Realizing the Value of FDA-Approved Therapies


This paper examines actuarially equivalent benefit designs private payers can use to provide FDA-approved innovative therapies. We include all FDA-approved innovative therapies in our study regardless of chemical structure or mode of administration. This includes therapies administered as oral medicine, ointment, injections, infusions or inhalation. These therapies may be proteins or enzymes or other complex chemicals that may be manufactured through genetic alteration of yeast, bacteria or other organisms, or through other complex synthesis. Our analysis does not consider generic drugs, herbal therapies, devices or durable medical equipment.

Today's innovative therapies are the fruits of many recent scientific achievements and the industry is hopeful that during the next five years, additional innovative therapies will create improved treatments and enhance the quality of life for thousands of people.

New and improved medical treatments have a cost. This cost is often absorbed by the patient's health insurance program creating employer and payer concerns regarding potential rising costs. As a result, employers and other payers are concerned about increased healthcare costs. This paper considers the following questions:

  • How much will innovative therapies add to costs for employers and their insurers?
  • How will individuals' costs vary with coverage design?
  • How can private payers use benefit designs to provide innovative therapies?

This study considers these questions with a time-line of only the next five years (2006-2011), because long term predictions regarding innovative therapy approvals are too uncertain. The authors believe that all parties interested and excited by the promise of 21st Century medicine will find this study useful and informative.


Many seriously ill patients currently use innovative therapies as an essential part of their treatment. We expect Food and Drug Administration (FDA) approval of many new therapies over the next few years. In this document we use the term "innovative therapies" instead of "biotechnology products" because the latter is sometimes interpreted as applying to only injectable or infused drugs, while we include innovative therapies administered in any form. These therapies may be proteins or enzymes or other complex chemicals that may be manufactured through genetic alteration of yeast, bacteria or other organisms, or through other complex synthesis. Our analysis does not consider generic drugs, herbal therapies, devices or durable medical equipment.

Our study, funded by the Biotechnology Industry Organization (BIO), concludes that

  • The costs of innovative therapies will generally not create a large cost burden relative to other costs for private healthcare payers by 2011.

  • As employer and health plans struggle to control the cost of providing health care, benefit designs are moving towards limiting coverage for innovative therapies or requiring high cost sharing ("out-of-pocket" expenses) for these products.

  • Private payers can generally make minor changes in their benefit designs that will assure the affordability of innovative therapies for their members.

Even though an innovative therapy can be more expensive than most current medicines, our projections suggest that, in aggregate, the costs of new innovative therapies will not create large cost burdens on the healthcare system by 2011.

In this report we focus on private commercial payers, which are usually associated with employer-based health insurance. We briefly discuss the impact of the Medicare Part D benefit structure on patient out-of-pocket costs for innovative therapies, but we do not specifically analyze the impact of innovative therapies on federal programs. We conclude that private commercial payers can expect an increase in total healthcare costs of a little over 1% due to new innovative therapies by 2011.

By 2011, the total cost for all innovative therapies (existing and new) will probably be about 6% of total private commercial payer costs. This compares to about 5% for 2006. Given the current, high annual rates of medical cost inflation, it will be hard for many payers to discern the cost increases directly related to innovative therapies.

However, we find that certain coverage and cost-sharing structures can push catastrophic costs onto individuals who require treatment with innovative therapies. Cost sharing refers to the portion of medical spending absorbed by the patient and not the insurer. For example, insurance coverage may specify that the patient will pay for 20% of certain prescription drugs.

Many of today's insurance coverage designs include a combination of copays, coinsurance and lifetime limits and were designed before FDA-approved innovative therapies were widely used. We expect that some benefit design modifications may be needed to accommodate these new therapies. The benefit modifications should be similar to previous benefit modifications that were used to adopt other new medical technologies. In addition, some of today's "consumer driven" designs can have features that can shift much of the costs of innovative therapies to the patient. Cost-neutral options exist, including redesigning benefit structures, for private payers to reduce the impact of the cost of innovative therapies.

We use the term "private commercial payer" or "payer" to refer to an employer providing health coverage or to some other non-employer plan sponsor such as a union or a Taft-Hartley group. These terms may also mean an insurance company or Health Maintenance Organization (HMO).

