Commercialization, marketing and social media

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.

Pharma companies are on the edge of a looming patent cliff over the next several years, and generic competitors could topple sales of some of the industry’s best-selling drugs. For some Big Pharma companies, generics are already on the doorstep.

Bristol Myers Squibb could soon see some headwinds for its blockbuster leukemia drug Sprycel as the first generic is expected to hit the market in September.

Sprycel (dasatinib) is a kinase inhibitor that was approved in 2006 to treat chronic myeloid leukemia, a type of blood cancer that impacts 1 in 526 people in the U.S. The tablet has been a reliable blockbuster for BMS over the years, reaching $1.9 billion in sales in 2023, BMS reported in its latest quarterly update. Sales topped $2.1 billion in both 2022 and 2021.

BMS has faced several challenges to Sprycel’s patents, and filed a patent infringement lawsuit again Swedish biopharma company XSpray Pharma in 2022, as well as a handful of other companies that were challenging “two FDA Orange Book-listed monohydrate form patents expiring in 2025 and 2026,” the company said in a recent SEC filing.

BMS settled with XSpray last year, settling all pending litigation, but also paving the way for XSpray to launch a generic dasatinib as soon as Sept. 1, 2024. XSpray has previously billed itself as able to get around secondary patents before they expire thanks to its technology. The contested patents were not set to expire until Sept. 28, 2026. However, XSpray has a PDUFA date of July 31 for its “optimized version of dasatinib,” Dasynoc.

If Dasynoc is OK’d, it will join dozens of new generics approved across indications in 2024, including several cancer drugs. With this wave of generics, branded oncology treatments could see a price drop overall, according to IDP Analytics. 

There are more dasatinib generics in development from a handful of other pharma companies, including Biocon and Teva. 

What’s next for BMS

Amid the upcoming patent expirations and onslaught of generic competitors, BMS has kept its oncology pipeline warm with new candidates and also sought expansions for Sprycel — the FDA granted approval for Sprycel to treat pediatric patients with chronic CML in 2017

The pharma giant’s cancer drug Breyanzi, a one-time CAR-T cell therapy, won approval for large B-cell lymphoma in 2022, and the therapy has since seen expansions for patients with relapsed or refractory mantle cell lymphoma, as well as accelerated approval in May for those with chronic lymphocytic leukemia or small lymphocytic lymphoma. The FDA also granted accelerated approval of Breyanzi for follicular lymphoma the same month. However, BMS faces competition in the same therapy space from Gilead’s Yescarta.

Elsewhere in BMS’ pipeline are two candidates for acute myeloid leukemia in early clinical stages. AML is considered a hard-to-treat disease, and a drug developed by Celgene and picked up by BMS in 2019 when it acquired the company was approved in 2017 to treat the condition.

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Generative AI (GenAI) in healthcare and Life Sciences promises important advancements in efficiency, scalability, and quality of care. 11TEN Innovation Partners' recent Demand-Driven Innovation report outlines how industry players are poised to leverage GenAI to accomplish these goals. Here’s a concise overview of the key findings and insights from the report, which is available in full HERE.

The state of GenAI in healthcare

Healthcare systems worldwide face numerous inefficiencies, particularly in administrative processes. Physicians spend an average of 15.5 hours per week on paperwork, detracting from patient care. GenAI-driven document automation presents a viable solution, significantly cutting down the time required for these tasks. For instance, Grand Mental Health in Oklahoma reported reducing documentation time from 10 minutes to just over 3 minutes using GenAI tools offered by Eleos Health.

Data and projections for the potential impact of GenAI underscore the dramatic changes ahead of us:

• Global spending on GenAI in healthcare is projected to increase from $1.64 billion in 2023 to $5.23 billion by 2027
• Approximately 75% of GenAI use cases in healthcare today revolve around administrative tasks such as claims administration, customer care, and back-office support.
• The average cost of drug discovery, currently at $400 million per drug, could be reduced to one-tenth through GenAI, significantly lowering the financial barriers to developing new treatments.

Enhancing patient communication

Static and impersonal patient communication approaches can be associated with low engagement but highly dynamic, personalized approaches have traditionally been complex and cumbersome to execute. GenAI-powered tools can revolutionize this process by providing automated, personalized communications. Kaliber, a surgical-focused AI software company, has developed a GenAI tool that delivers personalized clinical insights, enhancing post-operative care and recovery.

Accelerating drug discovery

The drug lifecycle, from discovery to regulatory approval, typically spans 10-15 years, delaying access to life-saving medications. GenAI is expected to contribute to the development of 50 new therapies over the next decade. Companies like Almirall are already leveraging GenAI in collaboration with tech giants like Microsoft to accelerate drug development.

