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Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
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Rare Diseases and Orphan Products: Accelerating Research and Development
C
Medicare Part D Coverage and Reimbursement of Orphan Drugs
Laura Faden and Haiden Huskamp*
INTRODUCTION
Given the small potential market for medications that treat rare conditions, pharmaceutical manufacturers may have reduced incentives to develop new medications for rare diseases. To increase incentives for manufacturers, the Orphan Drug Act (P.L. 97-414) provides the following provisions for drugs that receive an orphan drug indication1 from the U.S. Food and Drug Administration (FDA): a 7-year period of market exclusivity, a tax credit of 50 percent of the cost of conducting clinical trials, eligibility for federal research grants, and a waiver of user fees (21 USC 360bb, OIG, 2001). However, health plan coverage and reimbursement also influence a pharmaceutical firm’s decisions to invest in the development of a drug or biologic for a rare disease.
The purpose of this report is to examine stand-alone Medicare Part D prescription drug plan (PDP) coverage of a set of drugs and biologics that
*
Laura Faden., M.P.H., is a doctoral student in the Harvard University Program in Health Policy. Haiden Huskamp, Ph.D., is Professor of Health Care Policy in the Department of Health Care Policy at Harvard Medical School. Responsibility for the content of this paper rests with the author and does not necessarily represent the views of the Institute of Medicine or its committees and convening bodies.
1
The Orphan Drug Act of 1983 defines an orphan indication as follows: “in the case of a drug, any disease or conditions which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug” (21 USC 360bb).
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treat rare diseases or conditions—which we will refer to collectively as “orphan drugs”—in the Medicare population. This report does not address Medicare coverage and reimbursement of medical devices.
We focus on the Medicare population because the program covers approximately 15 percent of the U.S. population, including adults who have a disabling rare condition and who have Medicare coverage based on their qualification for Social Security Disability Insurance (SSDI). Furthermore, data on Medicare prescription drug coverage and reimbursement are more readily available than similar data from Medicaid and private plans—because Medicare is a public, federal program, the data are public and centrally collected.
There have been no comprehensive studies of Medicare PDP coverage and reimbursement of orphan drugs. In 2005, the National Organization for Rare Disorders (NORD) conducted a similar study that examined coverage of orphan drugs in 10 national Medicare Part D plans (NORD, 2006). This report extends the NORD report by including drugs approved since 2005 and by analyzing coverage of all Medicare prescription drug plans. Furthermore, this report goes beyond the NORD analysis, which only analyzed plan coverage, by also analyzing factors that may reduce access to covered drugs (i.e., formulary tier placement, utilization management).2
Medicare Beneficiaries
Medicare, which was created by the Social Security Act of 1965, is a federally administered health insurance program for people who are 65 years of age or older or who qualify for SSDI. There is typically a two-year waiting period before people who qualify for SSDI can receive Medicare benefits. Congress has waived that requirement for people with end-stage renal disease or Lou Gehrig’s disease.
As of January 2010, Medicare covers 46 million Americans, 17 percent of whom are under 65 years and are permanently disabled (KFF, 2010c). Almost half (47 percent) of Medicare beneficiaries have low income (below 200 percent poverty), and 7 million beneficiaries meet income and asset criteria to quality for Medicaid—these beneficiaries are known as “dual-eligibles.” Among the Medicare population, there is a high prevalence of comorbid conditions (44 percent suffer from three or more chronic conditions), and 29 percent have a cognitive or mental impairment (KFF, 2010c).
It is not known how many Medicare beneficiaries have a rare disease or, conversely, what proportion of people with a rare disease is covered by Medicare. However, the Social Security Compassionate Allowances program—
2
Although the NORD report notes the tier placement and utilization management tools used for each drug, the authors do not provide any analysis of these aspects of drug coverage.
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which guarantees immediate SSDI benefits for people who suffer from certain conditions—covers many rare diseases (Social Security Online, 2010).
Currently, more than 27 million Medicare beneficiaries are enrolled in a Medicare prescription drug plan, two-thirds of whom are enrolled in a stand-alone PDP (KFF, 2009d, 2010b).3 In 2009, 36 percent of these beneficiaries received low-income subsidies (LIS) that cover their premiums and deductibles; LIS beneficiaries are responsible only for a small copayment that is determined by their income level (KFF, 2009a). Dual-eligibles and those eligible for Supplemental Security Income cash assistance are automatically eligible for LIS, and other low-income beneficiaries can apply for the subsidies. All LIS beneficiaries are enrolled in plans that have monthly premiums below the benchmark premium amount established for each region (hereafter referred to as “benchmark plans”).
Medicare Prescription Drug Plans
Until 2006, Medicare did not cover outpatient prescription drugs. It covered hospital and physician services (Part A and B), which included coverage of inpatient drugs and drugs administered by a physician (e.g., infusions). The Balanced Budget Act of 1997 created an option for Medicare beneficiaries to receive insurance coverage from private health plans that contract with Medicare (Part C)—these plans are currently referred to as “Medicare Advantage Plans.” The Medicare Prescription Drug Improvement and Modernization Act of 2003 created the Medicare Part D program, a voluntary drug benefit that is administered through private health plans or pharmaceutical benefit managers. As of January 1, 2006, Medicare beneficiaries could voluntarily enroll in either a stand-alone PDP or a Medicare Advantage plan with prescription drug coverage (MA-PD). Dual-eligibles are automatically enrolled in a benchmark plan.
