In recent years, the healthcare industry has experienced a notable shift, with patients increasingly advocating for their rights while seeking collaboration with pharmaceutical companies.1 This evolving landscape represents a growing opportunity for pharma companies to consider and respond to the expanding emphasis on patient advocacy. Let’s delve into this transformation and opportunity by examining the role of patient advocacy groups (PAGs) in the rare disease (RD) space.
Why have PAGS become more important in the rare disease space?
Living with a RD comes with many obstacles, ranging from barriers to diagnosis to the complexities of integrating treatments into daily life.1 The COVID-19 pandemic highlighted and magnified the needs of RD patients, as it shed light on gaps within the global healthcare system. PAGs became a source of support and information for those living with RDs during the pandemic, allowing patients to find a sense of community and reassurance, despite facing limitations in their usual disease management options.2,3
RDs present complex and multifaceted challenges that are difficult to generalize, making them challenging to capture through conventional market research. Small population sizes in combination with limited travel availability can reduce the amount of research participants, therefore undermining the validity of results. Consequently, patients with RDs can feel misunderstood through traditional research methods.1,4
PAGs are therefore indispensable for patients with RDs, who find themselves at the intersection of a unique set of challenges that need to be understood accurately for meaningful change.3,5 These groups can help address the gaps that are missed through conventional research, by providing insights into the patient journey and the obstacles that they face at each stage.5,6 Their efforts have also historically contributed to the recognition and development of treatments for RDs.7
What do patient advocacy groups want to achieve in the rare disease space?
PAGs aspire to reduce the barriers that patients face in accessing care and support, by achieving broader advocacy and awareness. Given the limited prevalence of these conditions, the obligation of effective awareness-raising falls upon both PAGs and the pharmaceutical industry, as their collaboration could produce a greater influence than individuals or PAGs can achieve alone.3,5,6
In raising awareness of the RD patient journey and its complexities, PAGs are contributing to the paradigm shift towards patient-centred care. Although the specifics of this shift might look different depending on the therapeutic area, PAGs are generally trying to shape drug development research and disease management strategies to ensure that these align with individual needs and lifestyles.1,3,6
RD patients often encounter a lack of supportive resources due to the rarity of their conditions.1 PAGs work to rectify this issue through awareness campaigns and the production and distribution of educational materials.5,6 This lack of awareness also extends to the general public, where patients can struggle to find understanding from their peers.1,5 PAGs address the isolation that stems from small patient populations and geographical distances by creating community spaces where patients can connect with others who have had similar experiences.5,6
Beyond the individual needs of RD patients, PAGs seek to collaborate with pharmaceutical companies to drive changes that extend beyond treatment strategies and disease education. RD patient groups are often run by the family members of patients, meaning they can experience a lack of funding and access to key stakeholders – this is a key area in which pharma companies can support them, by helping with access to funding and stakeholders from their wider network of resources. This partnership also becomes particularly vital when advocating for systemic changes beyond the pharmaceutical realm, including the promotion of new laws and government policies.5, 6, 8
What does this mean for the pharma industry in rare diseases?
Through engagement with PAGs, pharmaceutical companies have the opportunity to cultivate strong relationships with patient communities, by amplifying the collective voice of patients and creating a collaborative environment.1,6,7
Patient advocacy also provides pharma companies with insight into the intricate and multifaceted nature of the RD patient journey. This gives them the chance to gain an intimate yet comprehensive understanding of complex patient journeys, which can illuminate where RD patients encounter unique barriers.3 In doing so, the role of patients can be transformed from passive recipients to engaged collaborators. Their insight can inform the creation of materials and programs tailored to address their unique barriers, thereby providing essential support while fostering trust.6,7
At TVF, we have collaborated with PAGs to develop patient support materials, allowing us to gain insight into RD patient experiences and the work that PAGs do to represent them. Through these experiences we’ve been able to examine the RD space and explore the need for PAG advocacy, as well as the progress that has been made by encouraging tailored solutions.
In a later article in this series on patient-centred-care, we'll be looking at how the pharma industry can effectively collaborate with patient organisations and the potential benefits that this holds for both groups.
By Carolina Zuluaga-Prola
References
1 - https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5702559/
2 - https://www.patient-view.com/2021/how-are-patient-groups-shaping-the-future-of-healthcare/
3 - https://meridian.allenpress.com/aph/article/21/2/35/483290/What-Rare-Disease-Patient-Advocacy-Groups-Are
4 - https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3964003/
5 - https://www.rarebeacon.org/rare-diseases/why-patient-groups-matter/
6 - https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10184204/
7 - https://www.jmcp.org/doi/full/10.18553/jmcp.2017.23.10.1078
8 - https://rarediseases.org/policy-issues/newborn-screening/
