[VAR_PRACTICE_NAME] logo for print

6359 Tribute Lane
Dora, AL 35062


Phone: 205-648-8429
Cell: 205-566-6090

Contact Us
shadow
Southern Society for Clinical Investigation - Dedicated to the advancement of medical research
Uncategorized

ZIKA funding failures: Executive branch gridlock

Before the Congressional recess:  Before the Congress left Washington for their summer recess, I reported on the partisan battle over additional funding to combat a potential ZIKA virus public health crisis (SSCI HeLP line for June 2016).  Recall that the Obama Administration requested $1.9 billion in new emergency funding, and the Congress worked that number down to a $1.1 billion funding request by removing funds not directly related to the ZIKA crisis.  Unfortunately, Congress was not able to pass the Senate compromise bill, despite a 52-48 vote in favor of the bill–recall that to advance through the Senate, a bill must receive 60 votes in favor of the bill.  I also wrote that as much as $622 million of unused Ebola appropriations could be repurposed to support ZIKA eradication and research.  This is about half the amount contained in the compromise bill that was defeated in the Senate, but it is still a great deal of money to initiate action on this emerging public crisis.

Current status of ZIKA expenditures:  The Obama Administration and the CDC have repurposed successfully over $600 million to be used to combat ZIKA.  However, the Administration has disbursed only about one-half of its reprogramed dollars or about $201 million.  Similarly, the CDC has released a little more than $100 million (again about one-half) of its available funds.  As of early August, the Executive branch Office of Management and Budget confirmed that $385 million of repurposed funding is available for the Administration to allocate to the fight against a potential ZIKA outbreak.  If the public health situation is dire, and if the funding need is urgent, how is it that the Administration is not disbursing all of the available funds rather than just half?   This is as lot of money, hundreds of millions!   In the August 3 editorial page of the Wall Street Journal, the editorial writers assert that the Department of Health and Human Services spokesman Bill Hall attributed the delay of disbursing available funds to combat this urgent public health crisis to “bureaucratic federal procurement regulations”.  Is it unconscionable, or not, that the Administration cannot manage to allocate hundreds of millions of dollars already in its accounts to combat this emerging ZIKA outbreak because of “bureaucratic federal procurement regulations”?  How can the President berate the Congress for going on recess and not doing its job, when the Administration is not doing its job either!  This partisan political story just gets worse and worse while a public health emergency festers.  In fact, the CDC recently issued its first ever travel alert in the US for parts of Miami concerning the increased risk of ZIKA infection, especially for pregnant women.

Government fails us:  The Congress has failed to appropriate what the Administration asserts is the necessary funding to combat an emerging ZIKA public health crisis.  The Administration has failed to disburse the hundreds of millions of dollars languishing in OMB accounts.  Sounds to me like the gridlock in Congressional is being matched now by bureaucratic ineptitude in the Executive branch.  This has to be the quintessential example of the government and our elected officials failing us, their constituents!  Perhaps after a crisis develops, Congress will pass a bill with any needed additional funds.  Perhaps if a crisis develops, the President will sign the bill and then instruct his executive departments to disburse the funds appropriately and expeditiously.  Of course, each branch of our government will blame the other branch for their public health failure!  Will the public’s health suffer because the ZIKA issue has become a “political football” in the midst of a major election year where the parties seem to be focused on scoring political points rather than on serving the public’s health?

Biomedical Research, Legislation

ZIKA virus funding fails: Partisan Politics 1, Public’s Health 0!

ZIKA virus funding fails: Partisan Politics 1, Public’s Health 0!

 

Bipartisan compromise fails again.  Once again, Republicans and Democrats in the Congress demonstrate that they would rather quibble and argue than act in the best interests of the people they serve.  In February, the White House requested $1.9 billion in emergency funding to combat an emerging Zika virus public health crisis.  In an effort at bipartisan compromise, funding not directly related to the ZIKA crisis was removed from the original Administration request leaving $1.1 billion of new funding for prevention, research, education, health services, international aid, and vaccine development.  This bipartisan compromise measure passed the Senate in May by an 89-8 vote in favor of the compromise.  This week, the House-Senate conference report compromise, which had just passed in the House of Representatives, was rejected by the Senate on a vote of 52-48 in favor of the compromise bill.  However, to pass Senate procedural hurdles, the vote in favor of the compromise bill needed 60 or more votes, so fell 8 votes short.

