In This Issue:

The Debate over Specialty Pharmaceuticals: Is the Promise Worth the Price?

Authors: Ian Spatz, Senior Advisor, Manatt Health Solutions | Nancy McGee, Managing Director, Manatt Health Solutions

EDITOR’S NOTE: Specialty pharmaceuticals hold great promise for patients with chronic, serious or life-threatening illnesses. At the same time, they can cost thousands of dollars a month—and are key drivers of rising healthcare costs. Manatt Health examines both sides of the debate around specialty pharmaceuticals in a new paper, available in the Health Policy Briefs section of the “Health Affairs” web site. Below is a summary of the key issues. Click here to read the full brief.  

What’s the Issue?

Specialty pharmaceuticals are a rapidly growing share of total drug expenditures by public and private health plans. These drugs--typically used to treat chronic, serious, or life-threatening conditions, such as cancer, rheumatoid arthritis, growth hormone deficiency and multiple sclerosis--are often priced much higher than traditional drugs. Total costs can be in the thousands of dollars a month and can exceed $100,000 a year for some products. There are usually few if any low-cost generic equivalents.

Research into specialty drugs is attractive to biopharmaceutical companies from both a medical innovation and a business perspective. The ability for the market to sustain the relatively high cost of these products creates substantial incentives for companies to research and develop products that address serious, unmet health needs. Also, in some cases, companion diagnostic tests are used to identify patient subpopulations in which the specialty product generates a differential response, either negative or positive. As a result, specialty products have stimulated diagnostic research. Given the level of investment, this means that patients and payers can expect continued innovation and research to develop specialty products and companion diagnostics in the future.

Payers are interested in steps they can take to control the contribution that specialty pharmaceuticals make to the growth in premium costs while ensuring that patients can access the drugs that will improve their health, as well as their quality and length of life. Biopharmaceutical manufacturers and patient advocates are concerned that restrictions on specialty pharmaceuticals could discourage research and harm patient care. Pharmacists also are concerned that some plans to control the use and cost of specialty pharmaceuticals will limit their opportunities to serve patients.

What’s the Background?

Specialty pharmaceuticals are generally defined as drugs and biologics (medicines derived from living cells cultured in a laboratory) that are complex to manufacture, difficult to administer, may require special patient monitoring, and sometimes have Food and Drug Administration (FDA)-mandated strategies to control and monitor their use. Increasingly, they also are defined by exceeding a certain cost threshold, such as $600 a month, that may place them on higher cost-sharing tiers.

From a distribution perspective, specialty pharmaceuticals may require specialized shipping and temperature-controlled storage and handling. To meet these special needs, a class of providers, known as specialty pharmacies, exists to distribute and dispense these products. Specialty pharmacies can be owned by stand-alone companies, pharmacy benefit managers (PBMs) or large pharmacy chains.

Because they may include oral, self-injectable or physician-administered infusions, specialty pharmaceuticals may be covered under either the pharmaceutical or the medical benefit of health plans. Although estimates vary depending on data sources, Express Scripts’ drug trend report claims that in 2010 approximately 47% of U.S. specialty drug spending occurred in the medical benefit.

Experts expect that share will continue to rise as specialty pharmaceuticals represent a critical pharmaceutical growth segment in drug development. Two of the largest PBMs, CVS Caremark and Express Scripts, report specialty pharmaceuticals are the fastest-growing segment of their drug spending. By 2019, they expect specialty products to account for half of their plan sponsors’ drug spending. IMS data reflects the same rapid growth, showing that 65% of new drug spending in 2012 was on specialty pharmaceuticals. That trend is continuing. In 2013, 60% of the new drugs the FDA is expected to approve will be specialty pharmaceuticals.

Because specialty pharmaceuticals treat chronic, serious, or life-threatening conditions, physicians try to initiate treatment sooner. As a result, costs accruing to the health plan occur earlier and last longer, particularly for products used to treat chronic diseases. From a patient perspective, however, these new therapies represent a more effective way to treat diseases in categories that offer few options.

