Drug Price Forecast January 2019 - Vizient

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Drug Price Forecast January 2019 - Vizient
Drug Price Forecast
   January 2019

© 2019 Vizient, Inc. All rights reserved.
Drug Price Forecast January 2019 - Vizient
Table of contents

Executive summary                                                                                  3

Projected timeline and approvals                                                                  10
A closer look by segment                                                                          12

Acute care                                                                                        12

Non-acute care                                                                                    13
Specialty pharmaceuticals                                                                         16
Pediatrics                                                                                        19
Key therapeutic class summaries                                                                   24

Oncology                                                                                          24

Infectious disease agents                                                                         29

Immunomodulators and disease-modifying therapies                                                  36

Plasma critical care products: IgIV and albumin                                                   40
Hot topics                                                                                        42

Overcoming the shortage of solutions to the drug shortage problem                                 42

Will favorable polices usher in a ‘biosimilar wave’ in 2019?                                      46
New technology add-on payment                                                                     52

Combating counterfeit drugs                                                                       53

Exploring the reasons for rising drug prices                                                      56

Continued challenges and new options in the opioid crisis                                         57
Conclusions                                                                                       60
Contributors                                                                                      62

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast    2
Drug Price Forecast January 2019 - Vizient
Executive summary

The January 2019 Drug Price Forecast is our best estimate of the change in the cost of pharmaceuticals that
participants in the Vizient Pharmacy Program will be purchasing between July 1, 2019, and June 30, 2020. The
forecast focuses on pharmaceutical products used across multiple health-system settings, including inpatient and
non-acute environments, and provides a year-over-year estimate of the expected price change.

Price change predictions for contract and noncontract product segments are shown in Table 1, along with the
overall drug price inflation number for existing drugs as calculated by Vizient.

Table 1. Summary of projected drug price inflation

                                                           Estimated price
                                                        change weighted by
                         Product group                  Vizient purchases, %
 Contract purchases                                              1.12
 Noncontract purchases                                           3.16
 Total weighted average drug price inflation estimate            4.28
Estimates based on Vizient member data.

In addition to price changes, the American Journal of Health-System Pharmacy advises that other factors — such
as volume changes and new product introductions — must be considered when preparing a drug budget.1
According to a May 2018 article, volume and mix decreased 5 percent in nonfederal hospitals in calendar year
2017, but increased 4.6 percent in clinics (including both physician offices and outpatient clinics).1 In addition, the
introduction of new products accounted for 2.8 percent of expenditure growth for nonfederal hospitals and 5.3
percent of growth for clinics during the same time frame.1 Both sets of statistics reveal the increasing influence the
expansion of non-acute care continues to have on health-system practice and expense. Table 2 shows the
therapeutic classes that account for the most spend among Vizient members.

© 2019 Vizient, Inc. All rights reserved.                       January 2019 Drug Price Forecast                          3
Drug Price Forecast January 2019 - Vizient
Table 2. Summary of highest-spend therapeutic classes among Vizient members

    Therapeutic category                    Key products in class
    Antineoplastic agents                   Rituxan, Keytruda, Avastin
    Disease-modifying antirheumatic Remicade, Humira, Enbrel
    agents
    Immunomodulatory agents                 Tysabri, Copaxone, Ocrevus
    Anti-infectives
        Hepatitis C                         Harvoni, Epclusa, Zepatier
        Antibacterials/antifungals          Daptomycin, Invanz, AmBisome
        (systemic)
    Plasma critical care
        Immune globulin, intravenous        Gamunex, Privigen, Gammaguard
        Albumin                             AlbuRx, Albutein, Flexbumin
    Hematopoietic agents                    Neulasta, Aranesp, Procrit
    Vaccines                                Prevnar, Gardasil, Pneumovax
Vizient member data for September 2017 to August 2018.

Table 3 shows the ten drugs with the highest total spend for Vizient members. Table 4 shows the drugs with the
largest projected increases in member spend due to price growth.

Table 3. Top 10 drugs by total spend among Vizient members

    Rank              Drug (brand name)
    1                 Adalimumab (Humira)
    2                 Infliximab (Remicade)a
    3                 Rituximab (Rituxan)a
    4                 Pegfilgrastim (Neulasta)a
    5                 Etanercept (Enbrel)
    6                 Alteplase (Activase)
    7                 Pembrolizumab (Keytruda)
    8                 Denosumab (Prolia, Xgeva)
    9                 Nivolumab (Opdivo)
    10                Ustekinumab (Stelara)

Vizient member data for September 2017 to August 2018.
a
    Biosimilar currently available or expected in 2019.

© 2019 Vizient, Inc. All rights reserved.                                  January 2019 Drug Price Forecast      4
Drug Price Forecast January 2019 - Vizient
Table 4. Top 10 drugs by size of projected price increase

 Rank            Drug (brand name)
 1               Adalimumab (Humira)
 2               Etanercept (Enbrel)
 3               Denosumab (Prolia, Xgeva)
 4               Ustekinumab (Stelara)
 5               Alteplase (Activase, Cathflo Activase)
 6               Vasopressin (Vasostrict)
 7               Vedolizumab (Entyvio)
 8               Pneumococcal 13-valent conjugate
                 vaccine (Prevnar 13)
 9               Abatacept (Orencia)
 10              Octreotide (Sandostatin LAR Depot)

Forecast highlights and overview
As can be seen by the analysis above, two things are consistent: (1) Oncology medications, disease-modifying
agents and immunomodulatory agents continue to account for the majority of spend, and (2) the projected rate of
price inflation continues to moderate. Although the FDA has approved many new specialty, orphan and other
high-cost drugs, these agents are used in very small populations. No other category has yet created a scenario
similar to the hepatitis C market, in which an expensive therapy is used across a rather broad patient population.
But even small price increases can have a substantial impact on drug spend, given the overall total of drug costs.

