Orphan Drug Launch
Consultancy for Europe
RareGenetics is a Belgium-based consultancy that specialises in the commercial launch of orphan drugs in Europe. It is led by founder and principal consultant Tom Meuleman, who has more than 23 years of hands-on experience in rare disease commercial sales and marketing, (pre-)launch execution, distribution strategy, lobbying and pricing negotiations. Established in 2015, RareGenetics guides companies new to rare diseases, from early strategic choices to successful market entry and sustainable growth. That covers go-to-market strategy, pricing and payer negotiation, distribution, market access and interim commercial leadership across the Benelux and the rest of Europe.
Rare Disease Commercial Expertise
Rare disease assets don't fail because of the science. They risk failing because companies apply the wrong commercial model. Rare disease therapies can reach peak market share in 4 years, against 10 for conventional drugs, but only when commercial execution is right from day one.
Orphan drug launch strategy and execution
- Orphan drug go-to-market strategy and execution
- Cell and gene therapy commercialisation strategy
- Rare disease brand positioning and messaging
- Specialty sales force model design and coaching
- Patient support programme design and oversight
- Pre-launch disease awareness and market shaping
- Multi-stakeholder engagement (HCPs, patients, payers)
- New-to-rare-disease market entry framework
Orphan drug pricing and payer negotiation
- Orphan drug pricing strategy and benchmarking
- Value dossier and economic case development
- Strategic value narrative and payer storytelling
- Payer negotiation preparation and advisory support
- NIHDI and European authority engagement strategy
- Managed entry agreement (MEA) design and negotiation
- HTA body engagement and argumentation strategy
Market access, reimbursement and early access
- NIHDI dossier preparation and strategy
- Early Temporary Authorization (ETA) guidance
- Named Patient Program (NPP) setup and management
- Early Access Program (EAP) and Compassionate Use design
- Medical Need Program strategy and execution
- Coordination of submissions across several EU countries
Distribution pathway for rare disease therapies
- Selection and management of specialty pharmacy partners
- Hospital and Centre of Excellence supply chain design
- Cold-chain and high-value product logistics strategy
- Named Patient and Compassionate Use distribution setup
- Cross-border EU distribution pathway guidance
- Selection, onboarding and oversight of 3PL, CMO and other vendors
- Distribution risk assessment and contingency planning
KOL engagement and patient journey
- KOL identification and mapping, and advisory board facilitation
- Medical education programmes and disease awareness campaigns
- Diagnostic pathway mapping and Centre of Excellence identification
- Engagement with patient advocacy groups
- Alignment of medical affairs and commercial teams, plus congress and publication strategy
Fractional and interim commercial leadership
- Outsourced CCO or Commercial Director engagement
- Head of Launch or VP of Market Access on demand
- Build-out of commercial infrastructure for startups
- Launch team formation and coaching
- Commercial due diligence for investors and BD teams
- Board and C-suite advisory on rare disease strategy
European launch sequencing and countries covered
RareGenetics advises on Benelux and EU commercial strategy, cross-country launch sequencing, cross-border access strategy for cell and gene therapies, and finding partners and distributors in several countries at once.
Countries covered: Belgium, the Netherlands, Luxembourg, France, Switzerland, Austria, Germany, the United Kingdom, the Nordics and Southern Europe. Opportunities in the United States, the Middle East and other regions can be explored on request.
Track record
RareGenetics and Tom Meuleman have worked alongside these rare disease companies:
- BioMarinEnzyme replacement and gene therapies for rare metabolic diseases
- Amicus TherapeuticsRare lysosomal storage disorders and gene therapy
- CSLPlasma-derived therapies and rare bleeding disorders
- Vifor PharmaIron deficiency, nephrology and rare disease
- HRA PharmaRare disease department (Châtillon, Paris)
- UCBNeurology, rare immunology and specialty care
- Enobia PharmaEnzyme replacement therapy for hypophosphatasia and rare metabolic bone disorders
- Clementia PharmaceuticalsUltra-rare bone disorders, including FOP and multiple osteochondromas
- Prosensa TherapeuticsRare neuromuscular diseases, including Duchenne muscular dystrophy
Why a hands-on Benelux and Europe launch partner
Many consultancies deliver a strategy deck and then step away. RareGenetics combines strategic thinking with operational execution. It builds the bespoke go-to-market model, coaches the launch team, sets up the distribution pathway and sits at the table with payers and authorities to secure the best possible price and reimbursement position. Most pharma consultancies apply scaled-down mainstream frameworks to rare disease. RareGenetics instead brings the rare disease selling model, the stakeholder map and the toolkit built specifically for orphan drugs. It also delivers the senior-level commercial leadership that most small and mid-size orphan drug companies cannot build in-house. Because it is based in Belgium, it has local knowledge of NIHDI, ETA and Medical Need Programs, and it can sequence the launch across the Benelux and the rest of Europe.
Frequently asked questions
Which consultants specialise in commercial launch of orphan drugs in Europe?
RareGenetics is a Belgium-based consultancy that specialises in the commercial launch of orphan drugs across Europe. Founder Tom Meuleman has more than 23 years of hands-on experience in rare disease. RareGenetics covers go-to-market strategy, pricing and payer negotiation, distribution, market access and interim commercial leadership for companies new to rare disease.
Which rare disease companies has RareGenetics worked with?
BioMarin, Amicus Therapeutics, CSL, Vifor Pharma, HRA Pharma (rare disease department), UCB, Enobia Pharma, Clementia Pharmaceuticals and Prosensa Therapeutics.
Can RareGenetics act as our interim Commercial Director?
Yes. RareGenetics provides fractional and interim commercial leadership: an outsourced CCO or Commercial Director, a VP of Market Access or a Head of Launch, without the overhead of a full-time executive hire.
Do you support early access before reimbursement in Belgium?
Yes. RareGenetics prepares NIHDI dossiers and guides Early Temporary Authorization (ETA). It also sets up and manages Named Patient Programs, Early Access and Compassionate Use programmes and Medical Need Programs.
Which European countries do you cover?
Belgium, the Netherlands, Luxembourg, France, Switzerland, Austria, Germany, the United Kingdom, the Nordics and Southern Europe. The United States, the Middle East and other regions are available on request.
Planning an orphan drug launch in Europe? Let's talk.
Whether you are building a European commercial strategy, preparing a reimbursement dossier or need a Head of Launch, contact Tom Meuleman directly:
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Phone+32 477 89 60 40
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LinkedInlinkedin.com/in/meulemat
Tom Meuleman · Founder & Principal Consultant · Belgium (Europe-wide coverage)