Our latest infographic breaks down a critical, but under-discussed, implication of the Medicare drug price negotiations: the risk to orphan medicines. While the IRA aims to lower the cost of some therapies, its structure may unintentionally discourage biopharma companies from... View More
This ISPOR Europe 2024 panel report explores pricing and reimbursement (P&R) of multiple medicines in Europe. Work has culminated from the panellists and audience of ~800 participants, where they discussed key challenges and forward-looking solutions for multi-indication products. The insights... View More
In recognition of Rare Disease Day 2025, we turn our attention to EU Joint Clinical Assessment (JCA), to explore the potential impact of JCA across three key dimensions important to sustaining investment and launch of new rare disease treatments in... View More
In this report, explore six themes Dolon believes will impact pricing and access success for rare disease and oncology medicines in 2025. Theme 1: Rising pricing tension Theme 2: Protectionist Policies Theme 3: GLP-1 Wave Theme 4: Oncology Crowding Theme... View More
EU Joint Clinical Assessment (JCA) has the potential to transform the evaluation of medicines in Europe, including orphan drugs. While thereās hope that JCAs will streamline fragmented processes and improve patient access, the road ahead is far from simple. Concerns... View More
Willingness to pay for new medicines has steadily declined in real terms in countries that incorporate cost-effectiveness thresholds in their pricing and reimbursement (P&R) processes. Nations such as the UK, Australia, Canada, and Sweden have not adjusted their thresholds for... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
This ISPOR Europe panel explored the evolving landscape of CAR T therapies, their high potential for lifelong benefits, and the economic and logistical hurdles they face. From complex manufacturing to unique pricing and reimbursement challenges, CAR T therapies require novel... View More
On March 12th 2024, Richard Sear and Emilie Neez explored alternative pricing approaches for orphan medicines in a presentation at the World Evidence Pricing and Access Congress in Amsterdam. Supporting slides are available here; this companion piece provides our (the... View More
While the revision of the General Pharmaceutical Legislation (GPL) targets EU-level frameworks and processes, proposed changes are sure to have immediate as well as downstream consequences at the country level. This paper explores these potential consequences, focusing on a selection... View More
We are looking for a Contract & Compliance Coordinator to join our team. This role is essential for providing internal support to the business on a range of commercial contracting activities. The successful candidate will be responsible for monitoring and... View More
In recognition of Rare Disease Day 2024, we explore four critical elements to sustaining rare disease innovation in Europe. These elements are outlined below, building on published work by Dolon team members.
The European Commission has proposed revisions to the Pharmaceutical Legislation, with the view to tackle unmet medical needs, competitiveness, access and affordability. This update represents a once-in-a-generation opportunity to strengthen the European biopharmaceutical ecosystem, if fit-for-purpose policy options are implemented... View More
Addressing remaining unmet needs in rare diseases hinges on a positive and enduring policy environment which helps to achieve the necessary clinical and economic conditions for drug development in small, hard-to-treat populations. This white paper examines through historic case analysis... View More
Despite the tremendous therapeutic advances delivered in rare diseases since the adoption of the Orphan Regulation in 2000, the European Commission has proposed a set of legislative revisions aimed at bolstering innovation whilst enhancing access and affordability. This study, based... View More
The European Amyotrophic Lateral Sclerosis (ALS) Coalition has published a paper that sets forth 10 policy recommendations aiming to improve the lives of people living with ALS and their caregivers. The paper sheds light on what it means to receive... View More
The past 5 years have been a period of great innovation for cell and gene therapy with a wave of very exciting and effective therapies approved in Europe. While the clinical potential of these products are great, there are recognised... View More
Dolon sponsored World EPA Congress 2023 and had the opportunity to present at the conference. In this infographic, we provide a summary of key discussion points from the roundtable ‘Paid early access in Europe – Is it needed, and what... View More
In recognition of Rare Disease Day 2023, we explore four key reasons why rare diseases warrant special treatment. These reasons are summarised below, building on published work by Dolon team members.
The Dolon Institute aspires to contribute to the policy conversations around patient access and promoting innovation in rare and severe diseases. Our work is grounded in the fundamentals of orphan medicine economics and experience engaging with stakeholders around the launch... View More
Dolon is pleased to announce the launch of the Dolon Institute, which aspires to contribute to the policy discussion that surrounds the development and availability of innovative medicines for rare and severe diseases globally. We hope to do our part... View More
This paper brings together a multi-disciplinary panel of expert co-authors who share perspectives on the current challenges, implications and potential solutions for absence of comparative survival data in health technology assessment (HTA) of oncology therapies.
