Commercialization, marketing and social media

Madrigal Pharmaceuticals’ March approval for its metabolic dysfunction-associated steatohepatitis treatment Rezdiffra marked a significant milestone. Not only was Rezdiffra the first drug to make it to market for Madrigal, it was also the first-in-disease approval for MASH, previously known as NASH, the most severe form of nonalcoholic fatty liver disease that affects an estimated more than 115 million people globally.

Since launching in April, Rezdiffra sales have already been promising, besting Wall Street analysts’ estimates during the second quarter of 2024 and generating $14.6 million in the U.S., Madrigal reported in its quarterly earnings. Despite the disease’s wide patient population, Madrigal is focused first on capturing 315,000 patients diagnosed with MASH with moderate to advanced liver fibrosis who are already under the care of a liver specialist.

That targeted patient population has been at the core of Madrigal’s marketing strategy and launch campaign, dubbed “Way Forward,” according to Kristin Bucklen, vice president of consumer marketing at Madrigal. 

“We're not trying to drive the masses,” she said. “The intention to be so focused with a specific population is something that I think sets us apart from a lot of medicines.”

The campaign, which was “a year-plus in the making,” has been active for about six weeks, according to Bucklen. But it’s not just diagnosed patients Madrigal is aiming to reach.

“Our other target patient actually thinks they just have a fatty liver and don't realize they could already have [MASH], which is a much more serious condition with even more serious consequences,” Bucklen said. “They might not realize they have [MASH] because from the patient perspective, [it] didn't resonate. They didn't connect with the words [MASH]. They [are] more connected with this idea of, ‘it's just a fatty liver.’”

The two sets of patients have their own challenges for Madrigal to overcome. For those who know they have MASH, it’s about medication awareness.

“With Rezdiffra, part of the challenge is because [MASH patients have] been told that there hasn't been anything for them — all they had was diet and exercise — they don't know how to look.”

For patients who think they just have a fatty liver, Madrigal has to educate them about MASH, a more serious condition that, once understood, can kick patients into action to have treatment discussions with their doctor.

“It could progress to cirrhosis or liver transplant, which we have found is quite motivating for patients,” Bucklen said.

On the physician side, there is “an equivalent campaign” to ensure doctors know Rezdiffra is available and there is a MASH treatment option for the first time, Bucklen said.

Madrigal’s ‘way forward’

In order to reach its target patient population, Madrigal decided to go all digital in its initial campaign launch with targeted internet ads and connected TV spots.

“Our CEO has communicated very clearly that the overall research that's been done from a corporate perspective says there are approximately 315,000 patients in the U.S., so it just makes sense to be very focused and targeted,” Bucklen said.

When it came to messaging, Madrigal engaged “hundreds” of MASH and fatty liver disease patients and asked what they wanted out of a MASH treatment.

“What struck me is how hopeless patients said they felt in the absence of a medicine,” Bucklen said. “Frankly, diet and exercise is not very specific in terms of being actionable for patients.”

Patients overall said they felt stuck with their diagnosis and were looking for a way forward. That sentiment became the heart of the drug’s messaging. 

“They said that they wanted to feel strength, control and confidence … so that they could do the things that they love with the people that they love,” Bucklen said.

Leveraging patient ambassadors, Madrigal hopes the ads will help other patients see the lived experience of MASH.

“I have [MASH], a serious form of fatty liver disease that’s scarring my liver,” a patient says in one of Madrigal’s ads. “I was doing all I could and it wasn’t enough.”

After Rezdiffra’s strong start in the U.S., Madrigal is pursuing commercialization in Europe as well, CEO Bill Sibold said in August, with an approval decision from the European Medicines Agency expected in mid 2025.

At a company the size of Johnson & Johnson, it would be easy for departments to become siloed, accomplishing only their own objectives without a vision of the bigger picture. But even in the earliest stages of science, units as fundamentally divided as clinical research and commercial strategy work together to achieve the pharma giant’s common goal.

