Radar on Specialty Pharmacy

Less than a year after the FDA approved the first chimeric antigen receptor-T cell therapy without a Risk Evaluation Mitigation Strategy program, the agency has eliminated the REMS for the other six CAR-Ts. At the same time, the FDA loosened other restrictions on patients, moves that should increase uptake of the agents, say industry experts.

“A REMS is no longer necessary to ensure that the benefits of these CAR T cell immunotherapies outweigh their risks and to minimize the burden on the healthcare delivery system of complying with the REMS,” said the FDA on June 26. The programs initially were in place because of the risks of cytokine release syndrome (CRS) and neurologic toxicities, including immune effector cell-associated neurotoxicity syndrome (ICANS), potentially fatal reactions.

People who undergo treatment with cell and gene therapies (CGTs) face a long, complex and often grueling process, from sickness to diagnosis to pretreatment requirements to the treatment itself to post-treatment monitoring and follow up. On top of all that, many people must grapple with a consideration that for some means walking away from treatment: the loss of fertility.

Before people can be treated with many of the available CGTs, they must undergo myeloablative conditioning, which involves being administered high doses of chemotherapy or radiation to destroy their bone marrow in order to cut down on the chance of their immune system rejecting the new cells. Unfortunately, infertility very often is a complication of this process, which may be a reason for people to not undergo treatment.

In a first, the FDA recently approved a drug administered every six months as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV. While some potential speed bumps to uptake exist, the therapy’s efficacy and less onerous dosing schedule compared with other similar medications could spur its use, making a tremendous difference in the fight against the infectious disease.

On June 18, the FDA approved Gilead Sciences, Inc.’s Yeztugo (lenacapavir) as PrEP to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35 kg. A health care provider administers the medication via subcutaneous injection into the abdomen or thigh. With the initial injection, people also take two tablets of the medication on days one and two, and then subsequent dosing is an every-six-months injection.

Many ultra-high-cost cell and gene therapies come with the promise of a cure — but it’s not that simple. It’s unclear with many of the agents exactly how long that “cure” will last. And the process a person must go through both before and after treatment is an extensive one, necessitating coordination among various components. So while many employers may not have had a claim for one of these products, they still need to prepare for that possibility.

“These are not the typical therapies that we’ve seen in the past where you get a pill or you inject a drug, and you keep it at home, or you go to a location near your home and have the drug administered like a specialty drug for cancer or something,” explained Tom Sondergeld, managing partner at TS Consulting Group LLC, during a June 5 webinar from the Midwest Business Group on Health (MBGH). “This is much more advanced.”

As insurers pledge to reduce the number of medical procedures subject to prior authorization (PA), industry experts say those efforts could trickle down to prescription drugs. According to Avalere Health research, Medicare Advantage plans’ increasing reliance on step therapy (ST) for Medicare Part B-covered therapies has become particularly burdensome for patients and providers, highlighting one potential reform target. But a policy banning its use was reversed by CMS during Donald Trump’s first presidency, and an effort to undo that during the Joe Biden administration did not pan out.

In the prescription drug benefit, both PA and ST are utilization management (UM) tactics used by insurers to guide enrollees toward more cost-effective therapies. ST typically requires patients to try a lower cost alternative or a drug with preferred formulary status before moving to the therapy their physician initially prescribed.