The legislation does not require PDPs to have uniform cost sharing requirements or formulary design. However, PDPs are required to offer a plan that is at least actuarially equivalent to a standard benefit package as determined by the Centers for Medicare and Medicaid Services (CMS) (CMS, 2010). In 2010, the standard benefit package is
$310 deductible;
25 percent coinsurance up to $2,830 of total drug costs;
3
Of the remaining Medicare beneficiaries not covered by a Part D plan—approximately 19 million—most have drug coverage, either through a retiree drug plan (through an employer or union) or another form of drug coverage (e.g., Veterans Affairs, Indian Health Services, state pharmacy assistance programs, employer benefits for active workers, etc.). Approximately 10 percent of beneficiaries have no prescription drug coverage (KFF, 2009d, 2010b).
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no coverage from $2,830 to $6,330 of total drug costs—a coverage gap that is commonly referred to as the “doughnut hole,” which, starting in 2010, will be partially subsidized (beneficiaries will receive a $250 rebate);4 and
5 percent coinsurance, or a flat copayment of $2.50 for a generic drug and $6.30 for a brand drug, above $4,550 out-of-pocket expenses (i.e., catastrophic out-of-pocket spending limit) with no maximum limit on out-of-pocket expenses.
In addition, CMS requires that PDP and MA-PD formularies include at least two drugs in every drug class5 and all, or substantially all, drugs in the following six “protected” therapeutic categories: antidepressants; antipsychotics; anticonvulsants; immunosuppressants (to prevent rejection of organ transplants); antiretrovirals (for the treatment of infection by retroviruses, primarily HIV); and antineoplastics (only those chemotherapy drugs that are generally are not covered under Medicare Part B) (CMS, 2010).
Even with these requirements, PDPs and MA-PDs have a considerable amount of flexibility in formulary design. First, plans decide whether or not to cover a drug. Second, plans can use a tiered formulary structure to create financial incentives for beneficiaries to choose lower-cost or preferred drugs. In 2010, approximately three-fifths of plans have the following fourtier structure (KFF, 2009c).
Tier 1: generic drugs
Tier 2: preferred brand-name drugs
Tier 3: nonpreferred brand-name drugs
Tier 4 (“specialty tier”): specialty drugs6
Each tier has a different cost sharing requirement—most plans assign a flat copayment to the first three tiers and a coinsurance to the specialty tiers (although a growing number of plans are requiring a coinsurance for the first three tiers) (KFF, 2009c). Almost all (94 percent) of plans have a
4
The Patient Protection and Affordable Care Act of 2010 (P.L. 111-148) included provisions to reduce cost sharing in the doughnut hole. Starting in 2011, Medicare and manufacturers will phase in subsidies for generic and brand drugs with the goal of reducing out-of-pocket expenditure in the doughnut hole in 2010 to the same 25 percent coinsurance that applies to costs below the lower threshold of the coverage gap.
5
The U.S. Pharmacopeia has developed a therapeutic classification system that serves as a guideline for Part D formularies. Model guidelines are publicly available: see http://www.usp.org/pdf/EN/mmg/modelGuidelinesV4.0WithFKDTs.pdf.
6
CMS guidelines stipulate that drugs placed on the specialty tier must cost at least $600 per month and prohibit enrollees from requesting cost sharing exceptions for specialty drugs (CMS, 2009b).
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specialty tier (KFF, 2009c).7 In 2010, the specialty tier coinsurance ranges from 25 to 33 percent of the full cost of the drug (KFF, 2009c).
In addition to coverage decisions and tiered formularies, a third way in which plans influence prescription drug utilization is by employing utilization management tools such as prior authorization (PA) requirements, step therapy (ST) requirements, and quantity limits (QL). PA requirements create an administrative barrier to accessing a drug—a patient or provider must follow a certain procedure, created by the plan, to request coverage of the drug and then await the plan’s approval of coverage. ST requirements establish a chronological course of recommended treatments for a condition that must be tried before coverage of the drug is approved. QL requirements set explicit criteria for the quantity of a drug that will be covered during a given period of time.
METHODS
Medicare Part D Plans Characteristics and Orphan Drug Coverage
We used the CMS Formulary and Pharmacy Network Information File (January 2010 quarterly release) to determine the coverage, tier placement, and utilization management requirements for each orphan drug. We classified drug plans as stand-alone drug plans (PDPs) and MA-PD plans.8 Within PDPs, we identified national plans (i.e., plans offered in every region of the country) and benchmark plans. National plans were identified by contract number (CMS, 2009b), and benchmark plans were identified using the regional premium limits (CMS, 2009a).
