Phacilitate Leaders World 

Are We Ready for a $5m Gene Therapy?

Are We Ready for a $5m Gene Therapy?

Episode two of the Phacilitate Leaders World super plenary sessions with Dave Lennon, President of Avexis, Inc

Episode two of the Phacilitate Leaders World super plenary discussions sees Anthony Davies quizzing Dave Lennon, President of AveXis about the projected cost of (the now FDA-approved) Zolgensma, which, at the time of filming was in the $4-5m range.

However, the reported cost upon FDA approval last week of $2.1m per patient has further pushed the drug pricing boundaries, paving the way for a new era of pricing and reimbursement.

So, how does this compare to the spend of our healthcare on caring for long-term diseases? Think dialysis, enzyme replacement therapies, oligonucleotide therapies and the like... Dave gives his take on this, ICER's health economics evaluation and how they are working with the healthcare system and payers around reimbursement from instalment plans to amortisation.

Anthony also gives some insight into his research, which hypothesised that if the three recently approved advanced therapies (Kymriah, Yescarta, Luxterna) reached 100% market penetrance at current North American pricing then the effect of that on North American drug spend would be LESS than a 0.1% increase. A comparable situation at 20% had the spend more than double.

Watch the interview to hear more about pricing, dosing challenges, AveXis' approach to manufacturing and more.

  • Real thinking forward combining experts in the field from manufacturing, regulatory and value vision” – “great network with strong experience and sharing risks & opportunities
    Anne-Virginie Eggimann
    bluebird bio
  • Great program, great people, great venue.
    Klaus Kühlcke
  • Dynamic, interesting and highly interactive event that promotes exchange and networking in highly specialized field of gene therapy.
    Nathalie Clement
    Powell Gene Therapy Center, University of Florida
  • Phacilitate provides a unique forum, bringing together research, process development, and commercial leaders on the cutting edge of cell, gene, and immunotherapy. A great conference for anyone wanting a comprehensive view of the field.
    Dr James Trager
  • It was all business. I’ve never been to an event where over 80% of the conversations I had were constructive to my business objectives.
    Kurt Gielen
    Chemelot Campus B.V.
  • Great way to expand network with global experts in cell and gene therapy who are facing similar challenges.
    Steven Goodman