What’s the Debate?

1. Cost Controls
Payers are seeking ways to manage the growing cost of specialty pharmaceuticals appropriately. One common tool for specialty medicines covered through the outpatient pharmaceutical benefit is to charge high cost sharing in the form of copayments (fixed amounts) or coinsurance (percentage amounts) by placing specialty drugs in their own formulary tier. According to the Kaiser Family Foundation, the percentage of employer-sponsored plans using specialty tiers increased from 14% in 2012 to 23% in 2013. The new health insurance Marketplaces formed under the Affordable Care Act (ACA) also will have specialty tiers, but their effect may be mitigated by the ACA’s out-of-pocket maximum.

Critics contend that high cost sharing discourages appropriate use of medicines. To mitigate the cost to patients and encourage treatment, many pharmaceutical manufacturers offer assistance programs. These programs provide coupons to patients for some, or all, of the amount of cost sharing. In the case of Medicare, where such assistance is not allowed under federal law, some manufacturers provide grants to independent non-profit organizations, which are able to reimburse patients a portion of their share of the costs of medicines.

Payers and PBMs criticize assistance programs, concerned they reduce incentives embedded in traditional benefit designs and may lead to overuse. Manufacturers and patient advocates defend these discount programs, arguing that the cost-reduction incentives of traditional benefit design should not be applied to specialty products because of the small patient populations involved and the seriousness of the diseases they treat. They also contend that making these high-cost payments available to patients can help reduce non-adherence and its consequences, such as emergency department visits and hospitalizations.

In addition to high cost sharing, some payers are requiring diagnostic testing as a condition of coverage to identify individuals who demonstrate a propensity to respond to a particular specialty product. There also is support for treatments that patients can administer at home rather than at more expensive sites of care such as outpatient facilities or physicians’ offices.

2. Medical Benefit vs. Pharmacy Benefit
Because specialty drugs are often reimbursed under the medical benefit and their costs may be bundled in with other services, payers have less direct influence over pricing. Therefore, payers and PBMs are exploring ways to move specialty products from the medical benefit to the more transparent pharmacy benefit in order to better influence pricing, as well as gain information on use and outcomes.

Critics of this trend are concerned that it could harm care by reducing physicians’ role in helping patients use their medicines properly. In many cases, time is of the essence in starting a patient on a specialty drug treatment regimen. As a result, the amount of time and effort required for physicians, office staff, patients and caregivers to navigate pharmacy benefits’ utilization restrictions can be onerous. Administering specialty products is often a driver of physician office revenue. Critics contend that new administrative burdens imposed as part of a pharmacy benefit may undermine office practice efficiency and profit.

Distribution channels also offer an area for managing cost. For some products, payers are seeking to limit the type and number of pharmacies that can distribute a specialty pharmaceutical to patients in order to gain economies of scale and concentrate purchasing power. These limited networks may also be able to improve the ability of health plans to implement care protocols, improve adherence, avoid product waste, and implement FDA-required use plans. These efforts to limit who can distribute specialty pharmaceuticals often clash with the interests of retail pharmacist and physician business models built, in part, on the revenue from administering specialty drugs.

3. Biosimilars
The Affordable Care Act directed the FDA to create a new approval pathway to allow the sale of clinically-equivalent versions of biologically-derived therapies known as biologics. Patient advocates and payers hope that the introduction of these follow-on versions of biologics – known as biosimilars – will force prices down in the same way that generic drugs compete with traditional brand-name drugs. In the United States to date, there have been no medicines approved under this new authority, and many experts believe that, even once approved, biosimilars will offer cost savings but not of the magnitude seen with generics. Nevertheless, some are concerned that patients will be switched to biosimilars, with or without their knowledge, which may not achieve the same results as the original drugs.

4. Comparative Effectiveness
The growth in the use of comparative effectiveness review has created another tool to control the growth of specialty pharmaceutical costs. Proponents believe that comparative effectiveness review can provide information that will allow payers to deny coverage appropriately for products that cost more than alternatives but do not provide any additional clinical value. Opponents question whether such reviews, especially those conducted by payers, can be unbiased and whether these population-based analyses take into account that how individual patients respond to treatment varies.

