Rare Disease Consulting

Medically relevant data and consulting to develop and commercialise treatments for rare diseases.
rare disease
Benefits
01

Prioritize pipeline and investments

Get highly specialized guidance to evaluate product opportunities based on the addressable population.

02

Reach the most patients

Use evidence of your product's safety, efficacy and value to achieve optimal pricing strategies and broad market access.

03

Accelerate launch and post marketing activities

Build from existing knowledge as your product progresses through the life cycle, with connected insight across disciplines. 

Greater understanding, treatment development and regulatory measures are introduced to encourage innovation in diseases with low prevalence and high unmet need. All of these aspects make it a very exciting time to work on rare disease.

Still, they represent a myriad of specifics that can be particularly challenging, including…

  • The knowledge gaps/disease natural history is often poorly understood
  • Not adapted standard trial designs
  • Sometimes combined with no standardized definition
  • The limited standard good practice guidelines
  • The lengthy and complicated diagnosis pathway
  • Specific regulatory pathways
  • The overall lack of awareness

Get the right expertise, at the right time, for a winning market strategy.

R&D

We provide disease-specific data and insights to help you prioritize assets and pipeline.

Business development and licensing, EpidemiologyForecastingHealth economics and outcomes researchKOL identification & influence mappingPatient PreferencesPatient reported outcomes,Patient, physician, payer insightsPortfolio assessments & planning 

Syndicated Data: Epi Database®, Forecast Architect®, CancerMpact® 

Clinical Trials

We provide connected clinical and real-world solutions to optimize clinical trials and prepare you for submission.

Phase II-IV trials, Market access & value definition, Medical writing, Non interventional studiesPatient reported outcomes, Registries, Scientific Publications

Submission & Approval

We generate evidence of your product's safety and effectiveness so you can file with the confidence of a well-defined value proposition for all stakeholders.

Health economics and outcomes researchMarket access, Pricing & Value definition, Risk-Benefit optimisation, Scientific Publications

Launch

We offer consultative approaches to ensure the success of your launch using connected commercial and real-world solutions.

Brand StrategyHealth economics and outcomes researchKOL identification & influence mappingMarket UnderstandingNon interventional StudiesPatient, Physician insightsPatient reported outcomes, Patient Preferences, Risk Management Plan, Medical writing

Subscription Data: Epi Database®, Forecast Architect®, CancerMpact®  

Post Marketing

We leverage real-world evidence to expand access through new indications with demonstrated product safety and value.

Brand Optimization, Health economics and outcomes research, Medical writing, Non interventional studiesPatient reported outcomes, Patient Preferences, Registries, Safety and Risk Management

Subscription Data: Epi Database®, Forecast Architect®, CancerMpact®