When Adm. Brett P. Giroir, MD, was sworn in as Assistant Secretary for Health at the U.S. Department of Health and Human Services (HHS) in February 2018, he selected sickle cell disease (SCD) as one of his top priorities – vowing to take advantage of the interagency resources and advances in patient care to improve the lives of patients living with this disease.
In his role as Assistant Secretary for Health, Dr. Giroir leads development of HHS-wide public health policy recommendations, overseeing 11 core public health offices – including the Office of the Surgeon General and the U.S. Public Health Service Commissioned Corps. He also serves as Senior Advisor to the Secretary for Opioid Policy. In this capacity, he is responsible for coordinating HHS’s efforts across the Administration to combat the nation’s opioid epidemic.
Dr. Giroir spoke with ASH Clinical News about HHS’ efforts to raise awareness about sickle cell disease, collaborating to work toward a cure, and the nine words that encapsulate his goals as HHS Assistant Secretary for Health.
What is your vision for your term as the HHS Assistant Secretary for Health?
As the Assistant Secretary for Health for the U.S. Department of Health and Human Services (HHS), Admiral in the U.S. Public Health Service Commissioned Corps, and senior science and public health advisor to the HHS Secretary, I am in a unique position to effect change via the offices I oversee.
On a regular basis, we bring together federal agencies, state and local partners, professional societies, nonprofit organizations, academia, patient advocates, and other stakeholders to develop shared goals, metrics, and plans to address a number of health-related issues. My overarching vision for most of what I do here is to try to transform the current “sick care system” into a “health promoting system.”
My philosophy can be summarized in nine words: “Health for all, health by all, health in all.”
- Health for all: Ensure that everyone has a fair and realistic opportunity to optimize his or her health. Here, we particularly focus on patients in the most need, including those suffering from health disparities.
- Health by all: Distribute and democratize health-care knowledge, capabilities, and delivery to empower individuals, families, and communities to advance and advocate for their own health.
- Health in all: Prioritize health considerations in all sectors and policy areas. Our focus in this realm will be the social determinants of health, and, perhaps in the future, the use of digital technology to advance overall health equity.
How are the HHS and other federal agencies working to raise awareness about sickle cell disease?
In expanding our efforts on behalf of patients with SCD, HHS is building an aggressive and bold patient-centered agenda that includes education, advocacy, payment reform, and the development of new therapies aimed to cure the disease within a decade.
This past September, for the first time since 1983, a U.S. president, Donald Trump, issued an official Presidential Message on SCD, reaffirming this nation’s commitment to patients with this disease. Since then, HHS has continued to raise the profile of this disease as a public health priority and to develop and implement tangible, meaningful, and aggressive programs that immediately impact people with this disease.
For example, in September, HHS hosted five public webinars and other events, and released 14 videos on sickle cell. We also released “Sickle Cell Stories,” a series of interviews with patients, advocates, and family members of those living with sickle cell. As part of that project, it was my personal privilege to speak with the U.S. Surgeon General, Vice Adm. Jerome Adams, MD, MPH. We discussed the challenges that patients with sickle cell face throughout their lives and our mutual commitment to leading change.
We also developed a new webpage to house videos, graphics, resources, event listings, and information for partners. On social media, we made 6.5 million impressions with #CuringSCD. Of those, our top three tweets reached nearly 1.4 million users. We had a great start in September, but this effort has only just begun.
What do you think are the biggest challenges to improving awareness and management of sickle cell?
The Cure Sickle Cell Initiative announced recently by the National Institutes of Health (NIH) builds on decades of scientific investments that have brought us to where we are today, with tools available to correct or compensate for the defective gene that causes the lifelong consequences of SCD.
However, discovering the science is only the first step in the arduous journey of bringing new treatments to patients and their families. New therapies must be proven safe and effective in rigorous scientific studies and receive approval by the U.S. Food and Drug Administration (FDA). This often is as difficult, as time-consuming, and as expensive as discovering the science itself.
Finally, we need to develop a strategy to ensure that treatments, when scientifically proven, are universally affordable and available to all who need them. That planning needs to start now, given the likely cost of gene therapies.
In addition to finding a cure, we must unite to fight the stigma, lack of knowledge, and stereotypes faced by patients with SCD. Primary-care providers – and emergency-room physicians in particular – must become more knowledgeable about this disease. Only approximately one-quarter of the 100,000 people with sickle cell in the U.S. receive the recommended standard of care. That is entirely unacceptable.
Fortunately, partners like the American Society of Hematology (ASH) have begun an aggressive plan to better inform health-care providers about this disease, and I have personally met with groups of providers via the Society to establish a plan of action. I am very impressed and moved by the passionate commitment ASH members have displayed for patients with sickle cell.
