Radar on Specialty Pharmacy

Issues around so-called alternate funding companies that carve out specialty drugs have existed for several years now, but only recently has a manufacturer taken legal action against such a company. On May 5, AbbVie Inc. filed a lawsuit (1:23-cv-02836) against Payer Matrix, LLC in the U.S. District Court for the Northern District of Illinois Eastern Division over its “fraudulent and deceptive scheme to enrich itself by exploiting AbbVie’s PAP [patient assistance program] through the enrollment of insured patients into a charitable program not intended for them.”

AbbVie states that it is “bring[ing] this action to stop Payer Matrix’s harmful conduct and protect its program so it can continue to serve its intended purpose — providing free drugs to uninsured and underinsured patients who otherwise could not afford their AbbVie medicine.”

On May 3, Option Care Health, Inc., the largest independent provider of home and alternate site infusion services in the U.S., revealed that it was acquiring Amedisys, Inc., which provides home health, hospice and high-acuity care, for $3.6 billion. While opinions on the deal differed, one industry expert contends that the transaction offers multiple long-term benefits within the ever-evolving health care space, especially the home setting.

The deal comes less than a week after Option Care unveiled its wholly owned subsidiary Naven Health, Inc., a nationwide home infusion nursing network and platform employing more than 1,500 nurses and serving all 50 states.

On May 5, AbbVie Inc. filed a lawsuit (1:23-cv-02836) against Payer Matrix, LLC in the U.S. District Court for the Northern District of Illinois Eastern Division over its “fraudulent and deceptive scheme to enrich itself by exploiting AbbVie’s PAP [patient assistance program] through the enrollment of insured patients into a charitable program not intended for them.”

Payer Matrix was not able to provide a comment on the lawsuit by press time. But prior to the filing and shortly before AbbVie updated its PAP language earlier this year, AIS Health, a division of MMIT, conducted an interview with Michael Jordan, Payer Matrix’s chief business officer (CBO), to learn more about the company’s practices.

The FDA recently expanded the use of BeiGene, Ltd.’s Brukinsa (zanubrutinib) to include its use in the treatment of a hematologic cancer. The agent is already approved for three other rare types of non-Hodgkin lymphoma. Respondents to a Zitter Insights survey said that while its availability will result in a lower level of unmet need in the treatment of chronic lymphocytic leukemia (CLL), there is still moderate or high unmet need for the condition.

On Jan. 1, the FDA expanded the label of Brukinsa to include the treatment of adults with CLL or small lymphocytic lymphoma (SLL). CLL and SLL are the same disease, a type of non-Hodgkin lymphoma, except CLL cancer cells are mostly in the blood and bone marrow, while in SLL, the cells are mainly in the lymph nodes. CLL is the most common adult leukemia.

Specialty drug management continues to be of utmost importance to plan sponsors, which are implementing a variety of levers to try to keep spending in check while making sure their beneficiaries are receiving appropriate care. Pharmaceutical Strategies Group (PSG), an EPIC company, recently released its 2023 Trends in Specialty Drug Benefits Report, which examines the use of these strategies and overall trends in managing these costly medications.

The report, released May 3, is the 10th annual report; it previously was published under the Pharmacy Benefit Management Institute (PBMI) brand. Conducted from Sept. 20, 2022, through Oct. 21, 2022, the survey included 182 benefits leaders from employers, unions/Taft-Hartley plans and health plans representing plan sponsors of approximately 86.7 million covered lives. Genentech USA, Inc., a member of the Roche Group, co-sponsored the report with PSG.

Celltrion Healthcare Co. launched Vegzelma (bevacizumab-adcd), the company said on April 17. The FDA approved the injectable last September for multiple types of cancer, including colorectal and non-small cell lung cancer. It is the fourth FDA-approved biosimilar of Avastin (bevacizumab) from Genentech USA, Inc., a member of the Roche Group, to launch in the U.S. While the company has partnered with other companies in marketing biosimilars in the U.S., its Celltrion USA unit is taking full responsibility for this launch.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 11-1 that AstraZeneca and Merck & Co., Inc.’s Lynparza (olaparib) in combination with abiraterone and prednisone or prednisolone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) should be limited to people whose tumors have a BRCA mutation. The FDA accepted a supplemental New Drug Application for a broader approval in mCRPC for the poly (ADP-ribose) polymerase (PARP) inhibitor last August. The class of drugs — which also includes GDK’s Zejula (niraparib) and Rubraca (rucaparib), whose rights Clovis Oncology, Inc. recently sold to pharma& Schweiz GmbH a few months after it filed for Chapter 11 bankruptcy — have been under some scrutiny after they withdrew some of their indications.

Biomarker testing is an important tool in cancer care, but a recent survey found payer coverage issues are creating access barriers. According to CancerCare, researchers found that biomarkers helped providers offer personalized care for various cancers for nearly all — 93% — respondents. Twenty percent of surveyed patients were able to forgo unneeded chemotherapy and/or radiation, while 10% found that they were eligible for a clinical trial.

However, the survey also found that 29% of people who had biomarker testing had insurance that did not cover it, prompting them to undergo appeals, obtain financial assistance or pay out of pocket for the service. In addition, 25% of patients said that their insurer required prior authorization (PA) for the process, a tactic that can delay access to treatment.

March 10: The FDA approved Acadia Pharmaceuticals Inc.’s Daybue (trofinetide) for the treatment of Rett syndrome in people at least 2 years old. The drug is the only FDA-approved treatment for the complex, rare neurodevelopmental disorder, which is estimated to affect 6,000 to 9,000 people in the U.S. The agency gave the drug fast track status and orphan drug designation; it also gave the company a rare pediatric disease priority review voucher. Dosing of the oral solution is weight based and can be given orally or via gastrostomy tube. The company has not disclosed the agent’s price, but analyst estimates are between $400,000 and $600,000 per year. Acadia said it expects the agent to be available by the end of April.

March 13: The FDA expanded the patient population of Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) to include the treatment of cholestatic pruritus in people with Alagille syndrome who are at least 3 months old. The agency initially approved the oral solution on Sept. 29, 2021. The starting dose is 190 mcg/kg once daily and then increased to 380 mcg/kg after one week. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $53,712.

In late 2022, the FDA approved the first gene therapy for bladder cancer, Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg). Almost three-quarters of oncologists surveyed by Zitter Insights expressed at least moderate interest in the agent, and payers said they expect to manage the drug to label. While the drug gives another treatment alternative to a much-needed area, it likely will continue to put financial pressure on payers, industry experts tell AIS Health, a division of MMIT.

On Dec. 16, the FDA approved Adstiladrin for the treatment of adults with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. The agency gave the novel adenovirus vector-based gene therapy priority review, breakthrough therapy and fast track designations. Dosing is once every three months into the bladder via a urinary catheter. The company said it expects the therapy to be available in the second half of 2023.

The FDA will withdraw Makena (hydroxyprogesterone caproate) from the U.S. market, the agency said April 6. The move follows manufacturer Covis Pharma Group saying on March 7 that it had requested an “orderly wind-down” of the drug from the market. However, “effective today, Makena and its generics are no longer approved and cannot lawfully be distributed in interstate commerce,” said the agency in its statement. “Approvals of these drugs have been withdrawn because the drugs are no longer shown to be effective and the benefits do not outweigh the risks for the indication for which they were approved.”

The decision brings to a close a long-running saga that started more than a decade ago, as well as shines a spotlight on the FDA’s often-criticized accelerated approval pathway.