The impact of parent advocacy groups, the Internet, and social networking on rare diseases: The IDEA League and IDEA League United Kingdom example

Abstract

Summary The development of the Internet and subsequent evolution of social networking has significantly changed the effectiveness of patient advocacy groups for rare diseases. The greatest degree of change has occurred at the patient level, with an increased ability of affected individuals to share experiences and support, and to raise public awareness. Other changes have occurred, not only in the way rare diseases are diagnosed, studied, and treated, but also in how they are addressed at the level of legislation and public policy. The International Dravet syndrome Epilepsy Action League (IDEA League) is the leading patient advocacy organization for Dravet syndrome and related genetic ion‐channel epilepsy disorders (hereafter referred to as Dravet syndrome or severe myoclonic epilepsy of infancy, SMEI). The IDEA League’s mission encompasses international support and outreach for patients and families, as well as collaboration with physicians, medical education, health care coordination, and research. The IDEA League is an excellent example of the impact of patient advocacy groups, the Internet, and social networking on the landscape of rare diseases.

Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery

“…Much has been written about the role of patient and disease advocates and foundations in drug discovery and development [16][17][18]. There are probably hundreds if not thousands of such rare disease organizations [19].…”

Genetic Information‐Seeking Behaviors and Knowledge among Family Members and Patients with Inherited Bone Marrow Failure Syndromes

“…The IBMFS are rare; patients and family members may struggle to identify local healthcare providers with sufficient experience with their disorder and may have few opportunities to interact directly with others affected with the same disorder. Indeed, online communities and resources have been identified as valuable sources of support for parents of children with FA (Zierhut & Bartels, 2012) and for those diagnosed with other rare genetic disorders (Black & Baker, 2011; Gundersen, 2011; Rivard & Mastel-Smith, 2014; Schaffer, Kuczynski, & Skinner, 2008). Each of the leading IBMFS family support organizations has a strong online presence including the Fanconi Anemia Research Fund, Inc. (founded in 1985; www.fanconi.org), Diamond Blackfan Anemia Foundation, Inc. (founded in 1994; www.dbafoundation.org), Shwachman-Diamond Syndrome Foundation (founded in 1994; www.shwachman-diamond.org) and Dyskeratosis Congenita Outreach, Inc. (founded in 2008; www.dcoutreach.org), and disseminating education and medical information are crucial elements of their missions.…”

Advocacy by Parents of Young Children With Special Needs: Activities, Processes, and Perceived Effectiveness

“…While many parents may focus on advocacy for their child only (Kalyanpur & Harry, 1999;Vincent, 2000), for some, the personal becomes political, as advocacy becomes activism (Zaretsky, 2004), and involves joining with other parents to transform systems and encourage more responsive services. At the macro level, parental advocacy can promote legislative changes, improved public awareness and community education, and more funding directed at disability services and research (Balcazar et al, 1996;Black & Baker, 2011). The authors hypothesize that as parents improve their knowledge and skills, the perceived effectiveness of their advocacy efforts may contribute to a developmental empowerment process that may ultimately promote positive outcomes, including coping and resilience.…”

“…Parents may gain access to information on disabilities and methods for approaching special education that can result in more strategic advocacy (Trainor, 2010). They may form social relationships with other parents to provide mutual support and reduce feelings of social isolation (Black & Baker, 2011). Parent groups can also provide specialized trainings and offer a chance to join with other parents for collective action (Madden, 1995).…”