Category: Uncategorized

Scrutiny on pharmacy benefit managers (PBMs) has just been ratcheted up considerably. The Federal Trade Commission (FTC) announced on June 7 the launch of its investigation into PBM practices, targeting the largest players. This investigation comes at an extremely crucial time for the biosimilar industry, as manufacturers prepare to negotiate with these same PBMs for coverage of adalimumab biosimilars.

The PBMs are the chief purchasers of medications for commercial plans and Medicare part D members. They negotiate drug pricing contracts with drug manufacturers for the vast majority of the insured population in the US. They are another unique feature of global health systems: No other advanced country allows private middlemen to serve such a role.

A 90-Day Deadline

The FTC will send “compulsory orders” to CVS Caremark; Express Scripts, Inc.; OptumRx, Inc.; Humana Inc.; Prime Therapeutics LLC; and MedImpact Healthcare Systems, Inc., who purchase medications and make pharmacy coverage decisions for approximately 90% of the commercial and Medicare part D insured population.

According to the FTC, these PBMs will be compelled to “provide information and records regarding their business practices,” which are notoriously opaque. The investigation will focus on how these PBMs affect access and affordability of prescription drugs. The FTC’s deadline for compliance will be 90 days from issuance of the orders.

“Although many people have never heard of pharmacy benefit managers, these powerful middlemen have enormous influence over the U.S. prescription drug system,” stated Federal Trade Commission Chair Lina M. Khan in the FTC’s release. “This study will shine a light on these companies’ practices and their impact on pharmacies, payers, doctors, and patients.”

How Do Net Prices and Rebate Contracts Buy Coverage?

At the center of the FTC investigation of PBMs is the PBMs’ encouragement of rebates to negotiate low net prices, and how this results in barriers to coverage of other drugs. For example, a PBM may allow a drug manufacturer exclusive formulary coverage for their drug if the rebates offered are sufficiently attractive.

This is potentially a huge obstacle for makers of adalimumab biosimilars. AbbVie passes millions of dollars in rebates to PBMs at present in exchange for Humira^® coverage. Biosimilar manufacturers who seek PBM coverage must contend with the PBM’s lost or reduced Humira rebates if their biosimilar is to be covered exclusively or at parity with the reference product. Further, how are these rebates passed along to payers and ultimately to patients?

The Influence of PBMs

“In these roles,” says the FTC, “[PBMs] often have enormous influence on which drugs are prescribed to patients, which pharmacies patients can use, and how much patients ultimately pay at the pharmacy counter. Many of these functions depend on highly complicated, opaque contractual relationships that are difficult or impossible to understand for patients and independent businesses across the prescription drug system.”

In addition to the impact of rebates, the FTC will also review practices that involve the PBM’s relationship with the pharmacy network, including reimbursement; their use of prior authorization and other administrative tools to manage access, and the PBM’s specialty drug policies.

This inquiry is the next step in the FTC’s request for information about PBMs issued in February (and elicited 24,000 public comments). It is not known when the FTC’s investigation will conclude or whether it will immediately impact the negotiations for the multitude of adalimumab biosimilars to be launched in 2023. However, this inquiry may begin to pry open the lockbox on PBM practices. There will certainly be more to report on these developments.

It is difficult to speculate on what FDA’s motivation might be to overrule the recommendation of its Advisory Committee and approve Biogen’s aducanumab for the treatment of mild Alzheimer’s disease. Relatively few providers are convinced of the value of this agent based on the clinical trial results reviewed, and many are outspoken in their opposition to it. Based on the providers’ perspective, what are payers supposed to think?

Research in the Alzheimer disease category has resulted in depressingly few bright spots. Biogen’s agent does not seem to break any new ground here—a drug that helps clear amyloid plaque from the brain seems to achieve that goal, but without showing meaningful differences in cognitive improvement (or delayed progression). The clinical trials proved this. FDA seems to be holding out hope that this mechanism of action will be proven beneficial at some point in the future. Certainly, they do not have this evidence to date. The fact that the approval will require a phase 4 clinical trial to support this hope is frankly uninspiring. Newly diagnosed patients will clamor for the drug, despite its poor record of efficacy and its significant risks, placing providers and payers in a difficult bind.

