Biogen Inc. (Nasdaq: BIIB) announced today that it is unable to participate in the Jefferies Healthcare Conference on Thursday, June 9, 2022, due to considerations related to COVID.
http://www.publicnow.com/view/E01C98BB4E54AEE17EFFA69AA114CB3329401D18
Alimera Sciences, Inc. (Nasdaq: ALIM) (“Alimera”), a global pharmaceutical company whose mission is to be invaluable to patients, physicians, and partners concerned with retinal health and maintaining better vision longer, announced today that its partner, Ocumension Therapeutics (“Ocumension”) received approval from the National Medical Products Administration for its IND application to begin the Phase III clinical study for fluocinolone acetonide intravitreal implant in support of a filing for marketing approval to treat diabetic macular edema in mainland China.
https://finance.yahoo.com/news/phase-iii-clinical-study-ind-120300229.html
Recently, RealClearHealth ran an article describing the vital role nurses from other countries play in filling nursing jobs in the United States. However, as the article made clear, these applicants barely make a dent in a persistent shortage of nurses facing this country. Much like other shortages we have endured during and after COVID-19 ranging from N95 masks to infant formula, the dearth of nurses is a result of regulatory coziness designed to protect a shrinking group of producers.
According to the U.S. Bureau of Labor Statistics (BLS), the United States is short 194,500 registered nurses a year. It is no surprise then that the epicenter of our nation’s nursing shortage is California, a state which excels in turning every regulatory body into a source of crony capitalism.
California has the highest nursing salaries globally at an average of $120,000 a year. So, if you thought the state would have no problem attracting nurses, you would be only half right.
Despite this high compensation, California has a shortage of 45,000 nurses, a need that existed even before the pandemic. Indeed, in 2020, California nursing schools received 54,823 qualified applications but could only accept 15,002. In other words, each year, 39,000 eligible nursing students are denied access to nursing education in California every year. Nationally, 80,000 nursing applicants were turned away, making California the biggest single contributor to our national nursing deficit.
Greed and self-dealing are the source of the shortage in California and, therefore, nationwide. The California Board of Registered Nursing (BRN), a state agency stacked with California Nurses Association (CNA) members and executives, deserves full credit for creating this nursing desert. The BRN controls the licensing of registered nurses and accrediting nursing schools in California. It has interpreted its authority to regulate the number of nursing students that any school can enroll. The BRN opposes substantially increasing nursing school enrollment, claiming it will lead to inadequate education and a shortage of clinical placements required for graduation and licensure.
The BRN claims that shortages are due to a limited number of qualified instructors to manage student nurses who must complete a clinical rotation to graduate and be licensed. It should know because the BRN is causing the shortage in faculty hiring and clinical opportunities. How is this possible? First, the BRN must approve the hiring of every nursing instructor in California – even though every nursing program is already required to meet national accreditation standards. This redundancy in oversight results in delays and the loss of potential faculty.
Next, the California BRN discourages the use of non-acute facilities for practical training. According to the California Department of Health Care Access, the licensed sixteen nursing programs in the state were using only 121 of the 708 facilities (or 17%). It also turns out that many of these facilities are in medically underserved communities, which have the worst nursing shortages in the state.
What have other states done to address their nursing shortages? Indiana passed the “Nursing Indiana Back to Health Act,” allowing private and public nursing schools to accept additional students. Like Maryland and other states, the Indiana law also addresses the need for additional faculty by allowing nursing programs to hire qualified instructors who are not full-time employees.
These are sensible solutions to a solvable problem that the BRN has opposed. A broad alliance of California health institutions has urged both – all of which have been proposed and opposed by the BRN and the CNA.
BRN policies are deepening health disparities. Indeed, the $1 billion California paid for traveling nurses during the first COVID-19 surge went to nursing working in the wealthiest communities. That money could have been used to hire nursing faculty and pay the tuition of the nursing students – most of whom are Black and Hispanic – who could eliminate the 45,000-nursing shortage. In February, the CNA said state legislators refused to vote on a bill to establish a single-payer health system. The CNA Association and BRN should know turning a shortage of patient care into a cash cow sets a gold standard for the politics of greed.
Robert Goldberg is Vice President at the Center for Medicine in the Public Interest (CMPI).
The two trust funds tracking Medicare income and outgo—for Hospital Insurance (HI) and Supplementary Medical Insurance (SMI)—are supposed to help policy makers see and address financial problems before they escalate into more difficult challenges. However, because Congress designed SMI so that, unlike HI, it can never become insolvent, the construct is weak. The risk now is that Medicare spending is pushing the federal government toward a debt crisis, which in turn may lead to abrupt and indiscriminate cuts in program benefits and other vital programs.
