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Key Launch Capabilities for a Rare Disease Launch

What makes a rare disease launch?

Authored by Stephanie Hall, Founder and CEO, Uptake. 

It’s a question that has fuelled our efforts in the pharmaceutical industry, as more and more rare disease indications and launches come to market in our industry. It’s inspiring to observe pharmaceutical teams achieve success in this domain, often by honing in on five key areas and maintaining a relentless commitment to efficiency, while never losing sight of the patients. Our Uptake Rare Disease and Early Commercialisation and Launch experts have put together 5 key launch capabilities for a rare disease launch, that teams should embed to ensure they are equipped with the right mindset and ways of working needed to transform outcomes and deliver maximum impact.

Discover the five key rare disease launch capabilities


1. Innovative Pricing and Access Strategies

  • Navigation of all orphan drug status R&D incentives, fast track status, RWE (Real World Evidence) to optimise speed to market and access
  • Ability to map national and territory funding flows, bodies and processes for all key local markets
  • Creation of a clear value proposition and price aligned to priority local markets with supporting services, education, use of technologies
  • Creation of flexible, innovative pricing and contracting strategies
  • Payer research and engagement plan for payer KOLs
  • Clear sequencing of global launch countries to optimise pricing negotiations
  • Competitor monitoring of launch dates, prices, discounts and access

2. Customer Segmentation and Micro-targeting

  • Ability to map all key stakeholders across payers / decision-makers, physicians, patients for each rare disease
  • Identification of key individuals and prioritised, specific customer segments for a mix of face to face and digital engagement
  • Clear focus on priority segments engaged at least 3 years pre-launch to shape launch strategic, decisions and tactics
  • Ability to reach wider customer segments with more generalist roles through impactful digital interactions to optimise limited budgets


3. Key Opinion Leader (KOL) / Patient Advocacy Group (PAG) Relationships

  • Key individuals across payer, physician and patient groups are identified for each priority country
  • Launch team members all have aligned relationships and a clear KOL plan in place for collaborative working
  • KOL / PAG engagement starts 2 – 3 years pre-launch with a consistent, sustained series of engagement activities
  • Tracking is in place to measure KOL / PAG activity, brand and company advocacy and overall customer experience with Pfizer interactions


4. Navigation of the patient pathway

  • The patient journey across different phases of symptom recognition, referral, diagnosis, treatment, follow-up is clearly mapped for each priority market
  • Clear identification of barriers in patient pathway
  • Highly customised, integrated patient-support services to reach the right treatment centre and optimise outcomes
  • Innovative use of technology to enable generalist physicians to recognise and refer a potential patient to the right specialist physician or centre with urgency


5. Expert, cohesive launch teams

  • Core launch team members in place at least 3 years pre-launch with clear objectives, dedicated (hybrid) roles and budget
  • Clear launch plan in place – at a strategic and operational level with key launch readiness milestones across global, regional and local teams
  • Launch team members have expert set of disease, product and patient knowledge
  • Clear governance and efficient ways of working set. Leverage wider organisation’s resources and relationships
  • Clear launch objectives, ambition, measures of success defined pre and post launch
  • Have a clear understanding of your launch readiness, using a launch excellence tool like LExLevel Pulse.


If you’d like to discuss any of these 5 themes in more detail, to accelerate the uptake of your rare disease brand, contact us.

Contact us

Read the latest rare disease launch guidance from our team of healthcare consultancy experts in our thought leadership article ‘Mastering the Art of Rare Disease Launches: Your Guide to Success.’

Introducing Uptake’s Rare Disease and Launch Excellence Experts

Melissa Dagless

Head of Growth & Innovation

Melissa Dagless

Deborah Lough


Deborah Lough

Jo Lopez

Practice Lead – Early Commercialisation & Launch

Jo Lopez

Stephanie Hall

CEO & Founder

Stephanie Hall

Maxine Smith

Managing Director

Maxine Smith