We calculated the “plan coverage rate” for a particular drug as the percentage of plans that cover the drug. Similar to the NORD report (NORD, 2006), we categorized each drug by level of coverage rate, which we classified as the following:
No or low coverage: <25 percent plan coverage rate
Low coverage: 25-49 percent plan coverage rate
Medium coverage: 50-74 percent plan coverage rate
High coverage: 75-99 percent plan coverage rate
Complete coverage: 100 percent plan coverage rate
We also examined tier placement and utilization management among drugs covered by PDPs. For each drug we calculated the “tier placement
7
This excludes plans that offer the standard benefit package (i.e., plans that have no tiering).
8
MA-PDs include both regional and local plans. However, for the analyses we removed duplicate MA-PDs by including each unique contract and plan combination only once.
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rate” as the percentage of plans that cover that drug on a given tier. For example, if 20 percent of the plans that cover a drug have placed that drug on tier 4, the drug has a tier 4 placement rate of 20 percent. The tiers range from one to four. A few plans have more than four tiers—for these plans we included all tiers greater than four in the tier 4 category.9
Similarly, we examined rates of step therapy requirements, quantity limits, and prior authorization requirements (“utilization management rate”).10 We categorized each covered drug by the rate of tier placement and use of utilization management, which we classified into the following categories:
No or low placement (or use): <25 percent of plans
Low placement (or use): 25-49 percent of plans
Medium placement (or use): 50-74 percent of plans
High placement (or use): 75-99 percent of plans
Complete placement (or use): 100 percent of plans
In terms of beneficiary access to drugs, a higher plan coverage rate will likely improve access, whereas a higher utilization management or tier 4 placement rate may pose barriers to access. These categories are subjective and were created only to simplify the interpretation of the results for all drugs along the three dimensions of access (i.e., plan coverage, tier placement, and utilization management). Therefore we also report the raw rates.
9
For the purposes of this report, we refer to tiers 4-6 as “tier 4” and assume that tier 4 is equivalent to a specialty tier. However, given the heterogeneity of the PDPs’ formulary structures, this assumption does not hold for all plans. As previously noted, three-fifths of the plans have a four tier structure—for these plans tier 4 is the specialty tier (CMS, 2009b). Some plans have no tiers (11 percent) or fewer than four tiers (7 percent)—for the latter, the specialty tier may actually be tier 3. Also, 21 percent of the plans have more than four tiers—for these plans, tier 4 may not be a specialty tier but rather a nonpreferred brand tier (CMS, 2009b) or a tier for injectible drugs (KFF, 2010a). Therefore, by collapsing tiers 4-6 and labeling this as a specialty tier, we may be misclassifying up to 28 percent of the plans. However, this misclassification may have a negligible effect in terms of concerns about beneficiary cost sharing because a large share (34 percent) of PDPs now use coinsurance rates for nonpreferred brand tiers (CMS, 2009b) and these coinsurance rates may actually be higher than specialty-tier coinsurance rates. Likewise, cost sharing for drugs placed in an injectible tier is also likely to be high because these drugs are quite expensive.
10
Note that for these analyses, the rates are based on only the number of plans that cover each drug (i.e., the denominator is different for each drug). Since each drug is associated with multiple entries in the National Drug Code (NDC) directory, a drug may appear on multiple tiers of a plan’s formulary (e.g., both the brand and generic version are covered, but the generic is on lower tier). For the purposes of our analyses, we assign drugs to the lowest tier on which they appear. A plan is counted as having a utilization management tool for a drug if the tool is applied to at least one of the covered NDCs associated with the drug.
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To determine if coverage policies differed by type of plan, we repeated these analyses for several subgroups of plans: all PDPs, benchmark PDPs, national PDPs and MA-PDs. Although we present data on all analyses, we focus on the analyses of all PDPs in the results and discussion sections.
List of Orphan Drugs
We created a list of drugs that were approved by the FDA with an orphan indication between 1983 and December 2008. We included only drugs approved prior to 2009 in order to provide adequate time for marketing and plan coverage decisions. We included only outpatient drugs (i.e., not covered by Medicare Part B as of July 2010) that have an approved orphan indication relevant to the Medicare population (i.e., not for a pediatric indication11) and that have not been discontinued or withdrawn.
Our list excludes drugs that are covered by Medicare Part A or B and blood products. We also excluded drugs that are available on the market (for other FDA-approved indications) and that have been granted an orphan designation but have not yet received FDA approval for an orphan indication. Lastly, we excluded drugs for the treatment of rare diseases or conditions that appear in Medicare compendia unless the FDA has approved the drugs for the same orphan indication (see Chapter 6 of this report).
The National Drug Codes (NDCs) for each drug were obtained from First Data Bank (FDB), which was up-to-date as of January 2010. We noted which drugs are available in generic form and which are biologics versus new chemical entities.
RESULTS
List of Orphan Drugs
Ninety-nine orphan drugs met our inclusion criteria (see Addendum Table C-A1). Drugs are listed in chronological order of the date of approval of the first orphan indication relevant to the Medicare population (some drugs have multiple relevant orphan indications). Twenty-nine (29 percent) of the drugs are available in generic form and eleven (11 percent) are biologics.
