A refined approach to orphan drug development is central to meeting patients’ needs

Achieving the optimum balance between accessibility and risk is vital for any drug development, but particularly so in the case of rare diseases, where unmet needs are acute and existing treatments often limited. And yet an imbalance persists, with too few orphan treatments reaching patients via traditional regulatory and access routes. Addressing this, regulators and HTA bodies/payers are looking to accelerate development and early access pathways for orphan drugs, with PRIME and other initiatives encouraging greater innovation around access and time to market.

As this paper argues, however, a much more integrated approach is required to take access to transformative orphan and specialty therapeutics to the next level. Satisfying multiple stakeholders is, of course, a challenge – clinical, regulatory and commercial priorities do not always neatly align. Yet, greater stakeholder co-operation and a more integrated approach will be key to evolving development and authorization lifecycles that put rare diseases and advanced patient-centered solutions centre stage.

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