Clinical News Roundup: Women Significantly Benefit From OB/GYN Trials

By Ed Miseta, Chief Editor, Clinical Leader

Women who participate in obstetrics and gynecology clinical trials experience improved health outcomes compared to those who are not involved in trials. Those were the results of research conducted by Queen Mary University of London in England. The researchers found this result to be the case regardless of whether or not the trial's treatment was found to be effective.

The researchers conducted a review of 21 studies that focused on pregnancy and reproductive health. The review included 20,160 women and found that clinical trial participants had 25 percent better odds of improved health outcomes compared with non-participants. In addition, the study demonstrated the beneficial effect of participating in a trial was greatest when it was a high-quality clinical trial, and when the trial tested an intervention that was unavailable outside of the trial. Researchers hope the results will lead to an increase in clinicians offering trials to their patients as well as an increase in women volunteering to participate in trials.

According to the study, research on the effect of participation in trials has not previously shown consistent evidence of benefit. This may be because previous work analyzed data from all medical specialties as one group. The result could include wide variations in the way care is given within and outside trials.

Sanofi Partners With Science 37 On Patient Recruitment

Today there are more than 40,000 clinical studies recruiting patients in the U.S. alone. Some of these trials will require thousands of patients, each of whom must meet precise criteria to join. Therefore, it’s not surprising that a study by the Center for Information and Study on Clinical Research Participation (CISCRP) found 80 percent of these studies to be delayed due to recruitment problems. This is a growing challenge, especially for treatments that target highly specific conditions affecting narrower patient populations.

To tackle this growing problem, Sanofi is applying a digital approach to clinical trials and partnering with Science 37, a clinical research services and technology company based in California. Leveraging mobile technology and telemedicine capabilities, this new approach will allow Sanofi to develop “site-less” or decentralized clinical trials. These studies are geared to be more patient friendly - easier for patients to access and eliminate many of the common impediments to participation. Using digital technologies to streamline finding and retaining participants for the entire length of a study has the potential to reduce the time required for a typical trial by at least 30 percent, according Science 37.

Going digital also eliminates a number of other hurdles to patient participation, including the most significant: Geography. Most people are eager to participate in relevant trials – 87 percent of patients want to do so, according to the CISCRP study. Yet, 70 percent of potential participants live more than two hours away from the nearest study center. Most clinical trials require patients to travel to those centers for regular tests and observations. This distance is a challenge to patient access.

Do Better Benefit Explanations Lead To Increased Recruitment?

Potential clinical trial subjects will be far more likely to participate if they are convinced doing so will benefit their overall health, says Jennifer Byrne, CEO of PMG Research. However, she stresses there must be action behind the words explaining trial-related concepts.

“Broadening the total value proposition of clinical research needs to go beyond buzzwords,” says Byrne. “Under the old paradigm, patients were enticed by the idea of giving back to society or gaining access to otherwise unattainable mainstream healthcare. But these days, that is not enough.”

Less than one percent of the general population participates in clinical trials. But surprisingly, 72 percent say they would participate if their doctor recommended it. A QuintilesIMS report on clinical research participation as a care option found patient and physician awareness of clinical trials to be hindered by the complexity of the healthcare system and the lack of an integrated approach.

Byrne has seen, anecdotally, that demonstrating clinical research can be part of a patient’s complete continuum of care and guides potential participants to the next level. For example, where possible, patients should be educated with a clear sense of how participating in a clinical trial can improve their own medical outcome and reduce their total cost of care. Byrne notes it can be a catalyst for behavior change.

Other bonuses of trial participation include higher patient satisfaction rates, lower trial costs, and recognition that patient care is moving toward a more active role for trial subjects. “My firm conviction is that clinical trials should be an equal opportunity choice with empowered patients,” says Byrne. “Let’s put clinical trials at the center of their [healthcare] journey.”

Are Digital Tools Increasing Patient Engagement?

By necessity, people living with a chronic condition are more familiar with the healthcare system than those who are not. A new poll from health IT company CDW Healthcare shows these same people are also more in tune with the technology available to improve their engagement with their health.

Building on last year’s efforts to examine what’s driving, influencing, and presenting barriers to the patient engagement space, CDW surveyed 200 patients and 200 providers (physicians and physician assistants) to assess the impact of digital tools and communication outreach efforts between the two. All patients surveyed were chronic, defined as having been to the doctor six or more times in the past year. Of this group, 70 percent said they have become more engaged with their care in the past two years, representing a 13 percent increase from 2016. In the past year, 74 percent of patients joined a patient portal (a nearly 30 percent jump from 2016), which resulted in 69 percent speaking with their healthcare provider more often and 69 percent of respondents accessing their healthcare information online.

Patients seem to embrace the idea that more frequent communication with their provider via digital tools could lead to improved health. If given the option, 64 percent of survey respondents said they would submit personal, real-time healthcare information to their provider, although that willingness to share online varies between ages.

Small Patient Groups Hamper Rare Disease Drug Development

For pharma companies seeking to develop drugs to treat rare diseases, the pricing of the drug is just one of the many challenges they face. Just as significant is how to manage the drug discovery and development process, which is more complex than for many mainstream drugs.

The development of any drug is an inherently risky business, requiring millions of dollars of investment and years of research and development. However, these risks can be even more extreme in the case of rare or “orphan” diseases, where inherently small numbers of patients make it harder to recruit particular subjects for clinical trials.

Because the patient populations are so small for each rare disease, trials become more intimate and drug companies are forced to work closely with their test groups and be more nimble when reacting to the needs of patients. Flemming Ornskov, chief executive of Shire, says trials for orphan drugs require a different approach and a longer-term perspective than for more common treatments. Shire is a rare disease drug specialist with more than two-thirds of its portfolio and three quarters of its new product pipeline dedicated to orphan diseases.

“If you look at the numbers, the trials are significantly smaller, but if you look at the time it takes to identify patients, get [them] to the treatment center, and the follow-up, which in many cases can be a decade, we are talking about a very significant effort,” he says. “The investment is of a different profile than if you did a short-period clinical trial for a well-known disease.”

Patient support groups, which raise awareness and represent the interests of sometimes no more than a handful of families, say that drug companies have made progress in recent years in terms of listening and responding to the needs of patients. The development of orphan drugs has also been helped by a more supportive regulatory environment, starting with the 1983 Orphan Drug Act in the U.S. that has enticed dozens of small biotech companies to develop treatments as well as some members of Big Pharma. The act sped up regulatory approval for orphan drugs and provided protection from potential competitor products for up to a decade.