Patient registries on the up
is now being recognised, according to an industry expert.
A patient registry is a large, uncontrolled research study, observational in nature, designed to capture data on what is happening to patients when a drug is used in the "real world." They are carried out post marketing approval, as a regular Phase IV study, although they are run differently to a Phase IV trial as they are less exploratory and hypothetical in design. According to Jeffery Trotter, vice president of Icon Clinical Research's Lifecycle Sciences Group, the data gathered from observational studies is important because regular controlled trials may have excluded patients who have a whole range of factors or conditions that could interfere with the workings of an active ingredient. "Pharma companies are now realising the important addition that observational data can bring to scientific understanding of their drugs," Trotter told OutSourcing-Pharma.com. "In addition, the data gathered in such studies could help firms avoid or minimise the damage from potentially embarrassing and costly market recalls by discovering safety problems earlier." There have been a number of high-profile product withdrawals of late after the surfacing of "real-world" safety data, such as in the case of Vioxx, costing the drug manufacturers involved millions of dollars and causing them endless headaches. "Regulatory bodies are now also increasingly asking drug manufacturers for patient registries to be completed as they are now recognising the benefits of such data, whilst still accepting their limitations," said Trotter. "Other reasons companies are now choosing to run such trials include attempts to gain a new indication, or gain a commercial, marketing or reimbursement advantage through new cost-effectiveness, quality of life, efficacy, safety or tolerability data being accrued." However, because the concept of running observational studies is still in its infancy, there are still many challenges the industry faces when conducting such trials, he said. When it comes to designing the trial, Trotter believes that many sponsors and contract research organisations (CROs) make the mistake of employing the same methodology as they would towards a randomised control trial (RCT), however, this is unnecessary and can cause observational trials to be more complicated than they need to be, he said. "Patient registries may be able to use a different approach to the way the site is set up, recruitment and monitoring is carried out and the data collected, for example," said Trotter. "In this way they can often be done much more cost-and time-effectively than typical Phase IV trials." One example given by Trotter is that a lot of monitoring is required in Phase IV trials to make sure the site is adhering to the protocol but this is not necessary in patient registries because of their more relaxed nature. "The challenge of patient registries is actually to observe them without introducing any unnatural elements," said Trotter. "Obviously the data gathered in this type of trial introduces more bias but this is something that needs to be accepted and compensated for somehow." He suggested the use of specially-tailored statistical tools, which can clean the "noisy" data collected so that biases can be excluded and it makes more sense.
