Rare diseases

Rare diseases Agency Consulting firm Experts Specialists Consultancy

Optimize your market access and care pathways for patients with orphan diseases

For more than 30 years, Alcimed has been supporting its clients in their projects to innovate, understand and gain access to orphan drug markets.

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    The challenges related to rare diseases and orphan diseases

    The rare disease market is experiencing strong growth and transformation across the world. Over 230 orphan drugs have been approved on the European market since early 2000.

    As a result, the typology of rare diseases, their challenges and their market situations are evolving significantly. This term now includes:

    • ultra-rare pathologies without any suitable medical solution,
    • relatively unknown rare diseases still awaiting solutions,
    • rare but well-known pathologies, with several treatment options, representing considerable progress for patients in established markets that are more similar to so-called “specialty” markets.

    Even though this market is developing, manufacturers, biotechs and research centers are facing many challenges, including:

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      How we support you in your projects related to rare diseases or orphan diseases

      For more than 25 years, Alcimed has been supporting its clients on many issues related to orphan and ultra-orphan diseases, in Oncology and outside Oncology. Indeed, we have carried out more than 150 projects in this field on more than 100 rare diseases for different actors such as:

      • National and European institutions and research centers (for example, the European Commission, INCa, AFM / Genethon)
      • European and North American biopharmaceutical and pharmaceutical companies, biotechnology companies and professional associations (e.g. Genzyme (Sanofi), Shire (Takeda), Endo, Amgen, Chiron, Actelion (J&J), Alexion (Astrazeneca), PTC Therapeutics, Vertex, Sanofi, J&J, Roche, Merck-Serono, Novartis, Novagali (Santen), Spark Therapeutics (Roche), Insmed, Bone Therapeutics, Cellectis, NPS Pharma (Takeda), Intermune (Roche), EFPIA, EBE, EuropaBio etc.)
      • Patient associations (e.g. SFPA).

      The diversity of our clients (industrial companies, biotechs, research institutes, professional associations, national or European institutes, etc.), the geographic areas we explore, and the types of projects we develop, give us a global and in-depth understanding of the issues related to rare diseases.

      Our projects cover areas as diverse as new technologies and therapeutic approaches for orphan diseases, search for funding opportunities, preparation for the launch of new orphan drugs, understanding of disease management and patient care pathways, assessment of market and licensing opportunities, market access, pricing and reimbursement models, regulatory developments and operational support (patient identification, care pathways optimization and many more).

      Examples of recent projects carried out for our clients in rare diseases

      • Support for a pharmaceutical player in optimizing the emotional journey of patients with a rare disease

        One of our clients, a leading pharmaceutical player, wanted to improve the care of patients with a rare disease by developing a high value-added service offering that improves their well-being and quality of life throughout their care pathways (from diagnosis to palliative care).

        To do this, our team interviewed patients, caregivers and associations in each country concerned in order to identify the levers for improving their well-being and their quality of life that could be addressed by the development of services. Following this, our team pre-tested these service ideas with healthcare professionals, before organizing a workshop to co-construct a service development roadmap with the medical and marketing teams of our client.

        Ultimately, several services were deployed in pilot countries and then extended to other countries in the area, improving the patient journey and positioning our client as a major player in public health.

      • Roadmap for launching a new treatment for a rare genetic disease in Europe

        Alcimed supported a US biopharmaceutical client to define the launch strategy for its orphan drug in Europe. It then helped its client to structure a concrete and operational plan, both at a European level as well for each target country.

        As a real roadmap for each of the key functions of the company (medical, marketing, production, regulatory, sales), the plan extends from pre-launch (before obtaining the MA, the Marketing Authorization) until the first sales.

        Our team is also involved in regularly updating this plan, depending on internal or external developments around this launch.

      • Optimization of the care pathway for patients with a rare disease in Europe

        We have supported the European Medical Department of a pharmaceutical manufacturer in optimizing the care pathway for patients suffering from a rare respiratory disease in several European countries. As this rare disease is difficult to diagnose and often confused with more common respiratory diseases, the patient diagnosis time was too long (2 to 3 years). Our client wanted to position itself as a public health player by providing reference hospitals with Alcimed’s service to:

        1. Diagnose the current coordination of care between the different health actors registered on the patient journey
        2. Co-define with this multidisciplinary team an ideal patient care model and an associated action plan
        3. Implement actions defined at local and national level.

        The result for our client? A reduction in the time taken to diagnose patients and a general improvement in physician coordination, an increase in therapeutic support and a change of perspective for the sales teams.

      • Study of the regulatory framework: accessibility of treatment for orphan diseases

        Support for a player in the pharmaceutical industry in understanding the fundamental factors of market access for orphan drugs in 9 emerging countries.

        By taking stock of internal knowledge and collecting missing information in terms of the market and regulatory analysis in particular, Alcimed has enabled its client to obtain an overview of the regulations on orphan drugs in emerging countries and to classify these countries according to their level of accessibility.

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