As J&J pursues clinical results, Dr. Craig Tendler, vice president of late-stage development, diagnostics and global medical affairs in oncology, has to consider not only what’s happening in a non-small cell lung cancer trial but what the commercial landscape will look like so patients can ultimately benefit.

“The way I look at the commercial contribution before we even get on the market is trying to define those key attributes that are important to physicians and patients and being able to integrate that into a development program where the results will hopefully support that profile,” Tendler said. 

So he teams up early with Biljana Naumovic, J&J’s president of U.S. solid tumor oncology, to ensure everyone is on the same page. Along with the research teams, Naumovic aims to define those key attributes at the outset.

“The primary end goal is to transform the lives of patients by changing the standard of care and supporting a long, fruitful quality of life,” Naumovic said. “We’re part of this discussion at the very beginning, so that before we go into the clinic, we’re defining where we want to go — for the past 20 years, we have done just that with very informed, passionate debates.”

If a program looks like it’s not going to change the standard of care, “we stop things that we do not think are going to hit the mark,” Naumovic said.

‘A huge responsibility’

When the baton makes its way from the clinical research teams to the commercial department, everyone needs to be on the same wavelength, Naumovic said.

“Bringing really strong data to then manage patients better is a huge responsibility,” Naumovic said, referring to the August FDA approval that stemmed from overall survival data in a late-stage study for EGFR-mutated non-small cell lung cancer with the drug combo of Rybrevant and Lazcluze compared with AstraZeneca’s Tagrisso, the current standard of care.

On the research side, patients stayed on the Rybrevant and Lazcluze combination treatment longer than with Tagrisso, Tendler said, noting that the combo provided better disease control, which is a selling point down the road. And that arises from early representation of the commercial perspective in clinical program design, he said.

“The beauty of having the commercial representation is that we have better insight as to what attributes should be integrated into the comprehensive development program,” Tendler said. “This idea that we have to look to not just bring a regimen that has enhanced progression-free survival but the factor that will convince physicians to use it in a higher proportion of patients is the survival data.”

At a conference with clinical experts like this past weekend’s annual conference of the European Society for Medical Oncology, Naumovic’s job is to distill the next steps into a commercial plan.

“Once the data is out, the oncology community has a million ideas for where to take it next,” Naumovic said. “And so we can create that together.”

In bladder cancer, for instance, J&J reported mid-stage data for a device called TAR-200 designed to sit in the bladder and release chemotherapy from within, showing a response rate of more than 83%.

“It’s a very clear example of an innovation being able to deliver an older drug in a new delivery device and having it get much more effective levels throughout the treatment cycle,” Tendler said.

Results like that give Naumovic some buzz to work with when the commercial strategy comes into play.

“This has never been seen before in localized bladder cancer, and to get there with a beautifully engineered device that stays in the bladder will be a radical change to what bladder cancer looks like once we bring it to the U.S. market in the beginning of next year,” Naumovic said.

The commercial influence

While science can only be done at its own pace, commercial demand can sometimes influence the direction in which a research team takes aim. A rise in colorectal cancer among young patients has troubled oncologists and caused companies like J&J to investigate new treatment options.

In a phase 1b/2 study for patients with metastatic colorectal cancer, a combination of Rybrevant and chemotherapy resulted in an almost 50% overall response rate.

“There has been no innovation in colorectal since [Eli Lilly’s Erbitux], and we’re talking several decades,” Naumovic said. “So like we did in bladder cancer and lung cancer, we’re coming to colorectal cancer the same way — there certainly is a resurgence of higher incidence in a younger population across the developed world because of diet, and we want to make sure we’re embracing that opportunity with the results that we have.”

Overall, J&J’s clinical and commercial teams have the same endpoints in mind — finding the right approaches to move the oncology field forward while keeping today’s patients at the center of the equation.

“We are a company that aims to put chemotherapy into the history museums where it belongs and focus on targeted therapies that will allow patients to live a longer and better life,” Naumovic said. “That’s where we think the field of oncology is going.”

