P4A Webinars

In hour-long, quarterly broadcasted industry-stakeholder discussions, P4A experts tackle complex subjects such as gene therapy and emerging technologies to help shape industry-wide conversations.

Webinar: EU-5 has become EU-4! What happened to the UK?

Focus on the EU5: that is the geographical scope we have become used to identifying market opportunities and preparing the ground for orphan drug launches. After all, these 5 countries (France, Germany, Italy, Spain and UK) represent approximately 80% of potential healthcare spend in rare diseases in Europe and are duly prioritised by pharma companies...

Webinar: Early Access Programs in Europe: ‘A Friend or Foe?’

In the EU, Early Access Programs enable patients to access therapies pre-commercialisation for diseases with high unmet need. Known as Expanded Access Programs in the US, these initiatives allow for orphan drugs to be made available to patients with rare diseases before they have been formally approved by regulators...

P4A’s three-part webinar series on Gene Therapy

Gene Therapy is emerging as the new frontier of human medicine with the potential to cure hard-to-treat rare diseases. The human genome project’s conclusion in 2003 followed by the first gene therapy approval in Europe (2012) and the US (2017) were key milestones.

However, very little is known about this approach to treatment, creating myths and expectations.

The P4A webinar series uncovered such myths – from clinical evidence to commercial concerns – and put forth the readiness of key markets to adopt and integrate gene therapies.

We gained expert insights from a cross-section of stakeholders from the medical community, patients to industry and payers.

Part 1: Gene-therapy a revolution or evolution of healthcare?

In the first of three webinars held in November 2017, P4A provided historical context, understanding of what gene therapy is; its clinical and environmental challenges and whether it has the potential to truly revolutionize the healthcare environment.

Part 2: U.S. Lessons Learnt from Kymriah & Luxturna

In the second of three webinars, P4A is focussing on the US environment for gene therapies. Single application cell and gene therapies in the US face health insurance challenges driven by a short-term focus, disconnection between price reporting and outcomes value, and complex distribution models...

Part 3: Innovative Access Agreements – A US & EU Perspective

In the last of the three webinar series on cell and gene therapy, P4A alongwith the MKO Group explored the role of access agreements in patient access. Payers are increasingly concerned over the high cost of cell and gene therapies accompanied by deep uncertainty in clinical data...