How the Pharmaceutical Industry Can Help Patient Advocates

I recently attended an amyloidosis scientific conference in Heidelberg, Germany, that was a productive and empowering experience. As a former caregiver to a spouse with hereditary ATTR amyloidosis and a patient advocate with New Zealand Amyloidosis Patients Association, learning about the advancements in the field of disease treatment is encouraging.

I hope my takeaways from the conference will benefit me as I review what I have learned and continue advocating for change in New Zealand. At the conference, which was organized by the International Society of Amyloidosis, various clinicians provided clinical data on the efficacy of breakthrough treatments for ATTR, such as Onpattro (patisiran) and Tegsedi (inotersen), and others that use the gene-editing technology CRISPR-Cas9. The results overall were very positive.

New Zealand’s current policies and legislation do not cater to the needs of people with rare conditions such as amyloidosis. Treatment approval takes a long time, and the process to fund therapies takes even longer.

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Working together

One particular area I found fascinating was the pharmaceutical industry’s verbal commitment to assist patient advocates like me in achieving what we need to in our work. Representatives from the industry, such as Alnylam Pharmaceuticals, Pfizer, Eidos-BridgeBio, and Intellia Therapeutics, spoke about the importance of working together, which I found promising and reassuring.

I am confident that I wasn’t the only one who felt that way. Patient leaders from all over the world attended the event. I met terrific advocates from Brazil, Canada, France, Ireland, Italy, the Netherlands, Spain, Sweden, and the United Kingdom. We all have experience as caregivers or patients and have similar agendas: making funded treatment available and enabling a supportive care network for sufferers. We want to be the source of current and reliable information. We want to advocate through education and awareness campaigns, and by hosting conferences, leading research projects, and pushing for treatment approval by regulatory entities.

For that assistance to happen, though, I believe it is important to target individual advocacy groups. As a patient leader, a critical area I work in is applying for grants. Without financial assistance, a considerable amount of my work wouldn’t be as effective, and in some instances, it would be hindered by an inability to manage awareness projects. So grants from pharmaceutical companies whose research focuses on an area of our expertise become vitally important to our work.

This isn’t a situation in which pharmaceutical companies are “buying us off,” as some might suggest. For rare disorder groups like ours, we are often volunteers who take time out of our daily lives to spread awareness because we have a personal stake in it. We aren’t taking a salary from these companies. We’re just trying hard to make a positive change.

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I genuinely hope that those who are committed to helping us can make the application and approval process for funding simpler. Currently, the process for some funding applications is tedious, and approval can take a long time. I understand that these processes must be rigorous because they involve the transfer of funds, but in my experience, application portals can be made to be more straightforward.

Additionally, advocates have to transcribe complicated scientific materials into information that is easily understood by patients and caregivers. This can take a lot of time and effort, such as when materials are in English and the patients only speak and read Spanish.

The famous Greek physician Hippocrates said, “Wherever the art of medicine is loved, there is also a love of humanity.” This aptly describes the conference’s upshot — that we are creating a space for medicine and humanity to come together to create miracles.

Note: FAP News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of FAP News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.