A bit of background: I’m a rare disease patient living with generalized myasthenia gravis (gMG). I have been on numerous treatments, however, I am still searching for the one which will provide me the best “quality of life.”
I sit on a number of patient advisory councils in the MG community. I’m also a patient advisor for a number of technology companies in the rare disease space. My background affords me a unique perspective on the intersection of technology and rare disease: I worked at Apple for almost 20 years, and headed up global sales for a life science consulting firm that leveraged applied technologies to reduce time to outcomes.
One of the areas that needs to be highlighted more is the importance of early access programs (EAPs). These programs give patients with serious diseases and limited treatment options access to drugs that are still being researched and not yet available on the market. Many patients know very little about these programs and how important they can be, not only for patients but also for pharmaceutical companies.
I believe it is imperative to improve the awareness of these programs in the patient community. Education is key to help patients understand the “why’s.” The better the EAP community can educate the patient community through existing channels such as support groups, the more the likelihood of success increases.
I was invited to speak at an the Compassionate Use & Preapproval Access (CUPA) conference in Boston in January of 2026. It was both an education for myself and for the audience, which was made up of academic researchers and physicians. I knew nothing about expanded access programs prior to becoming involved with Bionical Emas, a group that helps manage EAPs. They educated me as to the importance of these programs in rare and ultra-rare disease.
Most patents do not know these programs exist. And in rare disease, we need to do a better job extending the reach of these programs beyond the standard communications and conferences.
One suggestion is to connect with patients where they seek help — through support groups, and as importantly, through storytelling just like this article. Another is the use of search tools, which can summarize the content through social feeds much like we see when performing a search in a browser, with a summary at the top of the screen and then the opportunity to read the complete article.
These are new approaches many technology and pharmaceutical companies are beginning to employ for patient education and research. This really puts the patient first.
Extended access programs and new tools which can incorporate leading-edge AI search tools will help educate the patient community. Working with companies like Bionical Emas and incorporating ways patients can learn about and contribute to these programs will enhance the ability for patient locate and learn.
Living with a rare disease is difficult enough; not knowing where to turn for quality information makes it more challenging! Breaking down the barriers and opening up more access is the goal.
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