Dolon Institute
Furthering the debate through analysis and insight.

The Dolon Institute, our Value, Pricing and Access (VPA) think tank, provides contributions on topics that enhance patient access and ensure sustainable innovation in rare disease and oncology. We develop analysis and evidence-led insights based on our experience to meaningfully enhance conversation.

Outputs encompass white papers, external events, including Prescient initiatives, funded collaborations with clients, and contributions to external projects by VPA team members.

Publications powered by the Dolon Institute

Whitepaper

Dolon InstituteFurthering the debate through analysis and insight.

Publications powered by the Dolon Institute

ONWARD: Driving equitable access and system readiness in cancer care

ONWARD Driving equitable access and system readiness in cancer care

Taiwan Perspectives from the ONWARD Initiative: Advancing Equitable Access and System Readiness in Cancer Care

Featured articles

Why Orphan Medicines remain out of reach in low- and middle-income countries – and what can be done

Future-Proofing HTA: the case for flexibility in assessment frameworks

Access to multi-indication medicines in underserved autoimmune diseases: Are there better approaches to supporting sustainable access?

Pricing and reimbursement of multiple indication medicines: Can a balance be found between different stakeholder perspectives to optimise value and access for patients, while ensuring sustainable and affordable innovation?

Innovative industry perspective: Assessing the proposed regulatory changes and their impact on innovation and competitiveness of the EU regulatory framework as part of the EU General Pharmaceutical Legislation revision

Supporting patients with underserved autoimmune diseases: Challenges and opportunities for innovative medicines

The transformative value of cancer medicines in Europe

Rare disease policy in high-income countries: An overview of achievements, challenges, and solutions

From Launch to Legacy: Pricing excellence through forecasting & analytics

What do the new Medicare negotiations mean for pipeline orphan therapies?

Rare Disease Day 2025: How might JCA impact orphan sustainability in Europe?

Joint Clinical Assessment: The finger on the scale for orphan sustainability in Europe?

2025 themes for pricing and access success for rare disease and oncology medicines

Pulling back the curtain on Joint Clinical Assessment

Gene therapy mini-series introduction

Novel payment models to facilitate patient access to gene therapies

Facilitating effective health technology assessment for gene therapies

Enhancing the delivery of gene therapies through specific care models: examining centers of excellence (COEs)

Promoting multi-stakeholder education to support access to gene therapy

Evolution of willingness to pay in cost-effectiveness countries United Kingdom, Australia, Canada and Sweden

Revision of the General Pharmaceutical Legislation: Impact assessment of key orphan proposals on Member States

Exploring alternative pricing approaches for orphan medicines

Rare Disease Day 2024 – What does it take to see continued rare disease innovation in Europe?

Revision of the General Pharmaceutical Legislation: Impact Assessment of European Commission and EFPIA Proposals

How do we attain sustainability, affordability andavailability of CAR T therapies?

A critique of the critique: Pharmaceuticals value, price and profitability

Rare innovation: How it happens, when it doesn’t, and what can be done to sustain it

Revision of the Orphan Regulation: Estimated impact on incentives for innovation of changes proposed by the European Commission

Amyotrophic Lateral Sclerosis, A rare neurodegenerative disease: European landscape assessment and policy recommendations for improved diagnosis, care, and treatment

Innovative Contracting for ATMPs in Europe

Recommendations for better use of quality of life evidence in HTA for rare diseases

Mapping the burden of severe forms of epidermolysis bullosa: Implications for patient management

Paid early access in Europe –Is it needed, and what should it look like?

The ‘rituximab effect’: the challenge of low-cost and off-label comparators in rare diseases

Rare Disease Day 2023: Why do rare diseases warrant special treatment?

Introducing the Dolon Institute

Absence of comparative survival data in health technology assessment of oncology therapies

Beyond value: Why cell and gene therapy pricing requires new thinking

Facilitating more efficient negotiations for innovative therapies: A value-based negotiation framework

Double bad luck: Should rare diseases get special treatment?

Consideration of quality of life in the health technology assessments of rare disease treatments

Implementing outcomes‐based managed entry agreements for rare disease treatments: Nusinersen and tisagenlecleucel

How do we attain sustainability, affordability andavailability of CAR T therapies?

Double bad luck: Should rare diseases get special treatment?

Exploring the economics of gene therapy innovation and price

Consideration of quality of life in the health technology assessments of rare disease treatments

Implementing outcomes‐based managed entry agreements for rare disease treatments: Nusinersen and tisagenlecleucel

Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current issues and find potential solutions

Examining the impact of different country processes for appraising rare disease treatments: A case study analysis

HTA appraisal framework suitable for rare disease treatments

How did we get here and where do we go now? An economist’s perspective on EU Orphan Drug Policy

The use of patient‐reported outcome measures in rare diseases and implications for health technology assessment

The estimation of health state utility values in rare diseases: Overview of existing techniques

Estimated impact of EU Orphan Regulation on incentives for innovation

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in Europe

An analysis of orphan medicine expenditure in Europe: Is it sustainable?

HTA programme response to the challenges of dealing with orphan medicinal products: Process evaluation in selected European countries

Projecting pharmaceutical expenditure in EU5 to 2021: Adjusting for the impact of discounts and rebates

Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs: Evidence from four European countries

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

Scientific and social value judgments for orphan drugs in health technology assessment

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Estimating the budget impact of orphan drugs in Sweden and France 2013–2020

A pilot study of multicriteria decision analysis for valuing orphan medicines

What is wrong with orphan drug policies? Suggestions for ways forward

Estimating the budget impact of orphan medicines in Europe: 2010–2020

Work with the Prescient Value, Pricing and Access Team

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Dolon Institute
Furthering the debate through analysis and insight.