The Birth of Modern Disease Advocacy
As the first ever paid public policy advocate for the myeloma community, I’ve long believed that “advocacy” (and “advocate”) is a misunderstood concept. It’s a malleable word meaning different things to different people.
That's a big reason why I’m writing these Substack articles: I believe they can have an important role to play in service to the myeloma community, and disease advocacy in general. I’ll save some more arcane views for later this summer.
But first we’ll step away from myeloma completely to understand how modern patient advocacy began.
Prior to the 1990s, patient advocacy for diseases was limited to a few. It was rarely assertive or proactive. Organizations like the American Cancer Society and the March of Dimes weren’t created to educate patients or give medical advice. They were about doing good works, raising money with events ranging from high-society soirees to elementary school walkathons, helping those less fortunate who were fighting diseases.
Whatever political and legislative attention Congress and presidents paid to cancer and the National Cancer Institute (NCI) was due in large part to Mary Lasker, a politically active DC-socialite who made fighting disease and cancer her causes. Lasker’s nonpartisan parlor room politicking, beginning in the 1940s, culminated in President Nixon’s 1971 “War on Cancer” initiative.
In Animal Farm-like language, it made the National Cancer Institute (NCI) “more equal” than the other parts of the National Institutes of Health (NIH). Lasker was the template for philanthropic patronage of disease-related charities for most of the 20th century.
Throughout most of history until then, doctor-patient relationships were generally authoritarian. Patients were consumers, expected to obediently defer to physicians’ expertise and experience. The idea of disease advocacy organizations sharing information for patients was rare.
By the mid-1980s, AIDS upended everything. Being labeled a “gay cancer” when the disease’s causes were unknown only exacerbated political demonization and public neglect. Societal mores at that time tolerated or minimized homophobia. It was “their” problem, not ours.
Gay and bisexual Americans across the nation had fears of dying accentuated by fears of living. Their world was unraveling; some gave up, inviting fatalism by engaging in risky behavior. But could demanding gay voices about a “gay disease” be effective when no one in power wanted to listen? Desperation inspired a new political militance with new strategies and tactics.
ACT UP (AIDS Coalition to Unleash Power) was founded as a decentralized movement to address a crisis that policymakers and an American majority wanted to ignore; some even saw it as some sort of divine retribution for the “immorality” of being gay. ACT UP had no precedents to emulate, but had to do many things simultaneously, each seemingly impossible.
Their activism demanded government, medical institutions, and the pharmaceutical industry develop multi-pronged plans with urgency and money. To raise public awareness about AIDS, activists engaged in high-profile sit-ins, blocking doors and walkways at the Food & Drug Administration (FDA) offices in Silver Spring, Maryland and NIH headquarters in nearby Bethesda while simultaneously working the formal corridors of power on Capitol Hill and in the White House.
As ACT UP was gaining success, a relatively unknown, bright young doctor, Anthony Fauci, would coordinate the effort to find effective medications and therapies at the National Institute of Allergy and Infectious Diseases (NIAID). Under his leadership, gay advocates were encouraged to learn and become part of the drug development process.
Now more than forty years later, although no cure for AIDS has yet been achieved, HIV infections leading to AIDS have, in large part, become chronic, treatable conditions.
By engaging people affected by a specific disease, Fauci, ACT UP, and the movement engaged in public awareness campaigns that developed into patient education and support. Advocates partnered with pharmaceutical companies, biotech, and academia to develop new therapeutic approaches.
The AIDS experience changed how disease and disability was perceived, giving birth to patient advocacy, a term encompassing many meanings today. At its core, it is about making patients part of the research and treatment processes of their own diseases, channeling desperation into action. Few examples other than ACT UP existed until then.
An emerging leadership of breast cancer activists took note of the AIDS experience, realizing a breast cancer diagnosis could, with nurturing, be translated into a political act of substantial, positive change. They had a unique opportunity before them with the confirmation of Taxol as an effective treatment for certain breast, ovarian, lung, and other malignancies and the need to get it to patients.
But they had to overcome two obstacles, one practical, the other requiring limber political and legislative gymnastics.
As effective as Taxol was, it couldn’t be created synthetically. Frustratingly, it could only be derived from the bark of the Pacific yew tree, a protected species on federally owned lands and habitats of endangered spotted owls.
Environmentalists wanted to protect ecosystems. Forest workers wanted jobs. And cancer patients were desperate for the newest effective drug.
Working with broad, non-medical coalitions and Congress, breast cancer advocates carved out compromises allowing limited harvests to produce adequate supplies of Taxol. After 1993, the drug could be synthesized agents not requiring the cutting of Pacific yews.
The other obstacle was an arcane rule limiting temporary congressional flexibility within funding legislation that limited research funds for NIH and NCI.
Advocates successfully lobbied to create a unique new Department of Defense (DoD) breast cancer research program. Women served in the military, some developed breast cancer. Therefore, it made sense for DoD to have its own research mechanism to enhance combat readiness. Or so the justifying logic went.
AIDS and breast cancer advocacy experiences formed a template of sorts for other disease advocates. Educating communities to influence public officials could change regulatory policies like drug approval and increase investment in research and drug reimbursements.
As the decade played out, disease advocacy groups started to spring up, enabled in large part by the emergence of the internet to propel a new advocacy movement. The language of patient education became less academic and, therefore, more politically accessible.
Physicians, medical institutions, professional societies, and the medical device and pharmaceutical industries began to understand that patient advocates could be much more than consumers—they could be partners: partners to spread their messages and act as surrogates for the audiences they wanted to sway.
Advocacy became something more than political venting or fundraising. It had potential to change research, public awareness, treatment advice, and most importantly, it created new communities of disease interests. Topics and programs like patient education, fundraising, and public awareness became more refined to and add to linkages between medical professionals and pharmaceutical companies.
Myeloma and advocacy have a history going back to the early 1990s. Although not directly linked to the AIDS or breast cancer stories, myeloma advocacy was unknowingly built on the models and paradigms they created.
In retrospect, it seemed orderly and natural. In reality, a mixture of luck (bad and opportunistically good), timing, and personal commitment ultimately mattered. And because of the progress in myeloma research and treatment, it’s undergone some fundamental change. Some lessons are instructive, others are cautionary.
The experiences of the myeloma constituency are simultaneously mirrors, cautionary tales, and beacons for many disease interests. Explaining why will keep us busy for the next couple of weeks.
We’ll even see how some clogged sewer pipes in Switzerland played an unexpected role in nurturing the community at a crucial time.
Photo: Detail, Martin Johnson Heade, Newburyport Meadows (ca. 1876-81), The Metropolitan Museum of Art, New York