Alzheimer’s disease is a disorder heavily weighted toward the elderly population, and Medicare will be expected to bear the brunt of the bill for aducanumab. Early estimates have gone as high as $10 billion annually based on the prevalence and incidence of Alzheimer’s disease. This points to the immediate need for a national coverage determination (NCD) to help guide Medicare coverage. Health policy experts are calling for this to begin now. However, commercial payers will also need to make coverage policies pertaining to the agent.

In other words, payers will have their hands full trying to measure value against the projected $56,000 yearly cost of treatment. In an earlier analysis, ICER revealed that aducanumab may not be cost effective at one-tenth this price. Reacting to the approval, ICER sent out its own commentary, taking the FDA to task.

Payers, including Medicare Advantage plans, can utilize prior authorization criteria and step edits to manage appropriate access to this treatment. In this case, “appropriate access” may be very difficult to define, outside of an investigational clinical trial. Ordinarily, when products are approved by the Food and Drug Administration, there is an expectation of careful evaluation and decision making based on the efficacy and safety of the medication. In an accelerated approval scenario such as this, this expectation is not met. Judging from the cacophony of confusion coming from neurologists on this decision, I don’t believe that we have a clear understanding of whom this agent may benefit. Maybe it is just wishful thinking from the FDA that clearing amyloid plaque will somehow equate to better cognitive function (perhaps in a smaller subset of patients). Until we have real evidence that this is the case, a very conservative approach seems warranted. Let Biogen conduct the new study and prove the concept works. Until then, the number needed to treat, and the cost of that treatment, to gain benefit in one patient could indeed be enormous.

Research in the Alzheimer disease category has resulted in depressingly few bright spots. Biogen’s agent does not seem to break any new ground here—a drug that helps clear amyloid plaque from the brain seems to achieve that goal, but without showing meaningful differences in cognitive improvement (or delayed progression). The clinical trials proved this. FDA seems to be holding out hope that this mechanism of action will be proven beneficial at some point in the future. Certainly, they do not have this evidence to date. The fact that the approval will require a phase 4 clinical trial to support this hope is frankly uninspiring. Newly diagnosed patients will clamor for the drug, despite its poor record of efficacy and its significant risks, placing providers and payers in a difficult bind.

Alzheimer’s disease is a disorder heavily weighted toward the elderly population, and Medicare will be expected to bear the brunt of the bill for aducanumab. Early estimates have gone as high as $10 billion annually based on the prevalence and incidence of Alzheimer’s disease. This points to the immediate need for a national coverage determination (NCD) to help guide Medicare coverage. Health policy experts are calling for this to begin now. However, commercial payers will also need to make coverage policies pertaining to the agent.

In other words, payers will have their hands full trying to measure value against the projected $56,000 yearly cost of treatment. In an earlier analysis, ICER revealed that aducanumab may not be cost effective at one-tenth this price. Reacting to the approval, ICER sent out its own commentary, taking the FDA to task.

Payers, including Medicare Advantage plans, can utilize prior authorization criteria and step edits to manage appropriate access to this treatment. In this case, “appropriate access” may be very difficult to define, outside of an investigational clinical trial. Ordinarily, when products are approved by the Food and Drug Administration, there is an expectation of careful evaluation and decision making based on the efficacy and safety of the medication. In an accelerated approval scenario such as this, this expectation is not met. Judging from the cacophony of confusion coming from neurologists on this decision, I don’t believe that we have a clear understanding of whom this agent may benefit. Maybe it is just wishful thinking from the FDA that clearing amyloid plaque will somehow equate to better cognitive function (perhaps in a smaller subset of patients). Until we have real evidence that this is the case, a very conservative approach seems warranted. Let Biogen conduct the new study and prove the concept works. Until then, the number needed to treat, and the cost of that treatment, to gain benefit in one patient could indeed be enormous.

