RADAR on Specialty Pharmacy

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.

Researchers Examine CF, UC/Crohn’s Adherence, Say Specialty Pharmacies ‘Could Help Reduce Medical Burden’

Two recent studies of specialty-drug treated conditions examined the impact of adherence on hospitalizations and medical costs. Findings of the studies — one on cystic fibrosis (CF) and the other on ulcerative colitis (UC)/Crohn’s disease — from AllianceRx Walgreens Prime (which changed its name to AllianceRx Walgreens Pharmacy in late June) demonstrate the importance of specialty pharmacy interventions in helping keep patients adherent to therapy.

The study posters were presented at the recent International Society for Pharmacoeconomics and Outcomes Research 2022 Conference held in Washington, D.C.

News Briefs: Roe v. Wade Reversal Causes Methotrexate Access Issues

Following the U.S. Supreme Court’s recent reversal of Roe v. Wade, some people with autoimmune conditions are having access issues with certain medications, including methotrexate, according to Medical News Today. While that drug can be used to treat conditions such as rheumatoid arthritis, lupus and some cancers, it also is used to induce abortions to terminate ectopic pregnancies. Some pharmacists are not dispensing it for fear of being charged with a crime in states that have banned it for ending pregnancies. It also can lead to birth defects, so people of child-bearing age are advised to be on two forms of birth control while taking it, and some rheumatologists are no longer prescribing it due to the risk of accidental pregnancy and the inability for patients to get an abortion. The American College of Rheumatology released a statement noting that it is “aware of the emerging concerns surrounding access to needed treatments such as methotrexate after the recent decision in Dobbs v. Jackson Women’s Health Organization. We are following this issue closely to determine if rheumatology providers and patients are experiencing any widespread difficulty accessing methotrexate, or if any initial disruptions are potentially temporary due to the independent actions of pharmacists trying to figure out what is and isn’t allowed where they practice.”

U.S. Sees First Ophthalmologic Biosimilar Launch in Crowded, High-Cost Space

The U.S. market recently welcomed the first ophthalmologic biosimilar onto the market: Samsung Bioepis Co., Ltd. and Biogen Inc.’s Byooviz (ranibizumab-nuna), which references Roche Group unit Genentech USA, Inc.’s Lucentis (ranibizumab). While the agent is entering what is becoming a fairly crowded space, it will offer a cost-effective option for payers, say industry sources.

On Sept. 20, 2021, the FDA approved Byooviz for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO) and myopic choroidal neovascularization (mCNV). Under an agreement with Genentech, Samsung Bioepis and Biogen were not able to market the vascular endothelial growth factor (VEGF) inhibitor in the U.S. until June 2022. Biogen Inc. and Samsung Bioepis Co., Ltd. said on June 2 that they had launched Byooviz, and the medication became commercially available on July 1. The list price of the intravitreal injection is $1,130 per single use vial, which is 40% less than Lucentis’ list price.

Report Shows Evolution in Utilization Management for Specialty Drugs

While plan sponsors continue to use traditional utilization management (UM) tools for specialty drugs, some of these tactics have evolved over the years, as well as been joined by newer ones, such as new-to-market formulary blocks, according to a new report from Pharmaceutical Strategies Group (PSG), an EPIC company. And plans’ tracking of specialty spend under the medical benefit has improved, with 70% of respondents having this capability, up from 50% in 2019, according to the 2021 Trends in Specialty Drug Benefit Design Report.

The report, which is co-sponsored by Roche Group member Genentech USA, Inc., is the ninth annual version. It previously was published under the Pharmacy Benefit Management Institute (PBMI) brand.

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.

FTC Reveals PBM Investigation, Issues Policy Statement Targeting Rebates

As scrutiny over what Americans pay for prescription drugs continues, the Federal Trade Commission (FTC) is joining the action. In early June, the agency voted unanimously to launch an investigation of the business practices of the six largest PBMs. Then a little over a week later, it issued a statement on its enforcement policy on certain rebates and fees that manufacturers give PBMs that result in the exclusion of lower-cost drugs on their formularies. Industry experts say that PBMs should heed this “aggressive approach,” which appears to be challenging them to show that these rebates are justifiable and being passed on to patients and payers.

The focus comes after President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Americans are paying too much for prescription drugs and healthcare services — far more than the prices paid in other countries. Hospital consolidation has left many areas, particularly rural communities, with inadequate or more expensive healthcare options. And too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”

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.

Pharma Patent Practices Come Under Scrutiny From Congress, FDA, PTO

As Congress again proposes drug pricing efforts, many of its members, as well as a couple of government agencies, have pharma manufacturers in their crosshairs for a somewhat related reason: their patent processes. The FDA and the U.S. Patent and Trademark Office (PTO) recently said they would be working together to scrutinize certain practices that could potentially lead to delays in competition from biosimilars and generics.

The move follows President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”

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.