Launching a Rare Disease Drug
6 Things You Must Know
As awareness and innovation in the rare disease space grow, we expect new product launches to continue increasing. In 2022, more than half of the drugs approved by the FDA were for the treatment of rare diseases. And having been immersed in this space for over two decades, we don’t see a slowdown coming anytime soon for these orphan drugs and other emerging treatments.
But that’s not to say there aren’t hurdles. Rare disease launches are unlike any other. Many of these products are so novel that payers and providers struggle to wrap their heads around them. Without precedent and with sometimes unheard of price tags, biotech manufacturers need to firmly know what they’re doing to avoid missteps.
Rare Disease Drug Launch Must-Knows:
1. Timing is mission critical.
Patients endure endless confusion and frustration as it takes an average of 4-5 years for an accurate rare disease diagnosis. By the time they know what they are facing, the disease has often progressed significantly. Lives are literally at stake when commercializing a rare disease product.
Not only that, but there’s a cadence to the market access world that can’t be ignored. From clinical trial timing to accelerated approvals; commercial and government payer review cycles to coding and compendia submissions, biopharma manufacturers don’t want to miss a beat. Because when it comes to rare disease, even days can mean the difference between life and death.
2. White space is not your friend.
When you’re talking about a first-in-class treatment–as many in the rare disease space are–people are often wading into unknown territory. There’s not the usual context and precedents for payers to draw on for making coverage decisions. In other words, white space abounds and it can lead to confusion and incorrect assumptions. The best payer decisions are the most informed ones, so the burden falls on manufacturers to start the conversation early and to fill in the blanks as thoroughly as possible. One way we help clients tackle this challenge is to consider a PIE (Pre-approval Information Exchange) strategy, taking advantage of the ability to share clinical information ahead of FDA approval.
3. Focus on clinical impact.
When it comes to getting payers on board, it all comes down to messaging. Knowing what language will resonate and clarify can mean the difference between coverage and non-coverage. For example, often payers will pay less attention to clinical trial biomarker details and focus more on clinical outcomes. They want to know:
- How will this therapeutic improve the life of the patient?
- How meaningful is the clinical impact?
When DKP crafts value messaging for clients, we want payers to immediately understand why this product matters for patients. We might even incorporate QOL (quality of life) metrics to paint a picture of clinical outcome. Making sure the impact is clear and compelling is key.
4. Be proactive with clinical compendia.
Clinical compendia–the CMS-recognized source of truth for clinical evidence–can have a major impact on market access in the rare disease space (and beyond). When we partner with clients, we always recommend engaging with clinical compendia early on and with a well-formed strategy. We focus on key questions like:
- What is the science showing?
- Where does this treatment fit in the therapeutic landscape?
- What do payers need to know most?
5. Look at streamlining administration.
Rare disease products are among the most complicated and expensive therapeutics out there. Payers want to see as much burden reduction as possible all around, so that means simplifying distribution and administration wherever possible. Manufacturers should ask: how are we prepared for the complexity of distribution associated with rare disease products? Can anything be simplified or streamlined?
6. Field teams need proper training and intel.
With niche products like those in the rare disease space, proper allocation of resources is everything. That means your field team needs to know the nitty gritty and where to focus. We’ve been equipping field teams in the rare disease space for decades and helping them handle nuances like:
- Buy-and-bill scenarios
- Navigating academia-driven centers
- The subtle-but-impactful differences between the state Medicaid agencies
- Payer-centric messaging
- Distribution strategies
- Coding intricacies
When it comes to rare disease drug launches, the devil can be in the details. Nuances and complexities associated with distribution, coverage and access, contract negotiation, price and clinical compendia, and more can throw the whole thing off course quickly. And when we’re talking about people with often catastrophic conditions, there’s little room for error. We owe it to patients to be focused and precise, and to remove as many treatment barriers as possible.