This 52-48 vote in favor of the compromise bill still fails to achieve the required Senate threshold. However, this does not necessarily mean the bill is totally dead.  The bill could come up for another vote when the Senate returns after the July 4 holiday break.  However, is there reason for optimism?  If the bill succeeds, then additional compromise will almost certainly be required—so will either party “compromise” to achieve a vote in favor of the public’s health.  If the bill fails again, then there will not be adequate funding for Zika research or infection prevention and no extra funds for mosquito eradication as we enter the mosquito season, especially in the deep South.  Funds from unused Ebola appropriations, and some unused ACA appropriations funds, could be allocated to Zika eradication and research—approximately $622 million, or about half that contained in the current bill and about a third of the original Administration request.

In any piece of legislation, there are always numerous points of disagreement. This is just one reason that bipartisan cooperation is always so important, institutionalizing the never-ending need for compromise.  Two of the issues that upset Democrats about the bill include some so-called “poison pills” in the current legislation. For example, the bill from the House of Representatives directs funds through hospitals and public health clinics, but excludes women’s health clinics like Planned Parenthood.  This is a “hot-button” issue for the Democrats who argue that those at greatest risk for serious adverse effects from Zika infection are the children of pregnant women!  So how can any bill exclude women’s health clinics from the funding stream?  But the Republicans have an intense disdain for that organization, Planned Parenthood.  Another point of difference is that part of the bill waves the EPA permitting process for use of certain pesticides for a 180 day period for emergency mosquito eradication.  Democrats argue that this component of the bill weakens the clean water and air protections.  But the Democrats have an intense disdain for any restrictions placed on the EPA.

Of course, there are other issues of mutual disagreement. The only matter that seems to be in short supply is a desire to compromise, with both parties getting some of what they want but not all they demand. So can our long serving, experienced, intelligent and clever, political leaders sublimate their political priorities to forge a compromise solution that serves the public good?  Stay tuned!  We should know the answer to this question within the next 30 days.

Legislation, Medicare

Physician payment reform reform: Replacing SGR with QPP, MIPS, and APMs—all part of MACRA

The Medicare Access and CHIP Reauthorization Act (MACRA) replaced the infamous Sustainable Growth Rate (SGR, see posting in September 2016) with a new Quality Payment Program (QPP).  The new QPP itself has two component payment models, a Merit-based Incentive Payment System (MIPS) or an Alternative Payment Model (APM).

Recall that the SGR formula was the method used by the Center for Medicare and Medicaid Services (CMS) to control the rate of Medicare spending for physician services.  The SGR was part of the Balanced Budget Act enacted by Congress in 1997.  This SGR formula was flawed from the outset.  It did not consider adequately the volume of services provided, the quality of patient care, nor the efficiency of health care delivery.  In April 2015, the Congress passed comprehensive legislation with bipartisan support to repeal the SGR formula and adopt a new value-based reimbursement system focused on patients and the quality of care—this is the Medicare Access and Children’s Health Insurance Program Reauthorization Act (MACRA, H.R. 2).  Congress wrote the law with general guidance, tasking the Executive branch with implementation, as is often customary.  CMS is the executive branch entity of the Department of Health and Human Services responsible for translating the general guidance in the enacted Congressional law into the rule making and regulatory apparatus that informs and regulates physicians and other health care provider groups and organizations.   CMS completed their task faithfully this year and produced 962 pages of new regulations for you to comply with in order to be reimbursed for services you provide to Medicare beneficiaries.  Are you delighted that I reminded you about all of this?

So, what is the new Quality Payment Program? Physician and other provider entities now have two payment system options to choose from—MIPS and APMs.

MIPS—this new merit-based payment system combines parts of the patient quality reporting system (PQRS), the value modifier (value-based payment modifier), and the Medicare Electronic Health Records (EHR) incentive program into a single program.  The success measures are clearly in quality of care, resource use, clinical practice improvement, and meaningful use of certified EHRs.  In this payment system, physicians will continue to be reimbursed by fee-for-service, but they will receive modest annual increases of 0.25% beginning in 2019.  Theoretically, these physicians could earn “bonus payments” also, but only if the federal government determines that their performance on quality metrics merits a “bonus”.