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Every time a new multimillion-dollar drug is introduced to the market, headlines trumpet the cost. And while eye-popping price tags look exorbitant on paper, the reality behind these figures is much more nuanced.

The most expensive drugs are gene therapies, typically one-and-done treatments aimed at rescuing patients from a lifetime of repeated medical interventions. Prices are set based on the overall savings throughout a patient’s journey and intended to represent the high value of the drugs and their long road to development.

Even though these treatments could be curative — assuming the therapeutic effect holds up over time — patients haven’t always clamored to take them.

Orchard Therapeutics’ $4.25 million Lenmeldy became the most recent gene therapy to take the “world’s priciest drug” mantle when it was approved in March for metachromatic leukodystrophy. The company has outlined its launch plans, including work with payers to implement innovative reimbursement structures that “appropriately balance the needs of patients and families for adequate access,” but it’s too soon to know how well it will be received in the marketplace.  

But other gene therapy stories hint at the potential uptake challenges ahead. Here’s a look at the sales performance for some of the most innovative and expensive therapies approved in recent years.

Casgevy

List price: $2.2 million

Vertex Pharmaceuticals and CRISPR Therapeutics in December scored the first FDA approval for a CRISPR-based drug. Initially indicated for sickle cell disease, the companies won an additional nod for beta thalassemia a month later.

The groundbreaking therapy has been on the market for several months, but the companies are still counting cells — not dollars — when it comes to measuring uptake.

As of mid-July, 20 patients have had their cells collected for treatment, CRISPR Therapeutics reported in its second quarter earnings. And more than 35 authorized treatment centers have come online to expand access.

Although neither company has reported specific sales numbers for Casgevy, Vertex raised its full-year sales forecast this month by $100 million to almost $11 billion. While growth is being driven by the company’s cystic fibrosis portfolio, Vertex also pointed to an expected uptick in Casgevy demand as a reason for the boost, and said it has identified about 35,000 potential patients for the treatment.

Analysts also predict Casgevy sales will pick up in the third quarter and could ultimately hit about $81 million.

Hemgenix

List price: $3.5 million

CSL Behring raised its profile when it won an FDA OK for Hemgenix in 2022, the first gene therapy of its kind for hemophilia B. Still, sales reports suggest uptake has been slow.

Although CSL is forecasting revenue growth across its extensive hemophilia portfolio from $1.19 billion in 2023 to $1.3 billion this year, the company has yet to list specific sales results for Hemgenix in any of its financial presentations.

The company won regulatory coverage in England and Denmark for Hemgenix under an outcomes-based payment model earlier this year, which could help the treatment gain traction outside the U.S. A company spokesperson also told PharmaVoice it’s seen an “acceleration” of patients being infused with Hemgenix as it works to educate the hemophilia community about the treatment and win 90% coverage from U.S. payers. 

“Even more encouraging is the continued inquiries from patients and physicians, who are actively engaging CSL Behring about Hemgenix infusion readiness and scheduling as well as positive feedback from the hemophilia treatment centers prepared to administer doses,” the company spokesperson said in an emailed statement. 

Roctavian

List price: $2.9 million

Hemgenix isn’t the only gene therapy struggling to build momentum in the hemophilia market.

Since the FDA approved BioMarin Pharmaceuticals’ Roctavian for severe hemophilia A last year, sales have been so sluggish the company has had to rework its commercialization strategy.

Just five patients — three in the U.S. and two in Italy — took Roctavian in the second quarter, BioMarin reported, generating $7 million in revenue. Given the high operating expenses associated with producing the treatment, the company is now limiting access to three countries — the U.S., Germany and Italy. Ultimately, the goal is to slash Roctavian-related costs by $60 million and make the treatment profitable by the end of 2025, the company said.

BioMarin CEO Alexander Hardy also indicated earlier this year that the treatment could ultimately be divested from the company’s portfolio.