Indeed, Medicare’s trust funds as currently constituted are so flawed that they could be impeding, rather than facilitating, sensible reforms. As has been the case for several years, the 2022 Medicare trustees report, just released by the administration, will generate understandable concerns about the projected depletion of HI reserves. While solving that problem is necessary, it is far from sufficient, as HI finances less than half of Medicare’s annual expenditures. An excessive focus on HI may lead policy makers to assume that SMI spending requires no changes because projections show its trust fund with a positive balance well into the future.
With Medicare now in its second half century, it should not be surprising that many aspects of its original design need updating and modernization, including the framework used to enforce financial discipline across generations. Reform should ensure total Medicare expenditures are matched with sources of funding that do not presume ever-increasing levels of federal debt.
When Congress approved Medicare in 1965, Social Security served as a partial template. The basic idea was to append a new publicly run health insurance plan to the cash benefits provided to the elderly and disabled through the Old Age and Survivors Insurance and Disability Insurance programs. As with Social Security, Congress opted to use trust funds to align Medicare’s outgo with income over time; a projection spanning 75 years can provide early warning of impending problems.
There was one important difference with Social Security, however; not all of Medicare’s expenses would be financed from payroll taxes collected from current workers and their employers and held in a single trust fund. Instead, Congress created two trust funds for Medicare, only one of which—for HI, or part A—conforms to the Social Security model, with revenue from payroll taxes on current workers. The other, for SMI, which pays for parts B and D of the program, gets its revenue from premiums paid by current beneficiaries, and, importantly, from the Treasury’s general fund as needed to fully finance all spending. Consequently, the SMI cannot become insolvent, while the HI can.
The use of two trust funds to finance Medicare is a reflection of the political and market dynamics of the 1960s. At that time, the dominant insurers were the Blues plans, with “cross” policies identified historically with hospital expenses and “shield” versions with physician care. Congress wanted to mirror what many Americans were experiencing in the private market.
There was another political consideration, too; organized physician groups were suspicious of an expanded government role in financing medical care. A separate Medicare trust fund dedicated to non-hospital care, with funding that was reserved for physician services and other ambulatory care and was partially subsidized out of general funds, was a factor in securing the profession’s support.
The 2022 Medicare trustees report, released on June 2nd, provides updates projections of the pace of depletion of HI reserves.
The new projections are likely to be viewed in Congress with some relief because HI insolvency is modestly delayed. In the 2021 report, the trustees estimated that HI would run through its reserves in 2026, after which its annual obligations would exceed incoming revenue on a permanent basis. Over 75 years, the trustees projected HI had a deficit of $4.9 trillion, measured in present value terms. In this year’s forecast, HI will deplete its reserves in 2028, and its shortfall over 75 years is projected to remain as it was in last year’s forecast -- $4.9 trillion.
The SMI trust fund faces no such shortfall because, as described above, it can be fully financed from general funds as needed. Thus, the same 2022 report that tags HI with a multitrillion dollar deficit shows SMI with steady reserves over the next 75 years even though it accounts for about 60 percent of total program spending.
And yet the Treasury’s contributions to SMI are not costless; they expand the government’s annual budget deficits, and thus also add to federal debt. The general fund works as a catch-all for government tax receipts, borrowed money, and spending. Taxes dedicated to specific purposes come into the general account and then are credited to the separate ledgers tracking those activities; spending is assigned in a similar fashion. When total federal spending exceeds revenue, as it does today and will for many years in the future, the deficit is covered with borrowing. Consequently, when the general fund makes its required payments to SMI, the money comes either from existing, non-dedicated taxes (such as individual and corporate income taxes) or from amounts lent to the government by creditors. Because money is fungible, assuming that SMI has first call on income taxes only means that other necessary federal activities must be financed with more debt.
None of this would matter if the amounts involved were trivial, but that is not the case with SMI. The 2022 trustees’ report estimates the general fund contribution to SMI would be $413.9 billion in 2022 and $6.0 trillion over the period 2022 to 2031, reaching the equivalent of about 30 percent of all individual and corporate income tax receipts at the end of 75 years.
Congress hoped beneficiary premiums would serve as a check on SMI cost growth because rapid inflation would fuel political complaints. Initially, the premiums were set to cover half of program expenses. As costs soared in the program’s early years, Congress backed off the policy and allowed premiums to grow more slowly than part B spending. Eventually, a new benchmark was set, with beneficiary contributions covering 25 percent of program costs. When the prescription drug benefit was created in 2003, it also was financed with a 25/75 split between beneficiary premiums and general fund contributions.
