Jeremy Schafer, PharmD, MBA

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Years ago, I was lecturing on the topic of formulary development to a class of pharmacy students at my alma mater, the 

University of Minnesota College of Pharmacy

. One of them asked why the United States did not have a socialized healthcare system like many countries in Europe. I quickly replied, “because Americans aren’t ready for it.”

Noticing the student’s perplexed reaction, I went on to elaborate with case examples of, at the time, recently released tyrosine kinase inhibitors for the treatment of renal cell carcinoma.

At that time, those therapies had been approved and were in use in the U.S. but not available in the U.K. because the 

National Institute for Health and Care Excellence (NICE)

 had determined them not to be cost effective-despite data showing improved overall survival versus standard of care.

“Can you imagine,” I said to the student, “the outcry if the government denied a life extending drug to a patient because they deemed it not being ‘worth it?’”

As the debate on Medicare for All heats up, it may be time to revisit the question: Would Americans be ready to wait?

Buried among the large dollar estimates and Herculean operational undertaking details in the Congressional Budget Office’s report on Medicare for All was the following point: “potentially longer waits for some treatments and technologies.”

The length of these delays, if any, can be debated, but there are real case examples with socialized medicine systems today. Probably the most publicized as of late has been the back and forth between the UK and Vertex Pharmaceuticals around the cystic fibrosis drug, Orkambi. Orkambi was approved in Europe in 2015 but still isn’t available in the U.K. because the two sides cannot agree on a price.

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Meanwhile, thousands of eligible patients in the U.K. wait, disease worsening, as the debate rages. On the U.S. side, Orkambi was approved in 2015 and covered by most payers shortly thereafter, albeit at higher cost than what will likely be paid in the U.K.

The example of Vertex and the U.K. may be one of the more public examples, but delays in access are not uncommon-especially considering the higher price tags of emerging therapies. Nivolumab, a member of the rapidly growing checkpoint inhibitor class of cancer drugs, received initial rejections by NICE for non-small cell lung cancer (NSCLC), adjuvant melanoma, recurrent melanoma, and advanced bladder cancer. Many of these decisions were later reversed after additional data and discounts were provided but it is a significant difference compared to the U.S. where therapies and new indications are covered shortly after approval and in some cases, even before.

Yescarta, one of the new CAR T agents met a similar fate in the U.K., but was granted access later in specific populations. While these cases all vary, there is one core attribute: waiting for innovation. And in these cases, the waiting is not in relatively benign conditions but rather in serious, life-threatening conditions where waiting may have fatal results.

There are certainly counter arguments-and the U.S. system is not perfect by any means. Advocates for Medicare for All argue that patients with no insurance or poor insurance do not have access at all. Rising coinsurance and deductibles have also been associated with spikes in nonadherence and prescription abandonment in the U.S. In addition, a lower price on drugs may allow more patients to be treated when budgets are limited.  But the discussion here is whether those who currently have insurance and access-a population significantly expanded under the ACA-will be willing to wait in order to potentially lower out of pocket cost and access barriers for everyone.

Will the suburban mother be willing to wait potentially months for her child with cystic fibrosis to get a new medication? Will (or can) an elderly person with metastatic bladder cancer be willing to wait for the next innovative therapy?  Will an individual be willing to wait for a new drug if in doing so, their friends or family get access to care that they didn’t have before?  These questions are particularly relevant for individuals with private insurance that may be giving up more rapid access, albeit at a higher cost, to expand access to others.

, the lobbyist organization for pharmaceutical manufacturers, has stated that concepts like price controls and price regulation could impair innovation. Medicare for All would include a provision of the government negotiating prices with manufacturers, similar to what is seen in other nations. Pharmaceutical manufacturers would have to come to the table due to the size of the U.S. market, but would they take the same risks in development?

The last few years have seen the introduction of immunotherapy in cancer, CAR T agents, and the burgeoning pipeline of gene therapies. One could reasonably question if manufacturers would pursue risky endeavors if the largest and most profitable market in the world suddenly became far less profitable. The result would be eternal waiting when research programs were discontinued or never started at all.

If Medicare for All is to have a real evaluation, the details of how such a bill would impact a person’s access to care and innovation  need to be provided so consumers can make informed decisions. Healthcare bills that are all flash but no substance and written for the purpose of campaigning rather than policy making are not helpful. Americans need to understand how a change like Medicare for All would affect their healthcare and the healthcare of their families. Would patients have access to the same drugs they do now? Would access be delayed? Would drugs not be developed at all?

