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Product Launch Success: Getting It Right In Rare Disease

As we wrote in our last article, “To Ace Your Product Launch, Don’t Fail At Market Access,” launching a drug into a crowded market without understanding the market access landscape is like failing to study for a make-or-break final.

Yet while inadequate patient access to a life-saving treatment is much graver than a disappointing grade, of course, our point remains: Pharma manufacturers aren’t doing their homework before launch, which prevents patients from accessing a much-needed, oftentimes long-awaited therapy.

This lack of preparation is all too common, unfortunately, and the stakes are even higher for companies launching products that target an unmet need. If manufacturers launching within a crowded market aren’t cracking open the textbook until it’s too late, then those who are first to enter a rare disease space lack the right textbooks to study in the first place—there’s simply no playbook for bringing their drug to market.

Forging a new path

Without a sufficient road map, what do rare disease manufacturers need to do to secure appropriate market access from the outset? And why aren’t many of them doing it?

While more and more pharma companies are being incentivized to pursue rare diseases, manufacturers’ reticence to developing a market access strategy much earlier in the drug development life cycle often stems from a lack of understanding. We’ve seen leaders at top pharma companies assume that their high-cost drugs will be covered by payers simply because there’s no other drug on the market for that specific indication. In the absence of alternatives, payers will react favorably to your product, right? Unfortunately for patients, that’s not always the case.

Consider one manufacturer that we worked with recently. The company’s product was fulfilling an unmet need and had excellent clinical data. As such, leadership assumed that payers would manage it favorably. However, they didn’t get the coverage that they expected after launch. Even though their product was first to market, the company’s payer strategy didn’t account for the actuarial elements that factor into drug coverage decisions: If patients are successfully managing their symptoms via the current standard of care, then why put them on a high-cost therapy to treat the disease? Is the clinical benefit meaningful enough to offset the cost of coverage?

Unfortunately, most manufacturers don’t consider questions like these until payers raise them. And at that point, there’s little that can be done; pharma companies and patients must live with the very real costs of hampered access to a critical, first-of-its-kind therapy.

The keys to driving access

Despite the many obstacles to bringing a rare disease drug to market, manufacturers can take key steps early on—at least two years before launch, ideally—to overcome barriers to access and ensure that their much-needed products will get into the hands of patients.

To start, smart manufacturers are seeking to understand the payer market beginning in Phase 2 or even as early as Phase 1 of clinical trials. While exact analogs aren’t an option for those who are first to market, studying the launches and sales volumes of products in other rare disease categories can help not only predict payer uptake, but also shed light on how payers might react to price and inform how coverage could affect sales at launch. For example, a rare disease drug might have seen limited coverage at launch, but still have high sales volume due to medical exceptions. Ultimately, insights like these help manufacturers launching in a similar therapeutic area understand the complete picture and build better go-to-market forecasting models.

What’s more, manufacturers can’t afford to develop their value propositions and conduct pricing research late in the game. The value story—what’s in it for payers—needs to be developed at the outset and clearly communicated: Will your product decrease the number of times a patient needs to be hospitalized or visit the doctor? Will it prevent the need for a future transplant? Is it an oral medication that can be easily accessed at the pharmacy? Will patient support programs be created to help ensure patient adherence?

And while hammering home the value story is key, manufacturers would do well to remember that payers are also human. It might seem like cost-effectiveness is the only important factor in coverage decisions—especially in indications with small patient populations—but educating payers on the unmet need and specific burden of disease is critical. It can be easy to forget when engaging payers in discussions about the bottom line, but at the end of the day, a drug that will improve the lives of patients and caregivers is going to resonate with stakeholders. Compassion can coexist alongside cost concerns.

While there’s much to think about ahead of launch—especially within rare disease—pharma companies that have gotten it right understand the role that timing plays when developing and executing on their market access strategies. Getting it wrong leaves money on the table and, what’s worse, puts vulnerable patient communities at risk. Ensuring adequate market access is a test that every rare disease manufacturer needs to pass.

By Jayne Hornung, Chief Clinical Officer, and Fiza Bari, Senior Solution Consultant

This article was originally published in the June 2022 issue of Life Science Leader.

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