Elevidys

List price: $3.2 million

Analysts once pegged Sarepta Therapeutics’ Elevidys as a gene therapy that could defy larger uptake trends and soar to blockbuster heights. But so far, sales for the Duchenne muscular dystrophy treatment approved in 2023 have landed below some analysts’ expectations.

Elevidys pulled in about $121.7 million in revenue in the second quarter — down from about $133.9 million in the first.

On an earnings call, Sarepta CEO Doug Ingram blamed flat sales on the months-long approval process for the therapy, rescheduled infusions and other treatment delays.

Company executives also put a positive spin on the situation by pointing to several indicators Elevidys sales could ramp up, including a recent expanded label approval, an increase in demand for the antibody testing kits needed for the treatment, along with “unprecedented demand” at treatment centers. Overall, company leaders predicted a one-third sales jump in the third quarter, and another doubling of revenue in the final three months of the year.

“With the broader label granted in June of this year … the opportunity to serve patients and in so doing reward committed investors will be enormous,” Ingram said during the call.

Drug launches are undergoing a shift with patent cliffs, regulatory oversight and a tight investment environment nipping at the heels of commercial strategies.

Amid the headwinds, pharma is transitioning into a recognizable cycle, said Jim Lang, CEO of commercial services company Eversana, where companies are slimming R&D efforts and putting budget-focused thinkers in leadership roles. 

"We are actually excited by a move we're seeing in large pharma, which is they're putting attention at the CFO and procurement level on their whole commercialization spans,” he said. 

Rising competition in the market for personalized and precision medicine and a shift toward patient-centered care are driving those changes, he said. Examining some of the ongoing challenges for drugs coming to market in emerging therapeutic areas highlights how company strategies could take shape for the rest of the year and into 2025.

Here, we caught up with Lang to discuss challenges in pharma commercialization, drug launch failures and why launch strategies need to start years before hitting the market.

This interview has been edited for brevity and style.

PHARMAVOICE: What’s changed in the commercialization market recently?

JIM LANG: The years 2021 and early 2022, as far back as 2019, were uniquely positive for our industry. We had very low loss of exclusivity, meaning the percentage of revenue [lost] to patent life expiration. We also had an exceedingly well-funded — and people would argue now in hindsight over-funded — biotech market. Naturally when you have these highs, you tend to then flip to some low cycles immediately after.

We've now had about 18 months of very low biotech funding, [which] is now coming back. And then we had our whole industry process the impact of increased loss of expirations. Those [LOEs] are going up to about 4% to 6% of the revenue annually, where it was like 1% to 2% in those prior years. 

[There’s] been hundreds of companies over these past few years that have had layoffs or even outright shutdowns. Pfizer [and] BMS [recently] announced some cuts. I think we’ll see through the rest of the year the final group of folks making decisions about allocations of portfolio and cost cutting, and then 2025 will be a return to normal.

What are some of the challenges you’re seeing in drug launches?

The industry is tackling rare disease. That's exciting, but by definition, those are all smaller populations. And that inherently means you have a much smaller portion of the population you're attacking, and you need to be much more nimble. In reinventing your commercial models, you need to be more thrifty, you need to be more agile. And you know, it's a lot more difficult to be economically successful when your peak sales are $300 million versus when your peak sales are $5 billion.

Are there any spectacular commercialization failures in the last few years that you see as a key learning opportunity for the industry?

Commercializing a drug is not an easy task. In fact, nearly two-thirds of launches fail to meet expectations. Yet, two come to mind that our team is familiar with that reinforce just how challenging it is.

The first was for a blockbuster drug about five years ago to treat macular degeneration, an eye condition. It brought great potential to the market, but safety risks hampered its launch. Analysts expressed concern, and six months after launch the American Society of Retina Specialists issued a warning against the therapy, creating major challenges for growth. The drug never met expectations.

A second drug that comes to mind was developed to prevent hallucinations in patients with Parkinson’s. The drug also had safety concerns, and the company invested in an educational campaign to support its growth. This backfired in the form of a federal lawsuit, creating even more challenges. 