Addressing data challenges

Incomplete and inconsistent patient data hinders effective medical research and treatment planning. GenAI can generate synthetic data, mimicking real-world data characteristics while mitigating privacy concerns. This approach not only augments existing datasets but also enhances the development of personalized medicine. Generated Health’s partnership with the College of William and Mary to create synthetic patient data for training their digital nurse ‘Florence’ exemplifies this potential.

Future directions and considerations

The adoption of GenAI in healthcare and life sciences is expected to evolve beyond administrative support to more complex clinical processes such as decision-making and diagnosis. However, this shift will require robust regulatory frameworks to ensure the safety, equity, and efficacy of these technologies. Additionally, companies must explore sustainable practices to manage the significant energy consumption associated with GenAI.

Conclusion

Generative AI offers transformative potential for healthcare and life sciences, addressing long-standing inefficiencies and driving innovation. As these technologies continue to evolve, cross-industry collaboration and a focus on delivering value to patients and providers will be essential. To delve deeper into these insights and explore the comprehensive analysis, download the full report by 11TEN Innovation Partners: DOWNLOAD.

About 11TEN innovation partners

11TEN is a leading healthcare innovation ecosystem and strategy consulting firm, specializing in helping healthcare, life science, payer, and digital health organizations launch new products, therapeutics, and partnerships. 

11TEN is now part of the ClinicalMind family of business, offering end to end strategy and tactical execution services to seamlessly support our client partners from pipeline to product maturity. Learn more about 11TEN: www.11ten.com

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.”

Another gene therapy broke the record in March to become the most expensive drug in the world, serving as a reminder to healthcare systems that achieving market sustainability will require new ways of thinking. Can payers accustomed to the traditional pharma model adapt to a new age of one-time cures?

Orchard Therapeutics’ $4.25 million list price for the one-and-done rare disease gene therapy Lenmeldy eclipsed the previous record holder — also a gene therapy. That treatment, Hemgenix from CSL Behring and uniQure, won approval in 2022 and launched last year at a list price of $3.5 million. In fact, gene therapies have been outdoing one another in list price since uniQure’s Glybera launched in 2012 at more than $1 million per treatment before being pulled from the market having been administered to only one patient outside of clinical trials.

Despite the sticker shock associated with gene therapies, their promise of an effective cure for difficult diseases is here to stay. And the Institute for Clinical and Economic Review, an industry watchdog that examines drug prices in relation to a treatment’s medical impact, teamed up with a Tufts Medical Center think tank to explore payers’ options in the brave new world of ultrapricey therapies.

In just seven years, the FDA has approved 17 gene therapies, and the Tufts group, called NEWDIGS, estimates that more than 85 new gene therapies in 12 therapeutic areas will gain regulatory nods by 2032.

In the white paper released last week, the organizations explored how payers can work with drugmakers and patients to promote further access to gene therapies while addressing the lengthy and expensive development process that went into bringing these treatments into existence.

A fair price

ICER and NEWDIGS pointed to the drugmakers’ list price as a primary lever for improving access to gene therapies. ICER’s cost-effectiveness model is one way to do that by determining the value of a drug in terms of its impact on the patient both in length and quality of life.

Prior to Lenmeldy’s approval, ICER recommended a list price of between $2.3 million and $3.9 million based on a “health-benefit price benchmark.” Orchard’s eventual price of more than $4 million exceeded ICER’s standard, but the watchdog’s panel of experts couldn’t deny the treatment’s efficacy.

Furthermore, a gene therapy that is used once in a lifetime as opposed to chronically for years could cost less in the long run — ICER and NEWDIGS noted that a cure for hemophilia could be considered cost-effective as high as $20 million due to what payers already shell out over a patient’s lifetime with the disease.

But how much is too much? After all, small employers or programs like Medicaid don’t have unlimited funding and need sustainable models for reimbursement.

ICER and NEWDIGS recommend an “uncertainty discount” on the launch price that would take into account a gene therapy’s novelty. The cost-effectiveness of a gene therapy could be determined by an analysis that stretches out five years or more to “share financial risk between plan sponsors and manufacturers,” lowering the price at launch due to the uncertainty and then recalculating when those data points are realized later in the therapy’s market lifecycle.

Ethical concerns surrounding the “value” of human life have always made ICER’s job difficult, and the organization doesn’t deny that any pricing model highlights “the challenging interplay of evidence and values in setting priorities and pricing levels for these treatments,” according to the report. But the line being drawn would be a “shared savings” that allows a drugmaker’s return on investment to align with the benefit a gene therapy provides to society.

Milestones and subscriptions

One way to ensure the cost of a gene therapy reflects its value is to tie price to outcomes, and ICER and NEWDIGS present a few ways that could be accomplished.