My office is committed to ensuring 100-percent adherence to clinical guidelines, and we will work hand in hand with organizations like ASH, other agencies within HHS, and all providers to achieve this goal. Improved compliance with guidelines will immediately reduce pain crises, reduce emergency-room visits, and improve outcomes. It also will set the stage for new practice guidelines when advanced therapies, like gene therapies, come available. Simultaneously, we must empower patients to advocate for themselves to ensure that the system is individually responsive.
Many patients with SCD use opioids for pain management – how do you think they are affected by the opioid crisis and policy to help curb the opioid crisis?
Our agenda at HHS is highly focused on reducing the inappropriate use of opioids, but also preserving the appropriate use of opioids and other therapies for people who experience chronic or intermittent severe pain. We are committed to educating health-care professionals on how to better identify and treat patients with sickle cell in pain crisis. That includes promoting best practices in pain management (including the safe and effective use of opioids tailored to the needs of these patients) and changing the culture of discrimination associated with pain treatment. Of course, this also entails planning a strategy to pay for new treatments when they become available.
We need to be very careful in our messaging about opioid misuse. We want to be clear that we need to decrease inappropriate use of opioids, but that there are patients who experience excruciating pain and, for these patients, opioids often are the best available treatment.
My goal – and absolute intention and obsession – is that, in the near future, we can provide individuals with SCD a long life, free from pain and complications. Now is the time to accelerate our efforts.
With new approaches to treating and managing SCD, more patients are living longer with this disease. How is HHS working to address the needs of adults living with SCD?
Transition of care is one of the major issues for this patient population: When patients are transitioned from children’s hospitals, which are highly specialized, nurturing, and generally well-funded, they are released into an adult health-care system that does not necessarily care for patients in the same holistic, coordinated manner. This transition period is a critical time when complications can occur.
Advances in medical therapies have brought us within sight of a cure that will work for all patients. I believe the NIH’s Cure Sickle Cell Initiative will indeed accelerate the discovery of a cure. This initiative also includes a data strategy that will unite patient registries to assist researchers and clinicians in establishing national networks that will make it easier for patients to engage in clinical trials and other activities.
Having a wide range of ages and backgrounds participating in these studies is critical. The future of individuals with SCD should no longer be in question. We can find a cure that is available to all patients. I believe we will do so soon.
The American Society of Hematology (ASH) is undertaking a multifaceted initiative to address the burden of sickle cell disease (SCD), both in the U.S. and globally. Throughout the initiative, ASH has engaged a broad array of public health organizations, patient groups, federal agencies, industry representatives, and foundations to identify the highest priorities needed to improve outcomes for individuals with SCD. Below are ASH’s priorities for SCD.
Sickle Cell Disease Coalition
In 2016, the Sickle Cell Disease Coalition (SCDC) was founded to help amplify the voice of the SCD stakeholder community, promote awareness, and improve outcomes for individuals with SCD. The SCDC’s growing membership of more than 50 groups includes public health, research, and provider organizations; patient groups; federal agencies; industry representatives; and foundations.
SCD Research Priorities
ASH continues to take steps to advance SCD research and is exploring opportunities to enhance SCD clinical research to improve outcomes for individuals with SCD, facilitate innovative approaches to clinical trials research, and expedite drug development. In 2017, ASH announced plans to develop a research registry that will harness the power of big data to conquer blood diseases worldwide. Since then, ASH established the ASH Research Collaborative (ARC), a separate non-profit entity focused on fostering collaborative partnerships to accelerate progress in hematology. The foundation of the ARC is its Registry, which will initially focus on sickle cell disease and multiple myeloma.
SCD Access to Care
ASH is in the process of developing and implementing a multipronged strategy to expand health-care professional education and training on SCD. Activities are aimed at four key audiences: hematologists, patients, hospitalists, and primary care clinicians. The Society is also working closely with the Emergency Department Sickle Cell Care Coalition to support their efforts to improve emergency care of individuals with SCD. Managing the acute and chronic complications of SCD also is the topic of one of ASH’s five new clinical practice guidelines, to be published in 2019.
SCD Policy Priorities
ASH works with congressional champions to raise awareness for SCD on Capitol Hill, to have legislation introduced to strengthen current federal SCD programs, and to increase funding for federal SCD programs. ASH also continues to encourage the Centers for Medicare & Medicaid Services to test innovative payment models for SCD care delivered by health-care professionals.
ASH is exploring the development of a consortium of African countries to address newborn screening for SCD. The consortium would introduce standard-of-care practices for screening and early intervention therapies at participating institutions, with the goal of decreasing childhood mortality rates for SCD.
Please visit hematology.org/SCD to learn more about these efforts.