This study investigates the impact of new costs of innovative therapies. We focus on costs paid by private payers for their members and present these costs with per capita measures using payer parlance - Per Member Per Month (PMPM). We also show the dollar impact of typical individual cost-sharing coverage options, such as copays and coinsurance.

Our study results point to the need for wise choices of coverage alternatives, choices informed by an understanding of the value that innovative therapies brings to individuals. Insurers, employers and, to some extent, consumers choose coverage options that may affect tens of thousands of available procedures and products. Not surprisingly, new bioscience creates challenges for existing benefit structures.

As with other growing segments of the economy, it is impossible to precisely predict the longer-term impact of the innovative therapy revolution on healthcare costs. Certainly, FDA-approved innovative therapies promise to play an increasingly important role in improving healthcare. We present some of the near-term challenges and opportunities payers face incorporating new innovative therapies into their benefit package.

However, the reader should consider that even the short-term forecasts in this report are based on assumptions and cannot capture impacts such as changes in the regulatory environment or scientific developments, so these forecasts should be reviewed carefully for their applicability for any particular purpose.

This report is based on national average estimates and assumptions as to future events. Actual results for any particular organization will likely differ for many reasons including fluctuation in reimbursement and future events.

The report reflects the authors' findings and does not reflect the endorsement of any particular legislation by Milliman.


Recent developments in insurance coverage designs include consumer-driven health plans and health savings accounts. One result of double-digit healthcare cost inflation is that payers are shifting more of the costs to their members through coverage changes. These coverage changes may coincide with the arrival of new FDA-approved innovative therapies. Unfortunately, some coverage designs - new and old - do not fit well with the new innovative therapies that treat serious disease.

In this section we present near-term cost estimates and coverage considerations for innovative therapies that are relevant to commercial insurers and employer-sponsored health plans. We start with aggregate population costs and then consider the impact on individuals.

Putting High Costs into Perspective

Current prices for innovative therapies average $5,000 to $20,000 per person per year /1 With trend and the creation of new innovative therapies in the future the average in 2011 will be somewhat higher. To put these costs into perspective, we created two categories: the number of patients with high costs for hospital/medical (non-drug) spending and the number of patients with high drug spending.

The following chart shows, for a typical employer with 10,000 employees /2 (plus covered dependents - a total of about 22,000 people), the number of individuals with relatively high annual claim costs. Consistent with today's cost distribution, by 2011, most expensive patients will still be expensive because of hospital/medical costs and not because of overall prescription drug costs, which include the cost of innovative therapies.

Number of People with High Annual Claim Costs in 2011

Most individuals with high healthcare costs will receive both hospital/medical care and drug treatments, possibly including innovative therapies. Payers bore the costs for members with very high claims well before the biotechnology era, as they do today, and we expect the new medicines will not fundamentally alter the number of such high-cost patients in 2011.

Estimated Aggregate Cost of FDA-Approved Innovative Therapies for 2011

By 2011, the total cost for all FDA-approved innovative therapies (existing and new) will probably be about 6% of total private commercial payer costs. This compares to about 5% for 2006. Given the current, high annual rates of medical cost inflation, it will be hard for many payers to discern the cost increases directly related to innovative therapies.

Based on the methodology and assumptions described in the Methodology section, we estimate that in 2011:

  • New innovative therapies (FDA approved after 2005) will add a little over 1%, or $5.00 Per Member Per Month (PMPM), to the claim costs of commercial private payers.

  • Existing innovative therapies (available before 2006) will remain at about the current level, close to 5%, of total claim costs for private commercial payers.

We estimate that new innovative therapies will add approximately $5.00 PMPM in new costs and continued growth of existing products will add approximately $4.80, totaling $9.80 PMPM. However, the $9.80 PMPM payer cost assumes relatively low cost sharing by the member. Given the trend toward increases in cost sharing, some portion of this $9.80 PMPM will likely be borne by patients. The new therapies will add approximately $14.5 billion dollars in expenses for the commercial population. Additional amounts will flow through Medicare, Medicaid and other programs, or will be implicit in hospital-based services. The elderly will likely incur a high portion of the cost of these therapies, because Medicare beneficiaries tend to have more significant medical needs. However, the age impact of innovative therapies will depend on the clinical effectiveness characteristics of yet to be developed therapies.