A number of events, both positive and negative, affected the pharmacy landscape in 2018. New product
approvals and increases in competition expanded treatment options and helped lower costs, while natural
disasters, exacerbation of drug shortages and reimbursement limitations added to already challenging
circumstances. Pharmacy’s critical role in coping with these changes to help manage the cost and quality of
health care make it a strong focus of improvement efforts.

A substantial number of high-spend agents are currently or will soon be subject to competition from biosimilars,
including pegfilgrastim, infliximab, rituximab and trastuzumab. The importance of continued adoption of
biosimilars to long-term efforts at mitigating price growth cannot be overstated. In addition, several of these
molecules are primarily used in the outpatient setting and therefore eligible for separate reimbursement, which
increases the importance of accurate billing and ongoing collaboration between the pharmacy and finance
departments.

Also, we would note that all significant expenditures are associated with outpatient and specialty pharmacy–
focused medications. Two agents, Vasostrict (vasopressin) and Cathflo Activase (alteplase),that are heavily used
in the inpatient setting will further challenge budgets due to expected price increases.

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                      5
Drug Price Forecast January 2019 - Vizient
Positive elements
As in the past, changes affecting pharmacy practice are a mix of positive and negative. Some recent favorable
events include:

  • As of November 17, the FDA has approved 16 biosimilars in several therapeutic classes, the most recent of
    which is a competitor for trastuzumab.2 Although only six of the 16 products have reached the market
      because of lingering patent protections and exclusivities, the increased competition has already resulted in
      lower relative prices. Payer coverage has also improved; more biosimilars are now included on formularies
      at levels at least equivalent to the originator brands. The Centers for Medicare & Medicaid Services (CMS)
      now assigns each biosimilar a unique reimbursement code and has granted pass-through status recognition
      to all biosimilars, steps that should also encourage the use of these products.3 In the last year, the FDA
      commissioner has made numerous statements about the need to remove regulatory and other barriers that
      can limit the uptake of biosimilars and has signaled a new strategic plan to improve the efficiency of this
      market. The FDA also held an open public hearing, in which Vizient participated, on additional steps the
      agency can take to advance the uptake and marketing of biosimilars.4 These efforts are critically important
      given the substantial growth of marketed biosimilars slated to take place in 2019.

  • Although there are fewer generic versions of blockbuster drugs reaching the market than in past years, the
    added competition for small-molecule drugs continues to help lower costs. For example, the pricing of
      daptomycin has eroded by more than 60 percent since its loss of exclusivity. While biosimilars have not
      caused price erosion of this magnitude, the imminent launch of competition has at least halted further price
      increases.

  • The CMS followed up its comprehensive initiative to lower the prices of medications5 with a proposal for
    comment on a mechanism to reduce Medicare drug costs to levels that are closer to what other developed
      nations pay.6 This new proposed concept also attempts to eliminate incentives to use more expensive drugs
      to obtain higher levels of reimbursement.6 Although the details of these strategies have not yet been
      clarified, the continued attention from the federal government increases the likelihood that changes will be
      implemented.

  • Last year also saw significant efforts to address drug shortages. In July, the FDA formed a drug shortages
      task force to look at long-term strategies to address the supply challenges that have plagued pharmacy.7
      The task force invited written comments to the FDA on ways to improve the current environment, conducted
      listening sessions with various stakeholders (including Vizient) and hosted an open public hearing to obtain
      additional feedback.7 Statistics shared by the FDA show that the agency has prevented more than 800
      shortages of injectable drugs since 2011.8 Nevertheless, drug shortages remain an ongoing threat to our
      capacity to deliver high-quality care.

Challenges
Ongoing and new challenges in the pharmacy landscape in the last year have included:

  • The fragility of the pharmaceutical supply chain, which has been subject to interruption for almost two
    decades and shows no signs of sustained improvement. For the first time since 2014, both new and existing

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                      6
Drug Price Forecast January 2019 - Vizient
shortages increased in 2018. Critical categories like antimicrobials, chemotherapy products, electrolytes and
      central nervous system drugs are still greatly affected by supply problems.9

  • Issues in the pharmaceutical supply chain have been exacerbated by efforts to address the opioid epidemic
    by reducing opportunities for diversion and misuse. One such effort was the quotas of active pharmaceutical
      ingredients imposed on pharmaceutical suppliers by the Drug Enforcement Administration (DEA).10 The
      quotas restricted manufacturers’ ability to compensate for unexpected supply interruptions, as those that had
      spare production capacity did not have access to enough ingredients to enable them to pick up any slack. As
      a result of advocacy efforts the DEA recognized the need to adjust these quotas,11 but the example
      highlights the fragility of the market.