The pharmaceutical industry sometimes forgets how payers calculate the value of their products. Payers do not view value solely through the lenses of clinical efficacy, safety, and utility. The case for the value of a COVID-19 vaccine to prevent disease or an antiviral to prevent COVID-related serious complications and hospitalization can readily be made. What about the following, less-obvious scenario? A pharmaceutical offers some (but not high) level of clinical benefits in a common disease state for which no other pharmacologic therapy has been proven to work?

It is surprising how many disorders do not currently have effective, approved treatments. This is particularly true in the orphan disease areas (especially ultra-orphan diseases), in which fewer than 200,000 people have been afflicted by an illness. Often, genetic mutation or deletion is the principal cause. In other cases, like pancreatic cancer, outcomes with known therapies are considered minimally effective. When speaking about a high-prevalence disorder, the same can be said for nonalcoholic steatohepatitis (NASH). At least two therapies are in late-stage trials, but many questions regarding their clinical and quality-of-life outcomes are yet to be answered.

When developing a new pharmaceutical for orphan diseases or more prevalent disorders that do not yet have effective treatments, the calculation of value is not so well defined. In our market research with payers, we find that the first treatment found to have any clinical effectiveness for a disorder is likely to gain some level of coverage. This does not mean it will be favored as a preferred product. One can assume that prior authorization requirements will be the principal way in which access is controlled, particularly when the drug is priced as a specialty pharmaceutical.  

This is why payers are increasingly looking to organizations like the Institute for Clinical and Economic Review (ICER) to help them set pricing standards in the sometimes ambiguous world of pharmaceutical value. The analysts at ICER take a direct approach to the consideration of pharmacoeconomics. They evaluate whether the price proposed by the drug maker is cost effective based on its predicted clinical improvement and potential medical cost offsets, and, perhaps more importantly, at what price would the drug be cost effective?

The standard pharma company method includes gaining payer feedback in several prelaunch phases. This consists of presenting a condensed profile of “Product X,” which highlights the new drug’s benefits (real or aspirational), risks of treatment, and the intended patient population. After digesting this information, payers (usually a mix of medical directors, pharmacy directors, and clinical pharmacists) are offered a wide choice of potential prices, in an effort to better obtain a narrower range of acceptable prices. Subsequent rounds of feedback are obtained regarding rebates necessary to obtain preferred formulary placement and what prior authorization criteria will be utilized to manage patient access.

Rarely, do pharma companies directly ask the payers what price would make financial sense based on the product’s apparent value. It seems an obvious question, and it does sometimes get asked, but not often enough. This leaves payers (and most other stakeholders) with the impression that the price is an actuarial business decision: let’s pick a price point that maximizes revenue. This doesn’t mean the price is indefensible, but it does imply there is little transparency or logic to the pricing.

There are ample examples where initial pricing was drastically changed once the agent was introduced and caused an outcry that affected managed care plan coverage. Despite promising the first medical cure for patients with hepatitis C, the initial price tag for Gilead’s Solvaldi caused an uproar. Cost increases to cancer products like Revlimid, which may be associated with only incremental benefits, have been debated by oncology centers like Memorial Sloan Kettering Cancer Center as well as by Congress. Manufacturers like Amgen cut the price of their PCSK9 cholesterol-lowering agents by 60%, because of payers’ perception that the initial price was not rooted in value (and their subsequent refusal to provide broad formulary access).

Too many specialty drugs today and in the pipeline tomorrow carry price tags that do not align with their claimed value. Biopharmaceutical companies should expend considerably more time and effort incorporating payers’ assumptions and opinions on the value of new medications before setting price, or at least really gauge their reaction to proposed pricing well before launch. This will result in at least some degree of price acceptance at launch or at least the impression that the company cares about the payer perspective.

In a value-based health system, this will matter a great deal. 

