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  • Writer's pictureLighthouse
    • 5 min read

The Value of Molecularly Defined Patient Advocacy Groups

Updated: Mar 8, 2023

Summary in Thirty Seconds

  • Patient advocacy groups (PAGs) provide support, education, advocacy, and information sharing while seeking to include the patient perspective in all aspects of direct patient care as well as in treatment research and development and healthcare policy decisions.

  • PAGs can help newly diagnosed people navigate the very foreign landscape of cancer treatment that is usually marked with fear and uncertainty, helping to provide hope, community, and guidance.

  • In cancer, PAGs typically form around specific tumor types or around specific genetic mutations/variants.

  • PAGs clustered around specific genetic variants advocate for biomarker testing early and consistently for people with cancer, not just for diagnosis but also for targeted treatment leading to improvements in survival and quality of life, resistance tracking, and research to develop more effective treatments.

  • It is crucial that drug development teams, academic and clinical leaders, and others involved in cancer care continue to support these mission-critical advocators and the vital role they play.

Patient Advocacy Groups in Cancer

People with cancer have a variety of needs, and a wide array of professionals and groups play critical roles in cancer care. Patient advocacy groups (PAGs) have been developed by patients, their families and friends, and other caregivers to provide support, education, advocacy, and information sharing.[1] PAGs seek to include the patient perspective in all aspects of direct patient care as well as in treatment research and development while expressing the values reflected by specific patient communities.[2]


In cancer, PAGs form in two sometimes overlapping ways: around specific tumor types and around specific genetic mutations/variants. In the past decade, PAGs formed around molecularly defined cancers have grown notably. A 2017 study by OncoKB reviewed genetic testing in all cancers and found more than 3,000 unique mutations, fusions, and copy number alterations in 418 cancer-associated genes.[3] Almost half of these genetic variations included potentially actionable mutations. Thus, the number of potential PAGs clustered around specific mutations/variants is huge.


PAGs in NSCLC

In the United States, non-small cell lung cancer (NSCLC) accounts for about 13% of all new cancer diagnoses and 22% of cancer deaths;[4] however, only 6.2% of the US National Cancer Institute budget for 2020 went to lung cancer research.[5] NSCLC is not a unitary disease; rather, it is a collection of subtypes, each driven by specific molecular mutations, with funding unevenly distributed between these subtypes.[6] NSCLC has historically led the cancer field in adopting novel technologies like biomarker testing (Next Generation Sequencing) and targeted therapies, providing a good example of leading trends in oncology.


PAGs clustered around specific genetic variants driving disease have worked to influence decisions about the funding for the subtypes of NSCLC research. Examples of these subtypes include those with mutations such as EGFR, ROS1, KRAS, and ALK genes, and even more specific subtypes within these variants. As an example, at least 70 mutations exist within the EGFR variant group alone;[7] thus, within EGFR-driven NSCLC, mutation-specific PAGs have formed which have become the central address for the mutations they handle. A few examples of these include the Exon 20 Group (pan-tumor EGFR exon 20 insertion mutations and HER2 exon 20 insertion mutations), the EGFR Resisters (NSCLC EGFR deletion 19 and EGFR L858R and compound/complex EGFR mutations), and Exon 18 EGFR Lung Cancer Support Group.


A survey of three Facebook-based PAGs for NSCLC in the UK[8] revealed almost 90% of respondents found mutation-specific support groups provided better support and placed higher values on group discussions than other support groups. Almost 80% of respondents who belonged to one of these three PAGs reported feeling better prepared and informed regarding their disease and care, and half felt being part of a PAG had helped with feelings of isolation.


PAGs and Biomarker Testing

A review of the websites of molecularly defined PAGs reveals a particular emphasis on advocating for biomarker testing early and consistently for people with cancer.[9] This emphasis is unsurprising, given that the people comprising these PAGs were identified through biomarker testing. However, the advocacy for this testing goes beyond diagnosis and includes its importance for targeted treatment leading to improvements in survival and quality of life, resistance tracking, and research to develop more effective treatments.[10]


PAGs and Treatment Development

Molecular-specific advocacy groups played little part in the arrival of first-generation targeted therapies. However, over the past decade, patients and PAGs have banded together to create online communities with names such as KRAS Kickers, ROS1ders, NTRKers, RET Positive, and ALK Positive. While these PAGs first provided support and education to people with molecularly identified cancers, many now also push to fund research for the development of new life-saving medicines.[11] One of their most important roles is helping to match patients with appropriate clinical trials and raising targeted therapy awareness.


PAGs and Research

PAGs have not only advocated for research; they have also helped serve as the primary research focus. As an example, Parikh et al.[12] studied the demographic, clinical, and environmental characteristics of ROS1-positive people. A ROS-1 PAG helped design the questionnaire used in the study and multiple PAGs were major contributors not just to outreach efforts to find study participants, but PAG members also served as subjects in the study. This study helped clinicians and researchers better understand the challenges that patients experience pre-, during- and post-diagnosis, and it provided a model for other studies to employ with molecularly specific cancer PAGs. A second example of PAG involvement in research is from Horn et al.[13] who surveyed patients in two EGFR PAGs to help identify the utility of two patient-reported outcome measures for use in future NSCLC clinical trials.


PAG Benefits

A 2017 review article[14] identified seven benefits of PAGs: “(1) meeting and befriending other people with the same rare disease and similar experiences; (2) learning about the disease and related treatments; (3) giving and receiving emotional support; (4) having a place to speak openly about the disease and one’s feelings; (5) learning coping skills; (6) feeling empowered and hopeful; and (7) advocating to improve healthcare for other rare disease patients.”


PAG Challenge

Importantly, many members of PAGs are engaged in ongoing disease treatment for the very condition for which they are advocating. In this context, Nasso et al.[15] quote a respondent in a survey of patient advocates, “Perhaps organizations could set the stage…by openly validating the toll that cancer itself takes, and acknowledge that advocacy takes energy and commitment, which may not always be possible to sustain in the face of ongoing treatment or other life complications.”


Help Navigating a Challenging Landscape

Most people come into their cancer diagnosis with little-to-no knowledge of the basics of the disease, let alone the specifics of molecularly-defined cancer subtypes—the complicated terminology, complex diagnostic and treatment options, clinical trial decisions, outcome statistics, etc. Molecularly defined PAGs can help these people navigate this very foreign landscape that is usually marked with fear and uncertainty, helping to provide hope, community, and guidance. Additionally, these groups work beyond the individual patient to push for more treatment research and molecular testing, provide education and awareness to healthcare providers and researchers, and advocate for healthcare policy decisions. It is crucial that drug development teams, academic and clinical leaders, and others involved in cancer care continue to support these mission-critical advocators and the vital role they play.

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