Double bad luck: Should rare diseases get special treatment? Journal of Medical Ethics. 2022 Feb 1;48(2):99-100.
Facilitating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework. International Journal of Technology Assessment in Health Care. 2022;38(1).
Gene therapies are revolutionising the treatment of severe conditions, offering previously unattainable advances in health gains. Simultaneously, this therapeutic potential is matched by concerns around price and affordability. Are these fears reflective of reality? To address this question requires an... View More
Consideration of quality of life in the health technology assessments of rare disease treatments. The European Journal of Health Economics. 2021 Oct 29:1-25.
Although the Orphan and Paediatric Regulations have been successful at stimulating innovation in these previously underserved areas, significant unmet medical needs remain. EFPIA held in May 2021 a workshop, which gathered Senior staff from member company R&D, Clinical, Strategy, and... View More
This page describes a tool that is available only to clients and partners of Dolon, for the purposes of transferring personal and/or protected data. Using the web interface Your contact at Dolon will provide you with account details in... View More
Implementing outcomes-based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel. Pharmacoeconomics. 2021 Sep;39(9):1021-44.
Privacy policy for experts Your privacy At Dolon Group (Dolon), we respect your privacy and are committed to protecting your personal information. This policy explains how we collect information, what we do with it and what controls you have over... View More
Examining the impact of different country processes for appraising rare disease treatments: a case study analysis. International journal of technology assessment in health care. 2021;37(1).
The divergence between the rapid pace of scientific progress in rare diseases and declining sentiment towards orphan medicines reflects a paradox that is at the heart of the European orphan drug debate. While the last twenty years have witnessed a... View More
The use of patient-reported outcome measures in rare diseases and implications for health technology assessment. The Patient-Patient-Centered Outcomes Research. 2021 Sep;14(5):485-503.
The European Commission is currently reviewing the 2000 Regulation on orphan medicinal products (OMP). It is critical that any consideration of reform of the Regulation should be informed by a robust understanding of the relationship between incentives, investment, innovation and... View More
The estimation of health state utility values in rare diseases: overview of existing techniques. International Journal of Technology Assessment in Health Care. 2020 Oct;36(5):469-73.
Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches. Orphanet journal of rare diseases. 2020 Dec;15(1):1-4.
An analysis of orphan medicine expenditure in Europe: is it sustainable? Orphanet journal of rare diseases. 2019 Dec;14(1):1-5.
Gene and cell therapies represent some of the most advanced treatments being employed in biomedical research to improve the length and quality of life of patients with rare and complex diseases. Patientsā access to these therapies has been hampered by... View More
Projecting pharmaceutical expenditure in EU5 to 2021: adjusting for the impact of discounts and rebates. Applied health economics and health policy. 2018 Dec;16(6):803-17.
Dolon is a trading name of Dolon Ltd, registered in England and Wales with company number 08809427. Registered office: 63 St Mary Axe, London EC3A 8AA, United Kingdom. Cookie Policy We use cookies to help us improve your experience of... View More
Privacy policy Your privacy At Dolon Group (Dolon), we respect your privacy and are committed to protecting your personal information. This policy explains how we collect information, what we do with it and what controls you have over your personal... View More
Dolon is a trading name of Dolon Ltd, registered in England and Wales with company number 08809427. Registered office: 63 St Mary Axe, London EC3A 8AA, United Kingdom. Terms of use 1. Terms By accessing the website at https://dolon.com, you... View More
Dolon is a trading name of Dolon Ltd, registered in England and Wales with company number 08809427. Registered office: 63 St Mary Axe, London EC3A 8AA, United Kingdom. Terms of Use 1. Terms By accessing the website at https://dolon.com, you... View More
Headquartered in London, Dolon has a team of 50 enthusiastic and experienced consultants located across Europe. While diverse in expertise, geography and language, we all share a positive outlook and a collaborative approach to working with our clients. Our Senior... View More
Use the map below to learn more about how Dolon can provide strategic market access support for your product or rare disease portfolio throughout all stages of development.
Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs: evidence from four European countries. Value in Health. 2017 Jul 1;20(7):919-26.