SM Health Communications provides writing, consulting, and innovative market research services for the payer markets. Its proprietary P&T Insight™ virtual P&T Committee program is the leading mock P&T Committee product in the field. We’ve participated in many market research projects involving biosimilar development and launch, from the point of view of the biosimilar and the innovator drug manufacturer. For more information, please visit www.smhealthcom.com or contact Stanton R. Mehr, President, at stan.mehr@smhealthcom.com.

For our archived posts on a wide variety of topics, see http://smhealthcomreports.blogspot.com/.

In our previous post, we documented the difficulties and challenges in considering a “Medicare-for-all” scenario to extend coverage to virtually everyone. We also discussed the potential for implementation of a “public option,” which was first proposed with the initial version of the Affordable Care Act. One of the least considered alternatives to extend universal coverage is Universal Catastrophic Care (UCC).

Any attempt at extending coverage to the fullest, appropriate American population should accomplish three objectives: (1) it should lower the risk of medical bankruptcy (or even eliminate the possibility), (2) vastly simplify the health system from the consumer’s point of view, and (3) lower overall costs (administrative and healthcare costs) from the society’s view. The chart below is a simplified view of how well the three mechanisms discussed in this series of posts attack these objectives.

Objective	Medicare for All	Public Option	Universal Catastrophic Care
Lower Risk of Medical Bankruptcy	+++	+	+++
Simplify Healthcare for the Patient	++	—	+++
Lower Overall Costs From Societal View	+	+	++

Health Insurance as Insurance Once Again

Insurance of nearly any type is supposed to protect people from unlikely, negative events. For auto or homeowners insurance, this means the unlikely prospect of a serious vehicular accident or fire in the home. A routine visit to a doctor is a regular occurrence for the majority of the population. Chronic prescription medicines are taken by a substantial portion of Americans. This does not fit the concept of insurance, wherein low costs are paid by the many so that they few have protection in times of need. With healthcare, most people have a regular need.

The problem for underwriters is that general health care delivery is not truly something that can be insured. Unlike auto insurance, it is a service that the majority of people utilize at some point of time, and the costs associated with that care are not limited. As it is, most people pay large sums each year on auto insurance and do not file claims in a given year. Health care, however, is a service that many people utilize at some point during the year, and some repeatedly. First-dollar coverage for many services, including preventive care, raises rates. A wide array of services covered raises rates. Increasing provider charges raises rates.

Universal catastrophic care enables healthcare insurance to act more like insurance once again. It resets the definition of health insurance to that of reinsurance. A design such as UCC prevents medical bankruptcy for most consumers who require expensive care–like in today’s traditional Medicare, government funding is used to pay for their expensive episode(s) of care. Instead of being funded through premiums, UCC will be financed through payroll and employer taxes, like the Medicare-for-all plans being raised today.

Under UCC, individuals would be responsible for paying for (or obtaining private health insurance to pay for) medical care up to a specific threshold expenditure. Everyone can buy this insurance through their own resources or through government subsidies (based on a sliding income scale). In simple terms, people with incomes qualifying for today’s Medicaid will pay nothing out of pocket; a person earning $300,000 a year must pay for all care up to the level defined as catastrophic care. Medical savings accounts like those seen today to pay for today’s care under a deductible can still be used to pay for medical care below the catastrophic care threshold. Beyond that level, the government pays all costs. Above this level, the government negotiates all bills and sets fees with physicians and hospitals, and other health providers.

For example, Mr. Jones chooses his doctor, any doctor he believes will serve him best (no referrals). His doctor says Mr. Jones needs surgical intervention. Prior authorization or precertification is not required (or is completed electronically, instantaneously for highly complex cases only). He chooses any hospital he wants to perform the surgery, because the hospital network is universal. He is treated by numerous professionals, none of whom are “out-of-network,” because every credentialed provider is part of the UCC network. All hospitals and providers must meet accreditation and quality standards to operate.

What Happens Before Reaching the Catastrophic Care Threshold?

