Rare Disease Consulting

Medically relevant data and consulting to develop and commercialize treatments for rare diseases.

Greater understanding, treatment development, and regulatory measures are introduced to encourage innovation in diseases with low prevalence and high unmet need.

We can address challenges that include:

  • Knowledge gaps and disease natural history that are often poorly understood
  • Unadapted standard trial designs
  • Occasional combinations with no standardized definition
  • Limited standard good-practice guidelines
  • Lengthy and complicated diagnosis pathways
  • Specific regulatory pathways

The hallmark of rare diseases is low prevalence and high unmet need, but the unique challenges in conducting research and planning treatment development and commercialization, must also be considered.

These challenges fall into 4 main areas: Data, Expertise, Reach and Empathy which are clearly often insufficient or lacking
We bring these 4 key components together to create a holistic, evidence-based offer to inform critical decision making. 

 

Collect

evidence of your product’s safety, efficacy, and value to achieve optimal pricing strategies and reach more patients.

Prioritize

your pipeline and related investments with highly specialized guidance.

Build

on existing knowledge to accelerate launch and post-marketing activities.

Actionable

recommendations and ongoing consulting expertise from our team of R&D experts.

> 80

country presence and local team flexibility assure the right expertise, at the right time, for a winning market strategy.

1

Research & development (R & D)

We provide disease-specific data and insights to help you prioritize your assets and optimize your portfolio.

Rare Disease Consulting Research and Development

We support you with...

Modularity & flexibility
Our team combines unique, modular, and flexible assets and approaches in rare diseases with a knowledgeable, seasoned, and integrated team. Our research approach brings synergies through ongoing consultations throughout the various steps of the process. 

Comprehensive understanding
In our research we seek to understand all stakeholders, attitudes, decisions, experiences, and preferences beyond the level of understanding that can be derived by existing tools and measures. Health Economics and Outcomes Research (HEOR) provides comprehensive research and data to evaluate patient outcomes, treatment patterns, resource utilization, and costs.

Specificity
Market and disease-specific expertise will ensure a smooth project flow, aligned with your research objectives, timelines, and strategy.

2

Clinical Trials

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

Rare Disease Consulting Clinical  Trials

We support you with...

Connections between the clinical world and the real world
We provide connected clinical and real-world solutions to optimize clinical trials. Future Trends and Insights evaluates current and future drug competitors and offers expert analysis of the competitive landscape. It explores potential changes in treatment practices in the U.S., Western Europe, and Japan based on an evaluation of recently published clinical data, regulatory advances and setbacks, and ongoing clinical trials.

Readily available data
Additionally, our comprehensive databases support clinical trials that provide real-world data and information to influence physician and other stakeholder strategies.

3

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.

Rare Disease Consulting Submision and Approval

We support you with...

Connectivity
We provide connected clinical and real-world solutions that prepare for your submission. Our customized services help inform local health technology assessment (HTA) and value dossier submissions and can generate publications in peer-reviewed medical journals.

Pharmacoepidemiology plans
We support the development of pharmacoepidemiology plans for regulatory approval and 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.

4

Launch

We offer consultative approaches to ensure your launch's success, using connected commercial and real-world solutions.

Rare Disease Consulting Launch

We support you with...

Launch readiness
Scientific and commercial insight combine for a winning market strategy and successful launch. We offer consultative approaches to ensure your launch's success, using connected commercial and real-world solutions. By understanding stakeholders’ behaviors, motivations, and unmet needs, we help you identify where your brand should enter the market and how to maximize uptake.

Continuity across stages
Building from existing knowledge as your product progresses through its life cycle, with connected insight across disciplines, we can help accelerate your launch activities.

5

Post Launch Marketing

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

Rare Disease Consulting Post Launch Marketing

We support you with...

Speed
We can help you accelerate post-marketing activities by generating evidence early to expand access through new indications, scientific differentiation, and product positioning.

PASS and PAES
We can also assist you with all steps of designing and executing a post-authorization safety study (PASS) or post-authorization efficacy study (PAES). Our solutions enable you to adhere to risk management requirements, post-marketing commitments, and establish evidence for safety and effectiveness, in a real-world setting.

Registries
Our specialized strategic consulting services can build disease registries to describe the natural history of a disease from patients suffering from, or at risk of, an illness. Our product registries focus on patient treatments with a product or device and often fulfill post-marketing authorization requirements that focus on assessing safety or harm. 

Completeness
Interacting directly with patients using a specific treatment, our active drug surveillance studies help evaluate the probability and frequency of adverse events as part of post-marketing risk assessment for medical products.