In order to conduct a clinical study successfully, research teams need to have a grasp of the challenges patients face—especially true when the condition at the center of a trial is a rare disease. The obstacles such participants may have to wrestle with include physical distance, site and work obligations, mental health concerns, and beyond.
During BIO International, a group of industry experts will discuss these considerations in the panel discussion Participant Sensitivity: Amplifying the Voice of the Patient in Clinical Trials on Monday, June 13. Outsourcing-Pharma spoke with Clincierge CEO Scott Gray, the moderator of the talk, to get a sneak peek at the session.
OSP: Could you please share your perspective on the importance of incorporating the patient voice?
SC: Patient recruitment and retention are among the most pressing challenges facing our industry. More than half of clinical trials are delayed by low enrollment, and only about 80% of patients remain in trials until the end.
When sponsors and clinical research organizations take a step back to consider the patient's experience in these trials – and put resources behind alleviating these burdens – patients are more likely to enroll and stay enrolled, the trials become more efficient and life-saving and life-enhancing therapies enter the market faster.
OSP: What are some of the challenges in reaching and engaging patients and gathering their feedback on conventional clinical studies?
SG: The logistics of participating in a clinical trial can take a heavy financial, mental, and even emotional toll on patients and their caregivers – often in addition to the stress that comes with dealing with chronic illness. Nearly half of patients enrolled in early-phase clinical trials face out-of-pocket costs of $1,000 or more every month. This is an enormous barrier for many patients and their families, and it is no wonder that people in households earning less than $50,000 per year are 27% less likely to participate in clinical trials.
While data integrity dictates anonymity between researchers and their subjects, Clincierge's patient coordinators are able to provide direct support to patients and their caregivers. This human touch makes all the difference. While some studies estimate industry-wide participant dropout as high as 30%, Clincierge maintains an average patient retention rate of 95%.
Patient support takes on many forms. It can be as simple as managing reimbursements more efficiently to more complex logistics involved with cross-border travel. For example, in a trial targeting a rare disease, an entire family may need help relocating to a remote trial site in another country. Their relocation might involve visa coordination, prepaid air travel, short or long-term housing, ground transportation, translation and interpretation services, and more.
OSP: Then, what new or exacerbated challenges might trial teams face when a rare or orphan disease is at the center?
SG: Though we now support trials of all sizes, Clincierge initially focused on trials for rare and ultra-rare diseases, which are often genetic and disproportionately impact children and young adults. It is not uncommon for families to have two or more children enrolled in the same study, and these families' only hope comes in the form of a clinical trial. However, the very essence of a rare disease often complicates these patients' ability to participate. In addition to a small eligible population, trial participants are frequently very ill or have cognitive or mobile impairments, and trial sites are often dispersed across the globe.
Running a successful clinical trial for orphan and rare diseases presents unique challenges. Still, if a patient facing a rare disease is fortunate enough to come across a trial targeting their condition, it is imperative we do everything in our power to help them overcome obstacles to participation.
OSP: Please give us a glimpse into what the panelists of your session will be discussing, including the patient/caregiver opinion study you’ll be sharing.
SG: I am looking forward to moderating a panel on the patient experience at the 2022 BIO International Convention. The session is called Participant Sensitivity: Amplifying the Voice of the Patient in Clinical Trials, and it is scheduled for Monday, June 13 at 1 pm PDT in Session Room 11A.
The plan is to explore how the patient experience impacts clinical trial outcomes and to provide practical strategies for overcoming barriers to participation. I will be joined by a diverse panel of industry experts, including David Harris of Insight and Measurement, Pam Cusick from Rare Patient Voice, and Elisabeth Selph from Ultragenyx Pharmaceutical.
Our discussion will be informed by a study commissioned by Clincierge examining the opinions and behaviors of patients and their caregivers. Data were collected in two parts over the course of a year. An initial qualitative phase used focus groups, interviews, and writing prompts to uncover patient and caregiver attitudes and determine how they affected their participation in previous or concurrent trials. A second quantitative phase looked at data collected via a survey of patients and caregivers. We are officially releasing our report on Tuesday, the second day of BIO, but we will offer early access to those who attend the panel discussion on Monday.
OSP: What insight and experience do each of the panelists offer on this important topic?
SG: David Harris has an extensive background in pharmaceutical research and conducted the study on our behalf with support from Pam's organization, Rare Patient Voice, which connects patients and caregivers to surveys and interviews. Pam is a long-time advocate for patients with rare diseases.
Elisabeth brings the perspective of a pharmaceutical trial sponsor. Her company, Ultragenyx Pharmaceutical, has been a Clincierge client for several years. She will speak about the role sponsors and researchers have in the patient experience and how patient-centricity impacts trial efficiency and commercialization at her organization.
OSP: Is there anything you hope attendees will come away from the session knowing/understanding that they might not have before?
SG: Everyone involved in clinical trials seems to agree on the importance of patient experience, but we are still figuring it out as an industry. In addition to some of the highlights of the study, we hope to share stories from actual patients that will leave an impression on the audience. We hope attendees will come away with a greater appreciation of how challenging clinical trial participation can be for rare disease patients and their caregivers, as well as some practical strategies for overcoming barriers to participation.
OSP: Do you have anything you would like to add?
SG: Please visit us in the exhibition hall at booth 2405! Readers can also learn more about Clincierge on our website. The results of our study will be available on Tuesday, June 14.
The BIO International Convention is scheduled June 13-16 at the San Diego Convention Center. Visit the show website for more information or to register.