APM—with this payment model, the government shifts regulatory control away from reimbursing individual physician to reimbursing organizations who manage patient care (such as Accountable Care Organizations, Patient Centered Medical Homes, or bundled payment models).  A stated goal of this payment model is to increase transparency of physician-focused reimbursement models.  Physicians who work for, or in, such health care management systems are eligible for annual payment increases of 0.75% plus bonuses distributed only if the organizations hit the government’s spending targets.

Regulatory environment—Physicians may worry that they are now facing a Catch-22 situation, culminating in reduced reimbursements for their services while coping simultaneously with an increasingly powerful and cumbersome government bureaucracy.  Patients too are facing problematic choices. Health care insurance premiums are rising rapidly.  For example, in Florida, insurers are requesting average premium increases of 17.7% for individual policies and 9.6% for small-group plans.  Virginia’s largest insurer is seeking a 15.8% premium increase.  In Iowa, Wellmark Blue Cross and Blue Shield has contacted customers warning them of potential premium increases of as much as 38-43% next year.  In addition, rising premiums will be exacerbated further as health insurers leave the Affordable Care Act (ACA) marketplace, leaving behind fewer health insurance options.  Recently, the largest healthcare insurer in the US, United Health, has left the ACA marketplace in 27 states.  Unfortunately, physicians and their patients will continue to encounter a burgeoning government health care bureaucracy.

For additional information see: CMS.gov/Medicare/Quality-Initiative-Patient-Assessment

Legislation

Veterans Access, Choice, and Accountability Act of 2014

Veterans Access, Choice, and Accountability Act of 2014 (113th Congress; HR3230; S2424).
The pervasive problems of access to health care for veterans in the VA health system precipitated new Congressional action. While the VA leadership has been working to correct problems with access to health care, they have been unable to put this crisis in care and loss of trust behind them. In 2014, Kizer (former VA Undersecretary for Health) and Jha recommended specific strategies to move the VA forward. Just this year, the current VA Undersecretary for Health published his perspective and plans in the New England Journal of Medicine. Notwithstanding hard work at the VA over the past 2 years, a recent Wall Street Journal (WSJ) editorial castigates what it calls “the ‘corrosive culture’ at Veterans Affairs”. Clearly, problems with access to health care persist at the VA!
Before passage of the Veterans Access, Choice, and Accountability Act (VACA), veterans had several other choices for receiving health care from non-VA sources. All veterans who are over age 65 years (especially Vietnam veterans) are eligible for all components of Medicare. These veterans are dually eligible and can receive care simultaneously from a private provider through Medicare and from a VA provider. Other veterans have access to health care through VA’s fee-basis system —veterans meeting certain criteria get a “fee-basis card” allowing them to access private health care providers — or through the Department of Defense Tricare program (military personnel, military retirees and their dependents).
Components of the VACA
VACA is a new attempt to improve further veterans access to health care. There are a few criteria veterans must meet to be eligible for this enhanced access. First, there must be no medical appointment available within 30 days of a veterans preferred date or the date determined as medically necessary by their provider. Second, veterans must reside more than 40 miles from the closest VA medical facility. In addition to these two criteria, a veteran must meet one of the following criteria. 1.) The veteran must enroll with the VA by August 1, 2014 and have contacted the VA for a medical appointment. 2.) The veteran must be a combat-theater veteran discharged or released from active duty 5 years prior to VA enrollment.
The new law has several key components (I discuss only a few).
Improving access
Veterans who meet the criteria and elect to seek care from an eligible non-VA health care provider are able to do so once the VA confirms their eligibility and vets the non-VA provider–those veterans receive a “Veterans Choice Card”. By law, this program operates for only 3 years or until funding in the “Veterans Choice Fund” is exhausted. The WSJ editorial observes, “The law allows veterans to see outside doctors, but only for 60 days. Then it’s back to the VA queue”. One wonders if VACA actually provides veterans new choices for non-VA care or merely provides time for the VA to resolve its health care access problems.
Health care staffing
This part of VACA directs the VA to increase its Graduate Medical Education (GME) residency positions up to 1,500, emphasizing residency positions that improve veteran’s access to primary care, mental health, and other specialties that the VA deems appropriate. Furthermore, VACA extends the VA’s existing Health Professionals Educational Assistance Program until 2020 and increases the maximum reimbursement ceiling for education debt reduction from $60,000 to $120,000. In addition to these GME enhancements, there are provisions to recruit and appoint qualified personnel to fill occupations within the VA with the largest staffing shortages. These provisions in the new law should help the VA recruit additional medical professionals, and by increasing staffing, improve access to services for veterans.
There are many other components of the VACA, including provisions affecting health care related to sexual trauma; provision of additional veteran’s educational benefits; and VA personnel authorities and disciplinary procedures. While the VA continues to experience serious problems with access to health care, one might question whether Congress has provided the VA all the tools needed to improve performance. This is most obvious in the case of so-called personnel authorities and the ability to reward high performing employees while disciplining or terminating poor performers. The initial results reported in the WSJ editorial are discouraging!
Also see:
Kizer KW and Jha AK. Restoring trust in VA healthcare, N Eng J Med 2014;371:295-297.
Shulkin DJ. Beyond the VA crisis—becoming a high-performance network. N Eng J Med 2016; 374:1003-1005.
K M Rotunda. The “corrosive culture” at Veterans Affairs. WSJ editorial April 11, 2016; A11.