Pressure from individual patients and patient advocates, as well as limited resources, has restricted the ability of comparative effectiveness review to alter coverage significantly for specialty pharmaceuticals. Such review is not currently allowed in Medicare Part B but may be considered by Medicare Part D drug plans and private health plans.

What’s Next?

Given that the growth of the specialty pharmaceutical market is expected to continue for many years, discussions about the right balance between cost containment and patient access will continue. These discussions will play out in private forums among payers and specialty pharmaceutical manufacturers. They also will spill over into public debates over the role of government in encouraging policies that will impact specialty pharmaceutical access and cost.

The Patient-Centered Outcomes Research Institute, created by the Affordable Care Act, could be a significant new source of funding for research on the effectiveness of specialty pharmaceuticals, although such studies are often costly and difficult. In addition, the Independent Payment Advisory Board (IPAB), also created by the ACA, has the potential to authorize changes in the Medicare program to reduce payments for specialty medicines. However, as of now, the law has not triggered IPAB’s authority to act.

Attention will focus on the ACA’s Marketplaces and the access to specialty pharmaceuticals in Marketplace health plans. Debates also will continue on questions that may impact the uptake of biosimilar products, once the FDA begins to approve them. Already state legislatures have begun to consider and enact laws to regulate biosimilars.

Finally, questions about the ability of specialty pharmaceutical manufacturers to assist patients in paying cost sharing through the use of coupons and other means will continue in private and public forums. Manatt Health will continue to monitor the debate and share the latest developments, as well as their implications.

Operationalizing the New Fast Track Enrollment Options: A Roadmap for State Officials

Authors: Jocelyn Guyer, Director, Manatt Health Solutions | Tanya Schwartz, Manager, Manatt Health Solutions

EDITOR’S NOTE: On May 17, 2013, the Centers for Medicare and Medicaid Services (CMS) issued guidance alerting states that waivers would be available to facilitate the enrollment of eligible individuals into Medicaid using data states already had available in their Supplemental Nutrition Assistance Program (SNAP) and Medicaid files. Manatt has prepared a “roadmap” for the Robert Wood Johnson Foundation State Health Reform Assistance Network, detailing the process for securing a fast track waiver from CMS and providing additional strategies states may want to pursue. Below is a summary of key points. Click here to download the full roadmap, including samples of enrollment forms. Manatt also prepared a related issue brief for the Henry J. Kaiser Family Foundation. Click here to download the issue brief.

The CMS guidance allowing waivers to facilitate enrollment into Medicaid using information states already have “on hand” in SNAP and existing Medicaid files offers several key advantages. States securing a fast track waiver can:

  • Efficiently and quickly enroll eligible individuals in coverage
  • Alleviate the administrative demands of new eligibility and enrollment systems for Medicaid and the Marketplaces
  • Reduce the staff time required to process Medicaid applications

With the fast track waiver, states expanding Medicaid to new adults with incomes up to 133% of the federal poverty level (FPL) have a powerful tool for streamlining the enrollment process.

How to Apply for a Fast Track Waiver

CMS has established a simple process for applying for fast track waivers. (States can apply any time through December 15.) The waiver request can consist of a short letter to CMS, accompanied by, if needed, clarifying emails or phone calls. States must provide at least the following information:

  • Why the state needs the waiver to improve its eligibility and enrollment system and meet is administrative obligations
  • How the state will secure a signature from fast track enrollees—physically, electronically or telephonically
  • How the state will obtain and verify missing information
  • How the state will ensure people receive information on their rights and responsibilities, third-party liability and medical support requirements
  • How the state can ensure it will evaluate fast track enrollees using the Modified Adjusted Gross Income (MAGI) rule within a year of enrollment—or earlier if there is a change in circumstances
  • What the duration of the waiver is

CMS has indicated it will provide states with sample waiver language, if needed. Once CMS approves the waiver, it will send a letter to the state confirming its eligibility.