  • Novel products continue to be introduced at higher and higher prices. Previous publications have noted the
    approval of the first chimeric antigen receptor T-cell (CAR-T) agents, tisagenlecleucel (Kymriah; Novartis)
      and axicabtagene ciloleucel (Yescarta; Kite Pharma/Gilead Sciences), both of which are notable for their
      high cost (reduced to $373,000 from an initial price of $475,000).12 Those costs were then surpassed by the
      introduction of voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics), the first in vivo gene therapy
      product to treat a rare form of vision loss, whose price for treatment of both eyes is $850,000.13

  • Reimbursement-related issues continued in 2018, including the reduced payments for disproportionate share
    hospitals imposed by CMS, which threaten many members’ ability to provide care for vulnerable
      populations.14 Payers are also increasing their focus on cost differences for administering medications in
      different locations (e.g., physician office vs. outpatient infusion clinic).15 And as described above, the
      administration continues to propose ideas to lower the cost of health care to the government.6 Some of these
      proposals could dramatically affect the way in which outpatient providers (health-systems and physicians)
      purchase and charge for pharmaceuticals.6

Our advice
The Drug Price Forecast is intended to help Vizient members anticipate and adapt to the specific and overall
challenges that continue to confront health care. The forecast includes reviews of the acute and non-acute care,
specialty pharmaceutical and pediatric markets, reflecting the diversity of practice environments and patient
populations served by our members. In addition, we include pricing projections for the therapeutic categories that
tend to drive the majority of spend and are consistent across care settings: oncology medications, anti-infective
agents, therapies for multiple sclerosis (MS) and rheumatoid arthritis (RA), and plasma-derived pharmaceuticals.

Critical areas that merit close attention include:

  • Strategic focus on pharmacy: Vizient has consistently held that pharmacy must be treated as an essential
      element of any organization’s strategic plan given its impact on the cost and quality of patient care across
      practice environments. Whether an organization is attempting to reduce inpatient length of stay, expand
      medication management to include retail or specialty pharmacy, promote population health or decrease
      opioid abuse, pharmacy is an integral component of those efforts. This concept was articulated convincingly
      in the April 1, 2018, issue of the American Journal of Health-System Pharmacy, in which several articles
      discussed the need for senior pharmacy leadership to support the concept of a “pharmacy enterprise” that
      effectively and efficiently extends across all sites of care.16

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                      7
• Specialty pharmacy: Health systems must have a well-defined approach to the management of specialty
      pharmaceuticals as these agents consume more spend and increasingly contribute to rising drug costs. The
      specialty pharmacy section of the forecast provides an update on this market and describes ways to
      integrate services to improve outcomes.

  • Biosimilars: We have stated this concept before and we will continue to vocalize it. We must take full
      advantage of the opportunity to lower costs presented by the expanding portfolio of biosimilars. A number of
      competing versions of blockbuster biologics are likely to reach the market in 2019. Pharmacists must be
      well-versed on these agents so that they can address clinical and economic questions of increasing
      complexity, particularly as oncology biosimilars reach the supply chain.

  • Advocacy: Successfully addressing the many issues confronting pharmacy requires advocacy. The 2018
      Vizient Pharmacy Charter, developed with the members of the Vizient Pharmacy Executive Council, can
      serve as a resource for targeting advocacy initiatives. Even with the changes in government resulting from
      the 2018 midterm elections, drug costs and prices will remain a hot topic for bipartisan scrutiny and potential
      additional intervention.

The pharmacy landscape continues to be extremely complex — regulatory issues, financial pressures, clinical
requirements and forecast expectations must all be anticipated and managed. However, organizations that can
successfully navigate these challenges will not just succeed, but thrive. Our goal, as always, is to help our
members thrive.

Forecast preparation, process and assumptions
Some things to keep in mind when reviewing the Drug Price Forecast:

  • The forecast presents the Vizient pharmacy team’s best estimate of likely drug price behavior during the
    identified period. However, it is important to recognize the uncertainty inherent in the projection process.

  • This analysis was conducted using data from Vizient Pharmacy Program participants’ purchases (price and
    volume) in hospital and non-acute facilities. The product mix covered by this forecast is based on
    participants’ aggregated purchases and will differ from that of any individual facility.

  • The products analyzed comprise the top 80 percent of pharmaceutical purchases (using dollars spent on a
    line-item basis) made through pharmacy Authorized Distributors by Vizient Pharmacy Program participants
      in hospital, non-acute and pediatric settings from Sept. 1, 2017, through Aug. 31, 2018. Purchases made
      through the 340B program were excluded from the analysis.

  • Purchasing sterile preparations from outsourced compounders is a sizeable expense for many health
    systems. This forecast does not analyze these purchases as they are not reported by our Authorized
      Distributors. If your facility uses outsourced compounding services, remember to factor those purchases into
      your budget plans. Vizient has noted regular price increases from our contracted suppliers in this area and
      we believe that this trend will continue.

  • Vizient uses price change history for the last 36 months (where available), as well as experience and
      knowledge of current contract allowances and marketplace factors such as expiring patents and anticipated

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                      8
new competition, to develop an inflation estimate for each line item in the projection. The analysis does not
        take into account other market dynamics such as raw material scarcity and finished goods supply shortages.

     • Information on possible patent expirations is provided solely as a courtesy and is based on sources available
       at the time of publication; actual expiration dates can change because of patent challenges and litigation
        processes. There is also no guarantee that an approved generic product will be ready to enter the market by
        the expiration date. Manufacturers also may file a request for exclusive marketing rights with the FDA for
        periods ranging from 180 days to seven years depending on the category. If granted, this period of
        exclusivity may or may not be synchronized with the patent status and can further delay the introduction of
        competition into the market.