Before reaching the UCC threshold, Mr. Jones pays up to the preset threshold amount for all his medical care (it is also possible that dental/ vision can be added to this benefit, depending on financing). This threshold will be calculated based on the initial target national health expenditure. Let’s say that this number is set at $8,000 for 2022. Mr. Jones, who earns a moderate income, would be asked to pay for all of his care up to this amount or purchase health insurance that covered this care up to $8,000 (family care limits may be something similar to today’s high-deductible limits). If Mr. Jones earned very little (i.e., qualified for Medicaid in today’s system), the government would subsidize 100% of his payments. Patients with preexisting conditions may be compensated with a credit towards their initial cost of care. Remember, relatively few patients would require care that costs more than $2,000 in any given year. Overall, the vast majority of people would pay far less in premiums and incur lower costs than they do under today’s system.

A person who wished to purchase coverage for an individual or their family from traditional health insurers up to the threshold amount should be subject to very low premiums. This is because health benefits costs are capped at a relatively low amount. And insurers may not find it necessary to require any tiered networks or other marginal mechanisms to hold down excessive costs. Furthermore, health insurance companies will need to market themselves differently than they do today to capture consumer marketshare—with this structure, Medicaid and Medicare can be folded into this system (or not), while providing the same or improved benefits to consumers.

Marketing to People, not to Health Plans

This also means that hospitals and other centers must be more consumer centric in their approach, as the patient is more directly involved with choosing a provider, without unnecessary gatekeeping referrals, or other forms of authorization.  

Optimally, the hospital would be paid a single global fee for its inpatient services, medication use, and follow-up care. Optimally, drug companies would be paid a standard fee for outpatient medications (they are also paid a standard negotiated fee for inpatient medications; payment does not vary by hospital).

For routine office visits and elected procedures, the patient chooses their providers, with as much information on quality that can be made available. The hospital and/or physicians must compete for that patient, especially for those initial, below-the-threshold dollars.

Today, we pay $3.7 trillion on US health care expenditures, or more than $11,200 per person, which is far more than any other nation pays for their advanced health care systems. In comparison, UCC has the potential to lower this amount, while providing care access for every US citizen.

Simplicity. Competing for patient dollars. Quality. No preexisting exclusions. The health care system can be refreshingly simple, wherein patient choice carries much greater weight (which would enable the market to work better), where physician practices would reap savings in administration, and where patients and consumers might actually see considerably lower costs.

Several complex issues would need to be addressed, such as setting fees, labor practices, medical malpractice, the role of pharmacy benefits managers, encouraging routine vaccinations, and others, but this may be the simplest, most refined and rational way to deconstruct the decades-old patchwork machinery that has promoted rampant costs, decreased physician satisfaction, increased underinsurance, limited a patient’s ability to navigate the system, and ceded a world-leading position in health care effectiveness and innovation.

For our archived posts on a wide variety of topics, see http://smhealthcomreports.blogspot.com/.

Some election-year topics are hotter than others, and now that impeachment seems to be behind us, many expect that the topic of health care access will heat up.

Not that this hasn’t gained plenty of press and airtime in the past few months. The Democratic presidential campaign has focused on the potential of “Medicare for All” to either spur the discussion of improving health coverage or kill it altogether. And the reasons are not simply about money. It’s also about a relatively quick closure of a massive industry and the jobs it would lose.

As of 2018, the Kaiser Family Foundation indicated that 16% of all adults 18 to 64 years of age were uninsured. Broken down by states, around one-fifth of nonelderly adults were uninsured in Texas (24%), Oklahoma (20%), Florida (19%), Georgia (19%), and Mississippi (19%). Far fewer are found in states that expanded their Medicaid programs under the Affordable Care Act. Underinsurance is a separate issue; this includes those families with unaffordable deductibles or especially who have coverage in plans that offer limited benefits and exclude benefits for preexisting conditions. The percentage of the population who are underinsured is growing.

