Driven by the advent of precision medicine and empowered patients, the current healthcare landscape stresses the “need for speed” in biomedical innovation: more rapid development, accelerated and conditional approval, and – crucially – timely patient access to medical innovations. Here “the science has overtaken the system”: innovations are coming to market with ever fewer data (e.g. Phase II POC) and uncertainties whether the anticipated clinical benefit will be verified in real-world practice and possibly undiscovered risks pose challenges to payers and HTA bodies. This can result in delays or restrictions in access to potentially transformative treatments for patients with debilitating and life-threatening conditions, whose prime focus it is to improve their quality of life.
Developers with orphan and advanced therapeutics in their pipeline, which may be eligible for accelerated approval, should therefore consider implementing effective early access strategies, which are crucial to informing, enhancing and supporting strategic thinking on
Early access strategy is developed through research, innovative thinking, and interactive discussion on the following key elements:
Unmet Medical Need
Patient Centricity and Engagement
Compassionate Use (Expanded Access)
Collaborative Strategy Development
ORPHA Strategy Consulting wishes to develop long-term partnerships with clients, for an individual compound or at the portfolio level. Every potential early access strategy is different. Success thus originates through collaboration, by merging our specific expertise with that of the client. We pride ourselves on our responsiveness to clients’ needs and our approach will flexibly adapt to individual requirements.
This favours an iterative and highly interactive strategy development process :
Thank you for your interest. To start a strategic discussion on early access, please contact David Schwicker.
ORPHA Strategy regularly publishes Executive Briefings which focus on news and current trends in early access, adaptive development pathways, transformative medicines, orphan drugs and designations, rare diseases, and real-world patient-centered evidence. They are available for download here: ORPHA Executive Briefings
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