Clinical News Roundup: What If You Set Up A Clinical Trial & Nobody Signs Up?

By Ed Miseta, Chief Editor, Clinical Leader

Recruiting patients to participate in a clinical trial remains a challenge. Many sites will underdeliver on their projected number of patients. Some sites will deliver none at all. This situation has an article on MedCityNews asking, “What if you set up a clinical trial and nobody signed up?” The question is not so far-fetched. Only 3 or 4 percent of oncology patients sign up for a trial, and 60 percent of oncology trials will enroll no more than 1 patient.

But two new clinical recruitment methods hope to increase recruitment rates by harnessing the power of Big Data and IT to streamline the process of matching patients with trials.

One is a smartphone app developed by Dr. Peter Elkin, a biomedical informatics professor at the University of Buffalo. The app was designed to be a sort of Tinder for clinical trial enrollment, helping patients find a trial in their geographic area that matches their health interests and needs. The app, called “Participants In Science,” will be available by the end of summer through Apple’s app store and GooglePlay. Once patients download the app, they can do a free keyword search to find clinical trials in their area focused on specific health conditions.  

Another approach, developed by Quintiles, also attempts to match patients with appropriate clinical trials. The oncology Precision Enrollment model scours electronic health records (EHRs) to find patients who are a perfect match for cancer trials. Quintiles will make use of its network of 80 cancer research sites. A site will only be opened if a patient meets the inclusion/exclusion criteria. All patient information mined from the EHRs is de-identified until appropriate patients are found.

3 Trials Target Triple Negative Breast Cancer

At the American Society for Clinical Oncology (ASCO) Annual Meeting 2016, researchers presented results of three clinical trials using new targeted therapies against triple-negative breast cancer (TNBC). An article on Science Daily reports each therapy uses a distinct strategy influenced by the immune system, and all three have real potential to extend the lives of women whose cancers have progressed after previous treatments.

By definition, TNBC lacks estrogen and progesterone receptors and the overactive HER2 that drive other forms of the disease. These three cancer drivers also present doctors with targets useful in controlling the disease, for example, tamoxifen and fulvestrant block a cancer's ability to drive its growth with estrogen. Because TNBC lacks a known primary driver, doctors have lacked targets for new treatments, instead largely depending on decades-old chemotherapies, surgeries, and radiation techniques. Now the first generation of targeted therapies for TNBC may have arrived.

"This is a disease in real need of a breakthrough. There's really not any drug specifically approved for TNBC," says Jennifer Diamond, MD, investigator at the University of Colorado Cancer Center, an author on all three studies.

Direct-To-Patient Recruitment Benefits Breast Cancer Initiative

A direct-to-patient-approach rapidly identified a large number of patients with metastatic breast cancer willing to share tumor and saliva samples and medical records to accelerate research, according to data presented at the ASCO Annual Meeting 2016.

Healio is reporting on the effort titled, “The Mestatic Breast Cancer Project,” which was a nationwide study that encouraged patients to share samples and records in an effort to hasten research. The researchers collaborated with patients and advocacy groups to develop a website allowing patients across the U.S. to participate. Enrolled patients received a saliva kit and were asked to return a saliva sample to extract germline DNA.

“An estimated 150,000 women and men in the U.S. are living with metastatic breast cancer,” says Nikhil Wagle, MD, assistant professor of medicine at Harvard Medical School, physician at Dana-Farber Cancer Institute, and associate member of the Broad Institute. “The median survival is about 3 years, and more than 40,000 people die annually, accounting for 7 percent of all U.S. cancer deaths. Although treatments are improving, metastatic breast cancer is currently not curable. In order to make advances in our understanding of metastatic breast cancer, we need to be able to study tumor specimens — ideally, tumor specimens that are linked to clinical information.”

FDA Issues Guidelines Covering EHR Data In Clinical Trials

Health IT Outcomes reports the FDA has issued guidelines on the use of EHR data in clinical trials. Although EHRs do not technically fall under the control of the FDA, the regulatory agency has issued new guidelines recommending proper use of protected data in FDA-regulated clinical trials.

