RARE DISEASE SEARCH
Why Rare Disease Is Different Rare disease and gene therapy development requires executives with specialized expertise that traditional oncology or pharma leaders don't have. Orphan drug pricing strategies, ultra-rare patient recruitment, patient advocacy partnerships, accelerated approval pathways—these aren't skills you find in mainstream pharma. We place the rare leaders who've done it before.
Why These Executives Are Rare
Most pharma executives work on diseases affecting millions of patients with large Phase 3 trials and established regulatory precedents.
Rare disease requires:
Trial design creativity (50-patient trials, natural history comparisons, single-arm studies)
Patient advocacy partnerships (working with foundations, families, patient orgs)
Regulatory innovation (convincing FDA/EMA with limited data)
Pricing sophistication (justifying $2M gene therapy costs)
Manufacturing expertise (autologous therapies, patient-specific production)
These skills don't exist in mainstream pharma.
They exist in rare disease divisions. That's where we recruit.
Our Rare Disease Search Approach
We target executives from companies with successful orphan drug approvals and gene therapy launches—leaders at Alexion, BioMarin, Sanofi Genzyme, Vertex, Spark, and Bluebird Bio who've navigated the unique challenges of rare disease development.
When you need proven rare disease leadership, we know which companies have the talent you need.
Roles We Place
VP Business Development
Orphan drug licensing and partnerships
Gene therapy platform deals
Patient advocacy organization relationships
Government funding and grant navigation (Orphan Drug Act, PRIME designation)
Strategic alliances for ultra-rare diseases
Chief Medical Officer (CMO)
Orphan drug regulatory strategy (accelerated approval, breakthrough therapy)
Ultra-rare disease trial design (natural history studies, single-arm trials)
FDA/EMA interactions for novel gene therapies
Patient registry development
Biomarker and endpoint selection for rare diseases
VP Medical Affairs
Patient advocacy partnerships
Disease education and awareness programs
Rare disease KOL networks (specialized centers of excellence)
Compassionate use and expanded access programs
Registry studies and real-world evidence
VP Market Access
Orphan drug pricing strategy
Payer negotiations for ultra-rare treatments
Value-based pricing for gene therapies (one-time cure vs. chronic treatment)
Reimbursement strategy across US, EU, and global markets
Health economics and outcomes research (HEOR) for rare diseases
Therapeutic Expertise
Gene Therapy:
AAV (adeno-associated virus) vector development
Ex vivo gene therapy (lentiviral, retroviral)
In vivo gene editing (CRISPR, base editing)
Manufacturing and scale-up for gene therapies
Long-term safety monitoring and registries
Orphan Drugs:
Enzyme replacement therapies
Small molecule orphan drugs
Rare oncology indications
Ultra-rare metabolic disorders
Neuromuscular rare diseases
Regulatory Pathways:
FDA Orphan Drug Designation
EMA PRIME (Priority Medicines) designation
Breakthrough Therapy Designation
Accelerated Approval pathways
Conditional approval (EMA)
Market Access Challenges:
Pricing for ultra-rare diseases (<1,000 patients globally)
One-time gene therapy pricing ($1M-$3M+ per patient)
Demonstrating value in rare diseases with limited data
Global reimbursement strategy
Our gene therapy platform could address 20+ rare diseases. Should we hire executives with "platform" experience or "indication-specific" experience?
Both, but at different stages. Early (Series A-B): hire platform builders—executives who've scaled AAV manufacturing, built multiple programs simultaneously, and understand the regulatory pathway for platform expansion (like Ultragenyx's multi-program strategy). Late stage (Series C+): add indication-specific leaders for your lead program—executives who understand the specific patient community, natural history, endpoints, and KOLs for your target disease.
We've seen biotechs fail by hiring only platform thinkers who can't execute on a single indication, and fail by hiring disease specialists who can't scale beyond one program. We help you sequence these hires correctly based on your stage and investor expectations.
We're a Series B gene therapy company. Every "experienced" gene therapy executive we talk to wants $400K+ base salaries. How do we compete?
You probably can't match Big Pharma cash, but you can win on equity and mission. Here's the reality:
Big Pharma rare disease executives earn: $350K-$500K base + bonus + RSUs
Series B-C biotechs can offer: $250K-$350K base + significant equity (0.5%-2% for VP-level)
The executives who will join you are motivated by:
Equity upside - They believe your therapy will succeed and want ownership
Mission - They've seen patients die waiting for treatments stuck in Big Pharma pipelines
Speed - They're frustrated by 18-month decision cycles at pharma and want to move fast
Impact - They want to be the CMO who got YOUR therapy approved, not employee #47 on a Novartis program
We help you find executives who prioritize equity and impact over cash maximization. They exist—but you need to tell a compelling story about why your platform will win and why now is the right time to join. We'll also benchmark comp so your offer is credible even if it's not the highest.
How do we know if a "Big Pharma rare disease" executive can actually operate in a biotech with limited resources?
Ask about their worst resource constraint and how they solved it. Big Pharma rare disease executives are used to:
Dedicated medical writers, statisticians, regulatory consultants
$50M+ clinical trial budgets
Large MSL teams and patient support programs
In-house CMC and manufacturing expertise
Biotech reality: You might have $30M total funding, 2 people in clinical ops, and are using CROs for everything.
Red flags in interviews:
"At my last company, we had a team of 12 doing this..."
"I'd need to hire 5 MSLs before launch..."
"We'd build an in-house manufacturing facility..."
Green flags:
"I've worked with lean teams and CROs managing multiple programs simultaneously"
"I know how to prioritize which endpoints matter vs. nice-to-haves when budget is tight"
"I've written regulatory sections myself when we couldn't afford consultants"
We screen for rare disease executives who've worked at resource-constrained biotechs (not just pharma) or who've operated international affiliates of Big Pharma with small budgets. These executives understand scrappy execution while maintaining regulatory rigor.
How quickly can you find a CMO with orphan drug approval experience?
2-3 weeks for initial shortlists. We maintain relationships with CMOs from successful orphan drug programs and actively track who's ready to move from pharma to biotech. When you raise your Series B-C and need proven rare disease regulatory expertise immediately, we deliver vetted candidates with BLA/MAA submissions in ultra-rare diseases—not a 6-month search starting from LinkedIn.
Our rare disease has no existing patient advocacy foundation. Does that make executive recruitment harder?
Yes, significantly—especially for VP Medical Affairs and VP Market Access roles. Here's why:
Executives with successful rare disease launches rely on:
Patient advocacy partnerships for natural history data, patient recruitment, disease education
Patient registries co-developed with foundations for regulatory submissions
Patient foundations as co-funders for trials (some foundations contribute $5M-$20M)
Advocacy groups for payer pressure and reimbursement support
Without a patient foundation, your path is harder but not impossible. We look for executives who've built advocacy relationships from scratch or worked in diseases where they had to create the patient infrastructure themselves (like ultra-rare metabolic diseases where no foundation existed).
We'll also help you identify candidates who can BUILD these relationships as part of their role—executives with experience founding patient registries, creating advocacy boards, and engaging with unorganized patient communities. This skill is rarer than you thi
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