,” Robinson said. “They have been embracing social media and are getting more savvy in leveraging digital communications.” She pointed out that 52% percent of respondents indicated access to patients via social media or electronic channels was “priceless” or “valuable,” a 12% increase from the 2013 benchmark survey.

“Advocacy groups provide access to patients,” Robinson noted, “making it much easier for all concerned.”

Big Picture Is Key to Approval Process

Robinson noted that Premier Research’s own engagement with advocacy groups was highly valued by sponsor companies. “We have access to the small patient populations required for rare disease and orphan drug research, and we have access to experienced site investigators. Of course, we have an extensive track record in managing trials through the highly complex regulatory process.”

She added that this complexity underscores why 78% of rare disease and orphan drug studies are outsourced to a contract research organization (CRO).

“We recognize how vital it is to see the big picture,” she said. “It’s not just finding a patient that’s important — it’s the entire process. You have to know how to manage the process every single step of the way to a successful conclusion.”

She noted that Premier Research thrives on helping biopharmaceutical and medical device companies transform life-changing ideas into reality. These challenges call for tight teamwork, nimble responses, and innovative thinking — and for finding the patients, knowing the sites, and having the confidence to solve problems.

Premier Research focuses on addressing unmet needs in the disciplines of CNS, analgesia, rare diseases, medical devices and diagnostics, and pediatric research.

About Premier Research

Premier Research is a leading contract research organization serving the needs of biotechnology, pharmaceutical and medical device companies worldwide. The company has a wealth of experience in the execution of global, regional, and local clinical development programs with a special focus on addressing unmet needs in such areas as analgesia, CNS, rare diseases, medical device and diagnostics, and pediatric research. Premier Research operates in 50 countries and employs more than 1,000 professionals, including a strong international network of clinical monitors and project managers, regulatory, data management, statistical, scientific, and medical experts. With its mission to improve productivity in clinical development, the company aligns itself with the mission of its customers to bring new medical treatments to patients promptly, accurately, and cost-effectively.