Laura Joszt, MA

After the United States has moved farther along the path to value-based care, it will need to contend with a host of new challenges and questions regarding how the different pieces of care delivery fit together.

Disease burden is substantial for patients with myelofibrosis, even those with intermediate risk, and a not insubstantial percentage of patients have low or intermediate adherence during treatment, according to 2 abstracts from an Italian clinical trial presented at the 61st American Society of Hematology Annual Meeting & Exposition.

Findings from 2 studies presented at the San Antonio Breast Cancer Symposium highlight the value of genomic profiling for women with breast cancer.

More than 7 months after Scott Gottlieb, MD, resigned from his position as FDA commissioner, the Senate confirmed Stephen Hahn, MD, FASTRO, to be the next head of the agency.

Hereditary transthyretin amyloid cardiomyopathy, which is caused by a genetic variant significantly associated with heart failure in individuals of African descent, is underdiagnosed, according to a new study published in JAMA.

Patients with mantle cell lymphoma (MCL) face a substantial economic burden and susceptibility to adverse events (AEs) in the real world, according to an abstract presented at the 61st American Society of Hematology Annual Meeting and Exposition.

Accountable care organizations (ACOs) have saved Medicare a total of $3.53 billion from 2013 to 2017, or $755 million after shared savings were paid out, according to a new report from the National Association of ACOs (NAACOS).

Historically, patients with severe acute graft-versus-host disease (GVHD) and severe chronic GVHD as determined by the National Institutes of Health have poor survival. New research being presented at the 61st American Society of Hematology Annual Meeting & Exposition shows that earlier treatment with novel therapies can improve outcomes for these patients.

There are no cures available to patients with polycythemia vera (PV), who are first treated with hydroxyurea (HU); ruxolitinib is approved as a second-line therapy in both Europe and the United States for patients who are intolerant of or resistant to HU. Two abstracts being presented at the 61st American Society of Hematology Annual Meeting & Exposition explore the use of ruxolitinib in patients with PV, either in patients who first tried HU or had ruxolitinib as a first-line therapy.

While there is an increased interest in using real-world evidence (RWE) to design, test, and review rare disease treatments, payers may not be as receptive to using RWE when making reimbursement and formulary decisions, according to a new report from Syneos Health.

Researchers may have identified a way to delay the progression of multiple sclerosis (MS) by blocking a molecule that controls the entry of B cells into the brain, which results in deterioration of tissue.

The FDA has approved a new treatment for adult patients with acute hepatic porphyria (AHP) a rare genetic disorder. Givlaari is an RNA interference therapeutic targeting aminolevulinic acid synthase 1. Simultaneously, Alnylam Pharmaceuticals announced a new framework for value-based agreements to help patients gain access to the treatment.

Precision medicine may offer new hope to children with high-risk cancer, but only if families and healthcare professionals are fully educated on the benefits and limitations of precision medicine trials, according to a study in Journal of Clinical Oncology.

Genomic profiling of tumors has become standard in oncology, but tumors in children often do not have actionable DNA aberrations, requiring another way to effectively target treatment for these patients. A study in JAMA Network Open found that RNA sequencing from pediatric and young adult patients may be a feasible approach.

Two new rules from CMS will take steps to increase price transparency for the purpose of empowering patients and increasing competition in the market.

Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients to have regular red blood cell transfusions.

Although patient-reported outcomes (PROs) are increasingly being used to understand treatment effectiveness, there is still a lot unknown about what measures patients find most important. Two abstracts presented at the American College of Rheumatology’s annual meeting evaluated PROs in rheumatology and how PRO measures can be used in clinical care.

Pain is common in patients with rheumatologic diseases, and 2 abstracts presented at the American College of Rheumatology’s annual meeting analyzed opioid use in these patients, examining patient features associated with chronic use and changing opioid use patterns in the wake of the opioid epidemic.

Exercise can be a potent therapy for patients with rheumatologic diseases and can result in improvements in inflammation, disease activity score, pain, stiffness, and fatigue. However, exercise needs to be modified for these patients to address the unique barriers they may have compared with the general population, said panelists during a session at the American College of Rheumatology’s annual meeting.

Patients with rheumatoid arthritis (RA) not only deal with functional impairment, but also pain, fatigue, and other symptoms driven by interleukin (IL)-6 levels. In a session at the American College of Rheumatology’s annual meeting, 2 speakers examined the role of IL-6 in RA and treatment using sarilumab (Kevzara) to target and block IL-6 signals.

New technologies that can monitor sleep, track itching patterns, or assist with pain are improving outcomes and quality of life for patients with rheumatologic conditions, according to panelists at the American College of Rheumatology’s annual meeting.

Monotherapy was a big topic of conversation at the American College of Rheumatology’s annual meeting, held November 8-13 in Atlanta, Georgia, and 2 abstracts highlight the efficacy of sarilumab as a monotherapy in patients with rheumatoid arthritis.

Patients with rheumatoid arthritis receiving sarilumab have lower odds of unacceptable pain and are able to reduce their dose of oral glucocorticoid; they also have lower costs per responder than most other treatments, according to a trio of abstracts presented at the American College of Rheumatology/Association of Rheumatology Professionals 2019 Annual Meeting.

Utilization management tools, such as step therapy and prior authorization, are not only time consuming for patients, but they are a burden on providers and their practices due to the time and effort spent on the process, explained Jessica Farrell, PharmD, and Madelaine Feldman, MD, FACR, during their session at 2019 ACR/ARP Annual Meeting, held November 8-13 in Atlanta, Georgia.

Grouping patients into clusters based on shared characteristics, such as disease control and general health, may be helpful in understanding and predicting clinical outcomes in patients with rheumatoid arthritis (RA), according to an abstract presented at the 2019 ACR/ARP Annual Meeting, held November 8-13 in Atlanta, Georgia.

This year has been an exciting time for rheumatologists with impressive clinical trial results and promising outcomes for patients, said Susan Manzi, MD, MPH, codirector of the Lupus Center of Excellence and chair of the Department of Medicine of West Penn Allegheny Health System, during a session at the American College of Rheumatology annual meeting in Atlanta, Georgia.

The evidence shows that chimeric antigen receptor (CAR) T-cell therapies are effective, but the price tags on these treatments are high and have raised concerns about how many patients will get treated. During a discussion at The American Journal of Managed Care®’s Patient-Centered Oncology Care® meeting, held Friday in Philadelphia, panelists outlined the efficacy of the 2 FDA-approved therapies, Medicare reimbursement for CAR T-cell therapies, and the pace of innovation in healthcare.

Patients with diabetes who participated in a program of group medical visits (GMVs) and intensive weight management showed improvements in glycemic control, according to a study published in JAMA Internal Medicine.

Researchers have identified complement genes that appear to play a role in vision loss associated with multiple sclerosis (MS), and this finding could help researchers monitor and predict the progression and severity of MS, according to a study published in Brain.

Approximately 4% of the total world population is affected by a rare disease at any given time, according to new research on 3585 rare diseases.