Jeremy Schafer, PharmD, MBA, is SVP, director Precision for Value.

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Will Americans Accept Longer Wait Time in Medicare For All?

The 21st Century Cures Act ushered in a number of notable changes for stakeholders in the healthcare landscape. In particular, one new inclusion was the expanded utility of real-world evidence (RWE). The 21st Century Cures Act even went so far as to allow RWE to be 

used in gaining new indications for marketed pharmaceuticals

The question many may ask is whether RWE is up for such a task. RWE has always been challenging, with its quantity and quality varying considerably by source, timeliness, and the ability of skilled personnel to pull and analyze the data.

Perhaps RWE’s greatest shortcoming, however, has been how easy it is to manipulate to obtain a specific result. Because of this, opposing parties often view RWE generated by the other side with distrust. To overcome this barrier, RWE studies should detail, as accurately and transparently as possible, the data’s origin, how the study was conducted, and any limitations. This is doubly true if the data is to be used in expanding the use of a pharmaceutical, as patients would then be taking the medication for a condition the FDA has deemed appropriate based on RWE.

Such an approval based on RWE did not take long. On April 4, the FDA granted approval for IBRANCE (palbociclib, Pfizer) in combination with an aromatase inhibitor in men with hormone receptor–positive (HR+), human epidermal growth factor receptor 2–negative (HER2-) advanced or metastatic breast cancer.

 noted that the approval was based on “data from electronic health records and postmarketing reports of the real-world use of palbociclib in male patients sourced from three databases: IQVIA Insurance database, Flatiron Health Breast Cancer database, and the Pfizer global safety database.”

This approval was an exciting moment for RWE and enabled the use of a medication in a high-need, underserved population. Payers and other healthcare stakeholders were undoubtedly intrigued by the approval and interested in seeing what data was used to support the approval. RWE, after all, requires an extra level of scrutiny.

Unfortunately, documenting the data supporting the label is where the FDA came up short. Scouring the FDA label for palbociclib, a reviewer will find only a few mentions of breast cancer in men. One is in the indication, of course. The 

 is of little guidance and contains only the somewhat generic statement: “Based on limited data from postmarketing reports and electronic health records, the safety profile for men treated with IBRANCE is consistent with the safety profile in women treated with IBRANCE.” There isn’t even a mention of how many men were included in the safety assessment.

To put the safety data into perspective, the information provided by men is one sentence compared to multiple paragraphs on animal data. A reader of the label has a better understanding of the safety of the drug in rats and rabbits than in men. The efficacy section is even worse, and devoid of any mention of men. For a reviewer seeking to understand how palbociclib works in men or how RWE supports new approvals, this is not a strong start by the FDA.

The reason the disclosure of data is important is because the information is instrumental to how formulary decision makers review medications and provide coverage. A clinical review by a Pharmacy and Therapeutics (P&T) committee is reliant on quality data being available that a committee can digest to help them determine which patients are appropriate and how best to make cost-effective choices. The lack of information in the label means that formulary committees are likely to ask: “What is ‘similar safety?’” “How do we know the drug works?” “How many men were evaluated?” Incomplete or missing information can make formulary decision makers impose more aggressive management than what would normally be expected in an effort to moderate use of the medication until more data is available.

Thankfully, the manufacturer has stated that more data in men treated with palbociclib is forthcoming and will be presented at a future meeting. However, we cannot count on a manufacturer to do that in every case.

The use of RWE for expanding the FDA label of medications is an important and needed step. RWE can help more patients obtain access to a drug that may help them, and obtain such access sooner. However, simply because it is RWE does not change the fact that formulary decision makers need complete access to the data so that they may evaluate it just as rigorously as they would clinical trial data.

The FDA and the pharmaceutical manufacturers pursuing expanded uses via RWE should consider how they can best share the data, preferably before or at approval. Doing so will help formulary decision makers conduct a more thorough review and may ease access for populations already challenged by limited options.

Health plans review and consider evidence from multiple sources in order to make formulary and coverage policy decisions. The plans owe this duty to their customers: employer groups who purchase insurance as well as the insured patients covered by those plans. In order to assure that coverage policy decisions are created, maintained, and reviewed in an evidence-based context, the RWE used by the FDA to grant new indications must be made available publicly at the time (or preferentially before) the time the new indication is approved.

