Susan Stein, Connexion Healthcare Founder and CEO, led a workshop entitled “Industry—Understanding Their Motivation” at the 2015 Global Genes Rare Patient Advocacy Summit , which was held at the Hyatt Regency Resort & Spa in Huntington Beach, California, on September 25, 2015. The Summit, held annually since 2012, provides patients, caregivers, and advocates with information, introductions, and actionable next steps to build their rare disease community, effect legislation, raise funds, and engage in the development of new therapies. The workshops given at the Summit focus on specific aspects of each of these topics.
Stein began her workshop with a synopsis of the Orphan Drug Act of 1983, then went on to moderate a panel discussion, which was followed by a Q & A session with Alan Gilstrap, Executive Director – Advocacy & Policy at Akcea Therapeutics, and Joanne Vanak, RN, MSN, Senior Director of Scientific Advocacy at Janssen Scientific Affairs, LLC . The panel discussion revolved around the importance of continuing to develop relationships between patient advocate foundations and the pharmaceutical industry. These relationships are vitally necessary because both are involved in the clinical trial process, in sharing data, and in helping to define clinical endpoints, which are all critical to the drug development process. The panel opined that it is critical for industry and patient advocates to continue to foster relationships based on trust. Both panelists recommended in-person interaction and updates that adhere to regulatory and compliance guidelines to foster this relationship. Industry’s balance between business perspective and advocacy has been a delicate one. “In the last 5 years, there is definitely a noticeable shift … away from the public perception that [pharmaceutical companies] are solely driven by profits, and more to patient outcomes; pharmaceutical companies should be commended,” an anonymous attendee said during the breakout session. To ensure that this trend continues, advocates and industry professionals should maintain transparency and a climate of mutual respect while working toward common goals. Working in this manner requires flexibility, open-mindedness, and patience, Gilstrap explained.
The legislative tools are in place to expedite the approval process for orphan products. Patients with rare diseases have more say than ever and can gain a seat at the table by understanding both the pharmaceutical companies’ motivations and the restrictions under which industry must operate. Focused discussions by advocates through their respective foundations seem to result in the clearest and most impactful messages to industry.
About the Connexion Healthcare Rare Disease Center of Excellence
Connexion Healthcare is a full-service global provider of strategic medical and scientific communications, with decades of experience providing exceptional service to the pharmaceutical industry. We provide services through 2 Centers of Excellence—Oncology and Rare Disease—and possess core expertise in these therapeutic areas and their marketplaces.
The Connexion Healthcare Rare Disease Center of Excellence specializes in communicating the science behind rare diseases and orphan drugs. We strategically partner with industry leaders in the rare disease community to proactively connect all stakeholders in launching products to underserved patient populations. Our team comprises skilled communications experts who plan, develop, and disseminate scientific communications based on their advanced knowledge of the science, the patient journey, healthcare providers, and the orphan drug approval process.
At Connexion, our cross-functional teams navigate complicated treatment and market landscapes by elucidating the appropriate pathways to brand success. We understand how brand strategy drives market share. Since launching its dedicated Rare Disease Center of Excellence, Connexion has developed prelaunch, launch, and postlaunch communications in numerous rare diseases and disorders: congenital and genetic, movement, metabolic, and lysosomal storage disorders; lung, blood, skin, and endocrine diseases; and rare cancers.
For further information regarding the Rare Disease Center of Excellence at Connexion Healthcare and how we can develop rare disease communications to differentiate therapies by their unique attributes, contact:
Susan Stein, MPH
Rare Disease Center of Excellence