Kelly Davio

Since biosimilars gained a US approval pathway as part of the Affordable Care Act, these follow-on drugs have been widely seen as a key way to address the problem of high-cost biologic drugs. However, cost savings have been disappointing as only 12 biosimilar products have been approved to date, and just 4 have become commercially available. That’s a reality that led FDA Commissioner Scott Gottlieb, MD, to call the US market for biosimilars “anemic.”

A Spanish study has found that low dose of rituximab, administered on-demand rather than at predetermined intervals, provides a good long-term treatment option for some patients.

A new, post-hoc study has reported that adalimumab, used as monotherapy, led to positive clinical, functional, and radiographic outcomes in patients with rheumatoid arthritis (RA) for up to 3 years in more than half of patients who reached low disease activity (LDA) following combination therapy.

As states seek to implement waivers that will allow them to require some Medicaid beneficiaries to work in order to remain eligible for their benefits, a key and so far unanswered question is whether work itself promotes better health.

In July 2018, the FDA approved iobenguane I 131 (Azedra), the first treatment for patients with unresectable pheochromocytoma or paraganglioma, which are rare tumors of the adrenal gland, that require systemic anticancer therapy. These tumors, which typically appear at an early age and are associated with premature death, can increase the production of epinephrines and norepinephrines, leading to a host of symptoms, including hypertension, vomiting, weakness, and chest pain.

Notably, in contrast to previous reports on palliative care in children, in which most children died at home, most of the children who died during the study period died in the hospital or in the intensive care unit. In fact, some of the children died while receiving interventions, such as the placement of a percutaneous endoscopic gastrostomy tube.

Because the majority of a patient’s time is spent outside the clinic, physicians may find it difficult to arrive at an accurate assessment of performance status (PS) for patients who have advanced cancer. Objectively evaluating PS, which measures a patient’s level of function and ability of self-care, can be challenging, but new research suggests that fitness trackers may help clinicians gain better insight into their patients’ PS.

Fenfluramine, a drug that was previously sold as an appetite suppressant before it was withdrawn from the market in 1997 over concerns about its link to heart valve problems, may have a new application; low doses of fenfluramine hydrochloride have now proven successful in treating Dravet syndrome, a particularly severe form of epilepsy.

Depression has been associated with poorer outcomes for patients who have acute coronary syndrome (ACS), including myocardial infarction. Now, new research indicates that treating depression may improve long-term cardiac outcomes for patients with ACS.

During the 2018 American Society of Clinical Oncology’s (ASCO) Annual Meeting, researchers presented findings on 3 biosimilar trastuzumab products: Samsung Bioepis’ SB3, Agmen’s ABP 980, and Biocad’s Herticad.

Unplanned hospital readmissions, which affect more than 18% of Medicare beneficiaries each year, are generally understood to be tied to gaps in care coordination between hospitals and community healthcare providers, and new research suggests that readmission more than a week after discharge may be tied to patients’ inability to access appropriate outpatient care after discharge.

During the European Hematology Association’s 23rd Congress, held from June 14 to 17 in Stockholm, Sweden, researchers announced that the ELOQUENT-3 phase 2 study, evaluating elotuzumab with pomalidomide and low-dose dexamethasone in patients with relapsed/refractory multiple myeloma, met its primary endpoint in showing a statistically significant, clinically meaningful improvement in progression-free survival.

On June 14, 2018, the Senate Judiciary Committee voted 16 to 5 to report the Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act to the Senate floor. Here are 5 key things to know about the proposed legislation.

While receiving a diagnosis of type 2 diabetes is common for US adults, African American and Latino adults over age 50 who receive a new diabetes diagnosis may have another health issue to be concerned about: pancreatic cancer.

Akcea Therapeutics has announced the publication of results from the Re-FOCUS study that sought to assess the burden of familial chylomicronemia syndrome (FCS) on patients and the impact of Akcea's proposed drug, volanesorsen, on disease burden.

A new analysis from the Kaiser Family Foundation finds that most Medicaid enrollees who are able to work are already employed, and that work requirements are unlikely to help enrollees rise from poverty but could negatively impact those who are already working or are unable to work because of medical conditions.

GBC0905, a small-molecule drug, suppresses DUX4 function and protects skeletal muscle cells from muscle fiber death. Amanda Rickard, lead scientist of Genea Biocells’ FSHD program, said in a statement that the company expects the drug to be curative.

While it is well understood that, in the general population, regular exercise is associated with a reduced risk of death, less is known about whether exercise is similarly beneficial for adult survivors of childhood cancer who may already have an increased risk of death and a shortened life expectancy because of late effects of treatment.

The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc’s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy, an X-linked genetic disorder caused by a defect in the gene ABCD1.

A new analysis of findings from the Framingham Heart Study by researchers at the Vanderbilt University Medical Center suggests that a smoker’s risk of lung cancer drops substantially within 5 years of quitting.

The FDA has approved BioMarin’s pegvaliase-pqpz (Palynziq), a drug for the treatment of phenylketonuria (PKU) in adults. PKU is a rare autosomal recessive disorder, affecting approximately 1 in every 10,000 to 15,000 people in the United States, that increases the levels of phenylalanine (an amino acid obtained through all dietary proteins and some artificial sweeteners) in the blood.

On the closing day of the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, stakeholders gathered to grapple with the role of value assessments in a healthcare landscape that is increasingly focused on the use of precision medicine in treating disease.

On the closing day of the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, stakeholders gathered to grapple with the role of value assessments in a healthcare landscape that is increasingly focused on the use of precision medicine in treating disease.

The number of US adults who have high blood pressure could grow by as much as 31 million—and the number of adults who will be recommended for antihypertensive treatment could increase by 11 million—if full implementation of the American Heart Association’s 2017 hypertension guideline is reached.

Advances in medicine have produced breakthroughs in the treatment of a number of rare diseases, but these advances often come at a high cost. A multi-stakeholder panel at the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, addressed the question of how to define value in the always evolving and ever more expensive treatment landscape.

In a Monday panel convened at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 23rd Annual International Meeting in Baltimore, Maryland, stakeholders grappled with the role that value assessments play—or should play—in US private payer coverage and formulary decisions.

Approximately 20% of patients with epilepsy have a co-occurring autoimmune disorder, and autoantibodies directed against the smaller isoform of glutamate decarboxylase (GAD65Ab) have been found in patients with epilepsy, as well as in patients with stiff person syndrome and type 1 diabetes.

The authors write that these differences among Veterans Affairs (VA) populations could reflect variability across the medical centers in terms of quality of care, adherence to evidence-based treatment and screening guidelines, access to urgent care, posthospitalization care protocols, chronic disease management, and access to specialty care, social work services, and behavioral health care.

While the number of drugs approved for rare diseases has increased significantly since the passage of the Orphan Drug Act of 1983, questions linger about whether is connected with innovation in treatments for rare diseases, especially given the fact that patent protection for new products often extends well beyond the 7 years granted to orphan drugs.