P4A is hosting a 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.

During the P4A webinar series, we will uncover these myths – from clinical evidence to commercial concerns and put forth the readiness of key markets to adopt and integrate gene therapies.

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

Webinar 2: U.S. lessons learnt from Luxturna & Kymriah

In the second of three webinars, P4A is focusing 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. To mitigate these challenges, health insurers and gene therapy companies are exploring different payment models to help improve access and spread costs.

Following Kymriah’s approval, Novartis announced a collaboration with the Centres for Medicare and Medicaid Services wherein children and young adults would face no charge if the patient was unresponsive to the treatment within a month.

Other models include a staggered payment structure with a fixed upfront payment at the time of treatment origin followed by regular instalments until the bill is covered or the disease returns.

In January 2018, Spark Therapeutics announced payer programs for Luxturna that were designed to overcome cost and value-based challenges. These include:

  1. Outcomes-based rebate arrangement with a long-term durability measure
  2. Innovative contracting model under which payments would be made over time

We will be exploring the validity of these programs from two perspectives – health insurance plans and gene therapy manufacturers.

Key topics to be discussed at the webinar are:
  • Is it important to have alternative mechanisms and payment models for gene therapy?
  • What are the key lessons learnt from Spark’s payer programs?
    • Payer perspective
    • Manufacturer’s perspective
  • What are the key considerations for patient assistance and payer programs by gene therapy companies?
  • What are the relevant and appropriate mechanisms?



Sophie Schmitz Managing Partner, Partners 4 Access

Sophie is a leading expert on rare diseases and orphan drugs. She is heavily involved in advising orphan drug clients on price and market access strategies, with the ultimate goal of optimizing their launch. In a 20-year career span, Sophie has worked in various pharmaceutical therapy areas and medical devices focusing on key product launches in both strategic planning & implementation.

Dr Edmund Pezalla CEO, Enlightment Bioconsult and former VP for Pharmaceutical Policy and Strategy, Aetna

Edmund is an innovator in payer strategy for pharma and device manufacturers. As VP for Pharmaceutical Policy and Strategy in Aetna, he developed and coordinated strategy for pharmaceutical evaluation and coverage across both the medical and pharmacy benefit, created Aetna’s framework for innovative contracts, and developed Aetna’s public policy positions on drug and device coverage. He is active as a payer expert on a number of policy working groups including the New Drug Development Paradigm Project at MIT and was previously a Scholar-in-Residence at Duke-Margolis Health Policy Center in Washington, DC

Prasun Subedi Senior Director at Center for Health System Innovation and Leadership Patient and Health Impact, Pfizer

Webinar 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 revolutionise the healthcare environment.

If you missed the event, you can download the webinar here.

Join us for our second webinar