Introducing the Evidence and Insights Programmes

Turning patient experience into evidence that drives change

At DHT.health, our mission is simple but ambitious: to drive change in rare and underserved diseases by addressing challenges through research. Our high-quality data, robust analysis, and actionable evidence make the realities of living with a rare or underserved disease visible, measurable, and actionable. 

Our Evidence and Insights Programmes represent the next step in that mission. These programmes capture the impact, voice, and outcomes of patients and caregivers across conditions where evidence has long been limited or fragmented. By combining rigorous methodology with direct community collaboration, the Evidence and Insights Programmes create a new foundation for understanding disease burden, improving care, and shaping policy. 

What Are Evidence and Insights Programmes?

Our Evidence and Insights Programmes are multi-country research initiatives designed to unlock hard-to-find data, and deepen our understanding of disease-specific patient voice and experience.

Developed in close collaboration with patient advocacy groups, key opinion leaders, and healthcare professionals, each programme aims to capture comprehensive information on:

• The burden of illness associated with a specific rare or underserved disease

• The patient voice and lived experiences of those affected

• Key outcomes, including health-related quality of life

At the heart of each programme lies the PIVOT methodology, a structured, patient-centred framework designed and assembled by DHT.health, with input from patients and other stakeholders. This approach brings together expertise from health economics, real-world research, patient advocacy, clinical practice, and policy.

PIVOT gathers information from a defined Population, focused on the Impact, Voice, and Outcomes associated with their condition, and integrates these elements Together to generate meaningful and actionable insights.

This unified design makes the Evidence and Insights Programmes one of the most comprehensive methods of generating patient-reported datasets in rare and underserved diseases.

Programme data have already been used by multiple stakeholders, informing national health policy, supporting advocacy efforts, and being presented at scientific and patient conferences.

Why Evidence and Insights Programmes Matter Now

The rare disease evidence landscape is at a critical inflection point. Scientific innovation has accelerated, with new therapeutic modalities transforming the possibilities of care, yet real-world evidence has not kept pace.

Decisions by regulators, payers, and health technology assessment bodies increasingly require a more complete understanding of patient experience and societal impact. As recently as September 2025, the European Medicines Agency (EMA) released its Reflection Paper on Patient Experience Data (PED), recognising how systematically collected patient insights can inform medicines development, regulatory submissions, and post-marketing decision making.

Modern datasets need to extend beyond clinical outcomes to include:

• The lived experience of patients and caregivers

• Disease burden, treatment burden and satisfaction

• Health-related quality of life and productivity

• Preferences and willingness to accept trade-offs in treatment

The Evidence and Insights Programmes were created precisely to meet these needs. Each programme combines clinical, economic, and patient-reported outcomes to produce a holistic view of the condition, a view that is relevant across countries, comparable between health systems, and reflective of real-world life.

By combining rigorous methodology with direct community collaboration, the Evidence and Insights Programmes create a new foundation for understanding disease burden, improving care, and shaping policy. They bridge the long-standing gap between clinical data and lived experience, generating insights that are both scientifically robust and deeply human.

How the Programmes Advance the Evidence Landscape

Evidence and Insights Programmes take a direct-to-community approach, partnering with trusted patient organisations and other stakeholders to reach individuals who may not be represented in traditional registries. This ensures that all those affected by a condition, including mild, moderate, and disengaged patients, as well as caregivers and women and girls, all have a voice.

Each questionnaire is written in clear, patient-friendly language and incorporates validated patient reported outcomes. Data are collected ethically and independently, with anonymised or pseudonymised identifiers allowing for future follow-up and longitudinal insights where possible.

The result is a set of datasets that are rich, analysable, and applicable across multiple domains from health economics and outcomes research (HEOR) and real-world evidence (RWE) analyses to patient preference studies and advocacy communications. These datasets are designed to deliver both immediate and long-term value: informing burden of illness assessments, enabling longitudinal tracking of outcomes, and supporting value communication strategies for industry and advocacy alike.

By ensuring that real-world experiences are systematically captured, the Evidence and Insights Programmes not only expand the scope of traditional data collection but also advance the quality, inclusivity, and applicability of evidence across the healthcare ecosystem. In doing so, they help align research, policy, and clinical development with the realities of patient life, turning lived experience into measurable, actionable insight.

Value Across the Ecosystem

For Industry

Evidence and Insights Programmes provide cross-functional value for Medical Affairs, HEOR, RWE, Market Access, and Policy teams. They deliver the patient-level insights needed to conduct activities including but not limited to modelling burden of illness, assessing treatment satisfaction, and informing value communication strategies. Companies gain actionable intelligence on switching behaviour, adherence, and quality of life all essential for ensuring patient-centred value.

For Patients and Advocacy Groups

The programmes ensure that lived experience directly informs the design and evaluation of future therapies. By co-creating the research, advocacy partners can use the evidence to drive awareness, inform health policy submissions, and strengthen their voice in healthcare decision-making. A recent example of this is available here: Underserved, Overlooked.

For the Scientific and Policy Community

The standardised methodology enables data suitable for peer-reviewed publication and presentation, contributing to the global evidence base and helping align research with the realities of patient life.

Current Programmes

The first Evidence and Insights Programme is already underway and findings being presented at scientific and patient led conferences :

• Evidence and Insights Programme: von Willebrand Disease (PIVOT-vWD)

  
  

Further programmes in additional rare and underserved diseases will launch in the coming year, each designed to address specific evidence gaps and provide longitudinal value for patients and partners alike.

A Shared Commitment to Evidence That Matters

Evidence and Insights Programmes are more than data initiatives. They are a collaborative model for building trust, transparency, and transformation in rare and underserved disease research.

By uniting patient experience with methodological rigour, DHT.health and its partners are redefining what real-world evidence can look like, and how it can be used to improve lives.

If you are a biopharma company, patient organisation, or research group seeking to build evidence that drives real impact, we invite you to connect with us.

Together, we can turn real-world experience into measurable, lasting change.