Key Steps for Early Adopters to Operationalize Fast Track

States have broad discretion in operationalizing fast track enrollment. The basic steps states have used to date include:

Step 1: Identifying eligible individuals
In the basic version of fast track aimed at enrolling non-disabled, non-elderly adults, states have identified eligible individuals as those between 19 and 64 with incomes below 133% FPL who are not currently enrolled in Medicaid or receiving Supplemental Security Income (SSI) benefits. Variations to the basic approach have been to include children or focus exclusively on adults without children.

Step 2: Designing a fast track enrollment form
Fast track enrollment forms advise SNAP enrollees of their new opportunity to enroll in Medicaid. They also ensure that states have the information and authorization they need to establish eligibility for coverage. States that already have implemented fast track enrollment have included the following information on their forms:

  • The good news about the chance to enroll quickly and easily for coverage
  • The specific steps for enrolling, such as signing and returning the form by a specified date
  • A place for applicants to sign and date the form, provide contact information and, if needed, supply any missing information, such as verification of citizenship or immigration status
  • The date coverage begins
  • The applicant’s rights and responsibilities, as well as information about third-party liability and medical support requirements
  • “Next steps,” such as when the applicant will receive confirmation of coverage
  • A phone number people can call, if they have questions
  • A client ID number to help identify returned forms

States may choose to supplement or modify forms. Variations on the standard form have included asking if the consumer has alternative coverage; sending one form for all eligible family members, rather than a separate form for each individual; and informing applicants that any uninsured family members not listed on the form can apply for coverage online.

Step 3: Processing returned forms
Once a state receives fast track enrollment forms, it must verify citizenship and immigration status in accordance with Medicaid requirements. Verification steps depend on whether the fast track enrollee is identified through SNAP or Medicaid.

  • For SNAP beneficiaries: Since SNAP and Medicaid use different citizenship verification rules, states must confirm the citizenship of fast track enrollees according to Medicaid. Medicaid rules require electronic verification through the federal data hub using social security numbers or the Systematic Alien Verification for Entitlements (SAVE) program if the hub is down. In contrast, SNAP uses the same verification requirements as Medicaid for legal immigration status, so states don’t have to verify that information for SNAP fast track enrollees.
  • For parents of Medicaid children: States often lack citizenship and immigration status information for fast track enrollee parents using Medicaid data for their children. When that data is missing, states need to gather it and confirm eligibility, unless they already have it on file.

If a state can’t verify citizenship or legal immigration status, it must deny coverage to the fast track applicant.

Step 4: Confirming eligibility and providing follow-up information
After an individual’s citizenship or immigration status is verified, states send confirmation of eligibility for coverage and, if applicable, information on how to select a plan.

Step 5: Conducting a full MAGI determination
CMS expects states to evaluate fast track enrollees under MAGI rules within a year of their enrollment in Medicaid. If enrollees report a change in circumstance before their renewal date, their eligibility must be assessed under MAGI rules.

Implementation Steps Identified by Early Adopters

The four states already implementing fast track have identified some additional strategies to increase its effectiveness. Some extra steps that can help improve the process include:

  • Following up with non-responders using phone calls or other outreach methods
  • Conducting citizenship verification prior to sending fast track enrollment forms
  • Re-checking whether fast track enrollees already are enrolled in Medicaid to avoid duplicate enrollments
  • Using a centralized unit or specially trained team to process fast track applications
  • Integrating Affordable Care Act (ACA) marketing and logos to help consumers understand how fast track fits into broader reform efforts

Conclusion

CMS has given states significant flexibility in how they choose to implement fast track. State can tailor their fast track strategies to their needs, regardless of how they choose to operationalize the Medicaid expansion. For example, Arkansas combined its use of fast track with a private option under which it uses Medicaid funds to support beneficiaries in selecting a private Marketplace plan.

Manatt is continuing to monitor Medicaid developments. We will be sharing the latest trends in Medicaid next month.