Contracts could call for a full upfront payment with an option for rebates if the therapy does not meet expectations, the organizations said. For bluebird bio’s sickle cell disease gene therapy Lyfgenia, priced at $3.1 million, payers who select this option would be granted a rebate if a patient is hospitalized with sickle cell complications within three years of receiving treatment. Similarly, a drugmaker could offer a warranty for future payer expenses associated with a failure to meet a therapy’s promise.

A third alternative would involve installment payments over time based on positive outcomes.

But for this to happen, payers also need to be ready for these kinds of strategies, ICER and NEWDIGS said. The Centers for Medicare and Medicaid Services need to clarify their regulations to adapt to new payment methods and build organizational capacity to do so, according to the report.

For any small insurer, a sudden bill for millions of dollars can still have a big impact on the overall budget, and that’s where ICER and NEWDIGS recommend a type of insurance for insurers — rather than excluding coverage altogether, payers should protect themselves against unexpected claims through “stop loss” insurance.

In the same vein of pooling resources to pay a big one-time claim, subscription models among payers could spread the cost over a wider group of patients. Called “financial protection programs,” large payers like Aetna/CVS, Cigna/Evernorth and United Healthcare/Optum have their own versions of this model. Although the premium for these programs remains high, ICER and NEWDIGS said the model could become useful as more gene therapies enter the market.

Finally, a public option from the federal government could make gene therapy payments more sustainable, carving out of tax revenues or payer fees to do so. However, ICER and NEWDIGS pointed to issues in past iterations of this type of program as a result of inefficiencies and potential for misuse.

As innovative gene therapies become more prominent in the treatment landscape, ICER and NEWDIGS said two or more of these strategies could be implemented at once through policy “stacking” to provide the best sharing model between drugmakers, payers and patients.

For biotechs in stealth mode, keeping a low profile is the name of the game. But once they enter the next stage of growth, companies must shed the cloak of secrecy to attract investor attention. And in the competitive biotech market, developing a strong marketing strategy can be critical to garner needed support and advance R&D.

“If you don't have any marketing, then nobody is going to know about you and you're going to have to rely on your board of directors’ and CEO’s relationships,” said Daniel Lofaso, CEO and founder of the biotech marketing agency Digital Elevator. “Good science alone is not going to get you to that next step.”

But raising awareness while coming out of stealth doesn’t have to be complicated.

Here, Lofaso provides tips for how companies can set themselves apart and increase their chances for exposure early on.

Develop a buyer persona

Before companies begin to develop marketing materials, they should understand the “who, what and why” of their business — who the audience is, what makes their technology novel and why people should care.

Once they can create a story around these questions, the rest of the communication strategy tends to “fall into place,” Lofaso said.

And to stay competitive in the current climate, he noted, it’s especially important to build a narrative around how a company’s science works and the professional accomplishments of its leadership.

“Those things are what a lot of these investors are looking at when these biotechs get in front of them,” he said. “Scientists and founders don't like to brag about themselves, but we're not bragging. We're just telling the truth,” he said.

Design an engaging website

Outlining the story is only half the battle. Next, Lofaso said he often recommends companies build visual-heavy websites that distill information into easily comprehensible blurbs.

As a company grows, the website acts as the hub where all stakeholders, from investors to payers and patients, can go for in-depth information about what they’re all about and making it easy to skim is vital to holding people’s interest.

“[Investors are] looking at dozens of companies a week and need to be able to glean information quickly,” Lofaso explained.

Visuals, like 3D animations that show how a drug functions, are among the best ways to describe the science and keep viewers engaged.

And while companies “don’t have to go crazy with $150,000 websites,” the typical recommendation is that they spend 8-10% of their budgets on marketing, Lofaso said.

Get the word out

Once the stage is set, companies can attract attention through earned media campaigns and SEO-driven content marketing, Lofaso said.

To kick-start these efforts, he recommended that biotechs create a public relations plan and pitch timely stories about their technology to industry publications frequently read by investors.

They should also consider creating a blog with keyword-heavy articles highlighting the problems they’re attempting to solve to attract web traffic and build an audience over time.

Ultimately, though, Lofaso said the best marketing campaigns are the simplest.

“Identify who you're talking to, and then how you're going to hit them,” he said. “Everybody thinks they have to do some super technical marketing campaign these days, because it sounds exciting. But traditional marketing works really well.”

Since the birth of social media nearly two decades ago, pharma companies have almost exclusively used it as a marketing engine. But, as the adoption of real-world data revolutionizes the field, online networks are now gaining recognition as a bountiful source to understand patient experience and insights.

Throughout their care journey, patients turn to online social communities to discuss treatment options, symptoms and side effects. Recent improvements to artificial intelligence and machine learning capabilities have also made the once-arduous task of combing through social media easier, putting it at the forefront of the industry’s push toward more patient-centric clinical trials.

