MORRISTOWN, N.J., April 13, 2017 /PRNewswire/ -- Significant knowledge gaps about gene therapies persist, despite surging interest and accelerating clinical development, according to new research presented by SmithSolve, a strategic communications agency specializing in rare and genetic disease, this week during the 2017 Gene Therapy for Rare Diseases conference hosted by the New York Academy of Sciences. The findings were summarized in a poster titled, Enhancing Awareness and Understanding of Gene Therapy among Rare Disease Communities: A Research-Driven Roadmap.
Researchers conducted a multifaceted study that included an analysis of peer-reviewed literature, a series of industry expert interviews, a patient and caregiver survey, and a one-year audit of social and traditional media coverage and conversation about gene therapy. Among the more notable findings:
Intensification of industry R&D efforts and clinical trial activity for gene therapies have been accompanied by increased media coverage and a surging volume of discussion on social media.
Although there is considerable positive media coverage of gene therapy, stories often lack critical commentary or contextual information, potentially engendering unrealistic expectations about the promise of gene therapy.
Lingering safety concerns, the lack of an approved gene therapy in the U.S., and the newness of genetic testing technologies appear to be fueling physician misperceptions of the field.
There is a growing thirst for knowledge about gene therapies among individuals living with rare diseases and their caregivers.
"The gene therapy revolution is well underway, bringing with it a potentially massive shift in how we treat disease," said lead researcher Chris Smith, president and chief executive officer of SmithSolve. "Yet, despite the promise of gene therapy, our research identified a clear need for improved education and outlines a roadmap that all stakeholders can use to improve communication about this incredibly complex area of medicine."
Smith listed several opportunities to help bridge the informational gap and promote enhanced understanding of gene therapies. These include securing alignment among all stakeholders, establishing a common nomenclature, and fostering realistic expectations.
"Across dozens of rare genetic diseases, we are learning that early intervention leads to improved outcomes," noted Maria Escolar, MD, MS, director of the Program for the Study of Neurodevelopment in Rare Disorders at Children's Hospital of Pittsburgh, and associate professor of Pediatrics at the University of Pittsburgh School of Medicine. "This finding provides strong motivation for patients, advocates, physicians, research institutions, and industry to join together to improve awareness of gene therapies, so understanding keeps pace with research."
"We hope our research spurs the kind of dialogue and collaboration we recommend, and we look forward to facilitating these efforts," added co-author Patti Engel, RN, BSN, of Engage Health in Eagan, Minn.
The authors initiated this research to address the needs of rare disease patient communities and to fulfill Patient Advocacy Certificate Training (PACT) requirements of the Professional Patient Advocates in Life Sciences (PPALS). To learn more about the survey findings, visit http://smithsolve.com/results/ready-for-a-revolution/.
About SmithSolve SmithSolve, based in Morristown, N.J., is a strategic communications firm exclusively serving the healthcare industry. The company specializes in the rare disease and orphan drug sectors and helps established as well as early-stage biopharmaceutical companies tell their corporate narratives and translate complex science across multiple stakeholders. Its hands-on, senior-level team provides a broad range of communications services spanning product and pipeline news, patient advocacy, media relations, corporate reputation, issues management, digital design, and more. Established in 2006, SmithSolve partners with an established network of independent public relations agencies to design and execute programs worldwide. SmithSolve is a founding member of the Rare Collective™, a group of seasoned experts in orphan drug development (www.rarecollective.org). For more information, visit www.smithsolve.com.
About Engage Health, Inc. Engage Health, based in Eagan, Minn., is an experienced market development firm serving the pharmaceutical, biotechnology, and medical device sectors in the commercialization of specialty medical products. The staff at Engage Health brings over 20 years of experience in understanding and developing rare disease markets. From market quantification, patient and caregiver identification/mapping, to critical market research, Engage helps clients make the right decisions in development to maximize the commercial success of rare disease and specialty products. Engage is a founding member of the Rare Collective™, a group of seasoned experts in orphan drug development (www.rarecollective.org). For more information, visit www.engagehealth.com.