Novartis Oncology Office of Grants & Education Professional Medical Education
The Novartis Oncology Office of Grants & Education (OGE) supports independent high-quality medical educational programs which provide fair-balanced, evidence-based, current scientific information to healthcare professionals in order to positively improve patient care. Activities should have an educational focus, be independent of commercial bias and be non promotional in nature. OGE will perform these duties in compliance with laws, regulations and guidelines as established by the ACCME, PhRMA Code, OIG, other regulatory agencies and in compliance with Novartis guidelines and policies.
Sickle Cell Disease
RFP Issued: April 21, 2021 Applications Due to Novartis: May 17, 2021 by 5 PM EST Notification of Grant Decisions: June 2021 Educational Programming Starts: Early Q3 – Q4 2021
Sickle Cell Disease (SCD) is an inherited genetic disorder that affects Hb synthesis, causing erythrocytes to become rigid and adopt a sickle-like shape upon deoxygenation (Habara A & Steinberg MH, 2016) (Rees DC et al, 2010). This leads to vascular damage and contributes to the pathophysiology of SCD (Gladwin MT & Sachdev V., 2012; Kanter J & Kruse-Jarres R. 2013). The National Heart, Lung, and Blood Institute (NHLBI) estimates that SCD affects 70,000 to 100,000 people, mainly African Americans, in the U.S. (NHLBI, 2014). Major advances in the treatment of SCD over the past 4 decades have improved outcomes and increased life expectancy; however, quality of life remains poor1 and life expectancy is still reduced by about 20 years in a high-income setting (Piel FB & Steinberg MH., 2017; Lanzkron S et al. 2013; Gardner K et al. 2016; US Centers for Disease Control and Prevention. Deaths: Final Data for 2014. 2016).
Vaso-occlusive crises are the hallmark of SCD. These recurrent episodes induce severe pain, decrease quality of life, can cause life-threatening complications, are the primary cause of hospitalization in SCD, and are associated with increased mortality (Ballas SK et al., 2012; Claster S & Vichinsky EP, 2003). In fact, VOCs are the cause of approximately 197,000 ED visits each year (Wilson and Nelson, 2015). VOCs, also known as sickle cell pain crises, are severely painful acute events that can last, on average, for 10 days and may lead to further complications such as priapism, acute chest syndrome, cerebrovascular accidents or sudden death (Ballas SK et al. 2012; Claster S & Vichinsky EP 2003; Piel FB & Steinberg MH. 2017; Puri L et al. 2017).
VOCs that cannot be managed at home are often treated in the emergency setting.1 Lack of access to specialist care in this setting means that many patients receive suboptimal treatment and that rates of hospital admission are high (Telfer P & Kaya B. 2017; Lovett PB et al. 2017; Molokie RE et al. 2018).
Most of these patients do not have access to the minimum standard of care recommended by the National Institutes of Health (NIH) in their consensus document. This lack of access, along with other health policies, has resulted in unnecessary morbidity and mortality and a decreasing survival rate for adults.
SCD has changed from a fatal pediatric illness to a chronic adult disease characterized by progressive multi-organ failure. The survival rate for pediatric patients continues to improve, however, the overall survival rate for adults has made little progress even in regions where the rate of disease has decreased. Decades before the landmark Cooperative Study of Sickle Cell Disease, the median age of death was 14 years. At the completion of the Cooperative Study in 1988, the median age of death was 48 years for women and 42 years for men. Recently, the NIH published population-based surveillance data for all causes of death among 12,000 patients with SCD.
This poor overall survival in adults is accompanied by deteriorating quality of life, increased morbidity from multiple complications, and preventable deaths. As the disease progresses with age, there is an urgent need for access to trained health professionals, a receptive healthcare infrastructure, research that improves survival, quality of life, and decreased costs. Unfortunately, this environment does not exist. There is a lack of transition for young adults from pediatric centers to receptive adult providers. The ER/hospitalization visit rate for the young adult, upon leaving a pediatric program, skyrockets from 1.8 ER visits to an average of almost 4.8 visits or hospitalizations per year.
The etiology for the worsening in quality of life and survival after pediatrics is multifactorial, but clearly a major factor is the lack of educated and available adult sickle cell providers who have ongoing access to multidisciplinary care and daily support. Non-compliance of the medical community with NIH recommendations for the treatment of SCD is routine. Recent studies indicate this problem is worsening. Greater than three-quarters of patients do not receive standard recommendations for pulmonary, neurology, prenatal, renal, transfusion, mental health, and genetic counseling services. Recent estimates indicate the cost of sickle cell patient care is in the billions of dollars. Most of the expenditure is disproportionately from hospitalizations and complications that could be prevented or markedly decreased. The development of adequate ambulatory services will therefore result in hundreds of millions of dollars in savings while improving health outcomes. The training and ongoing educational support of adult providers including primary care physicians, hematologists, nurse practitioners, case managers, social workers, and counselors is critical to improving the survival of adult SCD patients.