The new guidance document is designed to give clarity to clinical investigators, research organizations, institutional review boards, and others on the use of EHR data in FDA-regulated clinical investigations. It addresses issues such as how to use EHRs as data sources in clinical studies, quality of EHR data, and ensuring EHRs meet the agency's requirements for record keeping and retention.

The goals of the guidance are to facilitate the use of EHRs in clinical investigations as well as to promote the interoperability of EHRs and electronic systems supporting those investigations. Because EHRs have gained such widespread acceptance and use, the FDA says it provides new opportunities to improve patient safety, increase clinical trial efficiency, and combine data from a range of sources.

Is NIH Abandoning Vital Clinical Research Centers?

STAT News reports the Clinical Research Center (CRC) program, one of the most successful research enterprises funded by the NIH, is dying. Even worse, its productive life is being cut short with virtually no discussion from the scientific community. David Nathan, director of one of the centers, questions the need to abandon them.

American hospitals and medical schools once carried out little or no clinical research. The NIH opened its flagship 200-bed CRC in 1953. Others were later established at major teaching hospitals around the country. At the program’s peak, the NIH was funding almost 90 CRCs.

“Over the years, CRCs have provided human laboratories where diseases are studied and new treatments are developed and tested,” says Nathan. “The CRCs include not only specialized inpatient beds and outpatient facilities, but a highly skilled group of research nurses, dietitians, research coordinators, technicians, and physicians who study patients with complex disorders in a research setting.”

Nathan states the decision by the NIH to cut funding came as a surprise, as none of the five recent outside reviews of NIH external programs suggested defunding the CRCs. Yet NIH leadership has ruled that funding the space and staff of the CRCs will no longer be permitted.

Nathan believes this defunding is likely to lead to “repurposing” of the beds for general use and abandonment of this resource for clinical research. He notes the loss of the trained clinical nurses will be particularly damaging.

Adaptive Trials May be Game Changer For Alzheimer’s Treatments

At a session during the BIO International Convention in San Francisco, panelists stated a new approach to clinical trials for Alzheimer’s disease therapies may be a gamechanger. Bloomberg BNA reports Simon Lovestone, professor of translational neuroscience at the University of Oxford, discussed adaptive clinical trials—in which multiple treatments are tested at the same time and the results analyzed continuously—whose goal is curing or treating Alzheimer’s by 2025.  

Lovestone discussed the European Prevention of Alzheimer's Dementia (EPAD) adaptive trial that is designed to accelerate proof of concept of an Alzheimer's treatment. “We will use different interventions in combination,” he says. “We will adapt the trial on the basis of an intermediate phenotype. We are trying, through an adaptive clinical trial, to pick the treatment that is the winner.”

Are Menstrual Cycles Keeping Women Out Of Clinical Trials?

An editorial appearing on the British Journal of Sports Medicine states scientists may avoid studying women’s bodies because of their menstrual cycle. The blog notes drug trials have historically been conducted solely on men, partially due to concerns of potentially damaging unborn fetuses. It notes women were also perceived as more physiologically variable, therefore using only male participants would allow for meaningful results with fewer participants and less funding.  

Since men were viewed as adequate proxies for women, the years of exclusion of female participants from research were considered inconsequential. However, it is now known that women can respond very differently to drug treatments than men. Evidence suggests that women are almost twice more likely to have an adverse reaction to a drug than their male counterparts, and 80 percent of drugs withdrawn from the market are due to unacceptable side effects in women.

The editorial notes that when research involving exercise metabolism does include women, participants are often tested in the early follicular phase of their menstrual cycle, when hormone levels are at their lowest, in order to minimize the possible impacts oestradiol and progesterone may have on study outcomes. This type of research practice leaves much ambiguity around how such hormones may influence the unique physiological processes in women, from blood pressure to substrate metabolism, thus perpetuating the significant gap in understanding how the menstrual cycle impacts exercise performance. Sex differences in the physiological response to exercise, likely caused in part by ovarian hormones, create a lack of understanding and a need for further research.