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Let's Take a Walk

Labor Day weekend starts today. It's time to say so long sweet summer and hello to a busy fall. From Philly to Houston, Team Sick Cells will find itself across the map over the next couple of months as we gather more stories from the members of our sickle cell community for our Faces of SCD campaign. 

This month, we'll highlight conferences we're presenting at this fall, share information on the Institute for Clinical and Economic Review (ICER) and where you'll be able to find us this fall.

We'll have some News You Can Use, but first...

A Minute With Marqus

Health woes shook up Marqus' August but he didn't let it stop him from making progress. While each day may be a battle of its own, this month proved that the little things you do (even when you're hurting) will always add up in the end. 
Marqus kicking off conference season in Chicago with a smile

August was extremely busy. I started this month in fair condition, but this unfortunately changed a little later. I experienced low blood counts, my leg ulcers reopened and I had a very bad experience with a sickle crisis and epileptic seizures. This is the second time I experienced a seizure and a crisis at the same time. During that time, I was reminded that even though I’ve been taking good care of myself, sickle cell disease is very unpredictable. You can wake up, go to work or school and by lunch you can be in a full out crisis. The bed you wake up in will be just a memory for a few days while you receive the care that you need, but things will get better. 

Marqus gearing up for a photo shoot he participated in during August

The good news is, even with all that, I was still able to make progress. I participated in a photo shoot, attended a men’s conference and a few discussions about what sickle cell disease is and why we need more blood ⁠— specifically the important life saving aspects of blood and blood donations.  

 

Marqus visited Prairie State College for a SCD-related seminar

At one point, transfusions were our only treatment option and donations help to provide the blood needed for these treatments. It’s great to see the progress we’ve made as a community. I hope to have more months as busy as August (less the crises). Here’s to a fantastic, September. 

ICER and the Future of Accessible
SCD Treatments

The Institute of Clinical and Economic Research (ICER) is reviewing two sickle cell disease treatments. Read more below about how this review directly impacts the SCD community and how Sick Cells is involved. 
 

ICER is currently reviewing Novartis’ crizanlizumab and GBT’s voxelotor for the treatment of SCD. The goal is to assess the cost effectiveness and value of these new drugs - using health economics. ICER’s assessments may be used by payers (largely insurance companies) to make decisions that affect the price and coverage of treatment options. 

If this assessment is going to influence our community's access to treatment options, we believe the patient perspective must be at the forefront of the discussion. Sick Cells is working to ensure that our community's voices are heard during ICER’s assessment of SCD treatments.

See you soon, Detroit, Philly and Houston!

Detroit, Philadelphia, Houston — we're coming for you! We're happy to announce the three cities Sick Cells plans to visit for our #FacesOfSCD campaign.
 
Do you live in or near any of these cities? We'd love to meet you. Please come visit us, we'll be in our Sick Cells t-shirts collecting interviews from members of the community for our #FacesOfSCD campaign. View past interviews from Chicago, Indianapolis, D.C. and more here

Thank You for Your Donations

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Each month, Sick Cells highlights policy updates impacting the sickle cell and larger rare disease community. Why? It is Sick Cells' mission to elevate the voices of the sickle cell disease (SCD) community and their stories of resilience. In highlighting the grave disparities our community faces, we hope to influence decision-makers and propel change.

House Passes Newborn Screening Saves Lives Act: H.R. 2507/S. 2158 would reauthorize existing federal programs that provide assistance to states to improve and expand their newborn screening programs, support parent and provider education, ensure laboratory quality and effective surveillance. The current authorization expires on September 30, 2019.

The House passed the House version, H.R. 2507, by voice vote on July 24, 2019. The Senate will need to pass the bill next and then the House and Senate will need to conference to work out the differences in the two bills before it can be sent to the President to become law.

Sickle Cell Disease and Heritable Blood Disorders Treatment Act of 2018  
 
On June 19, the House passed their 2020 spending bill which included a request for $2 million in funding for the Public Health Approach to the Blood Disorders Program for sickle cell disease and to support the CDC sickle cell disease surveillance program. The Senate needs to pass their 2020 budget bill now. Once that occurs, Congress will create a final 2020 budget bill. 

