(Bloomberg Businessweek) -- Opioids. They’re a public-health pariah, leading to more than 80,000 overdose deaths a year. Patients worry they’ll get addicted to them. Doctors want nothing to do with them. And politicians of all stripes are calling for less dangerous options for treating pain.
“We are looking for absolutely anything that’s not an opioid,” says Seth Waldman, an anesthesiologist and director of pain management at Hospital for Special Surgery, a top orthopedic medicine center.
Against that backdrop, the success of a safer painkiller would seem assured. A new drug, which Vertex Pharmaceuticals Inc. is developing, has been hailed as a scientific breakthrough because it treats pain without entering the brain, where opioids create addicts. The drug, suzetrigine, met its goal this year in pivotal trials for acute pain and is poised to become the first new class of pain medication in more than two decades.
But all that may not be enough to loosen the grip opioids have on American medicine. Despite their dismal reputation, they have two powerful things going for them: They’re cheap, and they work. The number of opioid prescriptions has been cut by half over the past decade, but some 130 million are still doled out each year.
“Opioids are the most effective fast-acting meds that we have,” says Gerard Limerick, an assistant professor at Johns Hopkins Medicine.
Usually, a new drug must outperform what’s already on the market to be widely prescribed by doctors and paid for by insurers. Vertex’s drug didn’t quite do that. In late-stage studies of patients with acute pain after surgery, suzetrigine worked better than a placebo. But it failed to reduce bunion surgery patients’ pain faster than a combination of an opioid and acetaminophen, raising questions about its commercial potential.
“It’s hard to make a case for denying a patient a drug that has clearly superior efficacy to everything else out there,” Brian Skorney, a biotech analyst at Baird, wrote in a note this year about Vertex’s pain studies. “It’s much easier to deny an expensive branded drug when there are a myriad of effectively free, generic options, some of which have now been shown to even be superior to the expensive brand.”
Health insurers often require patients to pay a higher copay for a branded drug and try a cheaper generic first, a practice known as step therapy. And hospitals typically get the same payment from Medicare whether a doctor prescribes an opioid or a non-opioid, creating an incentive to go with the cheaper, more addictive option.
For instance, an intravenous version of acetaminophen during surgery costs more than $40, whereas a morphine drip costs about $1, says Jianguo Cheng, a professor of anesthesiology at the Cleveland Clinic. “The reimbursement won’t increase just because you use $45 acetaminophen,” he says.
Vertex is betting the nonaddictive properties of its drug will make it an attractive alternative to opioids. But it isn’t leaving anything to chance. Knowing it faces an uphill battle commercially, the company last year boosted its lobbying spending almost 50%, to more than $3 million. Vertex is pressing Congress for new policies that remove “structural impediments” blocking access to opioid alternatives, says Stuart Arbuckle, its chief operating officer.
Vertex has scored at least one legislative victory: The No Pain Act, which goes into effect next year, provides an extra Medicare reimbursement to hospitals that prescribe alternatives to opioids. Another bill introduced this year in Congress would prohibit step therapy and limit how much Medicare patients pay out of pocket for non-opioids.
Other types of new drugs face this cost hurdle, too. The rollout of new contraceptives or antibiotics has been hampered by insurers guiding doctors and patients to older, less pricey meds. The difference is that those generics haven’t been declared a public-health emergency.
Without new laws to promote the use of alternative painkillers, insurers will continue to steer patients to lower-cost opioids and fuel the addiction crisis, says Chris Fox, executive director of the nonprofit Voices for Non-Opioid Choices. And even with new legislation, other obstacles remain. Getting doctors to prescribe a drug such as Vertex’s may require a massive reeducation effort. “The challenge we have is, by and large, our health-care workforce is trained to treat pain with opioids,” Fox says.
Each year, about 80 million Americans are prescribed a medicine for acute pain, caused by the likes of broken bones, burns and the aftermath of surgeries. Some 50 million more have diabetic nerve damage or other types of chronic pain that lasts at least three months, an area Vertex is also studying. For the most part, patients have only two treatment options: anti-inflammatory drugs like ibuprofen, which aren’t very strong, or opioids, which are highly addictive. They frequently fight with insurers over coverage of safer pain medications, which Waldman calls “a cruel joke.” “There are times when we can’t get a non-opioid medication approved, but we can get approval for an opioid,” he says.
Vertex isn’t the only biotech company looking for an opioid alternative. Earlier this year, Latigo Biotherapeutics Inc. started with $135 million in funding to find a nonaddictive painkiller. South Rampart Pharma Inc. recently completed an early-stage trial for a nonaddictive pain drug that works through unique pathways in the brain and aims to be safer and more effective than Tylenol, ibuprofen and other traditional medicines, says Hernan Bazan, the company’s chief executive officer.
One of the furthest along is Tris Pharma Inc., which is starting late-stage trials in treating acute pain and hopes to develop the same drug for chronic pain and opioid use disorder, says James Hackworth, president of the company’s brand division. If Tris Pharma can prove its drug works and is nonaddictive, it can be scheduled differently by regulators so it’s easier to dispense and prescribe than opioids, which have restrictions, he says.
Founded in 1989, Boston-based Vertex transformed into a biotech giant with almost $10 billion in 2023 sales after turning cystic fibrosis from a debilitating lung disease into a manageable condition. Now it’s trying to diversify into other treatments, including for diabetes and sickle cell disease. Developing a nonaddictive pain drug, which Vertex has studied for more than two decades, has been “one of the holy grails in pharmaceutical research,” says Paul Negulescu, a senior vice president at Vertex.
Vertex’s drug blocks a sodium channel that helps control activity in the body’s neurons and sends signals to the brain when you’re in pain. Wall Street is giddy about its prospects: Vertex’s stock soared about 40% over the past year, adding about $40 billion to the company’s value, largely due to optimism about the pain med, expected to hit the market in 2025.
Still, Negulescu remains realistic. “We don’t imagine we’ll replace opioids with this medication,” he says. Instead, the company’s goal is “to reduce the need to have to take them.”Read next: Why Prescription Drug Prices in the US Are So High
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