And let’s put aside the rhetoric that such a move would amount to a socialist takeover of the country. Universal health coverage is an important, worthwhile goal. The real question remains how to close the remaining access gaps in our system and do so in a way that covers more people faster than we gain uninsured amidst rapidly rising health insurance premiums, cost sharing, and deductibles.

As many analysts have pointed out, gaining fully universal coverage through such a plan will either cost trillions more than the bloated amount we already spend on health care (over $3.6 trillion in 2018), or it will cost a somewhat small percentage more  than we already spend. The Sanders campaign believes it will save money over the long term, but there haven’t been as much support for this viewpoint. If it is phased in over a relatively short 4 years, that means that all health insurance employees and executives will need to find new occupations by 2024. I haven’t been able to locate consistent figures; the US Bureau of Labor Statistics does not specify numbers for “health service managers” that include health plans, insurers, PBMs, but of course many may overlap with physician, pharmacist, and other medical professional counts. However, if I could count all of the at-risk employees of UnitedHealthcare, CIGNA, CVS Aetna, Humana, and the regional health plans, the number of people affected could approach or surpass 750,000.

There are a number of different options, some of which have been well addressed and others have not.

The Public Option. This may have been an opportunity missed during the Obama Administration. The “public option” was originally planned as part of the Affordable Care Act but was dropped from the legislation to assure its passage. The public option was supposed to be a government-managed health insurance choice, apart from the private exchange plans. This has been raised again by certain Democratic presidential candidates, and there is polling evidence that it has more support than Medicare for all.

When first considered in 2009, the benefits of the public option were twofold: (1) the administrative costs of this nonprofit health plan would very likely be less than that for private health insurance, resulting in a lower cost alternative; and (2) with sufficient growth, it could force private plans to accept higher medical loss ratios to compete. The potential implication is that private insurance plans that have to show profits would not be able to compete in reality, and the public option would become a de facto national health insurance. And it may have driven private health insurers into narrow areas, such as integrated plans like Kaiser Permanente, which could still remain viable.

The public option remains available. If it was implemented in 2012, when the ACA took effect, consider how the coverage, cost, and deductible situation might have been different today.

Universal Catastrophic Care. This option has not been well described. It resets the definition of health insurance. In this iteration, it is more a type of reinsurance—it prevents most consumers who experience the need for expensive care from suffering medical bankruptcy. An added benefit is that it would help individuals and their dependents more easily navigate their acute care needs.

The problem for underwriters is that general health care delivery is not truly something that can be insured. Unlike auto insurance, it is a service that the majority of people utilize at some point of time, and the costs associated with that care are not limited. As it is, most people pay thousands of dollars per year on auto insurance, and do not file claims in a given year. Health care, however, is a service that most people utilize at some point during the year, and some repeatedly. First-dollar coverage for many services, including preventive care, raises rates. A wide array of services covered raises rates. Increasing provider charges raises rates.

Under this scenario, individuals would be responsible for paying for (or obtaining private health insurance to pay for) medical care up to a specific expenditure. Everyone can buy this insurance through their own resources or through government subsidies (based on a sliding scale). In simple terms, a person with incomes qualifying for Medicaid pay nothing out of pocket; a person earning $500,000 must pay themselves, again up to the level defined as catastrophic care. Beyond that level, the government pays all costs. Simple.

In our next post, we’ll detail more closely how the catastrophic care option can work.

For our archived posts on a wide variety of topics, see http://smhealthcomreports.blogspot.com/.

The rebates given to pharmacy benefit managers to secure a drug’s place on the formulary have become a difficult barrier to coverage for new products. The rebate income for these PBMs is sometimes passed on to health plans, insurers, and employer purchasers, but more often it is not.

A big issue is that managed care organizations tend to become addicted to millions of dollars in rebate “income,” and this mindset prevents serious consideration of new medications at competitive costs.