Legislation, Uncategorized

Senate Mental Health Reform Act (S2680): Legislation sans Appropriation.

The Senate Committee on Health, Education, Labor, and Pensions (HELP Committee) unanimously, and therefore with full bipartisan support, passed legislation to reform the provision of “mental health” care.
Goals of the legislation: The goals include efforts to increase access to “mental health” care; improve training for those who care for people with “mental illness”; and promote better enforcement of existing “mental health” parity laws—making insurance companies reimburse for “mental health” treatment the same as for “physical health” treatment. The bill is composed of six titles. For example, Title III proposes support for state responses to “mental health” and substance abuse disorder needs. Title IV promotes access to “mental health” and substance abuse care, and Title V does the same explicitly for children and adolescents. The bill authorizes (not appropriates—do you understand the difference) block grants to states for “mental health” services and for prevention and treatment of substance abuse disorders.
Limitations: Foremost is the fact that the HELP committee lacks jurisdiction over funding. Stated differently, the committee that passed the legislation cannot actually appropriate funds to support implementation of the legislation. The Congressional Budget Office (CBO) scored the bill and estimates that it would cost between $40 to $80 billion dollars over 10 years—a new appropriation that would have to be offset with cuts elsewhere to maintain overall budget neutrality. Nonetheless, the bill could move to the Senate floor as early as next week to debate funding and to entertain any amendments.
Amendments on the Senate floor: What happens next is not readily predictable. For example, Senator Collins lamented that “exclusions of reimbursement for certain residential mental health services is a concern.” The Senator might offer amendments to repeal the current prohibition on Medicaid paying for inpatient care for anyone over age 21years or under age 65 who resides in an institution for “mental disease” (IMD). Removal of this so-called IMD exclusion would be very costly even though it would remove a major barrier to treatment of “mental illnesses”. Other Senators are “concerned also about Medicaid’s prohibition against reimbursements to hospitals and facilities larger than 16 beds for mental health and substance abuse treatment.” These are difficult issues, especially concerning funding decisions. Given the harsh reality of the current budget deficit crisis, this bill may fail to gain any appropriations in the ensuing Senate debates.
Does all this make sense to you? One additional comment from Senator Collins caught my attention. She lamented, “It is stunning to me that in this day and age we do not treat mental illness in the same way that we treat physical illness.” Her observation has veracity, of course. However, physical diseases are never lumped under a label of “physical illness” the way that we lump diseases of the mind under a label of “mental illness.” I remain puzzled by the labeling of some diseases that affect the mind as “mental illnesses” while others are labelled as diseases of the brain. (Feussner JR. When diseases of the brain become diseases of the mind: A new frontier for clinical research. US Medicine 39:9, November 2000.) For example, Alzheimer’s disease has devastating effects on personality, cognition, and memory. Is Alzheimer’s disease a disease of the brain or a “mental illness”? A brain tumor, or a temporal lobe seizure disorder, can affect one’s cognition or personality—are these diseases of the brain or “mental illnesses”? Alternatively, depression is likely to be labeled as a “mental illness” despite studies demonstrating a neuro-chemical basis for depression. Are schizophrenia or bipolar disorder diseases of the brain or “mental illnesses”? Does our profession inadvertently contribute to the lack of equitable reimbursement for “mental” as opposed to “physical” illnesses in part because of our labelling practices? Perhaps all those treating these patients can change our nomenclature in the future–just stop labelling patients as “mentally ill” and stop using the catch-all phrase “mental illness” altogether. Stay tuned!
Also see: www.congress.gov/bill/114th-congress/senate-bill/2680