Medicare Plan Characteristics—Part D and Medicare Advantage Plans
In 2010, there are 1,620 stand-alone PDPs and 2,418 MA-PDs. Of the PDPs, 1,295 (77 percent) are national plans and 398 (23 percent) are
11
We performed a separate analysis of Medicare Part D coverage of orphan drugs with only a pediatric indication orphan approval. See Addendum Tables C-A3 and C-A4.
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benchmark plans. The 1,295 national plans represent 12 plan sponsor organizations, 26 unique contracts, and 88 unique formularies (a sponsor may use the same formulary for multiple plans).
The average monthly premium is $46.39 (range: $1.50 to $120.20; standard deviation: $19.75) (see Table C-1). More than half (60 percent) of the plans have deductibles. The median deductible for all plan types is $310.00 (range: $10.00 to $310.00). Benchmark plans and nonnational plans are more likely to have a deductible and to have a higher deductible than nonbenchmark and national plans. Compared to stand-alone PDPs, MA-PDs have a lower average premium and are less likely to have a deductible.
Medicare Plans’ Coverage of Orphan Drugs— Stand-Alone PDPs and MA-PDs
The coverage rate (percentage of plans covering a drug) for orphan drugs among Medicare prescription drug plans is high. On average, an orphan drug is covered by 84 percent (standard deviation: 24 percent) of stand-alone PDPs. Table C-2 shows a breakdown of coverage rate category by plan type. Of the 99 drugs, 44 (44 percent) are covered by all 1,620 PDPs (i.e., complete coverage category). An additional 29 drugs (29 percent) are covered by at least 75 percent of the plans (i.e., high-coverage category).
Table C-2 shows that 19 (19 percent) of the drugs fall into the medium-coverage category (i.e., only covered by 50-75 percent of plans) and that 7 (7 percent) are covered by less than half of the plans (i.e., no or very low coverage and low-coverage categories). As of January 2010 4 drugs are not covered by any PDP: citric acid, glucono-delta-lactone, and magnesium car-
TABLE C-1 Average Premium and Use of Deductible for Different Types of Medicare Prescription Drug Plans (99 drugs)
N
Average Premium (std. dev.)
% with Deductiblea
All stand-alone PDPs
1,620
46.39 (19.75)
60
Benchmark PDPs
398
28.70 (5.69)
94
Nonbenchmark PDPs
1,222
52.15 (19.27)
49
National PDPs
1,295
46.70 (20.14)
57
Nonnational PDPs
325
45.15 (18.07)
72
MA-PDs
2,418
20.12 (18.81)
23
a The median deductible across all plan types is $310.
NOTE: 2010 Data.
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TABLE C-2 Orphan Drug Coverage by Type of Medicare Prescription Drug Plan (99 drugs)
All Stand-alone PDPs (N)
MA-PDPs (N)
Stand-alone National PDPs (N)
Stand-alone Non-national PDPs (N)
Stand-alone Benchmark PDPs (N)
Stand-alone Non-benchmark PDPs (N)
No or very low coverage (<25% plan coverage rate)
4
4
4
10
4
4
Low coverage (25-49% plan coverage rate)
3
0
0
13
7
2
Medium coverage (50-74% plan coverage rate)
19
17
19
8
15
19
High coverage (75-99% plan coverage rate)
29
36
19
25
24
29
Complete coverage (100% plan coverage rate)
44
42
57
43
49
45
NOTE: Number of drugs that fall into each coverage rate category. Because the number of drugs in the analysis is 99, the numbers and percentages of drugs are identical; the percentages have therefore not been included in the table.
bonate (Renacidin Irrigation); clofazimine (Lamprene); glutamine (Nutrestore); zinc acetate (Galzin). Three other drugs—lodoxamide tromethamine (Alomide Ophthalmic Solution), tinidazole (Tindamax), and metronidazole topical (Metrogel)—are covered by 45 to 50 percent of PDPs (see Table C-3). As explained in the note for the table, a search of formularies conducted in late spring 2010 found a few plans had initiated coverage of Galzin and Renacidin Irrigation.
Overall, MA-PD plans have slightly better coverage of orphan drugs than stand-alone PDPs. Compared to PDPs, the percentage of drugs falling into the no-very low and low-coverage categories is slightly lower among MA-PDs (4 percent in MA-PDPs versus 7 percent in PDPs). On average, an orphan drug is covered by 87 percent (standard deviation: 22 percent) of MA-PDPs, compared to 84 percent of stand-alone PDPs.
