Clinical Trials Q&A: Improving the recruitment process by listening to patients

Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today's challenges and opportunities. The Catalyst welcomes guest contributors including patients, stakeholders, innovators and others to share their perspectives and point of view. Over the coming weeks and months, Clinical Trials Q&A, will highlight different aspects of the clinical trials process from the people and organizations on the front lines of bringing essential medicines to the patients that need them. Like our Conversations series, views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today. 

This is the third post in our Clinical Trials Q&A series with individuals and organizations on the front lines of bringing essential medicines to the patients that need them. Improving clinical trial recruitment and retention is essential to the timely availability of new medicines to the patients that need them. Yet, there are many practical barriers that need to be overcome. To learn more about what the International Cancer Advocacy Network (ICAN) is doing to help address this problem, we talked with Marcia K. Horn, J.D., who has been on the forefront of this issue with twenty years of in-the-trenches experience with direct patient navigation and clinical trials as President and CEO of the International Cancer Advocacy Network.

JOCELYN ULRICH: What feedback have you received from people who volunteered to participate in a clinical trial?

MARCIA K. HORN: The best feedback, of course, is from the most fortunate of our cancer patients who thank us and tell us that their lives were saved. And if not saved, then families and caregivers report that life extension took place and a higher quality of life was achieved. And for those patients who experienced disease progression while participating in a clinical trial, the feedback is invariably that they still believed that their enrollment in the clinical trial, based on the array of several options that they had considered with their oncology team, was, in retrospect, the best option for them.

One of our Stage IV patients, who was the subject of a Newsweek profile in 2011, has enjoyed six more years of complete remission because of a clinical trial of a breast cancer vaccine (bringing her total remission to ten years and counting).

Another patient, who was years ago battling a metastatic GI cancer, had been “written off” early on by her first oncology team in a major center and was told there was absolutely no hope for her. She has had no recurrence of the GI cancer after more than 10 years after receiving a drug that had not been broached as even in the realm of potential therapeutic options for her. Clinical trials had never been discussed with this patient until she sought out ICAN’s advocacy services. Another patient, battling melanoma, was similarly told how dire things were by her oncology team, and more than five years ago had the good fortune to enter a Phase I trial of a novel immunotherapy drug which ultimately was approved by the FDA. She just celebrated yet another set of scans showing the coveted “NED” status meaning “no evidence of disease.”

Virtually every patient, family member and caregiver we have dealt with says the same thing: that the peace of mind achieved after having entered a clinical trial underscores that their cancer journey has left no stone unturned in their battles for life. Of course, favorable patient outcomes are not at all certain, and not all patients receive a benefit by any means. We all know how few compounds actually receive FDA approval. Even though patients, when fully informed about a potential clinical trial understand that they may not achieve tumor regression once enrolled, they still want to help the research process benefit future patients. We educate our patients that it is important to remember that the main goal of clinical research is to create new knowledge.

JOCELYN ULRICH: What would you say to a potential volunteer that is afraid they won’t receive the same level of care in a clinical trial as what they receive under their doctor’s care?

MARCIA K. HORN: The most consistent feedback from our patients in clinical trials is that they are thrilled at the very high level of care they receive as trial participants. As a member of Banner Health Institutional Review Board (IRB) Oncology Panel, I meet monthly with outstanding surgical oncologists, medical oncologists, radiation oncologists, nurses, and pharmacists who are members of that IRB and who are passionate about the clinical trials process. I can see the extraordinary level of care in the clinical trials process which has brilliant clinicians who are principal investigators or co-investigators of clinical trials, all working in a system interacting with the hospital’s ace regulatory affairs team, pharmaceutical/biotech monitors, research nursing staff, and the internal checks and double-checks of a top notch research program.

JOCELYN ULRICH: What are the most frustrating things about working in the clinical trials recruitment and retention area?

MARCIA K. HORN: While dealing with patients and educating them about cancer clinical trials is continually uplifting for us and for patients, spouses/families, and caregivers—especially when an advanced patient achieves life extension or can be brought back from the precipice—there are still frustrating moments.

