Patient centric real-world evidence (RWE) is an increasingly crucial component of benefit/risk and value demonstration, particularly for innovative and potentially transformative orphan and advanced therapy medicinal products and in rare diseases, where the collection of data via traditional routes is difficult. Technological advances permit the collection of rich and continuous real-world data (RWD) from an increasing number of sources and platforms (e.g. wearables, sensors, apps, hybrid sources combining electronic health records and genomics), while next generation analytics enable the translation of these data into actionable RWE.
Strategically planning and implementing the generation of real-world patient centred evidence requires specialised expertise and experience. Observational studies - retrospective and prospective - must provide a design, outcomes, endpoint(s), data quality, transparency and methodological rigour that are adequate and actionable for regulatory and HTA decision-making in order to be relevant for both benefit/risk and value demonstration.
We propose pre-planning real-world and patient-centered research as an integral component of an early access strategy and evidence generation plan in a life-cycle approach (from very early in development to post-authorization). A central component of this planning process is to consider seeking joint scientific and HTA advice and potentially utilizing the various incentives and adaptive pathways emphasizing real-world evidence, which are open to transformative medicines, particularly those eligible for orphan designation in rare diseases.
We support our clients in the strategic evidence generation planning and design of real-world and patient-centric research from very early in development and throughout the life-cycle, including: natural history studies, historical or concurrent clinical trial controls, rare patient and disease registries, pragmatic studies, expanded access & compassionate use programs, retrospective research (electronic medical records, charts, claims), scientific surveys, Patient Relevant Outcomes Measures (PROMs), and the elicitation of patient and carer preferences and unmet medical need.
Services and Capabilities
Real-World Patient-Centric Research Strategy
Real-World Study Design
Patient Preferences, Unmet Medical Needs, Relevant Outcomes
Statistics and Data Analyses
Communication and Scientific Publication
Research and Expert Network
Responsiveness and Effectiveness
Therapeutic and Marketplace Experience
ORPHA Strategy Consulting has experience in a wide range of therapeutic areas, specialized in rare diseases and orphan indications with small, fragmented patient populations and challenging recruitment. Our marketplace experience spans all of Europe, with scientific expert contacts in leading research sites and rare disease centers of reference in the major countries.
In rare diseases, our experience includes:
acute myelogenous leukaemia (AML), multiple myeloma (MM), neuroendocrine tumours, pulmonary arterial hypertension (PAH), Niemann Pick disease, acromegaly, graft versus host disease (GvHD), primary sclerosing cholangitis (PSC), Mucopolysaccharidosis (MPS), Myasthenia Gravis (MG), Graves Orbitopathy (GO), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Antibody-Mediated Organ Rejection (AMR), Narcolepsy, and Friedrich’s ataxia. We are currently working in rare autoantibody-mediated diseases, ophthalmology oncology/haematology, metabolic, CNS and GI indications.
We provide creative and analytically rigorous advice on strategic problems and devise effective solutions to drive our clients' programmes forward. We do this through collaboration, by channelling and merging our expertise with that of our clients. Our approach has been honed in more than 25 years of consulting experience and will flexibly adapt to specific and unique challenges. Please contact us today to start a strategic discussion about early market access strategy.
ORPHA Strategy Consulting
Phase IV Programs LLC
David Schwicker, Principal
4051 Basle, Switzerland
Tel: +43 676 362 9571