Unfortunately, many physicians have difficulty staying up to date on current guidelines for a disease as rare and complex as SCD, much less on emerging therapies. This is particularly true of physicians practicing in the community setting, who are tasked with treating a variety of conditions, making it hard to keep abreast of developments for any one. To ensure that all providers are equipped to rapidly integrate novel SCD therapies into their practice, education is needed on the latest safety and efficacy data for emerging agents in late-stage development, as well as practical information about how to use these options alongside existing treatments to optimize patient outcomes. Hydroxyurea was once the only FDA approved treatment for sickle cell disease for over 20 years, and was approved to reduce the frequency of painful crises and reduce the need for blood transfusion in patients with sickle cell anemia with moderate to severe painful crises. It is under prescribed by hematologists/oncologists. Over 60% adult patients eligible for hydroxyurea do not receive it. In 2017, Endari (L-glutamine) was approved for the reduction of acute complications of SCD in patients 5 years and older. Similar to hydroxyurea, it was studied in patients with HbSS and Hbβº-thalassemia genotypes. In November 2019, Adakveo (crizanlizumab-tmca) was approved to reduce the frequency of vasoocclusive crises in SCD patients 16 years and older. This drug was studied across all genotypes. Also in November 2019, Oxbryta (voxelotor) was approved for the treatment of SCD patients 12 years and older. Unlike the other therapies, voxelotor was shown to increase hemoglobin by 1 g/dL with no data on crises. Hydroxyurea, l-glutamine and voxelotor are all oral products, while crizanlizumab is the only drug administered by IV infusion. Ideally, physicians would feel confident managing the symptoms of SCD and preventing and treating its complications—including vaso-occlusive pain episodes and acute chest syndrome (the most common causes of SCD-associated morbidity)—as well as SCD-related chronic pulmonary disease, retinopathy, nephropathy, and cardiovascular disease.[Chaturvedi 2016; Gladwin 2017] Specifically, they would feel comfortable creating personalized SCD treatment plans based on the latest evidence regarding current treatments, with the goal of preventing pain crises and minimizing tissue and organ damage.[NHLBI 2014]
They would also feel confident in their ability to rapidly integrate new therapeutic options for SCD into their practice, in order to improve patient outcomes.
More recently, new data have the potential to shift the treatment paradigm for sickle cell disease. However, many clinicians lack confidence in their ability to incorporate these agents into clinical practice, how to best assess and treat VOCs, and how to safely and effectively assimilate relevant information into clinical practice. Hematologists/oncologists, primary care physicians (PCPs), emergency medicine physicians, and other members of the team who care for patients sickle cell disease must understand how to interpret current and emerging data, as well as how to incorporate these emerging agents into their clinical practice for shared decision making with their patients.
OGE has identified the need for innovative continuing medical education programs that strive to optimize patient outcomes through education on:
Address gaps in scientific knowledge among SCD experts and general practitioners treating SCD patients on the burden of disease specifically vaso-occlusion mediated end organ damage in adult and pediatric populations
Latest safety and efficacy data of current and emerging agents in SCD
Understand the mechanism of disease and the different types of SCD therapies in vaso occlusive crises, how to use new agents alongside existing treatments to facilitate adherence, and the economic value in the treatment of patients.
Sickle cell pain crisis pathophysiology, diagnosis, management and chronic implications (i.e. end organ damage) for the ER and community
How to implement multidisciplinary collaborations to improve care for SCD patients
Effective means of reducing and preventing vaso-occlusive crises in SCD patients in the ER and community setting
Clinical management of COVID-19 and SCD patients
OGE is seeking to support:
Virtual/Live (dependent on state regulations) community-based educational series of case-based programs held in community sickle cell clinics, heme/onc academic centers, and/or pediatric hospitals, along with an enduring component.
Virtual/Live (dependent on state regulations) programs with enduring component (stand alone or in conjunction with SCD related medical societies, congresses)
Highlights of ASH
Note: Program placement is independent of Novartis. Program placement should reflect nationwide distribution in locations in which patients with SCD obtain treatment.
National, Regional, and/or Local
Hematologists/Oncologists, Pediatricians, ER Doctors, Pharmacists, PCPs, PAs, NPs/(ONC) Nurses, Social Workers
Educational providers should include target number of participants. Further, please include details on proposed audience recruitment.
Please note: Novartis will not participate in the distribution of invitations to the CME/CE event.
Multiple single-support or multi-support initiatives may be funded; Up to $500,000 in total support is available.
Grant applications must be submitted by the Accredited Provider (or the Office of CME if from an Academic Institution) electronically via the Online Portal: by 5 PM EST on May 17, 2021 to be considered.
The grant application should include “RFP Response” within the Program Title [example: “RFP Response: Program Title”].
Proposals that include collaborations with third parties, including (but not limited to), medical societies, health education companies/centers, not-for-profit organizations, and academic institutions, are encouraged, as appropriate.
If you have any questions regarding this RFP or the Novartis process for grant submission, you should only contact OGE at 1 877 ONC GTED (1 877 662 4833) or via email at: [email protected]. [Please title the subject of your email: “RFP 2021 Sickle Cell Disease”].