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.

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.

News Briefs: Cancer Replaced Musculoskeletal Conditions as Biggest Driver of Large Companies’ Health Care Costs

Cancer replaced musculoskeletal conditions as the biggest driver of large companies’ health care costs, according to the Business Group on Health’s 2023 Large Employers’ Health Care Strategy and Plan Design Survey. The survey found that “13% of employers said they have seen more late-stage cancers and another 44% anticipate seeing such an increase in the future, likely due to pandemic-related delays in care.” Between May 31, 2022, and July 13, 2022, the organization polled 135 large employers in various sectors that cover more than 18 million people in the U.S. The survey also found that 99% of respondents said that they are concerned about prescription drug trend. Last year, prescription drugs were responsible for a median of 21% of the companies’ health care costs, and specialty drugs accounted for more than half of pharmacy spend.

News Briefs: ICER Says Evidence for Beti-Cel Demonstrates Net Health Benefit

The Institute for Clinical and Economic Review (ICER) unanimously determined that evidence for bluebird bio, Inc.’s betibeglogene autotemcel gene therapy “is adequate to demonstrate that the net health benefit of beti-cel is superior to that of standard clinical management,” it said in a final evidence report published July 19. “Given the high costs of standard care, cost-effectiveness modeling finds beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price is subject to an 80% payback for treatment failure,” stated ICER in a press release. The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) unanimously voted in June in support of approval for the treatment of people with beta thalassemia who require regular red blood cell transfusions. The FDA is expected to make a decision on the application by Aug. 19.

New FDA Approvals: FDA Expands Patient Population for Diacomit

July 14: The FDA expanded the patient population of Biocodex, Inc.’s Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in people between the ages of 6 months to 2 years and weighing at least 7 kg who are taking clobazam. The agency first approved the treatment on Aug. 20, 2018. The drug is available as a capsule and an oral suspension. Dosing is 50 mg/kg/day for both routes of administration. Drugs.com lists the price of 60 250 mg capsules and 60 250 mg powder for reconstitution as more than $1,589.

July 14: The FDA expanded the label of Pfizer Inc.’s Xalkori (crizotinib) to include the treatment of people at least 1 year old with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The agency initially approved the kinase inhibitor on Aug. 26, 2011. The FDA gave the agent orphan drug designation and granted the application priority review; that review used the Assessment Aid, a voluntary submission from the applicant to assist the FDA in its analysis. Dosing for the newest indication of the capsule in adults is 250 mg twice daily. The recommended pediatric dosage is 280 mg.m2 twice daily based on body surface area. Drugs.com lists the price of 60 250 mg capsules as more than $20,657.

New FDA Approvals: FDA Grants Additional Indication to CellCept

June 6: The FDA expanded the label of Roche Group member Genentech USA, Inc.’s CellCept (mycophenolate mofetil) to include, in combination with other immunosuppressants, prophylaxis of organ rejection in people at least 3 months old who have received an allogenic heart transplant or an allogenic liver transplant. The agency first approved the drug on May 3, 1995. Dosing for the newest uses is based on body surface area and indication. The drug is available as a capsule, tablet, oral suspension and intravenous injectable. Website GoodRx.com lists the price of 60 500 mg tablets as more than $1,070.

June 7: The FDA granted another indication to Sanofi and Regeneron Pharmaceuticals, Inc.’s Dupixent (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in people between the ages of 6 months and 5 years whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The agency initially approved the subcutaneous injectable on March 28, 2017. The FDA gave the new indication priority review. Dosing for people weighing 5 kg to less than 15 kg is 200 mg every four weeks; for those weighing 15 kg to less than 30 kg, dosing is 300 mg every four weeks. The drug’s list price, regardless of dose, is $3,384.83 per carton, which includes either two prefilled pens or two prefilled syringes.

CMS Unveils New Oncology Care Model to Mixed Stakeholder Responses

Only days before the end of CMS’s Oncology Care Model (OCM), the agency unveiled a successor that will start next year. While oncologists have been overall positive about the new program, they still have had some complaints.

Offered through the Center for Medicare and Medicaid Innovation (CMMI), the Enhancing Oncology Model (EOM) is a five-year, value-based, patient-centered care model that will start on July 1, 2023. Participants may include oncology physician group practices, private payers, Medicare Advantage plans and state Medicaid agencies. The application submission period started when the voluntary model was introduced on June 27 and will close Sept. 30.

How Can Pharma Incorporate the Commercial Aspect Into Drug Development?

When pharma companies launch a successful product, the process leading up to that point contains many key decisions from various teams across a manufacturer, including commercial. And with science leading to more and more innovations and many drugs coming to market via an accelerated process, it’s critical now more than ever to understand when to bring in the commercial team and how it can help with the development of a drug and its ultimate success in the market.

During a recent webinar, which was part of the Fierce Leaders in Sciences Forum sponsored by Fierce Pharma, moderator Lisa Johnson Pratt, a board member for Assembly Biosciences, kicked off the discussion by asking what the biggest challenges are for companies that are trying to bring a strong commercial point of view and input into the product development process.

Medically Integrated Dispensing Chops Waste, Signals Expansion

Newly released results of a Prime Therapeutics LLC oncology program suggest that if the PBM were to expand its highly coordinated oral oncology dispensing model beyond the pilot population, cost savings could exceed $1 million. And these promising results signal that the model may be on the brink of expanding into more disease states.

Prime’s medically integrated dispensing (MID) model, which takes a high-touch, care coordination-intensive approach, cut waste by limiting overfills, according to a Prime study released June 2. Compared with the traditional central specialty pharmacy dispensing of oral oncology drugs, the MID pilot involving 627 patients across three commercial insurance plans showed the potential to cut $1,800 in costs “per medication dose change,” according to the results.

OCM Nears Its June 30 Conclusion Without Successor in Place

The Oncology Care Model (OCM) that CMS’s Center for Medicare & Medicaid Innovation (CMMI) launched almost six years ago is nearing its June 30 end. And while CMMI introduced its Oncology Care First model in November 2019 with an eye on the OCM successor launching before its predecessor’s end, it is unclear what the program’s status is at this point. OCM participants tell AIS Health, a division of MMIT, that their overall experience has been good as they await next steps from CMMI.

The OCM voluntary pilot started in July 2016 with 17 payers and 196 practices; five payers and 126 practices currently are participating. While it began as a five-year program, CMMI extended it for one additional year in 2020 due to the COVID-19 pandemic. The program reimburses providers for episodes of care in the form of a per-beneficiary per-month payment, as well as a possible performance-based payment, if Medicare expenditures are below a target price for an episode. The amount of the payment is tied to a provider’s achievement on various quality measures. All participants began with one-sided risk but could shift to two-sided risk in 2017. Following the 2018 introduction of an alternative two-sided risk arrangement, starting in January 2020, practices that did not earn at least one performance-based payment had to enter one of the two-sided risk options or leave the OCM. Practices that earned at least one performance-based payment could remain in one-sided risk.