|COVID 19 When will it be Ready?|
Saturday, October 24, 2020
Recommendations From the Advisory Committee on Immunization Practices for COVID-19 Vaccination Implementation
Wednesday, October 21, 2020
Pocket - A “robot” doctor told a patient he was dying. It might not be the last time.
Californians Asked Pony Up for Stem Cell Research — Again
SACRAMENTO — In an election year dominated by a chaotic presidential race and splashy statewide ballot initiative campaigns, Californians are being asked to weigh in on the value of stem cell research — again.
Proposition 14 would authorize the state to borrow $5.5 billion to keep financing the California Institute for Regenerative Medicine (CIRM), currently the second-largest funder of stem cell research in the world. Factoring in interest payments, the measure would cost the state about $7.8 billion over about 30 years, according to the nonpartisan state Legislative Analyst’s Office.
In 2004, voters approved a $3 billion, 30-year bond via Proposition 71 to get the state agency up and running and to seed research. That measure will end up costing taxpayers about $6 billion, including interest. The original bond issue Prop 71 is here.
During that first campaign, voters were told research funded by the measure could lead to cures for cancer, Alzheimer’s and other devastating diseases, and that the state could reap millions in royalties from new treatments.
Yet most of those ambitions remain unfulfilled.
“I think the initial promises were a little optimistic,” said Kevin McCormack, CIRM’s senior director of public communications, about how quickly research would yield cures. “You can’t rush this kind of work.”
So advocates are back after 16 years for more research money, and to increase the size of the state agency.
Stem cells hold great potential for medicine because of their ability to develop into different types of cells in the body, and to repair and renew tissue.
When the first bond measure was adopted in 2004, the George W. Bush administration refused to fund stem cell research at the national level because of opposition to the use of one kind of stem cell: human embryonic stem cells. They derive from fertilized eggs, which has made them controversial among politicians who oppose abortion.
Federal funding resumed in 2009, and thus far this year the National Institutes of Health has spent about $321 million on human embryonic stem cell research.
But advocates for Proposition 14 say the ability to do that research is still tenuous. In September, Republican lawmakers sent a letter to President Donald Trump urging him to cut off those funds once again.
The funding from California’s original bond measure was used to create the new state institute and fund grants to conduct research at California hospitals and universities for diseases such as blood cancer and kidney failure. The money has paid for 90 clinical trials.
A 2019 report from the University of Southern California concluded the center has contributed about $10.7 billion to the California economy, which includes hiring, construction and attracting more research dollars to the state. CIRM funds more than 56,500 jobs, more than half of which are considered high-paying.
Despite the campaign promises, just two treatments developed with some help from CIRM have been approved by the Food and Drug Administration in the past 13 years, one for leukemia and one for scarring of the bone marrow.
But it’s a bit of a stretch for the institute to take credit for these drugs, said Jeff Sheehy, a CIRM board member who does not support the new bond measure. He said the agency funded the researcher whose lab discovered and developed the drugs, but CIRM holds no rights to those drugs and doesn’t receive royalties from them.
The state has received about $518,000 in revenue from licensing other Institute-funded discoveries, such as devices, McCormack said.
McCormack also pointed to some promising stem cell therapies still in clinical trials, such as a treatment that has cured 50 children of severe combined immunodeficiency, a genetic disorder often called “bubble baby” disease, and others that have led to “dramatic” improvements in paralysis and blindness, he said.
The campaigns for both bond measures may be giving people unrealistic expectations and false hope, said Marcy Darnovsky, executive director of the Center for Genetics and Society. “It undermines people’s trust in science,” Darnovsky said. “No one can promise cures, and nobody should.”
Robert Klein, a real estate developer who wrote both ballot measures, disagrees. He was inspired to invest in stem cell research after he lost his youngest son to Type 1 diabetes. He said some of CIRM’s breakthroughs are helping patients right now.
“What are you going to do if this doesn’t pass? Tell those people we’re sorry, but we’re not going to do this?” Klein said. “The thought of other children needlessly dying is unbearable.”
Sheehy, who has served on the agency’s board for 16 years, said he’s proud of the work the institute has done but believes it should be funded through the legislature, not by borrowing more money.
“The promise was that it would pay for itself and it hasn’t,” Sheehy said. “We can’t really afford it, and this is the worst way to pay for it.”
Even if CIRM isn’t turning a profit, some researchers and private companies are benefiting from the public money. Take the company Forty-Seven Inc., named after a human protein and co-founded by Irving Weissman, director of Stanford University’s stem cell research program. The state stem cell agency awarded more than $15 million to Forty-Seven, and $30 million to Weissman at Stanford for research.
That money fueled research that uncovered a promising treatment for several different cancers. Gilead Sciences, the pharmaceutical giant, bought Forty-Seven in 2018 for $4.9 billion. Of that, $21.2 million went back to CIRM to pay back Forty Seven’s research grants, with interest.
“Gilead will make far more than that if it turns out to be lucrative,” said Ameet Sarpatwari, a professor of medicine at Harvard Medical School who studies drug development.
Because this kind of work is both expensive and risky, private companies are reluctant to pay for early research, when scientists have no idea if their work will yield results, let alone profits, Sarpatwari said. So the state pays for this work, and drug companies come in to finance later-stage research once a molecule looks promising — and ultimately reap the profits.