Within PDPs, there is some variation in coverage rates between benchmark and nonbenchmark plans, with nonbenchmark plans having slightly higher coverage rates. On average, an orphan drug is covered by 83 percent (standard deviation: 26 percent) of benchmark plans and 85 percent (standard deviation: 24 percent) of nonbenchmark plans. The benchmark plans have a higher percentage of drugs that fall within the no-very low
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TABLE C-3 Orphan Drugs with No, Very Low, or Low Plan Coverage (less than 50% coverage among all standalone PDPs) (7 drugs)
Generic Name
Trade Name
Stand-alone PDP Coverage (% plans that cover drug)
Route of Administration
Orphan Designation(s) (year of orphan approval)
Citric acid, glucono-deltalactone, and magnesium carbonate
Renacidin Irrigation
0a
Irrigation
Treatment of renal and bladder calculi of the apatite or struvite variety (1990)
Clofazimine
Lamprene
0
Oral
Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum (1986)
Glutamine
Nutrestore
0
Oral
For use with human growth hormone in the treatment of short bowel syndrome (nutrient malabsorption from the gastrointestinal tract resulting from an inadequate absorptive surface) (2004)
Zinc acetate
Galzin
0a
Oral
Treatment of Wilson’s disease (1997)
Lodoxamide tromethamine
Alomide Ophthalmic Solution
46
Ophthalmic
Treatment of vernal keratoconjunctivitis (1993)
Tinidazole
Tindamax
47
Oral
(1) Treatment of giardiasis; (2) treatment of amebiasis (2004)
Metronidazole (topical)
Metrogel
49
Topical
Treatment of acne rosacea (1988)
a These drugs had 0% coverage according to our analysis, which was limited to coverage of the drugs’ NDCs (listed in the FDB) in the January determined that Renacidin and Galzin are in fact covered by some PDPs as of June 2010. Galzin, which was a “high priority access problem drug” in the NORD analysis, is not on any national PDP formularies but appears on at least two nonnational formularies. Renacidin also appears on two national formularies and a few nonnational PDP formularies.
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and low-coverage categories (11 percent in benchmark versus 6 percent in nonbenchmark plans).
There is considerably more variation in coverage rates between national and nonnational plans, with national plans having higher coverage rates. On average, an orphan drug is covered by only 77 percent (standard deviation: 32 percent) of nonnational plans, compared to 86 percent (standard deviation: 24 percent) of national plans. Within nonnational plans, almost a quarter (23 percent) of the drugs are classified as having a no-very low or low-coverage rate, compared to only 4 percent of drugs within national plans. Aside from the four drugs covered by no PDPs, no other drug is covered by less than 50 percent of the national plans. Conversely, 19 of the 95 covered drugs are covered by less than 50 percent of the nonnational plans.
Formulary Tier Placement by Stand-Alone PDPs
The orphan drugs are commonly placed on high cost sharing tiers. Table C-4 shows the tier 4 placement rate by plan type. For these analyses, and the utilization management analyses below, we excluded the four drugs not covered by any plan. Of the 95 remaining drugs, 84 (88 percent) are placed on tier 4 or higher by at least one PDP. Twenty-eight (29 percent) are placed on tier 4 by at least 75 percent of the plans (i.e., high tier 4 placement), and another 15 (16 percent) are placed on tier 4 by at least 50 percent of the plans (i.e., medium tier 4 placement).
Utilization Management Tools Used by Stand-Alone PDPs
PDPs rarely use step therapy to manage orphan drugs. ST is used by at least one plan only for 18 (19 percent) of the covered orphan drugs. Of these 18 drugs, half (9) have a ST use rate of less than 10 percent. The drug with the highest use of ST—interferon beta-1b—is given ST requirements by almost one-quarter (23 percent) of PDPs. The use of quantity limits to manage utilization of orphan drugs is more common among PDPs than the use of ST, although most plans do not use quantity limits for these drugs. QLs are used by at least one plan for 57 (60 percent) of the covered drugs. Twenty-seven (28 percent) of these drugs have QL use rates greater than 20 percent, and an additional 4 drugs (4 percent) have QL use rates greater than 50 percent (interferon beta-1a, lidocaine patch, raloxifene, and modafinil).