Finally, this document is a projection of price behavior only. When preparing a drug expenditure budget, also
consider changes in volume and mix for your organization and the effects of introduction and adoption of new
drugs.
1.    Schumock GT, Stubbings J, Wiest MD, et al. National trends in prescription drug expenditures and projections for 2018. Am J Health
      Syst Pharm. 2018;75(14):1023-1038.
2.    Biosimilar product information. FDA website.
      https://www.fda.gov/drugs/developmentapprovalprocess/howdrugsaredevelopedandapproved/approvalapplications/therapeuticbiologicap
      plications/biosimilars/ucm580432.htm. Accessed November 18, 2018.
3.    CMS revises Medicare Part B biosimilar coding and payment policies. Covington & Burling LLC website. https://www.cov.com/-
      /media/files/corporate/publications/2017/11/cms_revises_medicare_part_b_biosimilar_coding_and_payment_policies.pdf. Published
      November 8, 2017. Accessed November 6, 2018.
4.    Sagonowsky E. Gottlieb calls out “rigged” system that’s hurting biosimilars, pledges FDA fight to boost uptake. FiercePharma. March 7,
      2018. https://www.fiercepharma.com/pharma/fda-chief-gottlieb-calls-out-rigged-system-thats-hurting-biosim-use. Accessed May 22,
      2018.
5.    American Patients First: The Trump Administration Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs. Washington, DC:
      Department of Health and Human Services; May 2018. https://www.hhs.gov/sites/default/files/AmericanPatientsFirst.pdf. Accessed
      November 6, 2018.
6.    Gingery D. US throws drug pricing weight around, but still depends on pharma decisions. The Pink Sheet. October 28, 2018.
      https://pink.pharmaintelligence.informa.com/PS124148/US-Throws-Drug-Pricing-Weight-Around-But-Still-Depends-On-Pharma-
      Decisions. Accessed October 31, 2018.
7.    Statement by FDA Commissioner Scott Gottlieb, MD, on formation of a new drug shortages task force and FDA’s efforts to advance long-
      term solutions to prevent shortages [press release]. Silver Spring, MD: Food and Drug Administration; July 12, 2018.
      https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613346.htm. Accessed October 31, 2018.
8.    Drug shortages infographic. Food and Drug Administration website.
      https://www.fda.gov/Drugs/DrugSafety/DrugShortages/ucm441579.htm. Updated August 13, 2018. Accessed October 31, 2018.
9.    University of Utah Drug Information Services. Drug shortages statistics. American Society of Health-System Pharmacists website.
      https://www.ashp.org/Drug-Shortages/Shortage-Resources/Drug-Shortages-Statistics. Accessed October 31, 2018.
10. Balick R. DEA mandates reduction in opioid manufacturing for 2018. American Pharmacists Association website.
    https://pharmacist.com/article/dea-mandates-reduction-opioid-manufacturing-2018. Published November 28, 2017. Accessed
    November 6, 2018.
11. DEA working to avoid US drug shortages [press release]. Washington, DC: US Drug Enforcement Administration; April 10, 2018.
    https://www.dea.gov/divisions/hq/2018/hq040918.shtml. Accessed November 6, 2018.
12. Bell J. Medicare to pay hundreds of thousands for CAR-T therapies. BioPharma Dive. April 6, 2018.
    https://www.biopharmadive.com/news/medicare-to-pay-hundreds-of-thousands-for-car-t-therapies/520806. Accessed November 6, 2018.
13. Sagonowsky E. Spark sets off gene therapy debate with $850K sticker on Luxturna. FiercePharma. January 3, 2018.
    https://www.fiercepharma.com/pharma/spark-prices-gene-therapy-luxturna-at-850k-grabbing-top-spot-pharma-s-costliest-drugs.
    Accessed November 6, 2018.
14. Forys A. 340B changes: what biosimilar manufacturers need to know. Center for Biosimilars website.
    http://www.centerforbiosimilars.com/contributor/amanda-forys/340b-changes-what-biosimilar-manufacturers-need-to-know. Published
    February 19, 2018. Accessed November 6, 2018.
15. Magellan Rx Management. Medical Pharmacy Trend Report 2017. 8th ed. https://www1.magellanrx.com/magellan-
    rx/publications/medical-pharmacy-trend-report.aspx. Accessed November 6, 2018.
16. Abramowitz PW, Chen DF, Cobaugh DJ. Multihospital health systems: growing complexity of pharmacy enterprise brings opportunities
    and challenges. Am J Health Syst Pharm. 2018;75(7):417-418.

