Drug Approvals

Accelerated Approval Prices Are Unrelated to Clinical Value, Study Says

The manufacturers of many drugs granted accelerated approval by the FDA do not complete timely confirmatory trials of the drugs’ efficacy, according to a recent study published in the journal Health Affairs — meaning that the pricing for many accelerated approval drugs has nothing to do with their clinical efficacy. The study’s author tells AIS Health, a division of MMIT, that “the market doesn’t work very well” for drugs that have received accelerated approval, and “what it leads us to is overpaying at the beginning and underpaying, potentially, later.”

After a drug is granted accelerated approval, the FDA mandates that the drug be evaluated using confirmatory clinical trials. The accelerated approval designation is given to new, unproven drugs that could potentially meet a dire need for a new or more effective therapy to treat a terminal disease. The intention behind the confirmatory trial system is to make sure that the drug actually does what its developer says it will.

News Briefs: Walgreens to Complete Acquisition of Specialty Pharmacy

Walgreens Boots Alliance, Inc. (WBA) will assume full control of specialty pharmacy Shields Health Solutions for approximately $1.37 billion cash, the firm said Tuesday. Walgreens already controlled the pharmacy, holding 71% of the company’s stock thanks to a $970 million deal that closed last year. WBA CEO Roz Brewer said in a press release that the deal will allow “further progress on our strategy through Shields’ integrated model, increasing our value to health systems, expanding access to payor partners and supporting improved outcomes and lower costs.”

Centene Corp. agreed to pay $165.6 million to the state of Texas over the state's contention that the insurer overcharged the state's Medicaid program for PBM services, according to legal documents obtained by Kaiser Health News (KHN). The settlement is the largest of a series of settlements from at least a dozen states over similar charges. The Texas settlement is the largest such arrangement, with Centene paying out at least $475 million so far to the states of Arkansas, Illinois, Kansas, Mississippi, New Hampshire, New Mexico, Ohio, Texas, and Washington; three other states have not released the value of their settlements with the Medicaid-focused insurer, per KHN. Centene has divested most of its PBM assets over the past year, and told investors in 2021 that it set aside $1.25 billion to wind down its liability to states in this area.

Payers Report Cost Savings From Shifting to Biosimilars

As FDA approval of biosimilars continues and agents are expanding into new indications, more payers are using these drugs and seeing cost savings through that utilization, according to Zitter Insights.

When the FDA approved Fresenius Kabi’s Stimufend (pegfilgrastim-fpgk) on Sept. 1, it was the sixth biosimilar of Amgen Inc.’s Neulasta (pegfilgrastim) that the agency had approved. It also was the 38th biosimilar approved since the first one, Novartis Pharmaceutical Corp. division Sandoz’s Zarxio (filgrastim-sndz), was approved March 6, 2015, referencing Amgen’s Neupogen (filgrastim).

New FDA Approvals: FDA Converts Accelerated Approval to Full for Tabrecta

Aug. 10: The FDA converted the accelerated approval for Novartis Pharmaceuticals Corp.’s Tabrecta (capmatinib) to full approval for the treatment of adults with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test. The agency granted that accelerated approval on May 6, 2020. Dosing of the tablet is 400 mg twice daily. GoodRx lists the price of 112 200 mg tablets as more than $19,667.

Aug. 11: The FDA gave accelerated approval to AstraZeneca and Daiichi Sankyo, Inc.’s Enhertu (fam-trastuzumab deruxtecan-nxki) for adults with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations as detected by an FDA-approved test (see below briefs) and who have received a prior systemic therapy. This is the first drug that the agency has approved for HER2-mutant NSCLC. The FDA first approved the antibody drug conjugate on Dec. 20, 2019. The drug received priority review and breakthrough therapy designation. Dosing for the newest use is 5.4 mg/kg via intravenous infusion once every three weeks.

FDA Committee Backs ALS Drug as ICER Questions Projected Cost

By a seven to two margin, an FDA advisory committee on Sept. 7 voted that there was enough evidence to support the approval of AMX0035, an oral medication that Amylyx Pharmaceuticals, Inc. is developing for amyotrophic lateral sclerosis (ALS). The vote represented a reversal for the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC), which on March 30 had recommended against the medication’s approval.

Despite the committee's decision, David Rind, M.D., chief medical officer of the nonprofit Institute for Clinical and Economic Review (ICER), tells AIS Health that Amylyx may have trouble getting the drug covered due to the medication’s likely high cost and lack of clear effectiveness.

Survey Finds That Payers Are Covering Sequential Use of SMA Agents

A recent FDA approval of a label expansion put the three marketed therapies for spinal muscular atrophy (SMA) on equal footing for the youngest patients. And recent survey found that many payers are covering sequential use of the costly agents, including a gene therapy.

The FDA initially approved Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. on Aug. 7, 2020, for the treatment of SMA in people at least 2 months old. On May 30, 2022, the agency expanded the drug’s label to include the treatment of infants less than 2 months old. The survival motor neuron 2 (SMN2) splicing modifier is an oral solution administered by mouth or feeding tube and can be administered by a patient or caregiver at home after a recommended consultation with a health care professional prior to the first dose.

Specialty Trend Rose in 2021, but Biosimilars Are Having Impact

Specialty drug trend in 2021 largely recovered from the hit it took from the COVID-19 pandemic in 2020, driven mainly by an increase in utilization. That’s according to the 2022 Artemetrx State of Specialty Spend and Trend Report from Pharmaceutical Strategies Group (PSG), an EPIC company.

Published in August, the report is sponsored by specialty pharmacy Reliance Rx. Findings are based on an Artemetrx analysis of 73.9 million medical claims and 55.1 million pharmacy claims from PSG’s book of business. It is the sixth annual version of the report.

Payers May Restrict Coverage of New Fast-Acting Oral Drug for Major Depression

The FDA on Aug. 19 approved the first and only rapid-acting oral drug for major depressive disorder (MDD), a treatment that is being hailed by its manufacturer as a “potential game-changer” for people struggling with the difficult-to-treat condition.

One pharma analyst expressed optimism about the drug’s ability to disrupt the MDD treatment market. However, experts from the PBM sector predict that payers may not embrace the drug warmly given how many generics are available to treat depression.

FDA Approves Gene Therapy, Manufacturer Prices Drug at Record $2.8 Million

After the FDA approved Zynteglo (betibeglogene autotemcel) on Aug. 17, the gene therapy’s manufacturer, bluebird bio, Inc., announced the drug would have a $2.8 million wholesale acquisition cost (WAC). That is the highest WAC for a drug hitting the market in the U.S.

Zynteglo is indicated as a potential cure for patients with transfusion-dependent beta-thalassemia, a rare disorder caused by genetic mutations in cells that carry oxygen throughout the body. Such patients typically require red blood cell transfusions every two to five weeks.

FDA Grants Tentative Approval to Narcolepsy Agent; Neurologists Say They Are Likely to Prescribe It

A new formulation of a narcolepsy drug may get uptake among providers, according to Zitter Insights research. But payers indicate that they are likely to require patients to fail at least one generic drug before getting access to the new agent and other branded medications.

On July 19, Avadel Pharmaceuticals plc said that the FDA had granted tentative approval to its Lumryz (sodium oxybate) for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. The drug — which is also known as FT218 — is a once-at-bedtime extended-release version of Jazz Pharmaceuticals plc’s Xyrem, which requires one dose at bedtime and then another dose between two-and-a-half to four hours later.