Tuesday, September 22, 2015

Nearly $1 Billion California Stem Cell Plan: 50 Clinical Trials, Therapy Cost to Consumers Not Addressed

The CIRM view of the dynamics of stem cell research over the next five years
$575 million for clinical, translational
FDA big impediment
Patients want greater voice
Therapy cost to consumers not mentioned

The California stem cell agency will spend the bulk of its remaining $890 million for more advanced research, including as many as 50 new clinical trials, according to a plan released today.

Randy Mills, president of the $3 billion state research effort, is expected to discuss the proposal Thursday during a teleconference meeting of the agency’s governing board. The public can participate from 18 locations, including one in Atlanta.

The figures represent projected spending by CIRM.
Mills expects to spend $400 million on clinical ventures and about $175 million on translational research, the effort to transform basic discoveries into potential clinical use. Basic research would receive about $180 million between now and 2020. That’s when money for new awards would run out under Mills’ plan, which did not address the fate of the agency beyond that date.

The proposal was unveiled in part this week as a discussion item on the agenda for a meeting that comes in less than two days. The information was offered in a 30-page PowerPoint presentation with no supporting documents.

The plan also offered an overview of past activities of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Mills indicated that he had received input from 217 “stakeholders” as part of developing the proposal. The number referred to the persons who responded anonymously to an online survey. 

One of his conclusions from the input: Patient advocates “want a more active role.” He also reported, 
“Many of CIRM’s programs are still not widely known to all potential partners.”
Seventy percent of the stakeholders viewed the FDA “as the biggest impediment to the development of stem cell therapies.” Mills said his plan would “drive implementation of a new FDA paradigm.”

Mills also identified five key risks. They were:
  • “There may be an insufficient number of meritorious therapeutic candidates to reach our goals.
  • “There may be insufficient interest from qualified applicants to participate in key competitions.
  • “The current limited funding of the agency could affect the ability to retain or attract personnel.
  • “Investors may be uninterested in stem cell therapies.
  • “The FDA may be unwilling to improve the regulatory environment. 
Some key parts of Mills’ strategic plan are well underway, such as the start of CIRM 2.0, the name for the effort to step up the funding pace for clinical awards.  At Mills’ request this summer, the board allocated $100 million this fiscal year for clinical work. He is proposing $85 million for next year.

By the end of this week, the agency will be involved in 15 clinical trials. The goal of taking part in 50 new trials does not include those efforts.

Basic research (termed discovery by CIRM) is scheduled for $15 million this year and $50 million in both 2017 and 2018.

Missing from Mills’ presentation is a discussion of the cost to consumers of potential therapies backed by the agency or whether the agency might want to focus on less costly efforts. That question was an early one for the agency. In 2006, the Consumer Watchdog group said,
 “Cures and treatments must be priced so all Californians can afford and benefit from them, not just a wealthy few.”
The issue is back in the news again with national stories highlighting huge drug costs and price increases. Reuters reported today,
“Americans are paying way over the odds for some modern cancer drugs, with pharmaceutical companies charging up to 600 times what the medicines cost to make, according to an independent academic study.” 
Democratic presidential candidate Hillary Clinton, who supports CIRM efforts, said this week she intended to eliminate “excessive profiteering” in the drug industry, triggering a sell-off in drug stocks.

Another strategic issue involves whether the agency should make choices between research that directly involves only a few thousand people versus research that would directly affect millions. Also missing is an overview of the agency’s move away from research involving human embryonic stem cells(hESC), the controversial human tissue that is freighted with moral and ethical questions. As of February, the agency reported that about 240 of its 667 awards involved hESC. 

The desire to fund research involving human embryonic stem cells was the motivating force behind creation of the agency in 2004 through a ballot initiative. The $36 million campaign used former President Bush's restrictions on federal funding for hESC research to build support. Bush's action had stirred an outcry among scientists and many patient advocates. Biotech companies also shied away from publicly backing hESC research because of the controversial issues.

Mills’ strategic plan is well along the way to completion and may well be approved by the board later this year with few significant changes. The public can weigh in, however, with comments on the proposal during the Thursday meeting from the 18 teleconference locations.  The addresses can be found on the agenda.

Comments, criticism and suggestions for the CIRM board can be emailed to Kmccormack@cirm.ca.gov.

The strategic plan revision is set for discussion only this week. Presumably board approval could occur at meetings in October, November or December, the next non-teleconference session.

(Editor's note: An earlier version of this item said 20 teleconference locations were available.)

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