© 2019 Vizient, Inc. All rights reserved.                                   January 2019 Drug Price Forecast                                   9
Projected timeline and approvals
New drug approvals
 Drug                                           Indication                                                      PDUFA date
 Ruxolitinib                                    Graft-vs.-host disease                                          Q1 2019
 Biosimilar trastuzumab (PF-0528001)            HER2-positive breast/gastric cancer                             Feb 2019
 Iclaprim                                       ABSSSI                                                          Feb 13, 2019
 Ravulizumab                                    Paroxysmal nocturnal hemoglobinuria                             Feb 18, 2019
 Tagraxofusp                                    AML                                                             Feb 21, 2019
 N8-GP (turoctocog alfa pegol)                  Hemophilia A                                                    Feb 27, 2019
 Siponimod (BAF312)                             SPMS                                                            Mar 2019
 Ozanimod (RPC1063)                             RRMS                                                            Mar 2019
 Herceptin SQ                                   Breast cancer                                                   Mar 2019
 Sotagliflozin                                  Type 1 diabetes                                                 Mar 22, 2019
 Meloxicam IV                                   Moderate to severe pain, including postoperative pain           Mar 24, 2019
 Fosphenytoin, captisol enabled                 Seizures                                                        Mar 30, 2019
 Iclaprim                                       ABSSSI/HABP                                                     Q2 2019
 RI-002 (IgIV)                                  RSV in patients with primary immune deficiency disease Apr 2, 2019
 Selinexor                                      Multiple myeloma                                                Apr 6, 2019
 Risankinzumab                                  Moderate to severe plaque psoriasis                             Apr 25, 2019
 Esketamine                                     Depression                                                      May 4, 2019
 Sacituzumab govitecan                          Metastatic breast cancer                                        May 21, 2019
 Biosimilar bevacizumab (PF-06439535)           Nonsquamous NSCLC                                               Jun 2019
 AXVS-101                                       SMA type 1                                                      Jun 18, 2019
 Erdafitinib                                    Bladder cancer                                                  Q3 2019
 Imipenem/relebactam                            MDR gram-negative organisms                                     Q3 2019
 Lefamulin                                      CABP                                                            Q3 2019
 Fosfomycin                                     MDR gram-negative organisms                                     Q3 2019
 Biosimilar adalimumab (SB5)                    RA                                                              Jul 2019
 Biosimilar rituximab (PF-05280586)             Non-Hodgkin’s lymphoma                                          Jul 2019
 Crenolanib                                     AML                                                             Aug 31, 2019
 Cefidercol                                     MDR gram-negative organisms                                     Q4 2019
 Biosimilar filgrastim (TX-01)                  Chemotherapy-induced neutropenia                                Oct 1, 2019
Abbreviations: ABSSSI = acute bacterial skin and skin structure infection; AML = acute myeloid leukemia; CABP = community-acquired
bacterial pneumonia; HABP = hospital-associated bacterial pneumonia; IgIV = immune globulin, intravenous; IV = intravenous; MDR =
multidrug-resistant; NSCLC = non–small-cell lung cancer; PDUFA = Prescription Drug User Fee Act; RA = rheumatoid arthritis; RRMS =
relapsing-remitting multiple sclerosis; RSV = respiratory syncytial virus; SMA = spinal muscular atrophy; SPMS = secondary progressive
multiple sclerosis.

© 2019 Vizient, Inc. All rights reserved.                                  January 2019 Drug Price Forecast                              10
Regulatory and accreditation events
    Event                                                                                                               Effective date
    CMS MACRA                                                Performance measures (MIPS) enforced Jan 1,                Jan 2019
                                                             2017
    CMS Conditions of Participation for Long-Term            Deadline extended for facilities to have an                Reimbursement penalties
    Care: Antibiotic Stewardship Program                     antibiotic stewardship program in place (original          will not go into effect until
    implementation                                           deadline Nov 28, 2017)                                     May 2019
    DSCSA                                                    Wholesalers must verify serialized product                 Nov 27, 2019
                                                             identifier for saleable returns
    Enforcement of USP Chapter 
    Enforcement of USP Chapter 
                                                                                                                        Dec 1, 2019
    Enforcement of USP Chapter                          Final versions to be published Jun 1, 2019
    Enforcement of USP Chapter 
    DSCSA                                                    Dispensers must receive only product with 2D               Nov 27, 2020
                                                             data matrix bar code and product identifiers
Abbreviations: CMS = Centers for Medicare & Medicaid Services; DSCSA = Drug Supply Chain Security Act; MACRA = Medicare Access and
CHIP Reauthorization Act; MIPS = Merit-based Incentive Payments System; USP = US Pharmacopeia.

Anticipated availability of new generics and biosimilarsa
    Generic name                                  Brand name                                      Earliest possible introduction
    Minocycline                                   Solodyn                                         Feb 2019
    Azithromycin (ophthalmic)                     AzaSite                                         Mar 31, 2019
    Atazanavir                                    Reyataz (oral powder)                           Jun 2019
    Thalidomide                                   Thalomid                                        Aug 1, 2019
    Fosaprepitant dimeglumine                     Emend (injection)                               Sep 5, 2019
    Hydrocodone bitartrate                        Zohydro ER                                      Nov 1, 2019
    Carfilzomib                                   Kyprolis                                        Jan 20, 2020
    Posaconazole                                  Noxafil suspension                              Jan 20, 2020
    Alvimopan                                     Entereg                                         Jan 27, 2020
    Enzalutamide                                  Xtandi                                          Feb 29, 2020
    Lanreotide acetate                            Somatuline Depot                                Mar 8, 2020
    Lenalidomide (5, 10, 15, 25 mg)               Revlimid                                        Apr 5, 2020
    Melphalan HCl                                 Evomela                                         May 9, 2020
    Apixaban                                      Eliquis 2.5 mg                                  Jun 28, 2020
    Oxycodone HCl                                 Xtampza ER                                      Jul 10, 2020
    Lenalidomide (2.5, 20 mg)                     Revlimid                                        Aug 26, 2020
    Dimethyl fumarate                             Tecfidera                                       Sep 27, 2020
    Vasopressin                                   Vasostrict                                      Oct 17, 2020
    Acetaminophen (intravenous)                   Ofirmev                                         Dec 6, 2020
    Tiotropium bromide                            Spiriva                                         Dec 26, 2020
a
    Projected dates of loss of exclusivity for originator drugs and generic or biosimilar entrants to the market are subject to change.