With the general fund covering most of the added costs, SMI spending has risen rapidly even as beneficiaries pay more too. In 2022, the standard beneficiary part B premium is $170.10, which is $21.60, or 15 percent, above what it was in 2021 (there is a separate premium for part D coverage, which varies by the plan selected; the base premium is $33 in 2022). Since 2000, the nominal value of the Part B premium has gone up at an average annual rate of 6.4 percent, more than 4.0 percentage points faster than the general inflation rate.
General fund support for SMI is now one of the most consequential line items in the entire federal budget, as shown in exhibit 1. Shortly after enactment, in 1970, it totaled 0.2 percent of gross domestic product (GDP). By 2020, it was 2.0 percent of GDP. The trustees estimate it will reach 2.8 percent by 2050.
Source: 2022 Medicare Trustees’ Report (Expanded and Supplementary Tables and Figures).
Spending on Medicare, along with Social Security and Medicaid, is central to the nation’s fiscal challenge. All three have rising obligations tied to population aging, and spending on Medicare and Medicaid is also sensitive to the cost pressures unique to the health sector. Together, their combined spending has become the dominant force in fiscal matters over the past half century. In 1970, the combined federal expenditure on them was equal to just 3.7 percent of GDP; by 2020, it had reached 11.7 percent, with Medicare growth jumping 3.8 percentage points of GDP during that period (exhibit 2).
The Congressional Budget Office (CBO) expects the pressure to intensify over the next three decades as the full baby boom generation moves out of the workforce and into retirement. By 2050, Medicare spending will have reached 7.3 percent of GDP, or 5.2 percentage points of GDP above the level of spending in 2000. The combined spending on the big entitlement programs (now including the Children’s Health Insurance Program and Affordable Care Act subsidies) will reach 16.5 percent of GDP in 2050.
Source: Congressional Budget Office (Historical Tables and The 2021 Long-Term Budget Outlook).
While federal revenue has risen and fallen over the years, it has never reached a level that would support the spending that is now occurring, and which will only grow in the future. CBO’s projections assume tax receipts will be determined by the requirements of current law and reach a maximum of 19.1 percent of GDP, even as total federal spending (driven by Medicare and other entitlement commitments, along with interest payments on debt) will rise to more than 30 percent of GDP by 2052. Consequently, in the agency’s long-term forecast, deficits soar, as does cumulative federal debt, which would exceed 175 percent of GDP by 2050 (exhibit 2). It is unlikely the federal government could borrow such sums without causing serious damage to the broader economy.
In 2003, Congress, with Republicans in the majority, was concerned about the growing burden SMI’s general fund payments were creating for taxpayers. To compensate, it created a “trigger” tied to the percentage of total program spending covered by general revenue payments to the trust funds. When the Medicare trustees determine in consecutive annual reports that general revenue will exceed 45 percent of program costs in the current year, or during one or more of the subsequent six years, then the president and Congress are required to develop, and consider under expedited procedures, legislative amendments to eliminate the breach.
The scheme has not worked as planned. One problem is that the law creating the trigger, which was focused on standing up the prescription drug benefit, was passed mainly by Republicans, which means the trigger was never embraced by Democrats as a legitimate modification to the program. Indeed, there has been strong objection to the trigger among Democrats from the first years of its implementation.
Furthermore, presidents of both parties have balked at being told by Congress to develop a remedy for consideration in the legislative process; administration lawyers have consistently argued that this requirement is unconstitutional. Consequently, although the trustees have determined that the threshold has been breached fairly regularly since 2006 (and every year since 2018, including in the 2022 report), only President George W. Bush, in 2008, submitted a plan to bring the general revenue share back below the 45 percent threshold.
Medicare’s two-part insurance and trust fund design may have made sense in the mid-1960s, but it no longer does. Modern coverage is not bifurcated in this way, and imposing stricter financial control over facility spending than clinician and outpatient services can distort the policy-making process, and thus also patient care.
Congress should enact reforms to prevent a near-term HI crisis, but it needs to go much further, too. Its primary focus should be on modernizing the program and creating a system of financing that is reliable and sustainable (modernizing the benefit by creating a single plan of coverage is long overdue too). If a trust fund is still the preferred tool for spending discipline, then its tap on the general fund must be strictly limited. For instance, these payments could be tied to growth in the economy irrespective of how fast Medicare spending escalates.
Trust funds are accounting devices that assist policy makers in the financial management of important federal activities. They are always imperfect. Medicare’s trust funds have proven to be particularly defective. Most of Medicare now appears fully solvent even as program spending pushes the entire federal government ever closer to a debt crisis. The coming discussion of HI’s shortfall offers an occasion for revisiting, and fixing, this flawed construct.
