By Morrie Ruffin, ARM Foundation for Cell and Gene Medicine
Cell and gene therapies hold the potential to transform medicine; however, if patients are to gain access to these therapies, we must increase public awareness and understanding of how these therapies can benefit them. According to a review of recent research studies that measured U.S. public opinion related to gene medicine,1 the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.
The knowledge gap that exists around cell and gene medicine could delay achievement of our shared goal of accelerating the development of potentially curative therapies for a host of life-threatening diseases. One of the top priorities of the ARM Foundation for Cell and Gene Medicine is to increase public awareness and understanding of cell and gene medicine through education projects that highlight the clinical and societal benefits of gene medicine and the life-saving possibilities that these medical advances could bring.
In April 2016, the New England Journal of Medicine published an analysis of 17 public opinion polls. More than half of those surveyed said they had heard either a little (48 percent) or a lot (9 percent) about gene editing before participating in the survey; 42 percent said they had not heard about gene editing.2 A 2017 Science magazine article concluded there is a “broad mandate for public engagement.”3 Preliminary research conducted on behalf of the ARM Foundation for Cell and Gene Medicine indicates individuals do not yet see anything fundamentally wrong with gene medicine but are hesitant about its unknowns. People also tell us they are more likely to trust information about gene medicine from a nonprofit organization than the government or pharmaceutical or biotechnology companies.
These findings also mirror a 2014 study, published in Human Gene Therapy, which found that more than 50 percent of respondents said their number one concern was “not receiving all the information.” According to its authors, delivering effective, evidence-based communications on gene therapy, engaging the research and medical communities as well as the public in a conversation about the ethics of gene therapy, is a priority.4
Call For Clarity
According to our interviewers, research interviewees admit they are confused when people talk about gene therapy. Most struggle to understand the differences in terms, such as somatic and germline gene editing, and concede that a better understanding may affect their opinions of gene medicine. They indicate they would appreciate more information on how gene therapy may be used. One major opportunity for education is information and data suggesting that gene medicine may help treat nonheritable diseases and conditions.
The risk of public misperception is, of course, significant. Gene medicine is advancing at an unprecedented pace with the potential to benefit millions of patients and even the potential to ease the burden of incurable diseases. Public engagement and education, research transparency, and independent level setting based on the facts can help smooth the path to societal acceptance of gene medicine as a viable, nonthreatening treatment option. As we have learned from the research published in Science and other journals as well as our interview-based research, participants who can answer factual questions about gene editing and CRISPR are more in favor of gene therapy.
Moving Gene Medicine Into The Mainstream
The foundation recently launched a strategic communications program designed to generate awareness and support of gene medicine.
The program will inform the public of:
- Benefits and limitations of therapies;
- Potential risks;
- Key scientific, healthcare, and regulatory issues that affect the development and commercialization of advanced therapies; and
- Opportunities for involvement, engagement, and empowerment through information sharing and discussion.
We believe the research is urging us to develop jargon-free content that’s easy to access and includes compelling, clear illustrations that summarize the science.
The foundation will support the industry with:
- Messaging and recommended language that resonates with the general public as well as specific audiences;
- Science briefings for journalists, payers, and policy makers;
- Toolkits for patient advocacy groups and medical societies to use with members;
- Social media campaigns that engage stakeholders in conversations about gene medicine;
- Public service announcements;
- Conference and event presentations and handouts; and
- Special reports, such as white papers, on the promise of genetic therapeutics.
How Gene Therapy Leaders Can Become Involved
Industry and other relevant organizations are invited to become a part of this important education program.
The foundation seeks volunteers to:
- Serve on committees;
- Develop the best communications strategies and compelling, clear, and easy-to-remember messages that serve as the message gold standard for cell and gene therapy/regenerative medicine;
- Develop a glossary of terms;
- Develop webinars and online guidance for journalists;
- Develop information for policy makers;
- Engage peers so we can gain acceptance of terms and definitions quickly;
- Serve as the information and/or project champion within a company or organization;
- Share ongoing research and development news;
- Share market and opinion research insights, when possible, to inform foundation messaging and targeting;
- Share videos, infographics, illustrations, and other content information that can assist in educating the public;
- Vet information to ensure it is accurate;
- Be willing to be speakers and presenters;
- Be willing to contribute information, write or edit white papers, articles, and opinion pieces;
- Be willing to monitor public opinion, including local articles and social media, and provide guidance to foundation communications efforts;
- Identify opportunities to share information with the public;
- Assist with funding identified projects;
- Critique communications efforts so the foundation can continually improve its impact.
The foundation serves as the educational and information catalyst on issues fundamental to making gene and cell therapies, tissue-engineered products, and other regenerative medicine treatments available to patients. By examining, quantifying, clarifying, and informing stakeholders of the clinical and societal benefits of these therapies, as well as convening discussions to raise awareness about the sector’s progress and challenges, we can accelerate patient access to safe, efficacious, and potentially curative therapies.
Visit thearmfoundation.org or contact firstname.lastname@example.org for more information.
- Versta Research, February 2018.
- Blendon R, Gorski M, Benson J. The Public and the Gene-Editing Revolution, (April 14, 2016). The New England Journal of Medicine 374;15:1406-1411.
- Scheufele D, Xenos M, Howell E, et al. U.S. attitudes on human genome editing, (August 11, 2017). Science 357 (6351), 553-554.
- Robillard J, Roskams-Edris D, Kuzeljevic B, et al. Prevailing Public Perceptions of the Ethics of Gene Therapy, (August 2014). Human Gene Therapy 25:740–746.
About The Author:
Morrie Ruffin is co-founder of the Alliance for Regenerative Medicine (ARM) and co-founder, board member and executive director of its 501c3 affiliate, the ARM Foundation for Cell and Gene Medicine. Ruffin is also co-founder and managing partner of Adjuvant Partners, a business development and strategic advisory firm. He has held executive and business development positions at various organizations, including what is now the Biotechnology Innovation Organization (BIO). Earlier, Ruffin worked as a senior legislative assistant for a U.S. senator. He received his MA in international studies and economics from the Johns Hopkins School for Advanced International Studies and his BA from the University of Virginia.