Many factors go into a successful launch, but ensuring the product is ready to meet the needs of patients and is supported throughout the launch process from educating healthcare professionals, patient communities, payers and more is critical.

As a category, digital therapeutics have had a really hard time demonstrating their value to payers. [It] was the darling of investors for the last maybe eight years, but hasn't been for the last year and a half. But it's, I would argue, because they didn't pay attention to the payer side nearly as much as the health economic argument.

How do you think about pre-launch expectations from Wall Street? There are some studies that show about 40% of drugs underperform their sales forecasts. 

The conclusions are often that the experienced launchers do better than inexperienced launchers. If you're a first time emerging biotech company, and you're tackling this on your own, your odds are against you relative to experienced launchers.

The studies concluded that those who started their work two and a half years before launch, versus more like one year before, were radically different in their commercialization success. Big Pharma knows that, and that's why their processes kick off three years before launch. The challenge in emerging biotechs is almost all of them are on lifelines for financial investment. It even got worse these last two years. By definition, they're gonna have lower odds of success. 

There's always what I call the bias inherent in those studies because expectations are nothing more than the collective forecasts of analysts, including the company around that drug. And there's a lot of reasons why people are biased upward early on. Their whole story to this day has been [about] attracting talent and raising money. Of course, you're gonna paint the high scenario. 

What's your advice to those emerging biopharma companies that may be reliant on an investment line?

Don't wait. Even if you can't afford much, start with what we all know is the most critical path, which is the medical affairs or key opinion leaders to warm the market, talk to payers and make sure you're going to get reimbursed for these particular products. Those don't often have to cost that much, but they're the critical path work that often people wait too long on.

What else is trending in commercialization?

You hear a lot of noise right now about the direct-to-patient model. The old days were about healthcare professional-centered design, where the doctor was the center of how you thought about commercialization. There were certainly other constituents you had to influence the payers, regulators, the patient's themselves and the engines around doctors like their integrated delivery systems. But it's required a big change to [center] around the patient. And if you talk to leading companies who are wanting to do this now, they realize if they center their thinking around the patient and make patient experience spectacular, they'll drive better outcomes and deliver solid sales for the product at a lower cost.

If drugmakers have learned anything in the last few years about the Alzheimer’s market, it’s that an FDA approval is by no means a guarantee of success.

So as Eli Lilly celebrates last week’s green light for the amyloid-plaque clearing Kisunla — formerly known as donanemab — there’s still work to be done to ensure patients who qualify can access the drug.

Predecessors in the space have had mixed success. From the outright disastrous launch of Biogen and Eisai’s Aduhelm, which payers declined to offer on the basis of muddy efficacy and safety, to the same duo’s Leqembi, which was met more positively but has been slow to meet market expectations, Alzheimer’s has been a tough nut to crack.

Can Lilly make a bigger splash? Some market watchers think it’s possible despite clinical results that showed a risk of brain swelling called ARIA in a subset of patients. Like Aduhelm and Leqembi, the drug’s label is limited to patients in early stages of the disease and with confirmed amyloid plaques in the brain.

For one reason in particular, though, Kisunla stands out in the crowd.

“Kisunla has Lilly behind it,” said Jayne Hornung, chief clinical officer at MMIT, a market access consultancy. “That’s a powerhouse that literally knows how to bring a drug to market in the face of [adverse drug reaction] information.”

Kisunla is estimated to reach $2 billion in annual sales by 2029, according to a July 2 note from Cantor Fitzgerald analyst Louise Chen. That’s similar to Cantor’s Leqembi estimates of about $2.2 billion in the same time frame despite hitting shelves more than a year earlier than Kisunla.

Lilly “is a marketing machine, and it will be interesting to see how the launch will progress relative to expectations,” Chen wrote.

Kisunla carries a slightly higher price tag than Leqembi at $32,000 for a year’s worth of 13 infusions, compared to Leqembi’s $26,500. However, Kisunla can be stopped once plaques have cleared, whereas Leqembi is infused every two weeks indefinitely. In Kisunla’s late-stage trial, 17% of patients completed treatment in six months, 47% in a year and 69% at 18 months, Chen said.