Many innovative therapies are already in use, and we expect an increase in utilization of these products. We estimate that the cost of existing innovative therapies will increase by about 35% from 2006 to 2011. This increase reflects both a continuation of drug price inflation along with increased use of existing innovative therapies.

We estimate total innovative therapy spending (all payers including Medicare and Medicaid) to reach above $100 billion by 2011, up from about $55 billion in 2005. The 2005 number represents about 3% of the projected 2005 national healthcare expenditure of over $2 trillion /3. We note that the high dollar sales projections appearing in stock market news items about particular firms may not consider the likelihood that some investigational products will not reach market.

The Challenge of Regressive Benefit Designs

"Regressive" /4 benefit designs require patients needing expensive care to bear a disproportionate amount of the cost. By contrast, most employers seek to attract employees by maintaining "Progressive" benefit designs that insulate very sick employees or their dependents from high costs. Innovative therapies have a cost and are often used to treat people with serious conditions. Providing progressive benefit designs that protect the individual from the costs of innovative therapies poses a challenge to underwriters, consultants, actuaries and human resource experts.

While the aggregate costs of innovative therapies seem manageable over the next several years, the cost to treat individuals with innovative therapies can be expensive. Many innovative therapies are expected to cost $20,000 per patient per year or more. This poses special issues for individuals because certain cost sharing mechanisms, such as unlimited member cost sharing or low annual benefit limits for prescriptions, could make innovative therapies unaffordable.

A typical prescription drug benefit might have the following structure:

1st Tier: Low cost sharing for generic drugs (typically, under $20 per filled prescription)

2nd Tier: Higher cost sharing for preferred brand drugs

3rd Tier: Highest cost sharing for innovative therapies and non-preferred brand drugs (subject to medical necessity review and approval)

The structure's goal is to provide financial incentives to encourage patients to use generic drugs and avoid higher-cost drugs. Depending on the details, this structure can be progressive or regressive.

High cost sharing for certain drugs creates a regressive structure because of the large out-of-pocket expense for a seriously ill patient. For example, the following prescription benefit structure poses a particular problem for patients requiring expensive innovative therapy:

1st Tier: $15 Copay (generics)

2nd Tier: $30 Copay (preferred brand)

3rd Tier: 25% Coinsurance (innovative therapies and non-preferred brands)

A patient that requires an $18,000 innovative therapy would pay 25% of $18,000, or $4,500, with the above benefit. This cost sharing amount is unaffordable for many patients. Depending on the details of their policy, members with seemingly generous insurance coverage can have large out-of-pocket obligations for medical costs including innovative therapies.

In an effort to control costs and offer lower-cost insurance, some payers offer coverage with unlimited member cost sharing, such as the 25% coinsurance for the 3rd tier, as in the above example. Another cost control technique with similar impact is to limit annual drug coverage to some amount such as $10,000.

Although we did not model the impact of innovative therapies on Medicare, we note that the Medicare Part D benefit provides significant coverage after reaching the 2007 annual patient out-of-pocket limit of $3,850 on all covered drugs. Low-income beneficiaries will receive a more generous benefit with less cost sharing. However, some infused or injectable therapies may be paid under the Part B benefit and subject to unlimited 20% coinsurance.

Benefit Solutions

Fortunately, benefit design can be altered to accommodate both the added cost of FDA-approved innovative therapies and the need to control cost. Benefit managers can make choices to control costs and provide valuable innovative therapies.

The following table compares the projected national average costs of selected benefits for 2011.

Innovative Therapy: Benefit Cost Comparisons
Category Est. 2011 Cost
Increase in Spending on Innovative
Therapies from 2006 (new & old products)
10% increase in Generic drug utilization -$3.00
Reduce inpatient days by 10% -$6.00
Chiropractor $6.00
Podiatry $2.00
Vision Exams $3.00
Glasses/Contacts $5.00

* This is the projected full cost; plans generally would not pay 100%.

Source: 2006 Milliman Health Cost Guidelines

The preceding table offers examples of choices available to payers. For example, a payer could almost offset the entire projected cost increase for innovative therapies ($9.80 PMPM) by removing coverage for chiropractor and podiatry, perhaps shifting these benefits into a flexible spending account. The reader should note that some of the above benefits may be subject to state mandate regulations for policies issued through HMOs or insurance companies and, if so, coverage may be required as part of an insurance policy.

