Signals Blog


A networking highlight of the conference is the boat cruise

Cell and Gene Therapy World, or “Phacilitate” as delegates like to refer to it, happened last week in Miami, Florida (January 22-25).

The conference, with 1,500 in attendance, brings together industry professionals from more than 30 countries. The event explores trends and opportunities that are shaping the future of cell and gene therapies (CGTs). As the organizers describe it, “we work together to improve, disrupt and drive the future of advanced therapies.”

This year’s event grew significantly in size. For the first time, Cell and Gene Therapy World partnered with World Stem Cell Summit (WSCS) to offer two very exciting programs in the same venue. With so many topics and speakers, there really was something for everyone.

A highlight of Cell and Gene Therapy World is Ed Field’s Top 10 events list. Each year he looks back at the preceding 12 months to select the most significant highlights and put them in context. Here are his picks:

  • 10 Uniqure abandons Glybera
  • 9 Hitachi $75M acquisition of PCT
  • 8 Clinical start and hold of Cellectis’ off-the-shelf CAR-T therapies (the studies were put on hold after two deaths and later resumed)
  • 7 Spark/Pfizer hemophilia B clinical data (“they presented “hot” data at ASH in December”)
  • 6 Gilead/Kite $567M acquisition of Cell Design Labs
  • 5 $11.9B acquisition of Kite by Gilead (this is the largest acquisition in the CGT space, even including Celgene’s recent acquisition of Juno)
  • 4 FDA issues comprehensive regenerative medicine (RM) framework and grants numerous RMAT designations (Ed says 10, ARM says 12)
  • 3 FDA approval of Gilead/Kite’s Yescarta ($373,000)
  • 2 FDA approval of Novartis’ Kymriah ($475,000). First approval for a CAR-T
  • 1 FDA approval of Spark’s Luxturna (first approval for an inherited (eye) disease. Will be priced at $850,000 for both eyes.)

A gene therapy wins #1 spot! On a related topic, read Deanna Petersen’s post about her faith in gene therapies, going back 25 years. She is the Chief Business Officer for AVROBIO and uses the post to reveal the exciting news of AVROBIO’s $60 million series B financing to advance its gene therapy pipeline for several indications.

But back to the conference.

Reimbursement was the topic du jour, which is not surprising given the top three events in 2017. This is always a relevant theme when you assemble members of industry, but reimbursement takes on even greater importance in the current climate.

We heard from Dr. Pascal Touchon, Novartis Oncology, on why the pricing of Kymriah is “fair” and on how Novartis is making the treatment accessible to any patient who needs it. Kymriah even comes with a money-back guarantee.

Dr. Touchon was followed by Dr. Kenneth Schaecher, University of Utah Health Plans, who shared his thoughts on how reimbursement of CGTs will happen in the United States with its mixed public-private system. Dr. Schaecher compared health insurance to car or home insurance. Health insurers must predict their costs and set a premium that still allows for some margin to bring back to shareholders. They set rates annually with each and every client/patient.

L-R: Drs. Sven Kili, GSK, Jim Richardson, Foundation Fighting Blindness, Kenneth Schaecher, University of Utah Health Plans

Dr. Sven Kili, GlaxoSmithKline, talked about Strimvelis, which gained approval from the National Institute for Health and Care Excellence (NICE) for the rare disease ADA-SCID or “bubble boy” syndrome in October 2017. According to a GSK spokesperson, “Strimvelis is the first corrective gene therapy for children to be approved anywhere in the world, and this draft NICE guidance will be encouraging news for those families in the UK who have a child with ADA-SCID and no suitable matched stem cell donor.” (FiercePharma)

Dr. Kili shared that reimbursement isn’t always a guarantee, regardless of how promising your therapy appears. You have to demonstrate to the payer that the treatment saves money in the long run. Quoting Dr. Kili quoting a payer, “a dead patient is a much cheaper patient than a living patient.”

A significant challenge for companies and payers is the lack of data with these new therapies. Since industry can’t provide efficacy data from 70 years of monitoring patients, payers have to have some faith that the treatments will actually last a lifetime, as promised. This is a substantial difference in the CGT space as compared to more traditional therapies.

L-R: Drs. Sven Kili, GSK, Chris Mason, AVROBIO, Panos Kefalas, Cell and Gene Therapy Catapult

Reimbursement was also a topic of discussion at the Translational Academy, co-hosted by CCRM again this year. Dr. Panos Kefalas, Cell and Gene Therapy Catapult, provided a reimbursement overview that led into a panel discussion with Chris Mason, AVROBIO, and Sven Kili.

As heard elsewhere at the conference, the lack of long-term data is one of the issues hampering CGT reimbursement, but Dr. Kefalas suggests early engagement with payers is key to counter this problem. One solution could be outcomes-based agreements to reduce some of the risk that make payers so uncomfortable.

The panelists discussed annuities in CGT reimbursement – learn more here – and reimbursement in Japan. In addition to changing their regulatory process, the Pharmaceuticals and Medical Devices Agency (PMDA) has made conditional marketing approval easier so companies can generate revenue while clinical trials are underway. As Professor Mason explained, your efficacy requirements are lower to obtain conditional approval. You are then given seven years to generate the efficacy data. RepliCel, a Canadian company that is very familiar with PMDA and the regulatory climate in Japan, summarizes the situation here.

This is only a very small glimpse into the conference. With multiple tracks over several days and two groups vying for delegates’ attention, there was lots to absorb and too much to include here. For more, I recommend you search the hashtag #CGTWorld on Twitter or watch RegMedNet’s highlights videos. Kevin McCormack, of CIRM, also blogged about a WSCS panel on Alpha Clinics. If you were in Miami and want to share your key takeaways, please leave a comment below!









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Stacey Johnson

Stacey Johnson

For almost 20 years, Stacey has been providing strategic communications counsel to government, corporate, technology and health organizations. Prior to that, Stacey was at the CTV Television Network, first as a researcher, then as a story producer for “Goldhawk Fights Back,” a special ombudsman segment that aired weekly on the National News and Canada AM. Before joining CCRM as the Director, Communications and Marketing, Stacey was the Director of Communications for the Canadian Arthritis Network. Stacey is editor of Signals. You can follow Stacey on Twitter @msstaceyerin.