A welcome treatment — mostly

Kisunla’s approval was met with enthusiasm from patient groups and medical organizations that support the entry of medications to help ease the burden of Alzheimer’s disease. Among them, the Alzheimer’s Association, Voices of Alzheimer's and the Alzheimer’s Drug Discovery Foundation voiced support for the decision.

“Any new treatment in the Alzheimer’s area is providing some hope to that community,” Hornung said. “There’s definitely a market for it — it might not be the perfect drug today, but cancer drugs when we first developed those weren’t perfect either.”

Among critics of the FDA’s approval, however, was the consumer advocacy group Public Citizen, which cited “modest” efficacy and “substantial” safety risks that should have led to a rejection. For these reasons, access to Kisunla is appropriately limited to a specific set of patients who will benefit, Hornung said, pointing to three deaths in the late-stage study as a result of the brain swelling complications.

“It’s not the best drug for everyone, and we’ll have to pick patients appropriately, but we do that for every drug,” Hornung said. “The benefits do outweigh the risks for this medicine, like the FDA committee suggested.”

Lilly can learn from similar complications of drugs like Aduhelm and Leqembi in Alzheimer’s — and even drugs that treat other diseases like multiple sclerosis can serve as a comparison. Sanofi’s MS drug Lemtrada held a black box warning for a brain infection called progressive multifocal leukoencephalopathy that patients and physicians shied away from, Hornung pointed out. But the setback led to newer drugs without risk of PML, a testament to the evolving treatment landscape.

“As we develop new drugs,learn from them and understand what’s causing adverse reactions, we make changes to the molecules and create better drugs that benefit patients,” Hornung said. “We’re seeing the same thing here.”

More targets outside of monoclonal antibodies could be where the Alzheimer’s field progresses down the line.

“Scientists understand there’s a connection between amyloid plaque and Alzheimer’s, and while it may not be the cure, it is helping patients slow the progression of the disease,” Hornung said. “If we can do that, then one day that might lead to a cure using more specific targets.”

And payers are likely to provide access to drugs like Kisunla and Leqembi as long as patients qualify, Hornung said.

“Payers aren’t limiting access the way they did with Aduhelm,” Hornung said. “They’re finding ways to get this product to the right patients, and not freely handing it out to anyone with Alzheimer’s.”

Portfolio management in pharma is difficult for many reasons, including a shifting competitive landscape, the push and pull of clinical value and development timelines that reduce long-term market predictability. In fact, from September 2019 to December 2021, 62% of newly launched products underperformed expectations in the first year, according to a recent analysis from industry consultant Trinity Life Sciences.

How can companies mitigate botched market entries and ensure products reach their full potential? The battle starts earlier on in the development process than many companies realize, said Leslie Orne, Trinity’s president and CEO.

“Frankly, the bar keeps going up, up, up on what these drugs need to deliver, and we see a lot of junk making it to market,” Orne said. “When you look at the list of 50-plus FDA approvals [in 2023], we know from pattern recognition that there’s going to be winners and losers, and some who have to work harder for it — we’ve seen increasing difficulty of achieving success.”

The two major reasons assets end up in the “junk pile” are me-too science and a lack of data-driven rigor, Orne said. So, pharmas need to “reverse engineer” the stratification of market launches that leads to winners and losers and be more forward looking at their portfolio from the start.

The ‘junk pile’ beckons

Me-too drugs that join an already thriving treatment landscape have reduced chances of living up to their market potential. The fifth PD-L1 inhibitor or the sixth TNF-alpha inhibitor to enter the market, for instance, have less of a chance to make a difference in medical unmet need.

“If you’re rushing to market to hit clinical thresholds and not being strategic about how that asset is going to differentiate, you’re going to end up with a lackluster launch, even if the science has potential,” Orne said. “That’s the category we want to zero in on the most — how do you take pretty good science, or even great science, and develop it in a way that the commercial market will accept it?”