Benefit managers can also adjust cost sharing to control costs. The following table compares some simple cost sharing changes to the increased costs of innovative therapies:

Innovative Therapy: Cost Sharing Trade-offs
Category Est. 2011 Cost
Increase in Spending on Innovative
Therapies from 2006 (new & old products)
Increase Office Visit Copays from $15 to $20 -$5.00
Increase Inpatient per Admit Copay from $1,000 to $1,500 -$3.00

* This is the projected full cost; plans generally would not pay 100%.

Source: 2006 Milliman Health Cost Guidelines

The tables in this section show choices that benefit managers have to keep their benefits current with changing technology while controlling costs.

Progressive Versus Regressive Benefit Designs

The following examples illustrate how two actuarially equivalent benefit designs can cover innovative therapies. Two different benefit designs are actuarially equivalent if, across a broad population, the expected cost of both benefit designs is the same.

Using established actuarial methods; the authors present the following 2011 actuarially equivalent prescription drug benefit designs. For each benefit design, we show the cost sharing for a member requiring a $1,500 per month ($18,000 per year) innovative therapy (assumed to be in the 3rd Tier in all designs):

Providing for Catastrophic Costs in a Stand-alone Prescription Drug Program
Plan Benefit Design Actuarial
Annual Cost Sharing for
Therapy Costing $1,500
per Month
A Tier 1: $15 Copay
Tier 2: $30 Copay
Tier 3: $50 Copay
Equivalent $600
B Tier 1: $15 Copay
Tier 2: $30 Copay
Tier 3: 25% Coinsurance
C Tier 1: $10 Copay
Tier 2: $20 Copay
Tier 3: $40 Copay
Equivalent $480
D Tier 1: $10 Copay
Tier 2: $20 Copay
Tier 3: 20% Coinsurance

In the above table, plans A and B are actuarially equivalent to each other, as are plans C and D. The payer with plan A can afford to pay more for the catastrophic benefit than with plan B, because plan A's members have slightly higher cost sharing for many products relative to plan B. Likewise, in plan C, the $40 copay will require some people to pay more than the 20% coinsurance in plan D for some drugs and subsidize the few people who need the innovative therapy. There are an unlimited number of pairs of similar "equivalent" benefit designs. /5

Similar choices apply to benefits where innovative therapies are covered through a medical benefit as opposed to a stand-alone prescription drug benefit. In the Appendix, we show the case of a comprehensive major medical plan with an inpatient per-day copay, a hospital outpatient per-case copay and coinsurance or copay for other services. The limit on member cost sharing ("out-of-pocket" expenses) drastically affects the payments made by the member.

The Concept of Actuarial Equivalence

The above examples illustrate "actuarial equivalence." On average, a payer can afford to pay for rare and catastrophic care if it pays slightly less for a number of more common, but less expensive services. However, sometimes it is not this simple.

Actuarial equivalence is determined across large populations, but the random occurrence of a rare event can have a great impact on small groups. For example, among a small group of people, one person can suffer catastrophic costs - and those costs can impose a hardship on the entire small group. Actuarial equivalence, and the cross-subsidies that can cover catastrophic costs, require additional mechanisms to operate successfully among small groups of people.

Insurance adjusts for catastrophic risks through pooling and other techniques. For example, an "average" group of 50 covered individuals might expect to pay about $165,000 in health insurance claims in one year. The unlikely event of one organ transplant in that small group could cause the cost to double. By contrast, a group of 5,000 covered individuals might expect to pay $16.5 million in claims in one year. A similar, organ transplant in that larger group would add only 1%. Consequently, insurance programs use pooling /6, stoploss /7 and other techniques /8 to spread the risk of unlikely catastrophic events.

Managing Adverse Selection and Fluctuation

Adverse selection occurs when individuals or groups make benefit selections based on their individual needs. /9 Therefore, healthy people are more likely to go without insurance or chose cheaper insurance, and individuals with particular ailments are more likely to choose plans that provide more generous coverage for their problem and are therefore more expensive to the insurer.