Call your Senators and request that $2 million in funding for the sickle cell disease CDC surveillance program is included in the Senate 2020 funding bill. For more information on appropriations, check out our March newsletter that explains it all. 

Voices from the Community: Mark

Sick Cells met Mark in Central Park in NYC. Mark is a SCD advocate and explained to us that he began working within the community by "accident." While he hadn't ever planned to become a SCD advocate, the experiences he had within the community led him to become a warrior for the SCD warriors he works alongside. We're always so happy to get to know sickle cell advocates, caregivers, parents and friends. 
Mark in New York City at the Sickle Cell Thalassemia Patients Network's annual Walk for Sickle Cell
"I could see it's definitely people living with a chronic health condition that need more help than they're getting. They need coordinated care and there's a lot of barriers in the healthcare system and with pain management and emergency rooms." - Mark, sickle cell advocate 

Upcoming 2019 Conferences

Rare Across America. July 29-September 8. Rare Disease Legislative Advocates (RDLA) staff organizes meetings for rare disease advocates with their Members of Congress and/or the Member’s staff in their local offices. 

EveryLife Foundation for Rare Diseases - 11th Annual Scientific Workshop. September 5. This year's topic will be, "Science of Small Trials in the Age of Biological Plausibility."

Cayenne Wellness Center and Axis Advocacy - 11th Annual Sickle Cell Disease Educational Seminar. September 5-7. The seminar is a three day seminar designed to address the multi-factorial aspects of sickle cell disease and sickle cell trait.


Annual Sickle Cell Disease Therapeutics Conference. September 10. A forum to discuss the latest advancements and future trends for treating patients with sickle cell disease.

RARE Patient Advocacy Summit. September 18-20. The RARE Patient Advocacy Summit is an opportunity to forge meaningful connections with other rare advocates and take home actionable strategies and tools to accelerate change. 


Rally for Medical Research Hill Day. September 19. The Rally for Medical Research’s goal is to ensure policymakers make medical research a priority and to ensure funding for the National Institutes of Health.

Sickle Cell Disease Association of America (SCDAA) 47th Annual National Convention. October 9-12. The conference fosters the exchange of the latest scientific and clinical information related to the disease.  

BIO Patient and Health Advocacy Summit. October 30-31. The summit brings together patient advocacy organizations, academia, regulators, biotechnology industry, and other stakeholders to discuss timely policy issues and share best practices. Travel Stipends close Oct. 30. Apply here.

ASGCT Policy Summit. November 4-6. Explore solutions to challenges experienced both by global regulators and by sponsors developing gene therapies across established and emerging markets. Daily registration available. 
This year, Sick Cells' co-founder and president, Ashley Valentine, will present at the 11th Annual Sickle Cell Disease Educational Seminar, the BIO Patient and Health Advocacy Summit and the Sickle Cell Disease Association of America (SCDAA) 47th Annual National Convention. We'll share more details across our social media pages, so make sure to follow along (Twitter and Instagram)! 
Do you have any walks or talks you'd like Sick Cells to include in our monthly newsletter? Please send details to avassall@sickcells.org. Please note: Sick Cells sends our newsletter out the last weekday of each month. 

News You Can Use

Sickle Cell Disease Still Tends to Be Overlooked: There is a question about what role race and wealth play in how much attention and funding the disease receives.

Trial underway in US uses CRISPR gene-editing in people with severe sickle cell disease: Several dozen patients with sickle cell disease will be among the first in the United States treated for a genetic disease with the experimental gene-editing technology CRISPR.

ICER Will Compare Lead Candidate Therapies Crizanlizumab and Voxelotor for Efficacy, Value: “It’s important to us that we know what matters to you — the patient — to make sure our reports can support policies and guidelines that help make sure patients can access, and afford, the most effective treatments,” ICER says at the start of its questionnaire. “We want to hear from you.”
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