A legacy of advocacy and activism that continues to save lives

At the height of the AIDS epidemic, I came out as gay. 

I was 18 years old, living in San Francisco area and finally embracing who I was. 

But with the HIV virus ravaging the gay community, my new identity was synonymous with illness, death and fear. The government and medical systems were as much to blame for these stigmas as the virus itself. For the first few years of the crisis, those with power failed to intervene. The gay community was not deemed valuable. Not worth saving.

With Pride month coming to a close, I’ve been thinking back on my own coming out story and the extent to which my career, the healthcare industry, and especially the rare disease space, were influenced by the communities forged in response to the AIDS epidemic. 

In the face of widespread hate and discrimination, the queer community mobilized in the 1980s to form mutual aid networks to care and shelter one another. Outraged by an executive policy of silence while thousands of people perished, they harnessed their anger and frustration and banded together to form organizations that urged and worked with the Food and Drug Administration to provide faster and broader access to experimental therapies. 

And this movement was supported by allies at the time, including many women who were protesting for reproductive rights in the 1970s. What a powerful reflection decades later that human rights and access to healthcare remains ever important. 

HIV and AIDS activists were bold and fearless, and the impact of their efforts was extraordinary. They succeeded in driving reforms designed to urgently deliver treatments to patients with few other options and limited time. They spurred the development of the FDA’s Expanded Access Program, as well as accelerated approval programs for Investigational New Drugs. 

While we still have a long way to go to make clinical trials more equitable, organizations like ACT UP pushed for the inclusion of women, people of color and intravenous drug users in drug studies. They also pressed for the inclusion of patient voices in clinical trial design. They insisted that people living with disease be treated as partners, not test subjects. 

When it came time to choose my career path, I knew I wanted to help others. I believe everyone should have reason to hope. I found my calling in rare disease. There is similar work to be done in rare disease to ensure that drug development and regulatory approvals are rapid, that the voices of the rare community are at the table every step of the way, and that there is equitable access to information, clinical trials, innovation and hope. 

Although individually uncommon, collectively rare diseases affect a huge number of people — roughly 1 in 10 Americans, 30 million in the US, 400 million globally are affected by rare disease. Of those affected by rare diseases, 50% are children.

People living with rare disease often feel misunderstood and marginalized, because, in many cases, their care needs have never been met. An estimated 95% of all rare diseases still do not have a single indicated FDA-approved drug treatment.

The extraordinary efforts of HIV/AIDS advocates, the LGBTQ community and their allies, ushered in an accelerated approval pathway that is critical today to providing access to new, safe, and effective drugs to patients with serious and life-threatening diseases and conditions. Since its implementation in 1992, the program has facilitated expedited approval of treatments for many severe, incurable diseases, including various cancers and bacterial infections, HIV, multiple sclerosis, and sickle cell disease. 

As a nation, we depend today on the advancements brought about by people working together in the 1980s and 90s to change the norms of drug discovery and development. AIDS and HIV activists were a vanguard bringing representation, information, and urgently needed research to people in need. Their work saved lives and made medical science leap forward. 

As an industry, we have a moral imperative to learn from patient communities how to listen to people living with disease, act upon their experiences, and bring breakthroughs in medicine and scientific understanding. In doing so, we are building upon the foundation laid by AIDS and HIV advocates.

This Pride month, and every day, I reflect on the individuals whom we have lost, and their contributions. And I am honored to continue advancing their legacy as we look to ensure quality access to healthcare for everyone.