Laura Joszt, MA

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.

Serum neurofilament light chain is associated with brain atrophy and disability worsening, which means it can be used as an objective surrogate of ongoing disease activity in multiple sclerosis (MS), according to research published in JAMA Neurology.

While freestanding emergency departments (EDs) can reduce wait times and reduce the burden on overcrowded EDs, they increase utilization of emergency care and local market spending.

Pairing a cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitor with fulvestrant significantly improved overall survival (OS) for women with hormone receptor (HR)-positive, human epidermal growth factor 2 (HER2)-negative advanced breast cancer, according to 2 abstracts presented at the European Society of Medical Oncology 2019 Congress.

Data from EXPLORE, a prospective, multinational, natural history study, is used to characterize disease activity and clinical management of patients with acute hepatic porphyria (AHP) who experience recurrent attacks. The findings highlight the high unmet need for effective treatments.

The change to the public charge rule has had a negative impact on immigrant patients, who are disenrolling or declining to enroll in Medicaid and are delaying or avoiding care, according to a survey of community health centers.

While measurement of minimal residual disease (MRD) at the end of induction is an important prognostic factor in acute lymphoblastic leukemia (ALL), there is a gap in education among community oncology providers, according to an abstract presented at the Society of Hematologic Oncology 2019 Annual Meeting.

Patients with multiple sclerosis (MS) have a higher risk in general of infections, but the risk varies depending on their treatment, according to a new study in JAMA Neurology.

Rare diseases may affect a small number of people, but they have fiscal impacts beyond just healthcare costs. A new study in Orphanet Journal of Rare Diseases used a public economic framework to identify how hereditary transthyretin-mediated amyloidosis has a public economic burden beyond just health costs in the Netherlands.

Two abstracts presented at ECTRIMS 2019, the 35th Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis, looked at patterns among patients newly diagnosed with multiple sclerosis (MS).

Randomized controlled trials (RCTs) may be considered the gold standard for generating clinical evidence, but there is growing interest in using real-world evidence. However, only a small portion of clinical trials could be replicated in the real world, according to a new study published in JAMA Network Open.

Although minimal residual disease (MRD) is increasingly being used to predict treatment outcomes and as a surrogate marker of progression-free survival, there remains controversy over whether it is ready to be used in treatment decision making.

While current guidelines recommend that only women with breast cancer who have a family history or who meet clinical criteria undergo genetic testing, a new cost-effectiveness analysis suggests that genetic testing should be expanded to all women with breast cancer.

President Donald Trump has issued an executive order that will protect Medicare and bolster Medicare Advantage (MA) in response to Democrats' Medicare for All proposals.

Ozanimod, under development to treat relapsing-remitting multiple sclerosis (RRMS), has greater efficacy on the annualized relapse rate (ARR) than most other first-line disease-modifying therapies (DMTs), according to 2 abstracts presented at ECTRIMS 2019, the 35th Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis.

During the second plenary at the National Association of ACOs fall meeting, Meridith Seife, deputy regional inspector general, Office of Evaluation and Inspections in the HHS Office of the Inspector General, presented results from a government report identifying strategies of high-performing accountable care organizations that had improved care quality while cutting costs.

In the next 5 to 10 years, providers and health systems need to be thinking about how all the pieces of a new system that delivers holistic, value-based care fit together, said Will Shrank, MD, chief medical officer, Humana, during the opening plenary at the National Association of ACOs fall meeting.

Patients with psoriasis who were treated with biologics were less likely to develop psychiatric illnesses compared with patients not treated with biologics; however, the researchers did note treatment selection may have influenced the findings.

As minimal residual disease (MRD) and other measures to detect cancer burden are increasingly used to predict outcomes and direct future treatment decisions, Amgen has chosen to partner with Adaptive Biotechnologies to use the clonoSEQ product to assess MRD across multiple drug development programs.

As 2020 Medicare Advantage (MA) premiums will decline 14.0% from 2019, beneficiaries will have access to greater benefits, which has contributed to enrollment continuing to grow year over year, according to CMS.