Tuesday, October 15, 2013

Partnering forum seeks to build the collaborations necessary to bring cell therapies to the market successfully

Named for the La Jolla Torrey Pines Mesa in San Diego, the Stem Cell Meeting on the Mesa opened its eighth annual meeting Monday. For the past three years, the original one-day scientific meeting at the Salk Institute, which will take place Wednesday, has been preceded by a two-day partnering forum organized by CIRM and the Alliance for Regenerative Medicine (ARM) (my colleague Cynthia Schaffer blogged about that event).

The goal of the forum is to foster partnerships at many levels, between young companies and venture capitalists, between biotech companies and big pharmaceutical companies, between academics and industry, and importantly between these science sectors and the regulatory and payer agencies that sign off on new therapies and the patient groups whose voices need to be heard by those regulators and payers.

Quite frankly, the first year of the partnering event felt like we were pushing these concepts ahead of a field that was not quite ready. Last year, the partnering portion of the event started to take off and you could really feel a positive momentum. This year the event has grown 25 percent from last year, with 400 people attending and a sense of excitement that several of the companies in the room will make it to market. A few already have.

Geoff MacKay, chairman of ARM, opened the morning. As CEO of Organogenesis he could address real success. The firm has two approved products, one for wound healing and one for rebuilding soft tissues in the mouth. He noted that ARM had recently done a phone survey of top leaders at all the major pharma companies and 100 percent said they were now into regenerative medicine in some fashion. That is a turn around from the past that few in the room expected.

The first panel of the morning discussed the global impact of the field from the perspective of affordability and availability of the products. The latter referring to the difficulties of manufacturing a cell-based product in large quantities, with high purity and cleanliness, and creating a system to ship the cells where they are needed quickly and safely. All admitted these are huge hurdles for the industry, but they seemed to think the hurdles could be leapt.

A regulatory pathway pioneered in South Korea and recently adopted in Japan drew considerable interest. In Korea, after early phase safety and initial tests of effectiveness products can be approved and commercialized on a “conditional” basis. This time-limited approval can be used to generate data on larger numbers of patients, and allow the company to be reimbursed while it gathers that data. This could reduce the cost of traditional late stage testing and open up potential therapies to more patients sooner in the process. After a set time, the government reviews the additional data and either offers permanent approval or withdraws approval altogether.

Some industry experts have talked about a similar system here, but no one here on the Mesa seemed to be expecting rapid adoption in the U.S.

Don Gibbons

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