© 2019 Vizient, Inc. All rights reserved.                                        January 2019 Drug Price Forecast                                11
A closer look by segment

Acute care
Prescription drugs are one of the fastest-growing elements of health care spending, accounting for approximately
17 percent of the $3.3 trillion in total annual spend in the U.S.1,2

Table 5. Acute care drugs with the highest share of spend among Vizient members

 Drug
 Rituximab
 Pegfilgrastim
 Alteplase
 Infliximab
 Pembrolizumab
 Nivolumab
 Trastuzumab
 Bevacizumab
 Immune globulin, gamma (IgG)/Proline/IgA
 Ocrelizumab
Vizient member data on inpatient spend from September 2017 to August 2018.

The recent approval of Truxima (Celltrion/Teva Pharmaceuticals), the first biosimilar for rituximab, the acute care
drug that accounts for the highest share of Vizient member drug spend, is expected to have a significant impact
on the U.S. market. Overall, however, the approval of biosimilars has so far not had the expected impacts on
market share and costs, due to prior authorization requirements of various payer protocols and formularies.

As noted in the July 2018 edition of the Drug Price Forecast, the increase in drug spend is due to higher drug
costs rather than increased utilization. And many additional high-cost cell and gene biotherapies have been
approved since voretigene neparvovec (Luxturna), tisagenlecleucel (Kymriah) and axicabtagene ciloleucel
(Yescarta), with their accompanying sticker shock, came on the market. Medication cost management is therefore
a critical concern for pharmacy leaders and at the top of their list of challenges.3

The health system’s sustainability is being threatened by the flood of high-cost drugs for rare and ultra-rare
disorders that are priced at levels far above traditional cost-effectiveness thresholds.4 FDA Commissioner Scott
Gottlieb expressed concern about reimbursement for rare disease treatments and the need for reform at the
Milken Institute’s annual Future of Health Summit in Washington in October 2018.5 His statement comes on the
heels of the federal approval of right-to-try legislation and the uncertainty about its financial effect on the health
system.

Another factor in rising drug spending is persistent shortages of important drugs, including small-volume
parenteral fluids, emergency medications and injectable opioids including morphine, hydromorphone and fentanyl.

© 2019 Vizient, Inc. All rights reserved.                              January 2019 Drug Price Forecast                  12
(Pfizer, which controls about 60 percent of the U.S. market for injectable opioids, expects to return to full supply
capacity in the second quarter of 2019.)

The current administration has made reducing prescription drug prices a top priority. One result of this focus was
the release of American Patients First: The Trump Administration Blueprint to Lower Drug Prices and Reduce
Out-of-Pocket Costs in May 2018. Since then, the FDA has created a working group to consider prescription drug
importation and a record number of generic drugs have been approved, including many biosimilars.
1.   National health expenditure data. Centers for Medicare & Medicaid Services website. http://www.cms.gov/research-statistics-data-and-
     systems/statistics-trends-and-reports/nationalhealthexpenddata/nationalhealthaccountshistorical.html. Updated January 8, 2018.
     Accessed November 1, 2018.
2.   A look at drug spending in the U.S. The Pew Charitable Trusts website. http://www.pewtrusts.org/en/research-and-analysis/fact-
     sheets/2018/02/a-look-at-drug-spending-in-the-us. Published February 27, 2018. Updated August 28, 2018. Accessed November 1,
     2018.
3.   Brown T. What keeps hospital CEOs up at night? Infor website. http://blogs.infor.com/healthcare/2018/04/what-keeps-hospital-ceos-up-
     at-night.html. Published April 26, 2018. Accessed November 1, 2018.
4.   Lasser KE, Mickle K, Emond S, et al. Inotersen and Patisiran for Hereditary Transthyretin Amyloidosis: Effectiveness and Value — Final
     Evidence Report. Institute for Clinical and Economic Review website. https://icer-review.org/wp-
     content/uploads/2018/02/ICER_Amyloidosis_Final_Evidence_Report_101718.pdf. Published October 4, 2018. Accessed November 6, 2018.
5.   Statement by FDA Commissioner Scott Gottlieb, MD, on FDA’s new steps to modernize drug development, improve efficiency and
     promote innovation of targeted therapies [press release]. Silver Spring, MD: Food and Drug Administration; October 15, 2018.
     https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm623411.htm. Accessed November 21, 2018.

Non-acute care
The ability to deliver high-quality, cost-effective care outside of an inpatient hospital setting continues to grow in
importance. Providers and payers are focusing their energies on finding the least expensive site of care for
increasingly complicated services, including the administration of many medications.1 Given the growth of health
care expenditures, this trend is unlikely to change. As a result, it is critical to understand the factors affecting the
non-acute environment. Some of the challenges facing non-acute providers are specific to those settings — for
example, changes in reimbursement for home infusion and long-term care — but in many cases, the concerns of
non-acute facilities are the same as those of hospitals and health systems.

Two challenges that both acute and non-acute providers alike must deal with are drug shortages and compliance
with regulations governing the safety and sterility of medications, especially injectable products.

Drug shortages update
After remaining at fairly consistent levels for the last few years, the number of drug shortages, both new and
continuing, has actually started to climb.2 Recent data from the University of Utah Hospitals and Clinics shows
that as percentages, more injectable than noninjectable drugs are in short supply. Therapeutic categories
commonly used in various outpatient infusion settings, such as antimicrobials, electrolytes, nutrition-related
agents and intravenous fluids, also continue to be affected by supply issues.2 Vizient works aggressively to
provide members with the most current information on drug shortages as well as expanded clinical materials for
therapeutic alternatives to products affected by supply problems. In addition, the Vizient team has worked with the
FDA and other industry stakeholders to find solutions to these challenges.

