There is a growing recognition that the world of rare disease is different and comes with unique challenges, but the body of knowledge around how to address those challenges is very much a work in progress. At rareLife solutions, we’re helping to build that knowledge base by sharing what we’ve learned from our work with patients, advocates, caregivers, healthcare professionals, treatment industry leaders, and beyond. We share our thinking with the community here and via email. To be added to our email list and get future resources directly in your inbox, please get in touch.
While it is accepted that patients, advocates, and caregivers (PACs) often have compelling personal experiences that may be appropriate for a lay publication, they are perceived as not having the appropriate expertise to assist in the development of medical manuscripts. Thus, PACs are considered not to be qualified as authors under International Committee of Medical Journal Editors (ICMJE) criteria. In recent years, there has been a push towards expanding the role of PACs (also referred to as patient and public involvement in medical literature; “patient” includes advocates and caregivers in this context) in medical research and publications on ethical, normative, and substantive grounds.
The new paradigm, as supported by the National Institute for Health Research in the UK and other similar funding agencies now includes patients and the public at every phase of the clinical program. This model was developed in response to PAC and public demands for greater transparency and engagement. This paradigm shift involves clinical research being conducted “in collaboration with” or “by” PACs and democratizes the research process since they, especially patients, ultimately take the risk either during the trial or later with the approved therapy.
Our most recent rareIQ explores this new paradigm, along with the potential benefits that patient, advocate, and caregiver involvement can have on the clinical research process, the implications of funding agency requirements around PAC involvement in research, and more.
Patients, advocates, and caregivers are Key Opinion Leaders in their rare disease space in their own right and also can be the key to unlocking nuanced insights and a compelling publication.
Dakota Fisher-Vance
Young Adult Cancer Connection co-founder, and patient