Without that evidence, it is impossible for decisions to be made based on evidence; in this case we thus far have to take the FDA’s word that the evidence was strong enough to approve the indication.

. Dan brings to Precision over 15 years of experience developing and leading pharmacy programs in the health plan and PBM industry. 

Jeremy Schafer, PharmD, MBA, is senior vice president, director, Access Experience Team at Precision for Value. Jeremy provides enterprise leadership across Precision’s Access Experience Team and leads integration with other enterprise teams including health economics outcomes research, provider marketing, access and analytics, and media outreach.

Is real-world evidence up to the task of gaining new indications?

Show Me The Data: Why Payers Need to See RWE Used in New Indication Approvals

	The call for antimicrobial stewardship is growing in the healthcare landscape. 

 of pan–drug-resistant infections have occurred and, without action, society risks returning to the pre-antibiotic era. An underlying cause of resistant bacteria is the overuse of antibiotics. The Centers for Disease Control and Prevention (CDC) 

 that 30% of antibiotic prescriptions are unnecessary. The federal government’s National Action Plan for Combating Antibiotic-Resistant Bacteria was released to provide guidance on reducing antibiotic use in unnecessary situations and help stem resistance development. The report’s goals include slowing the emergence and spread of resistant infections, strengthening resistance-prevention efforts, and accelerating the development of new 

	Health plans and PBMs face complications in how to contribute to antimicrobial stewardship. Patients receiving antimicrobials often receive a short course of treatment, require a specific product based on the infectious organism and site, and may be transitioning home to finish therapy from a healthcare facility where therapy was started. These factors make traditional management techniques difficult. In addition, antimicrobials tend to be generic and inexpensive, which reduces a payer’s focus and resources in managing antimicrobials. 

	Challenges aside, payers nonetheless have capabilities to assist in antimicrobial stewardship. The following four steps, when enacted by payers, may help facilitate appropriate antimicrobial use and reduce the 

rise of resistant bacteria and other microbes, such as drug-resistant fungi

. Health plans may wish to include any or all of these steps in their quality improvement programs and work plans.

	In November 2016, augmenting previously issued guidance for antibiotic stewardship in hospitals and nursing homes, the CDC released the 

Core Elements of Outpatient Antibiotic Stewardship

. The outpatient core elements include commitment, taking action for policy and practice, tracking and reporting, and education and expertise. The guidance provides recommendations on optimizing antimicrobial use, setting policies for effective antimicrobial prescribing, tracking success, and providing education to clinicians and staff. A payer can engage by rewarding network health systems that have a documented antimicrobial stewardship program and demonstrating improvement in outcomes. Payers can monitor participating systems’ bacteria resistance rates and overall antimicrobial usage as performance end points. Rewards could include improved reimbursement rates or preferred network status.   

	The National Committee for Quality Assurance (NCQA) includes quality measures related to appropriate antimicrobial use within its Healthcare Effectiveness Data and Information Set (HEDIS

	·      Avoidance of Antibiotic Treatment in Adults With Acute Bronchitis

	·      Appropriate Testing for Children With Pharyngitis

	·      Appropriate Treatment for Children With Upper Respiratory Infection

	Performance on each measure generally improves with appropriate antibiotic use. Evaluating performance on the Plan All-Cause Readmissions quality measure may be beneficial as well, considering that infection is a frequent cause of rehospitalization. Payers should assess their quality scores on these measures and identify practices, hospitals, or prescribers that may be contributing to suboptimal performance. The payer could notify providers of poor performance, reinforce the importance of antimicrobial stewardship, and provide guidance on how to improve. Repeat poor performers could be subject to penalties, including reduced reimbursement, exclusion from preferred high performance networks, or network decredentialing. Payers may encourage adoption or advancement of antimicrobial stewardship initiatives further with incentives to health systems and hospitals.

	NCQA included in HEDIS 2017 a new measure, the Standardized Healthcare-Associated Infection Ratio. This measure is used to evaluate a potential infection risk for members admitted to hospitals within the plan’s networks.   The four healthcare-associated infections addressed by the measure are (ratios publicly reported by CMS):

	·      Central line-associated bloodstream infections (CLABSI)

	·      Catheter-associated urinary tract infections (CAUTI)

	The measure stratifies facilities’ standardized infection ratios (SIRs) by risks of exposure (high, moderate, low or unavailable) and estimates a member’s potential infection risk based on the percentage of discharges that fall within each risk classification. The measure also reports a plan-weighted SIR that facilitates comparisons among plans for infection exposure risk.

