Commercial readiness Archives

Insights

Why HCP Engagement Can Make or Break an Ultra-Rare Disease Launch

Insights from Chris Mycek, Head of Commercialization at Benchworks, a Danforth Health Company

Launching in an ultra-rare disease market is not a volume game. Success depends on whether the right physicians have the confidence, clarity, and support to identify patients, initiate treatment, and continue prescribing.

That is what makes healthcare professional (HCP) engagement so critical.

In broader specialty markets, commercial teams can often rely on scale and repetition to build awareness and drive adoption. In ultra-rare disease, the equation is different. Prescriber universes are small, treatment decisions are highly nuanced, and each account can materially influence launch performance. When uptake falls short, the issue is often not awareness alone. More often, it is a gap in confidence, coordination, or capability.

A recent case study involving the launch of a first-in-class therapy for a rare pediatric metabolic disorder highlights this dynamic.

When Awareness is Not Enough

A mid-sized biotechnology company launched a therapy for a rare pediatric metabolic disorder affecting roughly 2,000 patients in the United States. The product entered the market with strong Phase 3 efficacy data and favorable payer coverage. On paper, the launch appeared well-positioned.

Yet in the first 18 months, revenue significantly underperformed projections.

Commercial analysis showed the company had correctly identified its core prescriber base: approximately 85 pediatric metabolic specialists. But actual prescribing was concentrated among just 22 of them.

The remaining specialists were aware of the therapy but hesitant to act. Their concerns centered on patient selection, timing of treatment initiation, comfort with the novel mechanism of action, potential long-term safety considerations, and uncertainty around insurance coverage and out-of-pocket costs. Some also viewed the therapy as relevant only for the most severe phenotypes rather than the broader eligible population.

This pattern is common in ultra-rare disease.

Awareness may open the door, but it rarely drives adoption on its own. In highly specialized markets, prescribing decisions require deeper clinical confidence, practical support, and coordinated engagement across functions.

A More Targeted Intervention

Working with Benchworks, a Danforth Health Company, the organization implemented a six-month HCP engagement program focused on sharper targeting and stronger field coordination.

The strategy our experts facilitated centered on five key moves.

1. Refining segmentation beyond simple prescriber lists

The team developed a more dynamic targeting model based on patient volume, growth potential, and alignment with treatment philosophy. In ultra-rare disease, effective segmentation must go beyond static prescriber lists to identify the accounts most likely to drive near-term adoption.

2. Enabling peer-to-peer learning

The company launched small-group dinners and virtual roundtables featuring active prescribers sharing real-world clinical experience. This peer-to-peer model helped move physicians from awareness to greater confidence in patient selection and treatment initiation.

3. Tightening MSL and field integration

Weekly account planning sessions improved coordination between MSLs and field teams, enabling more aligned and responsive engagement. In ultra-rare disease, seamless medical-commercial collaboration is essential to delivering credible, timely support.

4. Addressing access concerns directly

The company developed payer-specific guidance and hub services talking points to address coverage and affordability questions. Clear access communication helped reduce friction between prescription intent and therapy start.

5. Supporting treatment initiation with practical tools

The launch team created clinical support materials, including dosing algorithms, monitoring protocols, and adverse event guidance. These resources helped physicians feel more prepared to initiate treatment with confidence.

The Impact of Coordinated HCP Engagement

Within six months of engagement with our experts, the company saw meaningful improvement across key launch metrics:

Active prescribers increased from 22 to 58

New patient starts rose 47% quarter over quarter

Prescription abandonment fell from 23% to 11%

Time from first prescription to second prescription dropped from 8.3 months to 4.7 months

Net revenue increased 12% year over year despite flat pricing

The takeaway is clear: when commercial teams address the specific barriers holding physicians back, adoption can accelerate meaningfully even in a small, highly specialized market.

Looking to Strengthen Launch Performance?

From launch planning to HCP engagement strategy, Danforth Health’s commercial readiness experts help life sciences companies build smarter launch strategies, align cross-functional teams, and accelerate adoption in complex markets.

Reach out to our team today.

Insights

Get Ahead of Risk: How Integrating Regulatory Early Drives Commercial Impact in Life Sciences

Expert insights from Lisa Maffei Luther, Founder and Head of Regulatory Strategy at Advyzom, a Danforth Health company

For many biopharma companies, regulatory is often viewed as a critical – but contained – function: essential for approval, but less central once a product reaches the market.

In reality, regulatory plays an increasingly strategic role in how a product is communicated, adopted, and sustained over time.

From the earliest stages of commercialization planning through post-approval lifecycle management, regulatory sits at the intersection of science, compliance, and commercial impact. And when that connection isn’t fully integrated, companies can face real consequences — slower launches, inconsistent messaging, or increased regulatory risk.

The growing complexity of commercial regulatory

Today’s commercialization environment is more complex than ever:

Promotional claims must be not only compelling, but fully aligned with evolving labeling and guidance
Pre-launch scientific exchange requires careful navigation to avoid crossing into promotion
Global considerations introduce additional layers of variability and risk
Post-approval changes — from new indications to safety updates — require ongoing coordination across teams

At the same time, commercial teams are under pressure to move quickly, differentiate clearly, and engage stakeholders effectively.

