Kelly Davio

“Even though we have all these fancy, effective drugs,” said Shaji K. Kumar, MD, “supportive care still plays an important role in multiple myeloma.”

Alan Venook, MD, recalled a time when the National Comprehensive Cancer Network guidelines for treating colon cancer were just 4 pages long. “I don’t think we envisioned that these guidelines would take on the life that they have,” he said.

“HIV status alone should not be used for cancer treatment decision making,” said Gita Suneja, MD, Duke Cancer Institute.

Prior authorizations delay care, have a significant negative impact on clinical outcomes, and place a high burden on providers, according to a physician survey conducted by the American Medical Association (AMA).

Currently, the FDA is evaluating 2 investigational drugs, both of which treat the underlying pathophysiology of hereditary transthyretin amyloidosis.

To achieve a similar capture rate for nonwhite patients as for white patients at age 50, screening ages would need to be reduced to 47 years for black and Asian women, and 46 years for Hispanic women.

In Canada, there exists no distinct regulatory or reimbursement pathway for orphan drugs. Instead, these products undergo the same review processes as any other drug. When a medicine is submitted for review, the Common Drug Review examines the clinical and economic evidence, and its expert committee issues a nonbinding positive or negative recommendation for listing on publicly funded drug plans.

Reviewing 25 years of experience with accelerated approvals (AAs) for malignant hematology and oncology drugs and biologics, FDA officials say that the AA program has demonstrated that it can be used successfully to expedite approval of safe, effective cancer therapies that balance uncertainty with the need to provide faster access to promising agents for serious and life-threatening diseases.

Researchers have identified a molecular target that could allow chimeric antigen receptor (CAR) T-cell therapy to be used in treating patients with glioblastoma. Although the heterogeneous expression of tumor-associated antigens limits the efficacy for CAR-redirected T cells for the treatment of glioblastoma, chondroitin sulfate proteoglycan 4 (CSPG4), a cell surface type 1 transmembrane protein, is highly expressed in a majority of glioblastoma specimens with limited heterogeneity.

A newly published report, “Fairness in Precision Medicine,” by Kadija Ferryman, PhD, and Mikaela Pitcan, seeks to better understand how bias could impact biomedical research into precision medicine.

Kazia Therapeutics Limited, an oncology-focused biotechnology company, announced on Friday that it has received the FDA’s Orphan Drug Designation for its investigational drug, GDC-0084, for the treatment of glioblastoma multiforme, the most aggressive form of primary brain cancer.

Last week, the European Crohn's and Colitis Organisation held its 13th annual congress in Vienna, Austria. At the meeting, researchers presented new data on using biosimilar anti–tumor necrosis factor therapies in treating Crohn disease and ulcerative colitis, 2 manifestations of inflammatory bowel disease. Here are 5 things to know about the research presented.

African American patients and uninsured patients are more likely to have diminished employment participation 2 years after being cancer free than are those with no history of cancer, according to new research published in Cancer.

Drug maker Vertex has announced that the FDA has approved tezacaftor/ivacaftor and ivacaftor (Symdeko) for the treatment of the underlying cause of cystic fibrosis (CF) in patients aged 12 or older who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, or who have at least 1 genetic mutation that is responsive to treatment with tezacaftor/ivacaftor.

The American Society of Clinical Oncology (ASCO) and the National Comprehensive Cancer Network (NCCN) have developed new guidelines on managing immune checkpoint inhibitor-related toxicities.

Fibrocell Science has submitted an Investigational New Drug Application (IND) with the FDA for a gene therapy candidate to treat moderate to severe localized scleroderma.

Transgender and gender-nonconforming adolescents have poorer overall health and receive less healthcare than their cisgender peers, according to a new study published in Pediatrics.

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

One in every 4 drugs approved by the FDA over the past 4 years was a personalized medicine, and the agency approved a record-breaking 16 personalized therapies in the past year, according to a new report from the multi-stakeholder group the Personalized Medicine Coalition.

Only 5% of rare diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives.

A new data note from the Kaiser Family Foundation provides new, nationally representative estimates of insurance coverage changes under the Affordable Care Act (ACA) for the approximately 3% of Americans who identify themselves as lesbian, gay, and bisexual (LGB).

Benitec Biopharma, an Australia-based drug developer, has received the FDA’s Orphan Drug designation for BB-301, an investigational drug for the treatment of oculopharyngeal muscular dystrophy.

Republican Senator Chuck Grassley of Iowa, chairman of the Senate Judiciary Committee, is calling on regulators and Southern Illinois University to account for reports that a researcher conducted rogue trials of an experimental herpes vaccine.

This week, the investors and analysts turned their attention to presentations by business leaders at the 36th Annual JP Morgan Healthcare Conference, held in San Francisco, California. Executives from the pharmaceutical and healthcare worlds showcased their products, pipelines, and visions for the future.

Alex Azar, President Donald Trump’s nominee to be the next secretary of HHS, suggested during his testimony before the Senate Finance Committee that pharmacy benefit managers (PBMs) would be the most effective tool to negotiate for lower drug costs, and said that PBMs should negotiate physician-administered drugs covered under Medicare Part B.

When patients with rheumatoid arthritis experience failure of an anti–tumor necrosis factor (anti-TNF) therapy, clinical guidelines support either cycling to a different anti-TNF agent or switching to a treatment with a different method of action (MOA). However, payers often require cycling of anti-TNF options before they will reimburse for treatments with a different MOA.

Several studies have suggested that inflammation plays a role in Huntington disease (HD), and a recent review, published in Neural Regeneration Research, explored the anti-inflammatory and immunomodulatory agents that have been investigated as possible treatments for HD.

The US Chinese aging population has experienced a rapid growth in recent years. However, research about the social networks of older immigrant adults has been lacking. That scarcity of information has led to a lack of understanding that has limited the ability of US healthcare professionals and policy makers to provide the best strategies to improve the health and wellbeing of older Chinese adults.

In a new study, published in Arthritis Research & Therapy, a research team led by Soo-Kyung Cho, MD, sought to investigate the incidence of malignancy in early rheumatoid arthritis and found that the use of biologic disease-modifying anti-rheumatic drugs decreased the overall risk of developing malignancies, though it did not affect the risk of developing hematologic malignancies.

Asthma, a leading cause of emergency department visits and hospitalizations among American preschoolers, may be worse in children aged 2 to 5 who are overweight or obese.