Adverse selection also affects prescription drug benefit design. If a payer offers both Plan A and B, described above, people who need innovative therapies (costing $1,000 or more per month) will tend to choose Plan A because of the $50 copay, and people who want a third tier drug with a cost of $100 will choose Plan B, because the $25 coinsurance (25% of $100) is less than the $50 copay in Plan B. The cross-subsidy that Plan A uses to fund the higher cost therapy will not work well in this example.

Because a concentration of expensive individuals can add a lot of expense to a payer's cost, adverse selection and fluctuation are real concerns in benefit design. However, these issues are not new. Insurers have faced adverse selection and fluctuation since the first employer-based health insurance policy was issued. As a result, pooling, reinsurance, benefit mandates, underwriting and other techniques have been developed to address these challenges, and we are confident that these methods can be successfully used to adapt to the cost of FDA-approved innovative therapies. Actuarial literature offers fuller treatment of these issues /10.

Potential Cost Offsets

This study examines important cost issues for innovative therapies as a whole rather than for specific products. Although we did not examine the potential for innovative therapies to offset the use and cost of other medical products and services, such offsets are a potentially important ameliorating factor. We have been conservative by assuming that no such offset exists.

New and improved medical treatment does not necessarily cost more than traditional treatment, although obviously it can. The current standard of care for certain diseases can be very expensive. Therefore, treatments that replace or modernize the traditional standard of care can reduce a patient's medical costs, even if the new treatments are themselves very expensive. An example is the cost offset of a hypothetical product that would prevent or cure Alzheimer's disease and avoid years of very costly nursing home care. In this example, even if the therapy is expensive, it will be cheaper than years of institutional care.

We believe that potential cost offsets of innovative therapies need to be considered in two ways:

  • Traditional cost-benefit. Studies of this sort focus on isolated or single-factor decisions, such as comparing the costs and benefits for different treatments for high cholesterol or advanced cancer.

  • System change cost-benefit. Technologies such as advanced medical treatments can be the agent for dramatic changes in our current, inefficient medical system. For example, use of innovative therapies is often closely monitored according to the principles of evidence-based medicine, which, in turn, can lead to dramatically improved quality and efficiency and reduced costs.

Recommendations for Payers

We suggest that payers consider the following issues in choosing benefit designs:

  1. The compatibility of benefit designs with innovative technologies and needs.

  2. The trade-off between catastrophic benefits and coverage for more routine and low-cost services.

  3. The impact of benefit designs on individuals with serious illness.

  4. Ways to manage the risk of adverse selection and fluctuations.

  5. The cost of traditional treatments and potential cost offsets as innovative therapies replace or supplements other care.


Our work required us to estimate both future innovative therapy costs and future medical costs. This section explains our methodology.

FDA-Approved Innovative Therapies

We included all FDA-approved innovative therapies in our study regardless of chemical structure or mode of administration. This includes therapies administered as oral medicine, ointment, injections, infusions or inhalation. These therapies may be proteins or enzymes or other complex chemicals that may be manufactured through genetic alteration of yeast, bacteria or other organisms, or through other complex synthesis. We did not include generic drugs, herbal therapies or durable medical equipment.

Estimated Future Spending on Innovative Therapy

Wall Street projections of U.S. sales of innovative therapies have often proved to be high; analysts tend to be bullish about the sector they cover. As with any growth industry, particular innovative therapies may have huge potential sales, but they might not even get FDA approval or may have much more limited application than hoped. We used projections of innovative therapy sales as one source for our estimates.

We reviewed the cost of individual innovative therapies from these projections and the diseases targeted by the therapy. Using medical opinion we estimated the portion of each that would be used for the commercial population. Dividing the projected 2011 commercial sales by the projected 2011 aggregate commercially insured population produces a per-capita cost for innovative therapy.

We compared the results produced for 2005 from the Wall Street projects to the results from our health claims database analysis. We adjusted the results slightly for the optimism inherent in Wall Street. We believe the resulting projected costs for 2011 are a somewhat more conservative projection of expected costs.

Estimated Future Medical Costs

We used Milliman's Health Cost Guidelines (HCGs) to estimate current medical costs for a typical employer population. The HCGs are an extensive claims cost database containing detailed average charge and utilization experience and fee schedules for roughly 60 benefit categories. The estimates presented by the HCGs include current innovative therapies in the market.