The Right Mix: Congressional Compromise on Compounding Results in New Law

Author: Michelle McGovern, Associate, Healthcare Industry, Manatt, Phelps & Phillips, LLP

EDITOR’S NOTE: The Compounding Quality Act, requiring compounders to meet quality and sterility standards, has been signed into law but continues to draw mixed responses from stakeholders. In the December issue of Bloomberg BNA’s “Pharmaceutical Law and Industry Report,” Manatt’s Michelle McGovern examines the new law and its future implications. Below we summarize some key points. Click here to download the full Bloomberg BNA article.

After a year of public debate and a half-dozen failed proposals, the Compounding Quality Act (the Act) was signed into law on November 27, 2013.The new Act establishes federal oversight of and dictates quality standards for compounding pharmacies that choose to register as “outsourcing facilities.” It gives the Federal Food and Drug Administration (FDA) clear enforcement jurisdiction over registered compounding pharmacies-- facilities that prepare sterile or other high-risk drugs, and which have historically not been subject to regulation by the FDA.

One such pharmacy, the New England Compounding Center (NECC) located in Framingham, Massachusetts, prepared a tainted, compounded steroid that caused a spinal meningitis outbreak responsible for the deaths of 64 people and illnesses in more than 750 in the fall of 2012. That incident helped put the debate over compounding pharmacy regulation in the media spotlight.

The Act passed both the Senate and the House with bipartisan support. It represented a compromise, however, between those who believed that legislation was necessary to clarify the FDA’s role in regulating compounding facilities and those who felt the FDA already had the authority it needed under existing policy guidance. While the Act’s success in preventing future public health crises remains to be seen, many of its provisions provide a stronger framework for regulation than compounders have seen in decades.

What Is the Compounding Quality Act?

The Act standardizes the patchwork of compounding regulations that had historically been in place. It requires compounders to adhere to standards that ensure the quality and sterility of products. Under the Act, compounders may voluntarily register as “outsourcing facilities,” which would subject them to FDA requirements and enforcement actions. Compounders electing to register as outsourcing facilities will be subject to, among other things, restrictions relating to the quality of their products and to engaging in behaviors more traditionally associated with manufacturers.

For instance, outsourcing facilities will be prohibited from compounding certain drugs and drug products (such as drugs withdrawn or removed from the market for being unsafe or ineffective), preparing drugs that are essentially copies of FDA-approved products, and acting as wholesalers of compounded products.

Additionally, the law attempts to prevent public health crises like the spinal meningitis outbreak from occurring in the future. Outsourcing facilities will be required to report adverse events, helping the FDA address and contain public health issues quickly. Outsourcing facilities also will be subject to a risk-based inspection schedule, permitting the FDA to access their facilities and ensure their products are prepared in accordance with quality and safety requirements.

The law will require drug reporting, so the FDA and consumers can learn the types of drugs being prepared by compounders. It also will require labels on compounded products to alert providers and patients about the nature of the drugs being prescribed and administered. The Act leaves regulation of traditional compounders --those that prepare patient-specific products based on individualized prescriptions – to the states.

The Pros and Cons of Voluntary Registration

The voluntary registration provision of the Act –arguably the key driver of future enforcement action – is one of its more controversial provisions. Voluntary registration will provide the FDA with unprecedented information about compounders. Because registration is not mandatory, however, it is inevitable that some compounders will not register.

On one hand, the registration option will essentially provide an FDA “seal of approval” for certain pharmacies, encouraging providers to select their products. In addition, it is likely that the FDA will focus investigations on facilities that, for whatever reason, elected not to register.

Registration, however, may raise cost issues. Because registered facilities will have to comply with heightened quality and reporting standards, unregistered facilities will likely be able to sell compounded products for less money than registered facilities. While certain providers will insist on drugs prepared by outsourcing facilities, others may choose cheaper products, even if they are prepared by unregulated facilities. This is particularly true as more providers shift to capitated payment models and cost containment grows increasingly important.

