Rare Disease Report

Legislation & Advocacy Efforts Needed in the Fight Against Sickle Cell Disease

OCTOBER 18, 2018
Krista Rossi
Over the past few years, many advances have been made in the fight against sickle cell disease, a group of inherited red blood cell disorders. Many of these advancements are thanks to the work being done by advocacy groups, such as the Sickle Cell Disease Association of America (SCDAA).

In a recent interview, Rare Disease Report ® sat down with the president and chief executive officer of the organization, Beverley Francis-Gibson, who highlighted the research presented at the 7th Annual Sickle Cell Disease Therapeutics Conference, which was held this year in New York. Francis-Gibson also discussed remaining challenges associated with the disease and the need for more efforts to promote awareness.

RareDR® : What do you feel were the highlights and take-home messages of your presentation?

Francis-Gibson: I talked about the SCDAA and my vision for the organization, our goals and agenda for the next year, and also the reason why I felt it was important to be in attendance for the Global Blood Therapeutics (GBT) event.

For my presentation, I discussed the fact that right now, there’s a lot of excitement around sickle cell disease, also withstanding the fact that September is Sickle Cell Awareness Month. But from the standpoint of SCDAA, my agenda is that we need to increase awareness of sickle cell disease and sickle cell trait here in the United States. We still have a vast number of families and individuals who don’t even know they may have the disease.

Secondly, I discussed the need for legislation and advocacy efforts by all of us—all of the attendees in the room—to ensure that there is legislation that supports families having access to care and legislation that supports reimbursements and getting treatment in a cost-effective manner. Thirdly, I talked about the need for partnerships and collaborations amongst all of us who are walking to address sickle cell.  

Were there any other presentations at the conference that you felt were exciting?

There were a number of pharmaceutical companies that shared information on drugs or pending clinical trials, and I thought that was pretty exciting because, again, it gives me and the attendees in the room an opportunity to hear about what’s happening in the pharmaceutical space.

It was great to hear Ted Love, who is the president of GBT talk about where GBT is in terms of their clinical trials, but also his philosophy for the company. I felt that we heard that throughout the presentations made by the other pharmaceutical companies as well.

Modus Therapeutics, a company out of Sweden, was the last one that presented [at the conference], and it was great to hear some of the international work that’s happening. And so, it was actually nice to have some international presenters as well as the US presenters.

The other highlight for me was also being in the room with our patients, many of whom are people living with sickle cell disease and many of whom represent our community-based organizations across the United States who are also members of SCDAA. It’s great to meet many of them who I’ve spoken to over the phone or some of them who I had not met—it was really good to meet them for the first time.

What strides do you see being made in sickle cell disease research that providers and patients should know about?

I think there’s a lot of things happening in the pharmaceutical companies that will help sickle cell patients and their families. I like the fact that there’s also a lot of innovation that was being discussed in terms of models that were addressing the disease. The presentations by the National Heart, Lung, and Blood Institute and some of the other companies that talked about the developing therapeutics for sickle cell made it a very rich opportunity for me to learn and also hear about what’s coming down the pike.

What remaining challenges do you still see when it comes to sickle cell disease and how do you feel they can be addressed?

Well, a lot of what I spoke about when I talked about advocacy is that there were times when the senator and the legislators feel that there is not a lot of momentum behind sickle cell disease because the patients and their families aren’t showing up on the hill to advocate for their own wellbeing and care.

One of the things that we’ve talked about when I did my presentation was that we needed to show up, because other diseases show up, and there’s a misnomer or misunderstanding that there are over 100,000 people suffering and living with sickle cell disease in the United States because when it becomes an issue of advocacy, we don’t see those numbers showing in the way that they should. One of the things that I talked about was the need for patients, advocates, families, and companies to show up to represent the sickle cell communities and sickle cell families so that there is not this misconception that it is really not something people care about.

Can you discuss efforts to promote awareness for sickle cell disease?

Get connected as a patient to the registry that connects patients and researchers to care information. “Get Connected” is a platform that SCDAA launched in June, and, to date, we have over 6,000 patients and individuals in the registry. Patients can also log or document their disease history and medical care in this registry, so it gives them one place to do that.

The other is was our annual convention, which was October 10 to 13, 2018. Our theme was celebrating diversity within the sickle cell community, commitment, innovation, and practice. We had over 500 patients, providers, researchers, companies, advocates, who showed up in Baltimore for that event.

I have been all over the country this month in Sacramento, Texas, and New York, speaking about sickle cell disease and helping to raise awareness for the disease, as I mentioned previously.

There have been a lot of invitations and events in addition to a lot happening with the GBT conference.
There was the Access to Care Summit the day before, there were a number of events and walks that happened in September to commemorate sickle cell disease and to raise awareness about sickle cell disease around the country.

At SCDAA, for example, we had a couple of new partnerships that we announced, one with the National Red Cross to do a blood drive across the United States. We had a Twitter party or social media party with companies that we used to help raise awareness about the disease.

I think it’s important for people to just remember that there are families or individuals who are suffering silently and behind the scenes from a disease that is needlessly not getting the attention that it needs in our communities, and I appreciate your time and willingness to write about this in your article.            

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