Prior authorization is the most widely used form of utilization management employed by PDPs. Table C-5 shows PA rates by plan type. PA is used by at least one plan for 80 (84 percent) of the covered orphan drugs. Thirty-three (35 percent) of the drugs are given a PA requirement by at least
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Generic Name
Trade Name
% Plans That Cover Drug
Eltrombopag
Promacta
90.5
Cysteamine
Cystagon
91.9
Midodrine HCl
Amatine
92.3
Modafinil
Provigil
93.5
Trientine HCl
Syprine
94.0
Anagrelide
Agrylin
95.6
Riluzole
Rilutek
95.7
Mefloquine HCl
Lariam
95.8
Ambrisentan
Letairis
98.3
Tiopronin
Thiola
99.4
Betaine
Cystadane
99.8
Pegvisomant
Somavert
99.9
Tizanidine HCl
Zanaflex
99.9
Cromolyn sodium 4% ophthalmic solution
Opticrom 4% ophthalmic solution
99.9
Deferasirox
Exjade
99.9
Ofloxacin
Ocuflox ophthalmic solution
99.9
Alitretinoin
Panretin
100.0
Altretamine
Hexalen
100.0
Aminosalicylic acid
Paser granules
100.0
Amiodarone HCl
Cordarone
100.0
Bexarotene
Targretin
100.0
Bosentan
Tracleer
100.0
Calcium acetate
Phos-lo
100.0
Cinacalcet
Sensipar
100.0
Dasatinib
Sprycel
100.0
Desmopressin acetate
N/A
100.0
Exemestane
Aromasin
100.0
Glatiramer acetate
Copaxone
100.0
Hydroxyurea
Droxia
100.0
Imatinib mesylate
Gleevec
100.0
Infliximab
Remicade
100.0
Interferon beta-1b
Betaseron
100.0
Lamotrigine
Lamictal
100.0
Lenalidomide
Revlimid
100.0
Lidocaine patch 5
Lidoderm patch
100.0
Megestrol acetate
Megace
100.0
Miglustat
Zavesca
100.0
Naltrexone HCl
Revia
100.0
Nilotinib
Tasigna
100.0
Nitisinone
Orfadin
100.0
Pilocarpine
Salagen
100.0
Potassium citrate
Urocit-K
100.0
Raloxifene
Evista
100.0
Rifabutin
Mycobutin
100.0
Rufinamide
Banzel
100.0
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% Plans That Place on Tier 3
% Plans That Place on Tier 4
% Plans That Have ST
% Plans That Have QL
% Plans That Have PA
19.3
78.0
0.0
46.0
88.7
51.0
12.0
0.0
0.0
16.1
3.5
0.0
0.0
11.5
0.2
48.8
6.9
0.2
74.3
98.5
49.2
11.5
0.0
0.0
0.0
0.1
0.0
0.0
0.1
11.5
11.5
65.9
0.0
11.1
29.1
0.0
0.0
0.0
11.2
0.2
15.5
79.8
10.8
33.6
50.7
48.2
12.5
0.0
0.0
0.0
52.0
11.0
0.0
0.0
15.0
13.7
68.9
6.3
30.2
85.9
4.3
0.0
0.0
0.1
0.1
0.0
0.0
0.0
0.9
0.2
15.3
79.3
0.0
0.0
51.2
2.1
0.0
0.0
5.3
0.2
16.2
57.6
0.0
4.2
10.8
19.3
74.6
0.0
0.0
28.6
45.5
25.6
0.0
0.0
4.4
0.0
0.0
0.0
0.0
5.1
14.0
68.0
0.0
21.5
55.2
15.0
80.0
10.6
29.8
62.4
4.3
2.2
0.0
0.0
0.2
27.9
2.2
10.5
39.8
28.6
15.2
79.7
10.7
40.9
55.4
6.3
0.0
1.4
9.3
0.1
40.4
16.9
10.7
21.1
0.0
13.0
82.4
0.0
43.8
91.7
0.0
0.0
0.0
0.0
0.2
15.2
82.3
0.0
28.8
69.0
12.6
82.5
1.1
0.0
94.4
15.3
82.4
22.6
43.0
92.1
4.2
0.0
13.8
30.6
33.5
15.0
80.2
0.0
34.6
69.9
33.6
8.4
6.3
56.4
45.7
2.1
0.0
0.0
21.3
7.4
15.9
62.4
0.0
6.7
31.1
0.0
0.0
0.0
0.0
0.2
15.2
80.0
10.7
38.7
53.2
15.6
75.3
0.0
0.0
26.4
3.1
0.0
0.0
0.0
0.2
0.0
0.0
0.0
0.0
0.2
21.1
2.2
0.0
63.8
0.0
38.5
12.4
0.0
0.0
0.0
45.7
25.6
0.0
48.3
40.9
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Generic Name
Trade Name
% Plans That Cover Drug
Sacrosidase
Sucraid
100.0
Selegiline HCl
Eldepryl
100.0
Sodium phenylbutyrate
Buphenyl
100.0
Sorafenib
Nexavar
100.0
Sotalol HCl
Betapace
100.0
Sulfadiazine
N/A
100.0
Tacrolimus
Prograf
100.0
Thalidomide
Thalomid
100.0
Topiramate
Topamax
100.0
Toremifene
Fareston
100.0
Tranexamic acid
Cyklokapron
100.0
Tretinoin
Vesanoid
100.0
Ursodiol
Urso
100.0
Vorinostat
Zolinza
100.0
Zidovudine
Retrovir
100.0
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% Plans That Place on Tier 3
% Plans That Place on Tier 4
% Plans That Have ST
% Plans That Have QL
% Plans That Have PA
20.5
66.6
0.0
0.0
21.9
0.0
0.0
0.3
0.1
0.2
16.1
61.7
0.0
0.0
19.3
12.9
79.9
0.0
41.2
83.1
0.0
0.0
0.0
0.0
0.2
1.4
4.3
0.0
0.0
0.0
39.9
18.6
0.0
7.6
99.8
10.2
79.8
0.0
33.5
71.4
11.6
2.1
0.1
38.8
13.7
48.3
16.6
0.0
16.9
4.1
17.0
11.8
0.0
0.0
24.0
8.2
50.5
0.0
0.0
31.9
2.1
0.0
0.0
0.0
0.2
15.3
80.0
0.0
34.5
64.1
2.1
0.0
0.0
0.2
0.0
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TABLE C-A3 Drugs with a Pediatric Orphan Indication (1983-2008 Approvals) (27 drugs)
Exclusivity Start Date
Generic Name
Trade Name
Indication for Original Approval
10/17/85
Somatropin
Nutropin
For use in the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion
10/17/85
Somatrem for injection
Protropin
For long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion
3/8/87
Somatropin for injection
Humatrope
For the long-term treatment of children who have growth failure due to inadequate secretion of normal endogenous growth hormone
8/2/90
Colfosceril palmitate, cetyl alcohol, tyloxapol
Exosurf neonatal for intratracheal suspension
Treatment of established hyaline membrane disease at all gestational ages
1/30/91
Succimer
Chemet capsules
Treatment of lead poisoning in children
7/1/91
Beractant
Survanta intratracheal suspension
(1) Prevention of RDS (hyaline membrane disease) in premature infants less than 1250 grams birth weight or with evidence of surfactant deficiency. (2) Treatment of (“rescue”) premature infants with RDS confirmed by x-ray and requiring mechanical ventilation.