	Payers can monitor performance on the new measure as data become available and  identify facilities contributing to sub-optimal performance. Payers may request action plans for performance improvement from these facilities, many of which are subject, as of January 1, 2017, to The Joint Commission’s new 

. Facility hospitals performing well on the measure or demonstrating improvement could be rewarded as noted previously. Performance data on healthcare-acquired infections among Medicare-certified hospitals is presently available through Hospital Compare (according to CMS) and through other quality metric and performance aggregator tools such as Qnav. Plans may wish to review the performance of high-volume hospitals within their network and proactively develop strategic collaborations on infection reduction and antimicrobial stewardship initiatives.  Collaborations could potentially include health systems and other stakeholders, such as employer coalitions, patient safety advocacy groups, and pharmaceutical companies.

	Payers often utilize prior authorization programs when the goal is controlling inappropriate use. However, prior authorization for antibiotics can be problematic.  Antimicrobials tend to be inexpensive, making administration of the program costly. Additionally, prior authorization programs or other point-of-sale barriers require patients to wait for approval and can potentially cause a delay in therapy. A 

 of recently hospitalized Medicare patients found that linezolid claim reversals led to more rehospitalizations (23% reversal group vs 9% filled group; 

 found that recently hospitalized Medicare patients who did not fill a linezolid prescription had higher medical costs than patients who did (

 that focused on a linezolid prior authorization program in commercial patients did not find adverse consequences, but noted that 15% of patients exposed to the prior authorization program had no record of antibiotic use in follow-up.

	As alternatives to prior authorization programs, payers can implement programs that allow patients to get on therapy quickly while also reducing the potential for inappropriate use. Quantity limits can facilitate appropriate regimens while limiting daily doses and excessive durations of therapy. Payer pathway programs could be expanded to antimicrobials, allowing patients recently discharged from the hospital or with lab results indicating appropriate therapy to be approved instantly. Payers can use retrospective drug utilization reviews to identify prescribers who write excessive amounts of antimicrobial prescriptions or prescribe for inappropriate indications. Payers should communicate the issues with these prescribers and outline ways to improve performance. Finally, payers may introduce an antimicrobial stewardship program through existing pharmacy and population health management programs. Many chronic conditions are managed with the provision of high-touch interventions (e.g., medication adherence, immunizations, depression screenings, referrals, transition of care). Appropriate patient antimicrobial utilization and management could be introduced into the care management process.

	Providers frequently cite demanding patients as a reason that antibiotics are prescribed inappropriately. Prescribers refusing a parent’s request for antibiotics for a sick child may be faced with the loss of a patient or with negative reviews on prescriber rating sites. Patients and parents may not appreciate how the inappropriate use of an antibiotic in a given situation contributes to an overall growth in bacterial resistance. 

	Payers have access to broad patient populations and the opportunity to educate. A payer could implement a public education campaign communicating to members the threat of antimicrobial resistance. Social media may also be considered for payers with the capability. Language should be simple but impactful and could inform on recent cases of “super bugs.” Instructions on nonantibiotic methods to increase a child’s comfort during a viral illness may reduce a parent’s feeling of helplessness.

	Combating rising antimicrobial resistance is a responsibility of every stakeholder in the healthcare landscape. Payers have an opportunity to impact antimicrobial use on a population level. Payer action requires monitoring of the data to identify quality measure performance and inappropriate antimicrobial use trends. Additionally, payers can assist in holding other stakeholders accountable by identifying and communicating to underperforming entities, as well as by rewarding those furthering the cause of antimicrobial stewardship. By enacting the steps detailed here, payers can practice antimicrobial stewardship, but do so in a way that limits negative impact on patients.

Jeremy Schafer, PharmD, MBA, is senior vice president, Precision for Value and Maureen Hennessey, PhD, CPCC, is senior vice president,

The call for antimicrobial stewardship is growing. Enact these steps to prevent a return to the pre-antibiotic era.

Author | Jeremy Schafer, PharmD, MBA

Articles

Will Americans Accept Longer Wait Time in Medicare For All?

Show Me The Data: Why Payers Need to See RWE Used in New Indication Approvals

Four steps managed care can take to promote antimicrobial stewardship