Earlier this year Ilaria Di Resta, the Clinical Operations Leader at EspeRare foundation, provided CTA an excellent overview of the clinical development climate, saying the industry must seize the chance to develop new orphan drugs despite the inherent challenges.

Certainly in recent times, the pharmaceutical industry’s commitment toward developing orphan drugs has shown no sign of abating. In Evaluate Pharma’s 2014 Orphan Drug Report, orphan drugs accounted for 46 percent of the industry’s new drug output in 2013 compared to 35 percent the previous year. With more and more orphan drugs being developed, clinical trials are on the rise.

While that can only be described as a good thing, finding the right participants for a rare disease clinical trial could become even harder. According to Global Genes, 30 million people in the US live with a rare disease, equating to 10% of the US population. Similarly, in Europe there are approximately 30 million people living with a rare disease. Furthermore, it is estimated that 350 million people worldwide suffer from rare diseases. Considering the global population stands at around 7 billion, the pool for clinical trial participants is relatively small.

Generally, the patient recruitment landscape for clinical trials is constantly evolving with pharmaceutical companies and CROs alike all striving to find novel ways to recruit patients. However, when it comes to running clinical trials for rare diseases, recruiting patients appears to be the largest stumbling block.

According to a report by Beroe, 30 percent of Phase III trials fail due to enrolment challenges. The enduring problem with rare diseases is that they are all very different and each present their own singular challenges. The tactics sponsors or CROs deploy to recruit patients can often depend on whether the disease is acute or chronic.

A leading clinical expert, working for a pharmaceutical company in the US, said for clinical trials, his company’s tactics are determined by a number of factors, namely the type of study, the type of disease, the type of patient, and the type of site they use.

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By GlobalData

So they set up a separate clinical trials website, designed to attract patients to come to their site. Once online, they would be referred to an investigator conducting a clinical trial in their specific disease.

“We just did a study for a disease called myasthenia gravis and it worked,” the clinical expert said. “Out of the 80 patients we were studying, we probably got 15-20 people who were referred through that website.”

While in some cases that strategy was successful, for acute rare diseases, that approach proved less fruitful.

“When a patient has a chronic disease, they know they’ve got the disease, they’ll search for it on the Internet and stuff will happen,” he said. “With acute diseases, they’re in hospital before they know it and it’s too late. By that stage they’re not going to be referred to an investigator.”

To address that issue, the company follows patients, who they think are at risk of developing exacerbations of the disease, making them eligible for that clinical trial. The trial sites then develop a database of the patients to see if they meet the criteria for entry to the study. While the expert said his company is constantly adapting their patient recruitment methods for people with acute diseases, whether they prove successful remains to be seen.

Indeed navigating through the patient recruitment phase is only half the battle, especially for rare diseases. Retaining patients for orphan drug trials is another problem that dogs the industry. In a recent Forte Research study, 85 percent of clinical trials fail to retain enough patients, while the average dropout is 30 percent. Furthermore, the report noted patients drop out for a variety of reasons from financial constraints to being physically unable. So going forward more needs to be done. To maximise the chances of retaining patients, the study recommended sponsors and CROs:

  • Minimise burdens during protocol designs;
  • Set expectations upfront during the informed consent form (ICF) discussion;
  • Explain the importance of the patient’s participation;
  • Promptly respond to patient inquiries;
  • Send reminders for upcoming visits;
  • Provide a comfortable, friendly environment, and;
  • Accommodate their schedule as much as possible.

Source: Forte Research


1. Global Genes – http://bit.ly/1V0bzq1
2. Forte Research – http://bit.ly/1OjfolV
3. Beroe – http://bit.ly/1NBiHTR
4. Evaluate Pharma – http://bit.ly/1OCBcIc