12/24/91
Histrelin acetate
Supprelin injection
Treatment of central precocious puberty
2/26/92
Nafarelin acetate
Synarel nasal solution
Treatment of central precocious puberty
7/14/92
Teniposide
Vumon for injection
Induction therapy in patients with refractory childhood acute lymphoblastic leukemia
4/16/93
Leuprolide acetate
Lupron injection
Treatment of children with central precocious puberty
7/29/93
Felbamate
Felbatol
As adjunctive therapy in the treatment of partial and generalized seizures associated with the Lennox-Gastaut syndrome in children
12/27/93
Immune globulin intravenous, human
Gamimune N
Infection prophylaxis in pediatric patients affected with the human immunodeficiency virus
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Exclusivity Start Date
Generic Name
Trade Name
Indication for Original Approval
1/18/96
Respiratory syncytial virus immune globulin (human)
Respigam
Prophylaxis of respiratory syncytial virus (RSV) lower respiratory tract infections in infants and young children at high risk of RSV disease
9/26/97
Sermorelin acetate
Geref
Treatment of idiopathic or organic growth hormone deficiency in children with growth failure
5/27/99
Etanercept
Enbrel
Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying antirheumatic drugs
9/21/99
Caffeine
Cafcit
Short-term treatment of apnea of prematurity in infants between 28 and less than 33 weeks gestational age
6/20/00
Somatropin (r-DNA)
Genotropin
Long-term treatment of pediatric patients who have growth failure due to Prader-Willi syndrome (PWS)
7/12/02
Rasburicase
Elitek
Treatment of malignancy-associated or chemotherapy-induced hyperuricemia
7/29/03
Ribavirin
Rebetol
Treatment of chronic hepatitis C among previously untreated pediatric patients at least 3 years of age or older
10/23/03
Botulism immune globulin
Babybig
Indicated for treatment of infant botulism caused by type A or type B Clostridium botulinum
12/28/04
Clofarabine
Clolar
Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens
8/11/05
Meloxicam
Mobic
For relief of the signs and symptoms of pauciarticular or polyarticular course juvenile rheumatoid arthritis in patients 2 years of age or older
8/30/05
Mecasermin
Increlex
Long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH
12/12/05
Mecasermin rinfabate
Iplex
Treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH
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Exclusivity Start Date
Generic Name
Trade Name
Indication for Original Approval
4/13/06
Ibuprofen lysine
Neoprofen
For closure of a clinically significant patent ductus arteriosus in premature infants weighing between 500 and 1500 g, who are no more than 32 weeks gestational age when usual medical management (e.g., fluid restriction, diuretics, respiratory support) is ineffective
12/20/06
Balsalazide disodium
Colazal
Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older
2/21/08
Adalimumab
Humira
Treatment of juvenile rheumatoid arthritis
NOTE: This list includes drugs that received approval only for a pediatric orphan indication. Remicade, which received a pediatric orphan approval for the treatment of pediatric Crohn’s disease, is also approved for an adult orphan indication and was therefore included in a previous list of drugs. The drugs are sorted by the exclusivity date (i.e., date of approval of orphan indication) for first orphan approval with a relevant indication. Drugs with multiple orphan designations often have different exclusivity dates associated with each approved indication. The text for some indications has been abbreviated.