Section 503A of the FDCA

In 1997, Congress enacted the Food and Drug Administration Modernization Act (FDAMA), which added compounding-specific provisions to the Food, Drug and Cosmetic Act (FDCA) in Section 503A. This section of the FDCA featured quality controls for compounded products but also included a prohibition on marketing compounded drugs. The marketing prohibition was struck down by the U.S. Supreme Court in 2002 as an impermissible restriction on free speech in the case of Thompson v. Western States Medical Center1. The Court did not decide on whether the non-speech provisions of the law could stand, however, and in the majority of the country, the entire law was considered invalid.

The Act resolves the constitutional question of the applicability of Section 503A of the FDCA to compounders by striking the speech-related provisions. Therefore, it gives the FDA unambiguous enforcement jurisdiction over compounders. This will allow the FDA to carry out investigations and bring enforcement actions with full regulatory authority. It also will make it more difficult for compounders to create procedural barriers to enforcement actions based on lack of federal enforcement jurisdiction. To be effective, however, this enforcement jurisdiction must be supported by sufficient funding.

Funding for Enforcement

The Act requires all registered facilities to pay an annual fee that will start at $15,000 in fiscal year 2015, adjusted for inflation. Facilities that require re-inspection in any fiscal year will have to pay a re-inspection fee of $15,000 in the relevant fiscal year. Although the inspection and re-inspection fees will help fund enforcement actions, it remains to be seen how many facilities will choose to register under the Act, and how much funding these registrations will generate.

If fees paid by outsourcing facilities fail to offset the cost of increased enforcement actions, congressional appropriations will be necessary. There is no guarantee however, that Congress will prioritize regulating outsourcing facilities in future national budgets.

Communication between State and Federal Regulators

Under the Act, the FDA will receive submissions from states concerned about compounding pharmacies that may be violating the requirements set forth in Section 503A of the FDCA, and descriptions of actions taken against compounding pharmacies. Increased federal-state communication can bolster enforcement actions by encouraging state pharmacy boards to target and report “bad actors” that may be violating Section 503A.

important to note, however, that state and federal regulators may only be communicating about “known” entities. Because the Act does not require all compounders to register with the FDA, the benefits of increased communication may not be fully realized.

An Uncertain – But Brighter – Future

How much progress the Act will make in curbing abusive compounding practices depends on a number of factors, including the content of the Act’s implementing regulations, the number and type of compounders that choose to register as “outsourcing facilities,” whether federal and state regulators will be able to identify “bad actors” that elect not to register, and availability of funding for enforcement. Although the true impact of the Act remains to be seen, its enactment alone is a significant sign of progress.

New Report Available: To Expand or Not to Expand Medicaid?

Through KidsWell, Manatt has released a new report analyzing states’ Medicaid expansion decisions. Titled To Expand or Not to Expand Medicaid?--An Analysis of State Medicaid Decision across the Country, the new report examines states’ decisions to expand their Medicaid programs under the ACA. It also provides a closer look at the thinking of governors and legislators in states that are not expanding. Content includes:

  • A 50-state map with a “snapshot” look at where states stand on their decisions to expand Medicaid, as well as projected enrollment figures as a result of expansion (The map is also downloadable on the KidsWell National Snapshots page. Click here to view.)
  • A chart detailing which states will expand Medicaid in 2014
  • Profiles of states that have not yet expanded Medicaid, including insights into how governors and legislators have influenced that decision.

For a copy of the full report, click here

What’s Next for Megatrends?

We received overwhelming interest in last month’s article on the 10 megatrends shaping the next 10 years of healthcare. (If you haven’t yet downloaded our full megatrends paper, click here.) Many of you wanted to delve deeper into these transformational trends causing a seismic shift in our healthcare landscape—particularly around the implications they hold for your organization. To guide your team in examining what’s driving these megatrends—and what they mean for your future—Manatt Health has created a half-day retreat we’ll hold with your executive team, including developing strategic action plans, customized to your opportunities and goals. To learn more, email Ilene Siegalovsky at isiegalovsky@manatt.com.

1. Thompson v. Western States Medical Center, 535 U.S. 357 (2002).

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