Laura Joszt, MA

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.

Although the number of people with nonalcoholic fatty liver disease, which progresses to nonalcoholic steatohepatitis (NASH), is growing, the health system is still trying to get a handle on which patients to target and how to identify them before the first treatments come to market, explained panelists during a session at AMCP Nexus 2019.

Although eculizumab is only approved by the FDA to treat 3 rare indications, the high and increasing net sales for the drug have raised concerns about off-label use, according to a research letter in JAMA Internal Medicine.

In a session at AMCP Nexus 2019, Melissa Andel, MPP, vice president of health policy, Applied Policy, covered the current state of health insurance coverage in the United States, major actions from the Trump administration impacting the Affordable Care Act (ACA), public sentiment around the ACA, and what stances presidential candidates have taken on healthcare.

As the number of disease-modifying therapies available for multiple sclerosis increases, patients and physicians can struggle to identify the right one for the right patient, highlighting the need for a patient decision aid.

Increased competition is making its way into the specialty drug market, affecting orphan conditions, cancer types, and even common specialty conditions, which is presenting some cost savings opportunities, explained Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, who presented on the specialty pharmaceutical pipeline during her regular session at AMCP Nexus 2019.

As the number of high-cost orphan drugs and gene and cell therapies continues to grow, there will be a greater need for alternative payment models to help figure out the best way to pay for these treatments.

Use of digital health tools appears to have leveled off in 2019, according to one study, but another finds that use of telehealth is poised to grow considerably.

Childhood conditions can have a long-lasting impact on the health of those individuals into their adulthood.

Less than half of Medicare patients newly diagnosed with blood cancer are receiving treatment for their cancer shortly after diagnosis, which may be attributed to the high cost burden they face, according to a new report from Milliman commissioned by The Leukemia & Lymphoma Society.