“We’re socializing the risk of drug development and privatizing the gains,” Sarpatwari said.
On paper, the institute has stricter pricing regulations than the NIH, which does not require that drugs developed with public money are accessible to the public. In California, companies have to submit plans for how uninsured patients will get medicine and are required to sell those medications to the state’s public health programs at a specified rate.
But in practice, the regulations have never really been tested.
Proposition 14 would add a new rule. It would take the money California makes from royalties and use it to help patients afford those treatments. It also benefits drug companies: Whatever revenue the state makes from these drugs will go back to the companies in the form of state-financed patient subsidies.
The measure also would establish a new working group (complete with 15 new, full-time staffers) that would help make clinical trials more affordable for patients by paying for lodging and transportation to the trials.
And it would increase the size of CIRM’s governing board from 29 to 35. This contradicts recommendations from the Institute of Medicine, which suggested shrinking the board to avoid conflicts of interest. Klein argues the extra board positions are necessary to represent different regions and areas of expertise.
Certain issues have developed in the fifteen years since CIRM was founded: These include:
Conflicts of Interest
“Far too many board members represent organizations that receive CIRM funding or benefit from that funding. These competing personal and professional interests compromise the perceived independence of the ICOC, introduce potential bias into the board’s decision making, and threaten to undermine confidence in the board. Neither the board chair nor board members should serve on any working group. The board itself should include representatives of the diverse constituencies that have an interest in stem cell research, but no institution or organization should be guaranteed a seat.”
“The problematic perception of conflicts of interest has persisted for as long as CIRM has existed. The IOM committee would be less concerned about individual board members with actual or perceived conflicts of interest if the board membership included more truly independent members. The majority of board members should be independent, with no competing or conflicting personal or professional interest. Broader representation from a wider variety of stakeholders will inject new perspectives into the panel and will help to dispel the perception of conflicts of interest.
“CIRM also should revise its conflict of interest definitions to include non-financial interests, such as the potential for personal conflicts of interest to arise from one’s own affliction with a disease or personal advocacy on behalf of that disease. CIRM policies for managing conflicts of interest should apply to that broader definition. "Ultimately, California voters must weigh the possibility of new treatments against the cost of financing them with debt.
The IOM (Institute of Medicine) also recomended changes in governance, the role of economic impact in California and the protection of intellectual property rights.
USE OUR CONTENT
This story can be republished for free (details).
Subscribe to KHN's free Morning Briefing.
Monday, October 19, 2020
As federal agencies have made a quick pivot to virtual options in order to provide health care and medical attention online during the COVID-19 crisis, more lawmakers are calling to maintain expanded access to telemedicine—even in a post-pandemic world.
Sen. Brian Schatz, D-Hawaii, said in a statement that telemedicine options that have made care possible during the pandemic should be permanent changes to the health care system after the Centers for Medicare and Medicaid Services released new data Wednesday on the use of virtual medical services.
“Telehealth is popular and bipartisan because it reduces the cost of health care and improves quality and availability,” Schatz said in a statement Thursday. “The skyrocketing use of telehealth during the pandemic shows that we cannot and should not go back to the Stone Ages of telehealth coverage.”
Several provisions in the CARES Act helped strengthen telemedicine access by relaxing rules and providing funding. It’s these provisions Schatz wants entrenched in standard operating procedures post-pandemic.
The data from CMS shows a dramatic increase in the use of telemedicine services. Before the pandemic, according to the report, around 13,000 Medicare beneficiaries received telemedicine in a week. During the last week in April, that number was closer to 1.7 million.
Nearly 5.8 million CMS beneficiaries have had a typical office visit online since the coronavirus pandemic began. Telehealth has been particularly popular for mental health appointments, according to the report, with 60% of psychiatrist and psychologist visits taking place virtually.
The CMS data tracks with what other agencies have recorded as well. The Veterans Affairs Department reported encouraging telehealth numbers back in April. The department touted a 70% increase in mental health appointments taking place using VA Video Connect, which facilitates remote face-to-face interactions.
In testimony to the House Budget Committee Wednesday, Dr. Robert Wah, former associate chief information officer for the Military Health System, listed telemedicine as a top area in which the federal government should invest.
“COVID-19 has highlighted the value of virtual, remote health care as effective, efficient, and well accepted,” Wah said.
On July 2, a bipartisan group of 38 senators sent a letter to Health and Human Services Secretary Alex Azar and CMS Administrator Seema Verma requesting a written plan, including a timeline, outlining permanent changes to Medicare telehealth rules. The letter called rule changes that allowed for the increased use of telehealth options a “lifeline” for patients and care providers.
“As you stated, it is hard to imagine rolling back these changes,” the letter reads. “However, we are hearing from patients and providers who are concerned about when Medicare’s temporary changes to telehealth rules will be rolled back and whether they will receive any advance notice.”
Though Schatz’s name was not on the July 2 letter, the senator has been a telemedicine advocate since before the pandemic. Along with Sens. Roger Wicker, R-Miss, Ben Cardin, D-Md., John Thune, R-S.D., Mark Warner, D-Va. and Cindy Hyde-Smith, R-Miss, Schatz introduced a bill in October 2019 that would expand Medicare’s telehealth services.