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Sickle cell disease is a complex and debilitating genetic disease associated with significant morbidity and early mortality. There has been significant underinvestment in innovation for patients, and change is long overdue. As part of its commitment to amplify perspectives from the sickle cell disease community, bluebird bio sponsored this interview with Mary Brown, president and CEO of the Sickle Cell Disease Foundation, to hear her reflections on 40 years of advocacy for the sickle cell community and opportunities and challenges during a period of unprecedented attention and investment.

Q: What is your vision for the sickle cell disease community? 
A: I want people with sickle cell to have the same opportunities as everyone else, and to be able to live full lives and manage and conquer their illness.

We’re not dreaming here — this is not something that’s pie in the sky! We already know that quality care enables families to see their young people develop into who they want to become and achieve their dreams while living with this disease.

But there are real barriers standing in the way of many people accessing that care — including lack of information for patients and families and lack of awareness in the medical community. That’s something other diseases haven’t struggled with as much, so we have work to do to close that gap.

Q: In your time as an advocate, what’s changed the most in sickle cell disease, and what has stayed the same?
A: I started as a volunteer because I believed there was not enough information or education in our community. That was true then, and unfortunately is still true today.

Patients and their families do not always receive the information they need to make decisions about their care. Medical students and healthcare providers are not always receiving the proper education and training to care for these patients — too often people must fight for the treatment they need when they visit the ER during a crisis. This fuels mistrust among patients and healthcare providers, which has been a long-term challenge and is complicated by racism and attitudes about sickle cell disease that persist today.

I’ve seen firsthand how education can make a difference, for individuals and within the system. While advocating in California I found that many policymakers were shocked by outcomes of people with sickle cell disease in the state. I heard over and over, “I just didn’t know.” That awareness led to funding and policies that are changing lives today. Knowledge does lead to real progress, and we have to keep opening the minds of people who have the authority to make those changes.

The biggest change I’ll point to is longer life for many people with sickle cell disease. I remember when life expectancy was 17-21 years of age. Better care and new therapies have increased that substantially. There is a lot of research now, including work toward a cure, but that only goes so far without awareness and education so that patients and families know about clinical trials or new therapies that could bring better quality of life.

Q: What will it take to make lasting change for people with sickle cell disease?
A: Consistency is the key to lasting change.

Consistent communication can address these challenges with information, education, and awareness. That leads to consistency in care — which we know is how people with sickle cell disease will have the best health and quality of life. We also need to ensure that there is consistency in insurance coverage. I see people who get so much better as they access specialized care, and they start to have more stability and can work — and then lose their insurance coverage and lose all that ground. It doesn’t have to be that way. Finally, consistency in funding, to sustain the work of community-based organizations that have been working for years to improve patients’ lives.

As more groups start to work with this community, the focus must stay on truly listening to what the community needs and understanding their challenges. In particular, it’s so important for young people to have a voice. As one of my community health workers says, “We need to have big ears and little mouths.” That is something that community-based organizations understand, and policymakers, companies, and researchers can learn, too.

This interview was edited for length and clarity. Ms. Brown was not compensated for her perspective and the views expressed are her own. Sickle Cell Disease Foundation previously received grant funding from bluebird bio.