For biosimilars, Pfizer and Merck have had a difficult time dislodging Janssen, maker of Remicade^®, from its preferred formulary position, despite lower prices based on wholesale acquisition cost (WAC). Janssen has simply matched the net cost (through increasing rebates), while keeping its WAC costs high—tempting plans with ever-increasing rebate revenue. The health plans don’t see the benefit of incurring the administrative costs of moving masses of patients from the preferred product to a new one, or seeing this revenue stream interrupted, without an overall further improvement in net costs.

Managed care plans have long said that discounts of 25% or more will be necessary to release the rebate stranglehold of preferred products. In the case of infliximab, this has not yet occurred, based on recent minor inroads made by Merck’s Renflexis^® biosimilar, despite larger discounts. Until greater competition is available, which drives down the WAC prices (and then average sales prices [ASPs]), barriers to accessing new medications will remain. In fact, when competition does increase, makers of the originator products, like Janssen, can simply ratchet up their rebates to maintain a hold on sales (and a billion-dollar plus profit).

Perhaps the best way around this is to force a change in the marketbasket. This can be accomplished in a couple of ways. The first, by instituting separate tiers for biosimilars and reference agents, takes the biosimilars out of the 1 of 2 preferred drug contracting restrictions, and allows patients to access biosimilars as well as preferred brands.

A second way is to reconsider biologic agents according to indication-based contracts or mechanism-of-action (MOA) based differences. Therefore, the marketbasket is modified to consider anti-TNFs separate from interleukins, allowing preferred agents in each separate category. This would allow, for instance, for more effective psoriasis agents to be well covered, and maintain the preferred position of Humira^® and Enbrel^® for appropriate patients.

A third way is to work out some innovative value-based contract, in which the manufacturer and health plan/insurer reaches an agreement on (usually) the expected outcomes of drug use and additional rebates or performance guarantees if the medication fails to deliver on this performance. The most important consideration in this agreement is the practicality of measuring an outcome of interest or ensuring adherence.

The rebate trap seems to be ensnaring more manufacturers of new biologics and biosimilars. Without greater consideration of the overall good, this trap can cause systematic problems for the pharmaceutical industry and discourage drug innovation and accessibility.

For our archived posts on a wide variety of topics, see http://smhealthcomreports.blogspot.com/.

Years ago, the undeclared rule of law was that a Pharmacy and Therapeutics (P&T) Committee’s deliberations must be free of cost concerns. This rule is increasingly being overturned.

The P&T Committee, as a formulary decision-making body, was supposed to decide a new agent’s fate based only on the clinical evidence for efficacy and safety. According to the standard protocol, once the P&T Committee’s decided to add a product to formulary, it was then the pharmacy contracting executives’ responsibility to simply get it for the best price available. This created a handy shield to prevent public complaint that a health plan or insurer was really making decisions based treatment cost.

The preeminence of 2 trends has caused payers to rethink this firewall. First, over the course of 25 years, multitiered copayment designs have become the standard. The very idea of a 3-tier plan, for instance, is to prefer products that medical and pharmacy executives believe patients should be incentivized to use, based on some facet of value. In practical terms, if tier 1 is for generics only, what is the difference between tier 2 (preferred brands) and tier 3 (nonpreferred brands)? The clinical difference between a preferred drug and, say, “me-too” agents on tier 3 is often insignificant. Most often, it comes down to which offers the best net price. Medicaid and exchange plans seem to be reverting to 2 tier plans (generic and covered brands), but these, too, are based on perceived value.

The second trend is the movement towards value-based benefit design (VBBD). At its simplified core, VBBD tries to determine whether certain drugs have more value (in terms of proof of clinical effectiveness, safety, or cost) compared with others. As a result, formulary decision makers are increasingly seeking comparative-effectiveness information or head-to-head study results to help determine the value of a new treatments. This information is added to P&T Committee discussions (sometimes included, if available, in drug information monographs). The result of which is a determination of whether prior authorization or step edits may be warranted to access the drug and what those criteria might be. P&T Committee members will have a hand in those decisions as well.

It only then makes sense to integrate the discussion of a drug’s cost into that of its clinical merits at the P&T Committee level, rather than to keep the greater value picture separate from formulary decisions.

