Specialty Drugs

In a move derided by some health care stakeholders and applauded by others — including private payers — CMS on April 7 finalized its National Coverage Determination (NCD) for an Alzheimer’s treatment that has been steeped in controversy since it received accelerated approval last June.

The final coverage decision largely affirmed what CMS had proposed in its draft NCD: Medicare will cover Biogen Inc.’s Aduhelm (aducanumab) only for patients enrolled in randomized, controlled clinical trials conducted either through the FDA or the National Institutes of Health. Patients also must have a clinical diagnosis of mild cognitive impairment due to Alzheimer’s disease or mild dementia with a confirmed presence of plaque on the brain.

With its Feb. 28 approval, the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.’s Carvykti (ciltacabtagene autoleucel; cilta-cel) becomes the second chimeric antigen receptor T-cell (CAR-T) therapy to treat multiple myeloma. Payers report being less likely to prefer it over some or all other multiple myeloma treatments with a similar indication, but oncologists are showing more enthusiasm for prescribing the new agent, according to Zitter Insights.

The FDA approved Carvykti for the treatment of adults with relapsed or refractory multiple myeloma after at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. The product is a B-cell maturation antigen (BCMA)-directed CAR-T agent. The one-time treatment will have a phased launch and will be available through a limited network of certified treatment centers. The FDA gave the drug breakthrough therapy and orphan drug designations. The therapy’s wholesale acquisition cost is $465,000.

Optum, Inc. recently unveiled Optum Specialty Fusion, a specialty drug management solution that’s focused on streamlining care for people on those products and lowering medication costs. According to the company, which is part of UnitedHealth Group, the approach has the potential to deliver 17% total cost savings in health plans’ medical and pharmacy spend.

Providers are able to request prior authorization for specialty agents via a portal, which then compares treatment options, including their costs, across both the medical and the pharmacy benefit. According to the company, “informed by Optum data and insights, Specialty Fusion provides savings options such as preferred products, dosage management policies, best sites of care and additional cost-control levers such as available discounts and pharmacy networks.” The solution narrows down treatment options to the ones or one that “makes the most clinical and financial sense” and provides approval for that treatment in real time, regardless of the benefit the agent falls under. Optum maintains that this approach lessens “administrative hassle and results in an expected 50% faster access to therapy for patients, while lowering costs.” Specialty Fusion is available for large health plans.

When the COVID-19 pandemic struck, to say the health care system was disrupted is an understatement. Many people undergoing treatment for cancer rightfully were concerned about their potential exposure to the virus and were hesitant to leave their homes for care. In order to continue treating some patients, Horizon Blue Cross Blue Shield of New Jersey partnered with Rutgers Cancer Institute of New Jersey (CINJ) and RWJBarnabas Health to start a pilot to offer home infusion of cancer treatments and telemedicine support for eligible people, the first such program in the state.

In addition to not putting people at risk of acquiring an illness, home infusion means that people don’t have to travel for treatment. It also provides them with one-on-one care, close monitoring during infusions and the ability to schedule treatments around their personal schedule. These benefits are especially important for people with compromised immune systems.

The FDA has approved a handful of drugs to treat atopic dermatitis recently. Among them is LEO Pharma Inc.’s Adbry (tralokinumab-ldrm), an interleukin-13 (IL-13) antagonist. According to a Zitter Insights survey, payers may take a bit of a restrictive approach in managing the drug. And with multiple new biologics approved for the condition and more potential agents coming onto the market, payers may impose more utilization management strategies on the therapeutic class as a whole, say industry experts.

On Dec. 28, the FDA approved Adbry for the treatment of people at least 18 years old with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those treatments are not advisable. The decision made it the first biologic that LEO Pharma has launched in the U.S. Recommended dosing is an initial dose of 600 mg via four 150 mg subcutaneous injections and then 300 mg every other week.

Magellan Health’s PBM, Magellan Rx Management, recently unveiled a partnership with Zipline that will deliver prescription medications, including specialty drugs, to patients’ homes through the use of autonomous aircraft. The program will provide convenience to customers, maintains Mostafa Kamal, CEO of Magellan Rx Management, as well as reduce emissions compared with traditional delivery methods.

Social determinants of health (SDOH), which researchers have suggested account for up to 80% of health outcomes, can also complicate medication adherence, which can be particularly challenging for people taking specialty drugs. Specialty pharmacies are uniquely suited to address these issues and provide the support and resources that their patients need to overcome barriers to effective treatment, industry experts tell AIS Health, a division of MMIT.

According to the Healthy People 2030 initiative from HHS’s Office of Disease Prevention and Health Promotion, SDOH “are the conditions in the environments where people are born, live, learn, work, play, worship, and age that affect a wide range of health, functioning, and quality-of-life outcomes and risks.” These factors can be grouped into five areas:

Feb. 17: The FDA approved Agios Pharmaceuticals, Inc.’s Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It is a first-in-class, oral PK activator and the first FDA-approved disease-modifying therapy for the disease. The agency gave the drug priority review and orphan drug designation. Dosing for the tablet is 5 mg twice daily. Its annual cost is $334,880.

Feb. 21: The FDA gave an additional approval to Foundation Medicine, Inc.’s FoundationOne CDx as a companion diagnostic to identify people with microsatellite instability high status solid tumors who may be candidates for treatment with Merck and Co., Inc.’s Keytruda (pembrolizumab). The company says it is the first FDA-approved diagnostic for this use. The CDx has 26 companion diagnostic claims and two group claims across 27 targeted therapies.

Teva Pharmaceuticals Ltd. launched the first generic version of Bristol Myers Squibb unit Celgene Corp.’s Revlimid (lenalidomide) in 5 mg, 10 mg, 15 mg and 25 mg strengths in the United States on March 7. The FDA approved the drug from Teva U.S. affiliate Arrow International Ltd. and Natco Pharma Ltd. on May 21, 2021. The companies have tentative approval for the 2.5 mg and 20 mg strengths due to an exclusivity issue: The FDA has approved Dr. Reddy’s Laboratories Ltd.’s lenalidomide for those dosages. The product is approved for three indications: (1) multiple myeloma in combination with dexamethasone, (2) transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with a deletion 5q abnormality with or without additional cytogenic abnormalities and (3) mantle cell lymphoma that has relapsed or progressed after at least two treatments, including bortezomib. The launch is limited, and through an agreement with Celgene, the companies are allowed to sell “mid-single-digit percentages” of Revlimid’s total volume this month, a figure that gradually will increase to one-third of the volume. Beginning Jan. 31, 2026, Teva can sell the drug without volume limitation. Multiple companies are expected to launch Revlimid generics in the U.S. this year.

When the first targeted oncology therapy — Novartis Pharmaceuticals Corp.’s Gleevec (imatinib) — came to market in the U.S. more than 20 years ago, industry experts believed it was ushering in a slew of similar agents focused on a particular mutation, or biomarker. The field, however, did not quite live up to lofty expectations. And while more research has grown the industry exponentially in recent years, it still faces some challenges. But no one can deny that biomarkers have had a profound impact on cancer care.

Agents that target drivers of cancer “have fundamentally changed the way that we treat a wide range of cancers,” maintained Josina Reddy, M.D., Ph.D., global head and vice president of product development for lung, agnostic, skin and rare cancers at Genentech, Inc., a member of the Roche Group, during a recent webinar sponsored by her company. “And in this era of cancer care, identifying those biomarkers in a patient’s tumor tissue or using blood is an essential step in developing a personalized treatment plan, and for a growing share of patients, that treatment plan might include those targeted therapies.”