News & Events

The latest headlines, conversations, and gatherings from Ideogen.

What Are Managed Access Programmes? Definitions and Roles

What a managed access programme is, how it differs from trials and expanded access, and the roles of sponsors, physicians and regulators in the EU and US.

Italy Access Procedure: Legge 648/1996, Compassionate Use and the AIFA 5 % Fund

How a medicine reaches a patient in Italy before or outside its authorisation: the Legge 648 list, the 2017 compassionate use decree and the AIFA fund.

Pre-Approval Access Policies: What Biotech Sponsors Publish

What US law requires an expanded access policy to contain and when to post it, what studies of company policies found, and what EU rules publish.

Shortening the Rare Disease Diagnostic Odyssey in Europe

Why rare disease diagnosis takes years in Europe and what shortens it: genomic sequencing, decision support, European Reference Networks and newborn screening.

Rare Disease Epidemiology and Global Statistics

How many people live with a rare disease, how rare is defined in the EU, US and Japan, why data are hard to collect and what the figures mean for developers.

Commercialisation and Development of Rare Disease Medicines in the EU

Orphan designation, incentives, HTA and pricing, early access and legislation reform: what developing and commercialising a rare disease drug in the EU takes.

How Named Patient Programmes Work in Europe?

How named patient programmes work in Europe: the Article 5(1) legal basis, the physician's role, import and customs, national rules and the US comparison.

Planning Expanded Access Programmes for Biotech Sponsors

How biotech sponsors plan an expanded access programme: eligibility, supply readiness, the protocol, FDA and EMA rules, request review and safety data.

Compassionate Use Programmes for Sponsors: Scope and Obligations

What compassionate use programmes cover and what sponsors must do: requests, patient selection, supply, pharmacovigilance, charging and continuity of care.