© 2019 Vizient, Inc. All rights reserved.                                 January 2019 Drug Price Forecast                                  13
Mastering the requirements for sterile compounding
Another area requiring time, attention and resources from pharmacy providers in multiple sites of care is meeting
the requirements for safe compounding of sterile and hazardous pharmaceuticals.3 Standards established in the
United States Pharmacopeia (USP) general chapters  and  require a thorough understanding of
current practices and the capacity to plan the necessary investments in technology and possibly construction.3

Vizient has been working for several years to increase clinicians’ understanding of these requirements and help
them adapt to the changing standards and follow adjustments in the dates of enforcement. Now that the
enforcement dates are set for Dec. 1, 2019, definitive action is required.

Changing reimbursement across practice settings: focus on non-acute practices
Given the increasing expense of pharmaceuticals and the greater costs imposed by stricter regulatory
requirements, ensuring effective reimbursement is essential to maintaining the capacity to provide effective
patient care. Within the non-acute landscape, two recent reimbursement changes could greatly affect the
economic viability of home infusion programs and long-term care institutions.4,5 As with drug shortages,
awareness and advocacy are critical to advancing the perspective of the member community and preventing
regulatory action that could compromise patient care capabilities.

Home infusion: what looked promising is now a concern

One area where regulatory actions are prompting substantial worry is home infusion. One strategy for addressing
rising drug costs that has been proposed by many stakeholders is to migrate as much care as possible, particularly
for infused pharmaceuticals, to outpatient settings including patients’ homes. While not appropriate for all patients,
home infusion services can allow providers to maintain a high quality of care in a less expensive setting than a
hospital or clinic. Some patients, with proper training, may even be able to self-infuse certain complex medications.6

Two recent pieces of legislation, the 21st Century Cures Act and the Bipartisan Budget Act of 2018, appeared to
promote the financial viability of home infusion care by recognizing professional services as explicitly
reimbursable activities within Medicare.4 The 21st Century Cures Act instituted a professional services
reimbursement structure that would begin in 2021.4 In the interim, the Bipartisan Budget Act of 2018 provided for
temporary transition payments with specific rates for defined drugs, to be implemented prior to the 2021 date.

However, in the Home Health Prospective Payment System for calendar year 2019, CMS has proposed limiting
reimbursement for professional services to days when a nurse is physically present with a patient.4 As many
home infusion providers and their professional trade group, the National Home Infusion Association, have pointed
out, this requirement fails to take into consideration the numerous additional services provided by other
professionals, including pharmacists, for critically important activities such as compounding, preparation and
virtual monitoring of patient outcomes.4 The CMS approach is being challenged by numerous stakeholders and
even by bipartisan sponsors of the already-passed legislation.7 Vizient will continue to monitor the ongoing debate
and any potential future changes to this rule.

Long-term care: pulling the ‘RUG’ out from under skilled nursing facility reimbursement

Another change that appears less subject to debate, but nevertheless will be a huge transition, relates to the
payments for long-term care and skilled nursing facility services.5 Beginning Oct. 1, 2019, CMS is adopting a per-

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                      14
diem payment system called the Patient-Driven Payment Model (PDPM).5 The PDPM approach replaces the
Resource Utilization Group (RUG) methodology, which has been used since the Balanced Budget Act of 1997.8
However, one criticism of RUGs has been that utilization is driven by the number of therapy minutes provided,
which could encourage the delivery of additional intervention without clinical need.8 Under the new system,
therapy minutes will continue to be recorded but will no longer drive payment. Instead, patients will be assigned to
a case-mix group based on the clinical information in their admission minimal data sets (MDS).8

Most critically for pharmacists, in the PDPM system pharmacy services are considered a separate Non-Therapy
Ancillary Service (NTAS).5,8 The NTAS has a variable per-diem adjustment factor for the first three days of care
because of the increased use of services such as intravenous medication administration during this period.5,8
Therefore, pharmacists must ensure that all relevant data, including comorbidities, are adequately captured on
the patient’s MDS to receive appropriate reimbursement, particularly for the first three days of patient care.

Summary
As in other areas of practice, the non-acute care market continues to face many challenges. Drug shortages
continue to make the task of patient care more complex, and the increasing cost of specialty drugs, as
demonstrated by the most commonly used products in the non-acute setting (Table 6), will keep pharmaceutical
expenditures trending higher.

Table 6. Top 10 drugs based on non-acute spend among Vizient members

      Rank          Drug (brand name)
        1           Adalimumab (Humira)
        2           Etanercept (Enbrel)
        3           Ustekinumab (Stelara)
        4           Dimethyl fumarate (Tecfidera)
        5           Glatiramer acetate (Copaxone)
        6           Fingolimod HCl (Gilenya)
        7           Ledipasvir/sofosbuvir (Harvoni)
        8           Secukinumab (Cosentyx)
        9           Apremilast (Otezla)
       10           Abiraterone acetate (Zytiga)

Vizient member data for September 2017 through August 2018.

