Skylar Jeremias

The FDA Friday approved pemigatinib for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement.

Researchers found that the factors most associated with health related quality of life (HRQOL) in patients with facial palsy are age, bilateral facial palsy, severity of facial palsy, anxiety, and key personality traits, including extraversion and emotional stability.

Despite experiencing general fears, patients with rare diseases may be the most equipped to handle the coronavirus disease 2019 (COVID-19) pandemic, according to some experts.

AHIP wants the FDA and FTC to streamline product approvals, clarify the regulations governing biosimilars, put a stop to attempts to discourage people from using biosimilars, and start educating providers and patients about the safety and efficacy of these products.

The FDA released guidance on what sponsors should take into account when trying to demonstrate clinical effectiveness of new treatments in patients with slowly progressive, low-prevalence rare diseases with substrate deposition that result from single enzyme defects.

Payers view the National Comprehensive Cancer Network (NCCN) guidelines as a gold standard for making reimbursement decisions, according to a panel of experts who spoke recently about biosimilar uptake.

Cushing syndrome, a rare endocrine disorder caused by abnormally excessive amounts of the hormone cortisol, has a new pharmaceutical treatment to treat cortisol overproduction.

The analysis looked at 13 of America’s largest employers, including energy, hospitality, retail, transportation, and finance companies.

Limited data about the effects of psoriasis therapy during pregnancy make it difficult to know which options are possible for pregnant patients and which ones can be harmful, according to a review.

An expert panel discussed the systemic and policy barriers to the uptake of biosimilars.

The results are in line with earlier studies, and the current review found a link with bladder cancer and lung cancer.

Investigators say that inconsistencies in the way rare diseases are defined contribute to misdiagnoses, delayed treatment, and other ills that could be addressed with global standards.