Regenerative Medicine Sector ARMs Itself With New Advocacy Group

Article reprinted from "The Gray Sheet" - March 15, 2010

Companies and researchers developing cell-based and tissue engineering therapies have formed a lobbying group to improve the federal policy environment surrounding their efforts. Read more...

Regenerative Medicine Sector ARMs Itself With New Advocacy Group

Article reprinted from "The Gray Sheet" - March 15, 2010

Companies and researchers developing cell-based and tissue engineering therapies have formed a lobbying group to improve the federal policy environment surrounding their efforts.

The Alliance for Regenerative Medicine officially launched last September and is now preparing to make itself heard in Washington.

One of the group's top priorities is enactment of regenerative medicine legislation, which ARM is shopping around to lawmakers. The alliance hopes to have a bill introduced and debated in Congress this year.

"The legislation is designed to facilitate advances in regenerative medicine by increasing [and] coordinating federal funding efforts in research and moving towards a regulatory paradigm that enables approval of safe and effective regenerative medicine products," explained Michael Werner, a partner in the Washington, D.C., law offices of Holland & Knight and co-founder of ARM, in an interview.

He says the association is also engaging FDA, CMS and other agencies to push for clearer and more coordinated policies for regenerative therapies, including bioengineered tissues and organs, stem cell treatments and related technologies.

ARM will soon begin disseminating information that explains regenerative medicine and its significance to policymakers.

"There is a whole body of information that frankly has never really made its way into the media" in terms of "what the field is, what the economic impact is, and what its impact will be in terms of standard of care," said ARM co-founder Morrie Ruffin, managing partner for Adjuvant Global Advisors.

ARM: Focus On More Than Embryonic Stem Cells

Werner and Ruffin are former colleagues at the Biotechnology Industry Organization.

According to Ruffin, BIO and other trade associations touch on "bits and pieces" of issues impacting the regenerative medicine space, "but not really the embodiment of what this field is."

The alliance is intended to fill that gap.

"There is clearly the need to focus on the technology and the science and communicate what it actually is, what it means, beyond all of the media attention that has been focused on embryonic stem cells," Ruffin said.

"The field goes well beyond that and involves a lot of really incredible things."

ARM member companies include Tengion, whose first product in development is a bioengineered bladder grown on a proprietary scaffold from a patient's own cells (The Gray Sheet' April 10, 2006), Advanced BioHealing, maker of the DermaGraft bioengineered skin substitute, and Theregen, which is developing a cell-based patch designed to induce formation of new blood vessels in a diseased heart.

There are also firms focused more directly on stem cell therapies, notably Geron, which is attempting to initiate clinical development of human embryonic stem cells to treat spinal cord injury.

Most of the private-sector participants in the alliance are start-ups, but there two very significant exceptions: Johnson & Johnson and Pfizer.

Further, about one-third of the 45 members (as of Feb. 26) are either academic institutions or patient advocacy groups. Examples include the Wake Forest Institute of Regenerative Medicine, the Cleveland Clinic and the Juvenile Diabetes Research Foundation.

"We wanted this to look like more of a movement and not just a corporate lobbying effort," Ruffin said.

Four-Year-Old Gov't Proposal Cited As Model

Regenerative medicine is still an early-stage movement, though, that needs a kick-start from the government to reach the next level, ARM founders argue.

Some promising steps have been taken in the past, which they hope to build on.

An interagency government working group issued a report in 2006 proposing a coordinated federal regenerative medicine initiative that the alliance supports. But there has been too little follow-up since then, they suggest.

"That report was issued four years ago, and we think that there is a lot of stuff that is still applicable today," Werner said.

The federal working group proposed an increase in long-term funding for regenerative medicine research. It also recommended establishing a government council, modeled on councils formed for the Human Genome Project and a national semiconductor research initiative, to set milestones for advancing the science and to encourage collaboration among government agencies and between government, industry and academia.

ARM's legislative agenda "will incorporate those types of elements," Werner said.

FDA Combo Product Policies Are One Focus

Another of the group's major goals is to improve FDA's capability to address oncoming regenerative medicine technologies.

On this front, there has been more recent progress.

Early last month, FDA requested $25 million in its fiscal year 2011 budget to fund research into early-stage technology, including regenerative medicine and nanotechnology.

And on Feb. 24, FDA and the National Institutes of Health announced a collaborative regulatory science initiative to improve processes for evaluating next-generation therapies (The Gray Sheet' March 1, 2010).

"I think there is a recognition at the agency that they really need to ramp up some of their scientific base so that they will be able to appropriately evaluate the newer products that are coming forward," Werner said.

John Walker, CEO of stem-cell-technology firm iPierian and chair of ARM's board of directors, points to two principal areas of regulatory science where the alliance is seeking near-term improvements.

One is to make sure FDA is conducting research "to answer some specific questions that relate to this emerging field that would preclude the need for any individual sponsor that comes to the agency to have to answer it on their own," he explained.

As a prime example, Walker pointed to questions about safety issues associated with undifferentiated pluripotent stem cells and how long the cells have to be tracked to measure the potential risk.

This type of issue "can be answered a little bit more thematically by the agency and not have every individual company have to go back and readdress the same issue over and over and over again," he proffered.

The alliance will also seek clearer regulatory procedures for biologic-device combinations, which Walker says are a fundamental component of many regenerative technologies.

"When you begin to look at the delivery of cells, you also begin to get into the field of various types of new devices that will be necessary to be associated with the delivery of those cells," he explained.

FDA has made significant efforts in addressing questions in this area through its Office of Combination Products, but Walker suggests more needs to be done to set the stage for many cell-based therapies.

ARM also plans to lobby for Medicare reimbursement policies that recognize the value of regenerative medicine therapies. The alliance plans to engage with CMS on a range of coverage and payment issues as they arise.

But in the very near term, the alliance is focused principally on getting its message out.

The 2006 interagency proposal on regenerative medicine "is very clear about the economic impact, [and] it is very clear about the disease impact of conditions that can be addressed with regenerative medicine," Werner stressed. "We just have to keep people focused on that."

- David Filmore

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