James Capretta serves on the advisory board of the National Institute for Health Care Management, which is a forum for the executives of Blue Cross/Blue Shield insurers to discuss major policy questions. He also serves as a senior adviser to the Bipartisan Policy Center and on the policy advisory board of Free the Facts, which sponsors campus educational programs on the nation’s long-term fiscal challenges.
https://www.healthaffairs.org/do/10.1377/forefront.20220603.144424
Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to its candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS).
APOL1-mediated FSGS is a kidney disease in which scar tissue develops on parts of the kidney called glomeruli, which is associated with APOL1 genetic mutations. The APOL1 gene is associated with the innate immune system, and people who have two mutations in the gene are at an increased risk of developing kidney diseases such as FSGS.
This is due to increased toxicity of the APOL1 protein in the kidney, which is the result of inflammatory exposures. Currently, there are no approved treatments for the disease, although treatments such as high-dose steroids can delay the progression to kidney failure.
The FDA designation was based on results from Vertex’s Phase II clinical trials. Results from the trial were shared in December 2021, showing that treatment with the drug produced a clinically meaningful mean reduction in protein in the urine, a sign of kidney damage, in 47.6% of those treated at 13 weeks. Inaxaplin was also well-tolerated, with no serious adverse events observed during the study.
“These results are very promising. For decades, I have cared for patients who have suffered from the rapid onset of kidney disease that progresses quickly to kidney failure, which we now know may have been caused by APOL1 gene variants,” said Glenn Chertow, M.D., M.P.H., professor of medicine at Stanford University School of Medicine and chair of the Vertex APOL1 program steering committee. “This approach has tremendous potential, as it targets the underlying genetic driver of kidney disease in these patients. These data demonstrate the potential of VX-147 as a targeted treatment for a patient population at unusually high risk of progression to kidney failure.”
Inaxaplin is an investigational drug that aims to inhibit the function of the APOL1 protein, which is responsible for kidney cell injury, cell death and damage to the glomeruli in those with APOL1-mediated kidney diseases. The drug is the first therapy designed to target the underlying cause of APOL1-mediated kidney diseases and is the first genetically targeted approach to treating kidney disease.
Currently, a Phase II/III clinical trial is ongoing to evaluate the drug’s efficacy and safety in patients between the ages of 18 and 60 years old with APOL1 mutations. 66 patients will be enrolled in the Phase II dose-ranging portion of the study, with an additional 400 patients estimated to be enrolled in the Phase III clinical trial. The primary endpoint will evaluate inaxaplin’s efficacy in changing patients’ estimated glomerular filtration rate, while secondary endpoints will evaluate the composite clinical outcome, such as the onset of end-stage kidney disease.
In addition to receiving Breakthrough Therapy Designation from the FDA, Vertex also received Priority Medicines (PRIME) Designation from the European Medicines Agency. The designation covered the drug’s use in APOL1-mediated chronic kidney disease. Inaxaplin has become the second nephrology product to receive PRIME designation by the EMA.
https://www.biospace.com/article/fda-grants-breakthrough-designation-for-vertex-s-new-kidney-drug-/
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX9250 for the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP).
According to the FDA, the purpose of the Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. Companies that receive this designation are eligible for benefits including more frequent meetings with and written communication from the FDA, eligibility for accelerated approval and priority review, if relevant criteria are met, and rolling review of the new drug application (NDA).
https://finance.yahoo.com/news/fda-grants-fast-track-designation-110000700.html
PBCAR0191 Achieved 100% Response Rate (ORR), 73% Complete Response (CR) Rate and 50% Durable Response Greater than Six Months Among Evaluable CAR T Relapsed Subjects
PBCAR0191 Achieved Peak CAR T Cell Expansion Matching Data from Autologous CAR T Peak Expansion in Long Term Durable Responders
PBCAR19B CAR T Cell Manufacturing Optimization Process Completed; Dose Level 2 Expected to Commence in Third Quarter 2022
Ongoing PBCAR269A Program Combined with Gamma Secretase Inhibitor Initiating Dose Level 3
Company to Host Webcast and Conference Call Today at 8:00 AM ET
Precision will host a conference call and webcast today at 8:00 AM ET to review its ongoing allogeneic CAR T programs. The dial-in conference call numbers for domestic and international callers are (866) 996-7202 and (270) 215-9609, respectively. The conference ID number for the call is 5754683. Participants may access the live webcast, and accompanying presentation materials, as well as the archived webcast on Precision’s website in the Investors section under Events & Presentations: https://investor.precisionbiosciences.com/events-and-presentations.
https://finance.yahoo.com/news/precision-biosciences-provides-allogeneic-car-110000810.html