Uncategorized

Update on the Patient Protection and Affordable Care Act (PPACA): Good and bad

SSCI HeLP Line
Update on the Patient Protection and Affordable Care Act (PPACA): Good and bad.

What was the base state before passage of PPACA in 2010?
Before considering the current state of health care after passage of PPACA, it is important to define the “base state”. Fortunately, baseline data are available from the 2010 national census concerning the health insurance status of Americans. According to the 2010 census, 16.3% of Americans had no health insurance, nearly 50 (49.9) million Americans! In 2010, Americans receiving health insurance from government increased slightly from prior years to 31%, 95 million Americans. That same year, Americans covered by employee-based health insurance decreased to 55%, 169 million Americans. These data represent the “base state” from the same year PPACA passed but before implementation. Physicians and scientists know how important it is to have such robust “before” data. As with any new law, there are “good” features that are very popular, and “bad” features that are not so popular.
THE GOOD. Although the naysayers will not admit it, there are very good provisions within PPACA. What are some of them? For a start, the PPACA broadens health insurance coverage for adults with pre-existing conditions and eliminates pre-existing conditions for children. Resolving this “pre-existing conditions” issue is long overdue! In addition, PPACA allows dependent children to remain on parents plan until age 26, an almost universally popular provision. The PPACA also ends rescissions, that is, insurance coverage cannot be cancelled because of a new illness. Now, there is also no lifetime dollar limit on coverage. Finally, the PPACA begins to address closure of the so-called Medicare drug donut hole. All of these changes are very positive improvements for many Americans. Importantly, there are now many more Americans with health insurance than before passage of PPACA—nearly 12 million more! These are some of the most positive changes resulting from implementation of PPACA.
THE BAD. Of course, no law is perfect. If you compare the 2016 census data with the numbers of newly insured Americans, there are at least 35 million Americans still without health insurance. Furthermore, in 2016, the penalty for not having health insurance increases significantly–to the higher of either $625/adult or 2.5% of household incomes. This is a potent penalty for those not complying with the PPACA provisions. According to CMS actuaries, health expenditures increased in 2014. Notably, the costs of hospital care, physicians’ services, prescription drugs and especially insurance premiums have all gone up. BUT the most striking increase is in the cost of government administration for health care—up 5-fold. More recently, from 2015 to 2016 insurance premiums on the ACA exchanges rose on average 9%. In 29 States, the average premium increases were in double digits and in one third of States the average premiums increased 20% or more. In addition to premium increases, there are increases in insurance deductibles. Silver plans, the most popular in the health care exchanges, now have an average deductible of nearly $3000 in 2016. Then there is the performance of the so-called “Obama care exchanges”. Fully 12 of the 23 cooperatives failed and are defunct. Of the remaining 11 cooperatives, 8 are under enhanced federal oversight or are on a corrective action plan. None of this is good news for Americans who counted on the cooperatives as a vehicle to sign up for health insurance. What does all this mean for patients wishing to use their health insurance? Can these patients really afford routine primary care or access to care for management of chronic disease? There are issues also of access to health care. Within PPACA, there was no plan to address the physician workforce, especially the ongoing shortage of primary care providers. For practicing physicians worried about medical malpractice, the PPACA failed to address tort reform in any meaningful way.
So there you have it—the mixed bag that is PPACA, both the good and the bad! Is it possible to keep the good while diminishing the bad? We shall see what the congress and new administration might propose after the elections in November. Stay tuned!