Regulators are keeping a close eye on these developments. As part of a mandate from the 21st Century Cures Act to consider more experience data during the drug approval process, the FDA published guidance in 2020 with recommendations for how companies could use online platforms to collect patient input.

However, because of vague language in the guidelines, many industry leaders have been hesitant to fully adopt social media as a source of real-world data, said Thierry Escudier, leader of The Pistoia Alliance’s new community of experts investigating social media’s data potential.

“The FDA made statements about the fact that social media can be a source of real-world evidence. But there is some caution and some challenges,” Escudier said. “It's also up to the pharma industry and those who are helping the pharma industry to make those challenges clearer.”

With that goal in mind, The Pistoia Alliance convened a group of experts in November from across pharma to develop best practices and guidelines for how the industry can use insights from social media as data points during drug development.

“Our focus is to help the R&D teams across the world in pharma, or vendors and technology providers,” Escudier said. “The legal framework we are able to work and operate under is to make people meet and work together for the benefit of both the stakeholder on the industry side, but also on the patient side and the academic side.”

The Pistoia Alliance was conceived in 2007 by leaders from Big Pharma including AstraZeneca, GSK, Novartis and Pfizer to lower “barriers to R&D innovation” by enabling stakeholders from across the industry to work together. The idea for the social media project originated from one of Pistoia’s member companies Semalytix and now includes representatives from AstraZeneca, Bayer, Biogen, Boehringer Ingelheim, Chiesi, Johnson & Johnson and Roche, among other smaller companies.

During the first phase of the initiative, representatives met in roundtable discussions over the last year to share advice on how their companies currently handle real-world data from social media. Now, as they move into an official project phase, they’ll seek input from more alliance members and outside stakeholders — including patients and regulatory agencies — to develop best practices and guidelines for the industry to follow as companies increase usage of social media data.

“That's the beauty of (the alliance) is that the least experienced (people) can get the good feedback and the best practice from the most experienced people,” Escudier said. “Since this topic can be sensitive, it's very important that no one is doing the wrong things. That's why everyone wants to learn from those who have already done it for many years.”

Social media challenges

Top questions the community of experts plan to tackle include how to find data, how to aggregate and analyze data, and how to anonymize data to ensure patient privacy.

One of the biggest challenges companies face with social media evidence is consistency and organization. While some social platforms like Twitter have character limits on posts, there are few rules beyond that — and raw information from these sites can come in a variety of languages and formats.

“There is this famous statement: Garbage in, garbage out. It means that if you don't know what you get in your database analysis, you will not know what you will get when you want the results,” Escudier said. “If people want to use those social media communications that are absolutely not filtered or formatted by the process of the clinical trial operating model, they may have a lot of (data). But how do you make sure to have (data) that you can analyze?”

Verifying a person’s identity can be another roadblock. In its guidance, the FDA suggested that “to have a verified patient, there should be enough information to indicate the existence of a specific patient, including age, sex, gender, initials, date of birth, name or patient identification number.”

But depending upon the social media site, tracking down these data points may be difficult. And conversely, once the patient data is confirmed, companies must then anonymize the data for analysis. The tedious nature and time commitment of these processes “is why some people still do not want to go into this space, because they might be afraid of how to do it,” Escudier said.

A large part of the alliance’s work will be communicating with regulatory agencies around the globe, including the FDA, to understand what types of social media data they will accept during the drug approval process.

“We want to get in touch with them and say, ‘OK, this is the way we have understood what you are expecting from us. Is it exactly that?’ Once this is agreed upon, (we will) … translate that into best practices,” Escudier said.

Abundant opportunities

Despite the challenges, integrating social media insights with other forms of real-world data and clinical trial results poses numerous benefits for pharma companies.

“The good thing with social media is you access directly what the patient thinks. So it's complementary to what (data) already exists,” Escudier said.

Patients on social media may be more likely to discuss symptoms and side effects that otherwise might go unreported in a clinical trial. A survey conducted by the Pew Research Center found that 35% of U.S. adults have used the internet to self-diagnose a medical condition, and over a third of those respondents said they did not visit a clinician for a professional opinion.

Pharmas can extrapolate information from these daily online interactions to uncover patient insights that they “would never get a sense of through more classical ways of capturing data,” Escudier said.

He also suggested that social media listening could be fruitful “for pharma companies looking to dive into new therapeutic areas” so that they “understand what patients say about their disease,” and can look to fill unmet needs.

By increasing collaboration between industry stakeholders, Escudier hopes the alliance’s community of experts can discover new ways to harness the potential social media could offer in the R&D stages of drug development.

“Alone, you go faster; but together, you go farther,” he said. “When you go with more people, you are sure to have better experience and expertise, and therefore you will be able to go farther. That’s the magic of the alliance.”