But being first can also lead to a dead end. Orne pointed to Biogen and Eisai’s debacle in bringing the Alzheimer’s treatment Aduhelm to market. Despite being a highly sought-after asset and the first approved treatment theoretically designed to modify the disease, the market prospect petered out quickly when payers and physicians poked holes in Aduhelm’s clinical evidence, ultimately leading to the companies pulling it from shelves.

“I’m not saying they could have known exactly what the roadmap to success was, but there are probably a couple things they could have done to articulate the value of that asset in a more realistic way to convince payers not to block it,” Orne said. “Biogen is doing amazing science in Alzheimer’s, but they stumbled with the first one, and they could have been more eyes-wide-open about the commercial reality to prevent that.”

That’s where a closer focus on data could help facilitate a more successful launch, Orne said, suggesting a more rigorous approach to traditional market research using tools like AI and machine learning to provide an objective approach.

Building (and deconstructing) pipelines

Big Pharma is always on the lookout for new additions through major M&A, bolt-on acquisitions, alliances and asset purchases. And the sector is “bound to grow again” after a yearslong drought wherein pharmas were hunkering down in a volatile market, said Arda Ural, EY Americas life sciences leader.

“Pharma continues to have $1.4 trillion in firepower, and targets are inexpensive,” Ural said, referring to the industry’s dealmaking capacity, as laid out in a recent EY report. “Meanwhile, you get a counterbalance that pharma on the top end is losing $300 million [in patent losses] and that revenue needs to be replenished somehow by the pipeline.”

For example, J&J’s recent purchase of a Numab Therapeutics subsidiary for almost $1.3 billion provides access to an early-stage eczema drug, adding to a portfolio that includes older immunotherapies at the end of their patent protection next year. Numab CEO David Urech told PharmaVoice the bispecific antibody was getting attention from another “leading pharma company,” as well, highlighting the need for pipeline replenishment across Big Pharma.

But while additive deals to build pipeline prowess fulfill an urgent portfolio need for large pharma companies, the flipside of cutting candidates before sinking too much into costly development can prove just as important for the go-to-market batting average, according to Orne.

And pharma giants have recently been attentive to that need, Orne said. From Sanofi to AstraZeneca to Takeda, companies are having commercial conversations at earlier stages and reprioritizing pipelines as a result. For instance, Sanofi’s 2023 decision to pull a late-stage candidate for myasthenia gravis came about “after careful evaluation of the emerging competitive treatment landscape in MG,” according to a company release.

“[Pharmas] are trying to get better at holding themselves accountable to the thresholds and the criteria they’ve set,” Orne said. “We advocate for quick kills — it takes more guts to do that the right way, but some of the best leaders I’ve seen say, ‘It’s time to look someplace else.’”

Imdelltra. Lumisight. Zevtera.

These new, invented-from-scratch words are now the brand names for three newly FDA-approved drugs, representing a small cell lung cancer treatment, an optical imaging agent for cancer detection and an antibiotic, respectively.

A drug’s name not only has to be memorable and evocative in some way, but also must adhere to strict guidelines from the FDA, since it will be approved along with the drug label.

“There's a lot of legal and viability aspects in naming that need to be considered, and those take months and months,” said Jonathan Finer, director of strategy at Conran Design Group, a brand and design consultancy that he said counts major pharma companies like Johnson & Johnson, Sanofi, Pfizer, Novartis and Merck & Co. as clients. “In many ways, those are the more arduous, rigorous aspects of the naming process.”

Today, the R&D and commercialization process moves faster than ever, and the branded drug space has never been more crowded, with thousands of new drugs in development. That’s why Finer said companies should start the naming process “as early as possible” to ensure the name works from a regulatory, patent, trademark, language, URL availability and branding perspective.

“Think about it really early in the commercialization process,” he said. “A name is a really exciting thing. It's the shortest story that we can tell about a brand. It’s the way in which we identify the brand.”