Uncategorized

HR 1314, 114th Congressional omnibus spending bill: Something for everyone!

The new year is off to a most positive start with passage of a $1.1 trillion omnibus spending bill by the 114th Congress, and recently signed by the President. This new spending bill increases funding for biomedical research substantially.  Funding for the National Institutes of Health (NIH) will increase by about $2 billion, and the new NIH appropriation will total about $32 billion.  This exceptionally good news is not to be taken lightly.  In 2009, at the beginning of the current administration, the NIH budget was $29.5 billion.  From 2009 to 2015, the NIH budget stagnated, and actually lost ground in terms of inflation-adjusted dollars.  In 2015, the NIH budget was a modest (only by Washington, DC standards) $30.1 billion.  One only need to compare that budget with the 2009 budget to deduce that neither the previous Congresses, nor the current administration, was able to navigate a divisive political atmosphere to continue supporting the country’s critical investment in biomedical research. Fortunately, the 114th Congress gets it!  The current increase is highly positive and ends an unfortunate sustained lack of federal investment in research.  However positive, the current increase is just a beginning, and the Congress and the next administration must embrace the pragmatic need to keep the country’s investments in medical research ongoing and increasing in a predictable manner.  If you recall, HR-6, the 21st Century Cures Act suggested just that.  The 21st Century Cures Act recommended a multi-year, incremental investment in biomedical research funding to put the NIH on more stable footing, with the ability to fund research knowing what the out year budgets might be.  While the 21st Century Cures Act was passed overwhelmingly in the House, it continues to languish in the Senate.

And what about everyone else?

In addition to the increase in the NIH budget to about $32 billion (a 6.6% increase, largest increase in 12 years) other entities supporting medical research also received increased budget appropriations. The NSF will see a 1.6% increase ($119 million) to a new budget of $7.46 billion.  The research appropriation for the VA’s Office of Research and Development will see a 7% increase in its research budget to $631 million, its largest budget ever.  Only the AHRQ will experience a budget decrease, of 8% from $364 million to $334 million, (this could be viewed as progress as HR-6, the 21st Century Cures Act, proposed to terminate all funding for the AHRQ).  Let’s hope that congressional support for further investment in medically focused research continues for the next period of time, providing the academic enterprise, and its research faculty, some stability and the assurance that this most critical academic mission in not again shortchanged or caught up in partisan bickering.  The only remaining concern is that despite being passed by Congress and signed by the President, report language—what the Congress expects the funded departments or agencies to do with the increased revenues from the new budget agreement–is still not available for this Omnibus spending bill.

For additional information, see also news.sciencemag.org 12/18/2015

Coming next: Update on the Patient Protection and Affordable Care Act (PPACA):  Good and bad.

Legislation

HR 6, the 21st Century Cures Act, Title 2 “Development”

HR 6, the 21st Century Cures Act, Title 2 “Development”

 

What is the Congress proposing in this new act?

 

In the previous SSCI HeLP Line, I discussed Title 1, “Discovery”, of the 21st Century Cures Act.   This part of the Act invests in research by providing additional funding for the NIH.  However, the Congress perceives that application of scientific research discoveries is not efficient enough to meet patient needs.  Title 2 of the 21st Century Cures Act addresses that perception, requiring that the FDA streamline its processes to speed the pace of FDA reviews of new drugs and medical devices.

Currently, the FDA approves most new medications within 6-10 months, perhaps faster than other comparable regulatory bodies. The FDA approval process relies on data derived from pivotal clinical trials—the highest level of scientific evidence that demonstrates efficacy.  Many believe that the FDA process is already efficient, given the complexity of the research and the high quality of scientific evidence required to establish benefit for innovative therapies.  By requiring changes to the FDA’s current processes, an unintended consequence of the new 21 Century Cures Act may be an accentuation of the conflict between a desire for a speedier FDA review process and the need to demonstrate efficacy and safety with scientifically rigorous studies.