HTA programme response to the challenges of dealing with orphan medicinal products: process evaluation in selected European countries. Health Policy. 2019 Feb 1;123(2):140-51.
Recommendations from the European working Group for Value Assessment and Funding Processes in rare diseases (ORPH-VAL). Orphanet journal of rare diseases, 12(1), pp.1-15.
Evidence generation for an injectable chemotherapeutic with orphan designation for pancreatic cancer. For advanced, previously untreated, metastatic pancreatic cancer ā a severe illness with very short life expectancy.
A US-based biotech with a market-leading portfolio of drugs to treat rare and severe diseases, producing an innovative, chemotherapeutic agent approved for use in second line for a rare form of cancer.
Orphan drug pricing strategy. A small-cap, biotechnology company producing a biologic molecule for ultra-orphan indication; licensed and launched in US and Japan, applying for a license in Europe. Aim to explore the conceptual value of the molecule vs. other ultra-orphan... View More
A biopharmaceutical company planning to launch a newly acquired ultra-orphan product in the EU for the treatment of an extremely rare skin condition. In the EU, there were no currently authorised treatments for this indication.
A US-based biotech with a market-leading portfolio of innovative therapies for rare and severe diseases, facing a lack of consistency in payer assessment systems for rare diseases across countries globally.
A client wanting to create an interactive pricing exercise for external audiences to help educate them about the challenges and different perspectives associated with pharmaceutical pricing. Two different pricing simulations were created: one from the payer perspective and one from... View More
At Dolon, our specialist knowledge in rare diseases is our greatest asset and we want to share that with our clients. Below, we invite you to explore some examples of how we can work with you to translate our knowledge... View More
Treatments for rare diseases face a uniquely challenging pricing and reimbursement environment. To be successful, companies need a specialised approach: Pricing strategies need to be transparent, value-based, and defensible to all rare disease stakeholders Communication of product value must reflect... View More
Dolon is a strategic pricing and market access consultancy specialising in rare diseases. With headquarters in London and staff located across Europe, weāre dedicated to accelerating access to life-saving treatments for patients all over the world. Three core principles underpin... View More
Scientific and social value judgments for orphan drugs in health technology assessment. International Journal of Technology Assessment in Health Care. 2016;32(4):218-32.
Headquartered in London with staff across Europe, Dolon is a dynamic, fast-growing company with a strong reputation and a bright future. At Dolon, we place particular importance on emotional intelligence and the ability to get along with other people ā... View More
Headquartered in London with staff across Europe, Dolon is a dynamic, fast-growing company with a strong reputation and a bright future. At Dolon, we place particular importance on emotional intelligence and the ability to get along with other people ā... View More
Payers and policymakers are constantly revising the mechanisms and processes they use to make reimbursement decisions for rare disease treatments. There is, as yet, little consensus on the best approach for assessing the value and determining the price of rare... View More
Pricing of rare disease medicines is both controversial and complex. With small patient populations and a high level of risk in the R&D process, many payers recognise the tension between the price of new medicines and the need to incentivise... View More
Successful market access depends on demonstrating the value that innovative new medicines provide to payers, clinicians, patients and society. In rare diseases, that task is harder. Disease mechanisms are more complex, patient burden is poorly documented and information on natural... View More
Treatments for rare diseases face a uniquely challenging pricing and reimbursement environment. To be successful, companies need a specialised approach: Pricing strategies need to be transparent, value-based, and defensible to all rare disease stakeholders Communication of product value must reflect... View More
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat. Duis aute irure dolor... View More
Treatments for rare diseases face unique challenges in getting to market. Rare diseases are hard to diagnose, challenging to study and difficult to treat. Patients need access to effective therapies, but healthcare budgets are squeezed. The way that value is... View More
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Orphanet journal of rare diseases. 2015 Dec;10(1):1-9.
Estimating the budget impact of orphan drugs in Sweden and France 2013ā2020. Orphanet Journal of Rare Diseases. 2014 Dec;9(1):1-9.
A pilot study of multicriteria decision analysis for valuing orphan medicines. Value in Health. 2013 Dec 1;16(8):1163-9.
What is wrong with orphan drug policies? Suggestions for ways forward. Value in health. 2012 Dec 1;15(8):1182-4.
Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet journal of rare diseases. 2011 Dec;6(1):1-0.