Balancing these dynamics isn’t easy, and it’s no longer something that can be managed in silos.

Regulatory as a bridge — not a checkpoint

The most effective organizations are shifting their mindset: Regulatory isn’t just a checkpoint at the end of the process — it’s a strategic partner throughout.

When regulatory is embedded early and works closely with commercial, medical, and market access teams, it can:

Shape messaging strategies that are both impactful and compliant
Translate complex labeling into clear, usable claims
Anticipate and mitigate risk before it slows down execution
Streamline review processes and reduce rework
Support consistent, aligned communication across functions and markets

In this model, regulatory becomes a driver of efficiency and clarity, not just a gatekeeper.

The overlooked importance of post-approval strategy

Just as critical, and often underappreciated, is the role of regulatory after approval.

Maintaining a product’s value in-market requires continuous attention to:

Labeling updates and evolving safety information
Post-marketing requirements and commitments
New data, indications, or formulations
Shifts in the regulatory landscape

Without a proactive approach, these factors can create friction, delay opportunities, or even impact patient access.

With the right strategy in place, however, they become opportunities to extend value, strengthen positioning, and ensure continuity of care.

A more integrated path forward

As commercialization models evolve, the line between regulatory and commercial execution continues to blur.

Companies that succeed will be those that treat regulatory not as a constraint — but as an enabler of smarter, more effective commercialization.

That means building more integrated teams, aligning earlier in the process, and taking a lifecycle view of both compliance and value creation.

Because in today’s environment, getting regulatory right isn’t just about avoiding risk — it’s about unlocking the full potential of your product.

Insights

Commercial Planning: Building the Right Launch Team & Governance Model

Expert insights from Asymmetry Group, a Danforth Health Company

Product launches are amongst the most complex undertakings for biopharma companies—and there’s no shortcut to success. No matter how strong your strategy or detailed your plan, success ultimately depends on people. If you don’t have the right team in place, even the best-designed launch will fall short. That’s why launch governance and team structure aren’t just operational necessities; they’re strategic levers. Organizations that invest in building a high‑performing launch team and a governance model tailored to their unique needs don’t just execute—they create a competitive advantage.

Several considerations will shape your launch team structure and governance—and directly influence who you select, how the team operates, and what support they’ll need:

1) Launch Experience

Is this your company’s first launch? How much launch experience exists within your organization?

Implications: Internal experience will impact the level of expertise required on the team, whether you need to bring in external support, and how much structure and oversight your governance model must provide.

2) Desired Launch Team Dynamics

To what extent do you expect your team to operate as an integrated, cross‑functional group? Is this consistent with your current culture?

Implications: Your desired team dynamic will shape role selection, behavioral expectations, and whether new ways of working are needed to enable strong cross‑functional collaboration. Even if you have a highly experienced launch team, it’s likely the first time THIS TEAM is launching together.

3) Organizational Decision-Making

How does your organization collaborate and make decisions? Is authority centralized or shared across empowered teams? Are decisions made quickly or more slowly?

Implications: Existing decision-making patterns will inform governance design—defining authority levels, escalation pathways, meeting cadence, and required functional representation to maintain momentum. And then it’s up to the organization to live these decisions to create trust and efficiency.

4) Partnerships

Are external partners involved in the launch? What is the structure of the relationship, and how could it affect planning and execution?

Implications: Partnership arrangements will influence team composition, require clearly defined roles and responsibilities, and may necessitate additional coordination or joint governance mechanisms.

While there’s no single approach to structuring your launch team and governance, four best practices consistently position organizations for success:

1) Appoint a Launch Lead Who Bridges Strategy and Execution

This role varies by company and launch, but the Launch Lead must flex between strategic and execution‑focused work while maintaining a strong cross‑functional lens. Launch success requires alignment across all functions—not the efforts of just one or two teams.

2) Establish a Small, Expert Launch Management Team (LMT) to Drive Progress

Typically composed of the Launch Lead and a few launch‑experienced resources, the LMT drives critical work: shaping strategy, coordinating across teams, tracking progress, and flagging risks. Acting as the “quarterback” of the launch, the LMT ensures visibility, timely communication, and cross‑functional alignment.

3) Create a Lean, Empowered Governance Structure that Can Scale

Your model should include a Core Launch Team, Extended Launch Team, Steering Committee, and functional working teams. Core Launch Team members—one per key function—must be empowered decision‑makers who effectively communicate back to their functions. Keep the Core Team lean; leverage the Extended Launch Team to involve additional functions without slowing down execution. The Steering Committee plays a critical role by resolving escalations and making timely decisions that cannot be addressed within the Core Team, Extended Team, or individual functions.

4) Define Roles and Ways of Working to Enable Alignment and Decision‑Making

Roles and responsibilities should be clearly defined across all governance groups. Equally important is aligning on ways of working early—how decisions will be made, how escalations will be handled, how information will be shared, and what each governance member is accountable for.

Planning your next launch?

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