In addition to process improvements, the 21st Century Cures Act directs the FDA to develop procedures for use of data from other study designs and methods of data analysis. Specifically, the Act discusses clinical trial methodologies like adaptive trial designs and use of Bayesian analytical methods.  These are robust designs and analytical methods, but other suggestions are not.  For example, the 21 Century Cures Act directs the Secretary to develop programs to evaluate the use of “clinical experience” in the drug and device review process.  Clinical investigators know that clinical experience alone rarely contributes key scientific evidence concerning efficacy. In addition, the Act suggests use of smaller or shorter-duration clinical trials, observational studies like case-control studies, and registries.  All of these methods are much less rigorous than randomized clinical trials to establish efficacy and safety.

The bill also encourages the FDA to rely more on biomarkers and intermediate, surrogate measures rather than actual clinical end-points when assessing efficacy of both drugs and devises. However, improvement in intermediate variables does not always translate to improved patient outcomes.  For example, a treatment may show positive preliminary results by reducing tumor size but does not lead to improved patient survival.  In this case, improvement in a surrogate variable does not establish efficacy because it does not improve patient outcomes.

Under the Subtitle, “Streamline Clinical Trials”, the Act enumerates alterations of waivers for informed consent to patient volunteers in clinical trials. They offer a new exception when clinical testing poses “no more than minimal risk”.  This exception concerns some physicians and scientists given the vagueness in defining “minimal risk”.  Perhaps this exception is offered to facilitate comparative effectiveness trials where the competing treatments are already FDA approved drugs and devices with known risks and adverse effects.

Title 2 “Development” specifies 15 subtitles and other topics that deal extensively with review processes and decision-making at the FDA. For additional information about these other issues go to http://energycommerce.house.gov/cures

Also see J Avorn, AS Kesselheim. The 21st Century Cures Act:  Will it take us back in time?  New Eng J Med 2015; 372: 2473-2475.

COMING NEXT: 2016 Congressional Omnibus spending bill:  Something for everyone.

Biomedical Research, Legislation

21st Century Cures Act

HR 6, the 21st Century Cures Act:  Renewed optimism for patients and researchers.

What is this 21st Century Cures Act?

This new bill in the House of Representatives is in the early stages of the legislative process. However, it has gone through more than a yearlong developmental process.  The latest version of the bill has been reduced to 199 pages.  Importantly, the Energy and Commerce Committee approved the bill unanimously on May 21 by a vote of 51-0, with obvious and enthusiastic bipartisan support!  When the bill came up for consideration before the entire House, the vote remained overwhelmingly bipartisan—members who voted in favor 344, against 77, abstain 12.

What is the Congress proposing in this new bill?

The current iteration of the bill has three main components: Discovery, Development, and Delivery.

Title 1 focuses on “Discovery”.

The 21st Century Cures Act has several goals that should energize academic physicians and scientists.  The bill proposes to re-invest in discovery research by increasing the NIH budget—currently $30.3 billion.  The committee proposes to increase the NIH budget to $31.8 billion in 2016; $33.3 billion in 2017; and $34.8 billion in 2018.  Importantly, the proposed NIH budget grows predictably over the next 3 years:  total increase is $4.5 billion (nearly 15%).

The 21st Century Cures Act also creates a dedicated innovation fund of an additional $2 billion/year from 2016 through 2020. This represents another $10 billion “to support biomedical research through funding of basic, translational, and clinical research” at the NIH.  This component of the NIH budget allows congressional appropriators to invest additional resources in scientific research without impacting current budget caps (per sequestration).

The 21st Century Cures Act is prescriptive in that it directs the NIH to invest in specified priority areas.  The bill specifies support for innovative research in such areas as biomarkers, precision medicine, infectious diseases, and antibiotics, for example.  In other priority areas, the 21st Century Cures Act directs the NIH to reserve funding specifically for “young emerging scientists”.  This subtitle in the Act includes also specific language regarding improved loan repayment programs.  Furthermore, the bill directs the NIH to create “capstone awards” to support the work of particularly outstanding scientists previously funded by the NIH.