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TABLE C-A4 Medicare Stand-Alone PDP Coverage for Drugs with a Pediatric Orphan Indication: Inclusion on Formulary (i.e., Plan Coverage), Tier Placement, and Utilization Management (27 drugs)
Generic Name
Trade Name
No. of Plans That Cover Drug
% Plans That Cover Drug
% - Plans That Place on Tier 3
% Plans That Place on Tier 4
% Plans That Have ST
% Plans That Have QL
% Plans That Have PA
Beractant
Survanta intratracheal suspension
0
0.0
—
—
—
—
—
Botulism immune globulin
Babybig
0
0.0
—
—
—
—
—
Caffeine
Cafcit
0
0.0
—
—
—
—
Colfosceril palmitate, cetyl alcohol, tyloxapol
Exosurf neonatal for intratracheal suspension
0
0.0
—
—
—
—
—
Histrelin acetate
Supprelin injection
0
0.0
—
—
—
—
—
Ibuprofen lysine
Neoprofen
0
0.0
—
—
—
—
—
Immune globulin intravenous, human
Gamimune n
0
0.0
—
—
—
—
—
Mecasermin rinfabate
Iplex
0
0.0
—
—
—
—
—
Respiratory syncytial virusimmune globulin (human)
Respigam
0
0.0
—
—
—
—
—
Sermorelin acetate
Geref
0
0.0
—
—
—
—
—
Somatrem for injection
Protropin
0
0.0
—
—
—
—
—
Succimer
Chemet capsules
0
0.0
—
—
—
—
—
Teniposide
Vumon for injection
0
0.0
—
—
—
—
—
Somatropin [r-DNA]
Genotropin
622
38.4
57.6
25.6
0.0
27.8
99.5
Somatropin
Nutropin
625
38.6
16.6
77.6
0.0
28.0
99.5
Somatropin for injection
Humatrope
693
42.8
15.0
79.4
0.0
25.3
99.6
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Generic Name
Trade Name
No. of Plans That Cover Drug
% Plans That Cover Drug
% Plans That Place on Tier 3
% Plans That Place on Tier 4
% Plans That Have ST
% Plans That Have QL
% Plans That Have PA
Clofarabine
Clolar
899
55.5
25.0
66.6
0.0
0.0
19.4
Mecasermin
Increlex
1,347
83.1
21.4
73.1
0.0
0.0
92.0
Nafarelin acetate
Synarel nasal solution
1,445
89.2
30.0
61.7
0.0
0.0
31.4
Etanercept
Enbrel
1,450
89.5
11.5
83.3
1.4
45.2
93.7
Balsalazide disodium
Colazal
1,536
94.8
4.4
0.0
0.0
0.0
0.2
Meloxicam
Mobic
1,546
95.4
0.0
0.0
0.1
0.2
Leuprolide acetate
Lupron injection
1,554
95.9
11.1
9.8
0.0
18.1
71.1
Adalimumab
Humira
1,618
99.9
13.0
82.4
1.2
46.8
94.3
Felbamate
Felbatol
1,620
100.0
38.1
18.9
0.0
0.0
0.0
Rasburicase
Elitek
1,620
100.0
17.7
77.3
0.0
0.0
49.0
Ribavirin
Rebetol
1,620
100.0
6.9
6.4
0.0
17.4
73.6
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REFERENCES
CMS (Centers for Medicare and Medicaid Services). 2009a. 2010 Medicare Advantage Ratebook and Prescription Drug Rate Information: 2010 Low-Income Premium Subsidy Amounts. http://www.cms.hhs.gov/MedicareAdvtgSpecRateStats/Downloads/RegionalRatesBenchmarks2010.pdf (accessed September 2, 2010).
CMS. 2009b. 2010 Medicare Part D National Stand-Alone Prescription Drug Plans. http://www.cms.hhs.gov/PrescriptionDrugCovGenIn/Downloads/NationalPDPs.pdf (accessed September 2, 2010).
CMS. 2010. CMS Announces Course of Action to Identify Protected Class of Prescription Drugs. http://www.cms.hhs.gov/apps/media/press/release.asp?Counter=3409 (accessed September 2, 2010).
KFF (Kaiser Family Foundation). 2009a. Medicare: Low-Income Assistance Under the Medicare Drug Benefit. http://www.kff.org/medicare/upload/7327-05.pdf (accessed September 2, 2010).
KFF. 2009b. Medicare Part D 2009 Data Spotlight: Specialty Tiers. http://www.kff.org/medicare/upload/7919.pdf (accessed September 2, 2010).
KFF. 2009c. Medicare Part D 2010 Spotlight: Benefit Design and Cost-Sharing. December. http://www.kff.org/medicare/upload/8033.pdf (accessed September 2, 2010).
KFF. 2009d. Medicare Part D Spotlight: Part D Plan Availability in 2010 and Key Changes since 2006. http://www.kff.org/medicare/upload/7986.pdf (accessed September 2, 2010).
KFF. 2010a. Explaining Health Care Reform: Key Changes to the Medicare Part D Drug Benefit Coverage Gap. http://www.kff.org/healthreform/upload/8059.pdf (accessed September 2, 2010).
KFF. 2010b. Medicare: A Primer. http://www.kff.org/medicare/upload/7615-03.pdf (accessed September 2, 2010).
KFF. 2010c. Medicare at a Glance. http://www.kff.org/medicare/upload/1066-12.pdf (accessed September 2, 2010).
NORD (National Organization for Rare Disorders). 2006. Letter to Mark B. McClellan, M.D., Ph.D., Administrator of CMS: Orphan Drug Coverage in Medicare Part D Formularies January. http://www.rarediseases.org/news/pdf/Final_ltr_CMS_011006_V2.pdf (accessed September 2, 2010).
OIG (Office of Inspector General, U.S. Department of Health and Human Services). 2001. The Orphan Drug Act: Implementation and Impact. http://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf (accessed September 2, 2010).
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