Anyone with an involvement or interest in clinical research is likely to be aware of the challenges sponsors face in getting their compounds approved by the FDA. The low rates at which drugs make it all the way through the research process is a favorite topic of blogs and journalistic outlets covering clinical research. Many of the drugs that fall by the wayside suffer that fate because of unfavorable efficacy data in the middle and later phases.1 Many others, though, fall victim to administrative or technical challenges – and these issues often arise not as a compound nears the finish line, but as early as the nascent stages of Phase I. Here are three obstacles that researchers commonly face early in Phase I, as well as some insight into how the right eClinical software can nip some of those obstacles in the bud.
Across all phases of research, challenges in patient recruitment are often accounted for by a lack of awareness of clinical trials and a skepticism of the tested compound’s efficacy compared to existing treatments. Recruitment challenges in Phase I, however, have their own additional causes. Perhaps the most notorious of these is the “guinea pig” fear: as the drug’s safety and tolerability profiles have not yet been established, potential subjects often worry it may pose a danger to their wellbeing. A lack of motivation is another common factor, as most Phase I trials involve healthy volunteers with no medical necessity for the tested compound. The exception to this, of course, is Phase I oncology trials, which enroll sick patients. But even here, patients with an advanced condition and limited time are often reluctant to try a treatment with undetermined efficacy.
Before dosing begins in a clinical trial, there is a period of negotiation dedicated to hammering out details related to administrative matters like site payments. As we discussed in a post last year, the time spent on negotiations with clinical research sites tends to decrease as research moves from one clinical phase to the next. It follows, then, that the negotiation period tends to be longest in Phase I. In fact, Phase I site negotiations often last more than twice as long as many sponsors prefer.2 These lengthy negotiation periods threaten to derail timelines even before research has begun.
As Phase I oncology trials enroll sick patients instead of healthy volunteers, eligibility criteria can be strict, and dropout rates are notoriously high. This places the protocols of these trials at particularly high risk of unexpected changes. Indeed, a study by the Tufts Center for the Study of Drug Development found patient recruitment struggles to be a common cause of protocol amendments, with many amendments aiming to change patient eligibility criteria.3 Outside of oncology, there is another risk: the Tufts study reported that most Phase I protocol amendments take place before patient enrollment begins,3 and therefore create delays in the nascent stages of a trial that may be difficult to recover from as research progresses.
A software solution to these obstacles
With these challenges lying in wait, researchers can benefit from entering their Phase I trials equipped with the tools necessary to address them. The right eClinical software can provide both direct and oblique solutions to early Phase I challenges. Medrio’s EDC makes it easy to perform mid-study changes, a feature that serves as a bulwark against protocol amendments. And the software has a user-friendly drag-and-drop interface that enables researchers to build studies in weeks instead of months, creating time savings that can offset any delays stemming from patient recruitment or site negotiations.
The outcomes of Phase I trials set the tone for the rest of the research process. The value of a strong start, then, is difficult to overstate. While these trials face considerable challenges in their early stages, robust and intuitive EDC like Medrio is available to mitigate those challenges.
1 Mills, Roger; Why Do Drugs in Development “Fail” in Clinical Trials?; Pharmaceutical Processing; December 2016
2 Glass, Lucas Miller; Speeding Negotiations with Investigator Sites; Applied Clinical Trials; 6 November 2015
3 Getz, Kenneth A.; Protocol Amendments: a Costly Solution; Applied Clinical Trials; 1 May 2011