Here are three key considerations for the drug naming process.

Passing the regulatory hurdle

Before getting creative with a brand name, drug developers and branding experts must first ensure it fits within the FDA’s regulatory framework. For instance, it needs to be proprietary and newly coined, but it also can’t be too similar to any other existing brand names in spelling or pronunciation.

The main reason? The possibility of errors between drugs with similar names.

“The No. 1 legal parameter in naming is that there can never be any confusion, either in the patient's medicine cabinet or for the pharmacist at a large healthcare system,” Finer said. “From a legal perspective, it's creating a differentiation.”

The FDA’s newest guidance for drug naming, released in December 2020, lists other considerations, too, such as pronounceability; avoiding certain references to the drug’s ingredients and or how the drug is manufactured or administered; and steering clear of names that are too similar to brands in other countries.  

In fact, Finer said he always conducts a preliminary trademark screening of possible names due to the rigor of that aspect of the naming process.

“We will never show name candidates to a client before they have gone through preliminary legal screening,” he said. “It's extremely likely that candidates in the creative development phase will not be viable.”

Considering language and letters

In addition to considering existing brand names, pharma companies also need to think about other languages and cultural connotations in other countries, as well as current pharma naming conventions when naming new drugs.

The name not only needs to be “meaningful and attractive to the ear of an English speaker,” but also for speakers of other languages, too, from Romance to Slavic to Asian languages, Finer said. It also can’t translate to anything offensive or counterproductive.

In addition, there are pharma naming conventions that have evolved over the years and have become recognizable to and expected by consumers. In other words, they sound like a drug name.

“They feel like they have a similar construction: two to three syllables, often eliminating vowels, not having too many consonants in a row, truncating words and using alternative spellings and letters: the Y’s instead of I’s, the X’s instead of S's,” Finer said. “Those are the sorts of devices that have created a little bit of a convention in pharm naming.”

On the other hand, “bad” names might have too many consecutive consonants, making them confusing or hard to pronounce.

“Names like Evrysdi, Reyvow and Xywav reflect how innovative letter-strings and unique sounds are becoming the new standard,” Finer said. “Our challenge as namers will be balancing the need for names to both fit with this convention, and, as necessary, be disruptive.”

Evoking a feeling or idea

Although it sounds like drug naming is all about constraints, there are ways to get creative within those constraints.

“It takes us back to the most elemental part of those words, and that's the letter strings. It's how certain characters look together and sound together to form meaningful ideas — letter strings that allude to stories,” Finer said. “They allude to subject matters or some of the feelings … that we might have associated with the drug.”

A good example of this is the insomnia drug Lunesta, Finer said.

“A pharma name I really appreciate is Lunesta because it beautifully captures the essence of its function. The name evokes a sense of tranquility and restfulness, with 'Luna' referencing the moon, a universal symbol of sleep,” he said. “It's simple yet evocative, making it memorable and aligning perfectly with the product’s purpose of aiding sleep.”

The recently approved drug Lumisight seems to do this, too. It’s an IV-administered, fluorescent imaging drug that detects cancerous tissue after a lumpectomy, and its name alludes to both the fluorescence (lumi) and the way it helps with cancer detection (sight).

Finer also points to the name Skyrizi, the plaque psoriasis, psoriatic arthritis and Crohn’s disease treatment from AbbVie. It embeds the real word “sky” into the coined word to convey a feeling of lightness and clarity. That feeling is extended into other branding elements, too, such as the website, which shows an airplane soaring through a clear blue sky dotted with wispy white clouds.

The power of a name

Although it’s nearly impossible to quantify whether a name meaningfully impacts drug sales, it’s still important to come up with one that’s memorable, evocative and meaningful while still fitting within the FDA’s framework.

That’s because a name “is a very powerful thing in drawing connections,” Finer said.

“Once a drug has a commercial name, it really brings it to life in a distinctive way. It makes it more real and relatable, and that is the basis of building a connection, and branding is all about connection and relationship. This is that first, critical point.”