As Catalyst blog readers know, clinical trial patient recruitment remains a roadblock to trial completion. Some principal investigators and their study nurses are missing out on what should be one of the most important set of conversations that they can have—those initial pre-enrollment conversations with the patient and his or her family or caregiver which greatly impact whether the patient will ultimately sign the informed consent.

JOCELYN ULRICH: What do you think can be done to improve interactions between principal investigators and study teams and potential clinical trial participants?

MARCIA K. HORN: At ICAN, we are very interested in the issue of educating principal investigators and study teams to make sure that the PI’s interactions with potential clinical trials patients are characterized by a level of communication that answers all the questions of the patient and their families. Not only is this the key to recruitment, but it is the essence of patient retention once the patient enters the clinical trial.

We also find that some patients are not willing, and understandably so, to be poked and prodded and beholden to an unreasonably high number of clinic visits or travel to a very distant trial site. The whole prospect of entering the trial then becomes a heavy psychological burden, and it is hard for the patient and his family to contemplate remaining in the trial under those onerous circumstances. Additionally, many trial participants have travel costs and dislocation worries even if the trial drug, special testing, and additional physician visits are paid for, routine patient care costs are covered, and free flights and hotel accommodations are provided or arranged for—or their costs defrayed—near the trial site. It is physically stressful for a Stage IV patient to be “on the road” traveling thousands of miles away to a clinical trial in some instances. It is very sad when a patient, who is doing well in a clinical trial but can no longer tolerate the wear and tear of frequent cross-country flights, decides to withdraw from the trial.

For some of our patients who have limited options remaining in the standard arsenal, they worry that clinical trial participation is a big gamble. Some of these patients would like to know, prior to entering a trial, how well the trial is doing and if patients in the trial are getting benefits from the trial drug or intervention. At ICAN, we make sure that the patient understands that the clinical trial’s study team cannot possibly disclose such information. While this is frustrating for some patients, they come to understand quickly what the clinical trials process involves and what the ethical standards of clinical research entail.

JOCELYN ULRICH: What steps is ICAN taking to move the clinical trials process forward?

MARCIA K. HORN: 1)      ICAN has developed a revolutionary clinical trials search engine called Remission Coach® which is in the final stages of development. That system will tremendously help both patient recruitment and retention in clinical trials.

2)      We are very intent at ICAN to integrate the sophisticated patient voice and patient advocate voice onto the biopharmaceutical industry’s protocol review committees at the earliest stage of their work. Pharmaceutical and biotech companies will certainly save many millions of dollars if patient engagement happens early on. This is also the focus that I have working with fellow patient advocates at the DIA (Drug Information Association) Patient Engagement Community—advocating the actual insertion of the patient and patient advocate voice at every pivot point of the research continuum. If we can do a better job getting the patient voice and patient advocate voice heard earlier rather than later, the global oncology community will benefit from better clinical trials recruitment and retention stats.

3)      ICAN is working to improve recruitment and retention in clinical trials for underserved patient populations.

4)      We think it is critical to have far more trials to meet the need of patients battling rare cancers, and for rare sarcomas and appendix cancer in particular, we want to see additional clinical trials.

Marcia K. Horn, J.D., brings twenty years of in-the-trenches experience to direct patient navigation and clinical trials as President and CEO of the International Cancer Advocacy Network, a 501(c)(3) organization headquartered in Phoenix, Arizona. ICAN assists Stage IV cancer patients across the U.S. and abroad, seeking to improve patient outcomes and attain the highest achievable quality of life. ICAN has received Top Rated Health Care Nonprofit Awards for the last five years (Great Nonprofits), Gold Seal transparency (GuideStar), and its work has been highlighted in Newsweek, Pharmaceutical Executive magazine, Robert A. Nagourney, M.D.’s Outliving Cancer and Robert Byars, PhD’s Waging War on Cancer.

Marcia is a PCORI Ambassador and was a finalist for both the Phoenix Business Journal's Health Care Heroes Award and the Organization (2012) and the Nonprofit Executive's Director of the Year Award (2013). Marcia practiced commercial and appellate litigation for 16 years while holding leadership positions in major civic organizations. She is a graduate of Stanford University and Stanford Law School.