Denise Myshko

Olumiant is the first systemic therapy available to treat alopecia.

In February 2022, the FDA had issued a complete response letter in order to inspect a new third-party packaging and labeling facility.

If approved, beti-cel will be the first potentially curative gene therapy for people with beta-thalassemia who require regular red blood cell transfusions. The PDUFA date is Aug. 19, 2022.

In COVID-19 news, an FDA advisory committee recommends EUA for Novavax’s vaccine. The FDA has also granted approvals to Dupixent for children as young as 6 month with atopic dermatitis and to the biosimilar Riabni for rheumatoid arthritis. An advisory committee has also recommended approval for two gene therapies, one for a rare neurogenerative disease and another for a blood disease.

If approved, elivaldogene autotemcel will be the first gene therapy to address the underlying genetic cause of cerebral adrenoleukodystrophy, a rare disease that affects young boys.

A biomarker used in kidney disease screening has potential to identify people with sight-threatening diabetic retinopathy.

Two studies conducted by AllianceRx Walgreens Prime call attention to the importance of adherence for reducing risk of negative health outcomes and financial costs.

Perfluorohexyloctane prevents excessive tear evaporation and has the ability to restores tear film balance. A new drug application is expected to be filed this year.

In some cases, the exclusions favored brand medicines that were more expensive than the excluded generics.

This trend in prescription drug pricing outpaces growth in prices for other healthcare services.

The commission will review PBM business practices, including the impact of rebates on formulary design, the costs of prescription drugs to patients, and methods to determine pharmacy reimbursement.

A more accurate number of rare diseases is more than 10,000, and just 500 of these disorders have treatment options available for patients, according to RARE-X.

Xcenda’s review of the three largest PBMs—CVS Caremark, Express Scripts, and OptumRx—finds they are excluding medicines in classes where generics are not available or for serious conditions, such as oncology and autoimmune disorders.

An Xcenda survey of payers found that cost-effectiveness analyses from the Institute for Clinical and Economic Review (ICER) are used in price negotiations and to implement prior authorizations.

The FDA has withdrawn the approval of Ukoniq, which is used to treat two specific types of lymphoma. In approvals, the regulatory agency has approved a new indication for Kymriah, two addition Opdivo regimens, and extended the use of Evrysdi in newborns with SMA. The agency also accepted for review Dupixent to treat skin lesions but extended the review of a new ALS therapy, refused to file an application for a rare metabolic disorder, and put a hold on the trial for Cialis OTC.

The FDA has extended review time to allow for review of additional analyses of data from the company’s clinical studies. The new PDUFA date is Sept. 29, 2022.

The label now includes a new section about the risk of substituting an oral azacitidine product, Onureg, for the injection therapy.

More than 5,700 trials worldwide are investigating PD-1/PD-L1 inhibitors, and new trials of these therapies were of combination regimens.

The FDA indicated the risk of death outweighs the benefits of Ukoniq, which was approved in February 2021 to treat specific lymphomas.

If approved, Orasis’ low-dose pilocarpine would be the second product that improves presbyopia, which is the age-related loss of clear up-close vision. The company plans to submit an NDA in the second half of the year.

The bill would allow the Federal Trade Commission to impose penalties on PBMs of up to $1 million for unfair and deceptive practices.

The FDA has approved the first non-steroid cream for psoriasis, as well as another biosimilar of Neulasta, Tibsovo combination for older patients with leukemia and a new formulation of Tyvaso. The agency has issued a second CRL for poxvirus treatment and also accepted applications for several therapies, including a nasal spray for migraine, mirvetuximab for ovarian cancer, and a treatment for the rare disease Friedreich’s ataxia.

Molly Beinfeld, senior research lead, evidence synthesis at the Institute for Clinical and Economic Review, talks about the organization’s research assessing payer coverage policies of prescription drugs.

Doug Nemecek, M.D., chief medical officer, behavioral health at Evernorth, talks about the mental health issues teenagers are facing.

Launched last year, Prime’s MedDrive is an integrated drug management solution that leverages biosimilars to help reduce drug spend.

In COVID-19 news, FDA expanded EUA for Pfizer/BioNTech COVID-19 booster to children 5 to 11 years and cleared first at-home combo COVID-19, RSV and flu test, but declined an EUA for the antidepressant fluvoxamine to treat COVID-19. Regulators also approved Lilly’s novel diabetes drug and Dupixent eosinophilic esophagitis, modified Dsuvia REMS program and issued a CRL for bimekizumab for psoriasis.

Generics are now available for the two drugs: Vimpat and Combigan.

The FDA has changed how often AcelRx Pharmaceuticals is required to audit healthcare settings that administer Dsuvia, which is a synthetic opioid.

Aetna has made available four specialty drugs that were approved early in 2022 through prior authorization. These include a biosimilar, a CAR-T immunotherapy, and therapies for macular degeneration and a rare auto-immune disease.

While regulatory flexibility is important for drugs for rare diseases, investigators are concerned the trend toward surrogate endpoints decreases confidence that new drugs can improve patient outcomes.