Another priority area in the 21st Century Cures Act includes pediatrics research and directs the NIH to establish a “national pediatrics research network”. The network would be composed of research institutions operating as a consortium in order to pool resources and coordinated activities related to research on rare diseases or birth defects.  The bill also declares a “sense of Congress” that directs the NIH and FDA to work together and with the European Union, industry, and others to establish a “global pediatrics clinical trials network”.

The 21st Century Cures Act identifies other priority areas.  The bill specifies the need for the NIH to engage “in scientifically based strategic planning implemented in support of research priorities”.  This is a recurring requirement to be repeated at 5-year intervals.  In addition, the bill directs the NIH to improve access to clinical trials data by creating and then releasing de-identified clinical trials data sets, from qualified clinical trials, for use by other medical investigators.

These budget issues and priority areas represent some of the key features included in the current version of the 21st Century Cures Act.  Now that the House has acted, what will the Senate choose to do?

For additional information visit: http://energycommerce.house.gov/cures

COMING next: The 21st Century Cures Act, Title 2 “Development”

Legislation, Medicare, Sustainable Growth Rate

The Timely Demise of the Medicare Sustainable Growth Rate (SGR)

What was the Medicare Sustainable Growth Rate Formula? (the “Doc Fix”)

The SGR formula was the method used by CMS (Center for Medicare and Medicaid Services) to control the rate of Medicare spending for physician services. The SGR was part of the Balanced Budget Act enacted by Congress in 1997. This flawed formula was not influenced by the volume of services provided, by the quality of patient care, nor the efficiency of health care delivery. Every year since 2001, the formula led to the threat of cuts in Medicare fee schedule payments to physicians. Congress voted annually to defer action on the SGR-mandated cuts, the recurring “Doc-Fix” crisis. However, by 2015, the cumulative SGR-mandated reductions in physician fees paid by Medicare reached an estimated 21%. (Were you ever prepared to take a 21% reduction in Medicare reimbursement for your services?)

Why did Congress not repeal the SGR and fix the problem?

In policy and politics, money matters, and BIG money really matters! In past years, the Congressional Budget Office (CBO) estimated the cost to repeal the SGR formula at about $320 billion! That is, if Congress failed to implement the SGR-mandated cuts, Medicare costs would supposedly increase by about $320 billion. However, this year the CBO estimated that repeal of the SGR would only result in increased Medicare costs of about $130 billion. Technically, if Congress voted to repeal the SGR, it needed to identify the source for revenues to offset eliminating the SGR-mandated cuts. This year, however, Congress finally repealed the SGR – hooray! While Congress did repeal the SGR, it did not identify other revenues to cover the increased Medicare spending (known as deficit spending). The level of bipartisan cooperation was remarkable given the level of acrimony often on display in Congress. In the House of Representatives, the vote to repeal the SGR was 392 to 37, and in the Senate, it was 92 to 8.

How will this new legislation work?

In April 2015, the Congress passed comprehensive legislation to repeal the SGR formula and adopt a new value-based reimbursement system focused on patients and the quality of care – this is the Medicare Access and CHIP (Children’s Health Insurance Program) Reauthorization ACT (H.R. 2). The President promptly signed this new law. Therefore, no physician will face the threatened 21% reduction in Medicare fees scheduled for this year! With the permanent elimination of the SGR formula, physicians will experience stable payments during a transition period to the new payment system. The legislation specifies annual physician fee increases of 0.5%/year through 2019. After 2019, physicians will be incentivized (bonuses or penalties) according to their performance, as assessed by the federal government. A new “merit-based” incentive payment system (MIPS) will be constructed to reward (or penalize) performance in four areas: quality of care, clinical practice improvement efforts, resource use, and performance on meaningful use of the electronic medical record. Exactly how this incentive payment system will work is not yet determined. The new law includes several other important elements. For example, Congress extended the CHIP program with no cuts for another 2 years. The legislation extended funding also to support community health centers for the next 2 years ($7 billion).

For additional information, see HJ Aaron. Three Cheers for Logrolling – The Demise of the SGR. N Eng J Med 2015; 372: 1977-79.

Coming Next: Optimism for increased science funding – the 21st Century Cures Bill

ruler

The SSCI Thanks Its Sponsors

  • Lippincott Williams & Wilkins sponsors Southern Society for Clinical Investigation