We applied annual trends, starting at 10% for 2006 declining to 7% for 2011, to the 2005 HCG figures to project per-capita costs to 2011 for total medical services excluding the innovative therapies described above. We assumed a similar trend in per-capita costs for prescription drugs.

Modeling Benefit Designs

The costs borne by individuals or insurers for any medical treatment depend not just on the price, but also on the benefit design. This section describes how we modeled different benefits.

A large number of innovative therapies are administered by a physician in the doctor's office or in a hospital outpatient setting. As such they have traditionally been covered under the hospital or medical portion of a typical private commercial payer's insurance policy. There is a trend towards moving these drugs to the pharmacy benefit so as to take advantage of the deep discounts typically offered by a pharmacy benefit manager or specialty pharmacy.

We created two baseline models from the HCGs to allow for the traditional as well as "carve out" benefit designs. The first model assumed that innovative therapies would continue to be covered as a medical expense. The second model assumed that all innovative therapies would become part of a three-tier pharmacy benefit with the innovative therapies covered under the 3rd tier and having the highest cost sharing.

After our 2011 cost models were developed, we then crafted "progressive" and "regressive" benefit designs that have the same expected PMPM claim costs and therefore would be considered actuarial equivalent from the payer point of view. The HCGs contain standard actuarial tools that we used to determine the impact of various levels and kinds of cost sharing.

We did not consider payer administrative costs in our projections.


A comprehensive major medical plan provides coverage for inpatient and outpatient hospital care, physician care and other medical care such as radiology and laboratory. In the case of a comprehensive major medical plan, the total annual cost sharing will depend on the other services the patient uses. We show the drug cost sharing under various alternative plans for a person using a drug that costs $1,500 per month.

Avoiding Catastrophic Costs in a Comprehensive Benefit
Plan Benefit Design Actuarial
Annual Cost Sharing for
Innovative Therapy Costing
$1,500 per Month
E $200 / IP day
$100 / Outpatient
$50 Emergency Room
$20 other
Equivalent $240
F $300 / IP day
$100 / Outpatient
$50 Emergency Room
20% other
No limit on coinsurance
G $300 / IP day
$100 / Outpatient
$50 Emergency Room
30% other
$2,000 limit on coinsurance
or less
H $350 / IP day
$100 / Outpatient
$50 Emergency Room
$20 other
Equivalent $240
I $200 / IP day
$100 / Outpatient
$50 Emergency Room
25% other
No limit on coinsurance
J $200 / IP day
$100 / Outpatient
$50 Emergency Room
35% other
$3,000 limit on coinsurance
or less

The costs of E, F and G are the same for a large population, as are the costs of H, I and J.
IP = copay per inpatient day
Outpatient = copay per hospital outpatient visit
Emergency Room = copay per ER visit
Other = copay or coinsurance for other services such as office visits or drugs


1/ Express Scripts 2005 Drug Trend Report. Online at
2/ Milliman 2006 Health Cost Guidelines standard labor population
3/ Centers for Medicare and Medicaid Services, Office of the Actuary. National Health Expenditure Data, Retrieved December 6, 2006 from (PDF)
4/ This borrows terminology from tax policy, where a regressive tax is one where the taxes paid by lower income people represent a higher portion of their income than for richer people. Internal Revenue Service United States Department of the Treasury, retrieved December 6, 2006 from
5/ Adverse selection can affect that cost equality, if people who know they need expensive treatments leave Plan B or D and choose Plan A or C, respectively.
6/ Bluhm, William F., ed. 2003. Group Insurance: Fourth Edition, p. 677-686
7/ Stoploss programs can spread the costs of catastrophic events across a broad population. For example, a stoploss insurer may sell coverage for annual claims by any individual greater than $100,000. The premiums collected by the stoploss insurer from many such policies would go to pay for the few, random high-cost cases. Bluhm, William F., ed. 2003. Group Insurance: Fourth Edition, p. 708-710
8/ For example, a number of states have established mechanisms to promote risk sharing among insurers of small group and individual insurance. Bluhm, William F., ed. 2003. Group Insurance: Fourth Edition, p. 492-493
9/ Kongstvedt, Peter R. 2001. The Managed Health Care Handbook: Fourth Edition, p. 1358
10/ Bluhm, William F., ed. 2003. Group Insurance: Fourth Edition