The double-digit growth of specialty pharmacy spending has injected new urgency into the conversation about value for payers. The P&T Committee is (still) the arbiter of value for the pharmacy benefit and for specialty pharmaceuticals. That is why at least two-thirds of P&T Committees are now fully invested in cost considerations. The old firewall has been breached.

SM Health Communications provides writing, consulting, and market research services for the long-term care and payer markets. Its proprietary P&T Insight™ virtual P&T Committee program is the leading mock P&T Committee product in the field. For more information, please visit www.smhealthcom.com or contact Stanton R. Mehr, President, at stan.mehr@smhealthcom.com.

For our archived posts on a wide variety of topics, see http://smhealthcomreports.blogspot.com/.

This is one of the prickliest topics in the health care industry, and it has been as long as I can remember. One of the publications I worked with was called Product Management Today. Long defunct, this periodical followed the pharmaceutical industry quite closely. As a result, we were familiar with Tufts University’s Center for the Study of Drug Development. This was the first organization to publish what was considered credible figures as to what it cost to bring a drug to market.

In the early 1990s, that number was published in the Journal of Medical Economics to be about $231 million. Remember, these were generally small molecules, and the drug industry consisted of dozens of big manufacturers identifying their molecular targets, characterizing them, and cultivating them through the clinical trial process. In 2003, this number was revised upward to somewhere north of $800 million. In 2016, the Tufts team recalculated the numbers, and it found essentially a 10-fold difference (not accounting for the inflationary change in the value of the dollar between 1991 and 2016). They indicated that it costs $2.7 billion to bring a product to the market today, which also considers the cost of failed drug development. In other words, they added the opportunity cost to a pharma company for a drug that was withdrawn or failed clinical trials (any stage). In their 2016 analysis, they took as their drug sample, 106 investigational drugs, 87 being small-molecule compounds and the remaining 19 biologics. I’m guessing that if biologics represented more than 18% of the sample, the total average estimate could have been even greater.

The cost to produce a biosimilar is considerably less. This is partly the result of the lesser clinical trial requirements compared with new chemical entities. Although this figure has not been convincingly calculated, I’ve heard it cited to be anywhere up to $250 million. This seems to be reasonable, based on the development requirements. Unfortunately, several biosimilar manufacturers have to wait to market their agents because of patent litigation, and that in itself represents a cost.

Today, the results of another study was released, which offers a very different number. Published in JAMA Internal Medicine, authors from Oregon Health and Sciences University and Memorial Sloan Kettering Medical Center found that the cost to produce 10 cancer medications was really $648 million (range, $204 million to $2.602 billion)—add another $110 million or so for opportunity costs. This included 5 drugs that received fast-track approval. Not only did the researchers use US Securities and Exchange filings that cited manufacturer-reported costs, but they limited the analysis to only manufacturers without a previous approved drug on the market. The drugs evaluated included several monoclonal antibodies, so it was reflective of complex molecule development.

I’d like to point out another question that perhaps skews the calculation. Rarely these days does “big pharma” get involved with new drug identification and characterization. True, they are often involved in the expensive clinical trial phase, but do we read in the paper weekly that a drug discovery company has licensed or sold the product to a big pharma entity (or even sold the company itself)? And what is the guarantee that they are not overpaying for the price of the drug or the company?

The upshot of this is that the 10 companies evaluated in the more recent study had cumulative revenues resulting from their new agents of $67 billion from the time of approval (until December 2016 or the time it sold or licensed the product to another company). The range per product was ranged from $204.1 million to $22.3 billion. It sounds overall, that they were good buys for the big pharma companies but not necessarily for the health care system.

In other words, whether it cost $648 million, $800 million, or $2.7 billion, the research and development costs for these new agents are made back in a year or less. It is hard for us to listen seriously to pharma companies who use the cost to develop the agents, or their having to eat the cost of failed agents, as credible justification for the prices being charged.