The movement of patient care away from the traditional inpatient setting will continue as stakeholders look for
ways to offset rising costs. As a result, pharmacy providers must be knowledgeable about the differences in and
potential changes to reimbursement patterns in order to succeed in this market and provide consistent care to
their patients. Familiarity with and knowledge of the home infusion space is particularly critical, as is expertise in
the specialty pharmacy arena, which will be discussed in the next section.

© 2019 Vizient, Inc. All rights reserved.                       January 2019 Drug Price Forecast                         15
Vizientinc.com resource
Drug compounding and handling resources page
1.   Magellan Rx Management. 2018 Employer Market Insights Report. 1st ed.
     https://www1.magellanrx.com/media/746514/2018_mrx_employer-market-insights-report.pdf. Accessed October 29, 2018.
2.   University of Utah Drug Information Services. Drug shortages statistics. American Society of Health-System Pharmacists website.
     https://www.ashp.org/Drug-Shortages/Shortage-Resources/Drug-Shortages-Statistics. Accessed October 29, 2018.
3.   Thompson G. Complying with pharmacy standards: USP 797/USP 800. The Oncology Pharmacist website.
     http://theoncologypharmacist.com/top-resources/online-only/17542-complying-with-pharmacy-standards. Updated August 8, 2018.
     Accessed October 29, 2018.
4.   NHIA executive summary of CMS proposed rule implementing home infusion services temporary transitional payment and permanent
     payment, National Home Infusion Association website. http://nhia.org/new_site/docs/NHIA_Executive_Summary_of_CMS.pdf. Published
     July 9, 2018. Accessed October 29, 2018.
5.   Baldwin P, Smith E. New focus for Medicare SNF reimbursement: CMS PDPM final rule for FY 2019 and FY 2020. Long Term Care
     Pharmacy News. http://ltcpharmacynews.com/docs/PDF%20Docs/August%202018%20Elise%20Smith.pdf. Accessed October 29, 2018.
6.   Infusion FAQs. National Home Infusion Association website. https://www.nhia.org/faqs.cfm. Accessed October 29, 2018.
7.   Congressman Sessions letter to CMS relays concern over proposed rule on Medicare Part B home infusion reimbursement. National
     Home Infusion Association website. http://www.nhia.org/documents/Sessions_Home_Infusion_Rule_Letter_SIGNED.pdf. Published
     August 7, 2018. Accessed October 29, 2018.
8.   Patient-Driven Payment Model (PDPM) resource center. American Health Care Association website.
     https://www.ahcancal.org/facility_operations/medicare/Pages/PDPM-Resource-Center.aspx. Accessed October 29, 2018.

Specialty pharmaceuticals
The number and cost of specialty pharmaceuticals continues to draw the attention of the health care community
as providers, payers and patients work to manage the increasing expense of these medications. Since the
publication of the July 2018 Drug Price Forecast, the FDA has approved 21 additional new molecular entities.1 Of
those, 12 (57 percent) are considered specialty pharmaceuticals based on factors like indication and intended
population, administration and monitoring considerations, cost, or limited distribution processes.1,2

Over the five years from 2013 through 2017, total U.S. expenditures for specialty drugs nearly doubled, from
$83 billion to $157 billion.2 If orphan drugs (i.e., “ultra-specialty” medications) are included, the total spend on
specialty drugs reached $200 billion in 2017.2 Combined, the costs of specialty pharmaceuticals and orphan
drugs nearly equal the total amount spent on traditional medications in the U.S. (Figure 1).

© 2019 Vizient, Inc. All rights reserved.                                 January 2019 Drug Price Forecast                             16
Figure 1. Annual spend by drug type as a percentage of total U.S. drug spend

Data derived from IQVIA Institute.2

The increasing financial challenge presented by specialty pharmaceuticals has a substantial impact on health
care providers and an even greater impact on patients, for whom the burden imposed by high-cost drugs is
significant. A recent analysis of dispensed prescriptions and out-of-pocket expense for U.S. patients in 2017
reveals the size of the problem.2 Of the 5.4 billion prescriptions filled that year, 31 percent resulted in no out-of-
pocket expenditures for patients, and another 48.9 percent cost between $0.01 and $9.99.2 Only 0.2 percent of
prescriptions cost patients $250 or more, but although the percentage of prescriptions in this category seems
small, it actually translates to 14.1 million prescriptions with a price tag of $250 or more. Another 3.4 million
prescriptions cost $500 or more.

The size of the burden for patients will likely continue to increase as insurers and payers implement additional
strategies to limit their exposure to increasingly expensive medications.2,3 For example, payers have now added
coinsurance payments and pharmacy deductibles on top of the specialty pharmacy tiers, limited distribution
strategies and prior authorization requirements that were already in place.2 Another strategy is copay
accumulators — programs that apply only a patient’s own out-of-pocket payments towards the deductible,
excluding the value of manufacturers’ coupons.2,3

As a result, providers — particularly the academic medical centers, integrated health systems, community-based
hospitals and pediatric facilities that comprise the Vizient membership — must consider the impact of specialty
pharmaceutical costs not only on their organizations, but also on their patient populations.

Biosimilars in specialty pharmacy: waiting for 2023
As discussed in other sections of this forecast, 2019 will be a seminal year for biosimilars, as competing versions
of monoclonal antibodies commonly used in oncology, such as rituximab, bevacizumab and trastuzumab, are
expected to enter the market.4 In addition, a second biosimilar for pegfilgrastim will likely enter the market by

© 2019 Vizient, Inc. All rights reserved.                        January 2019 Drug Price Forecast                        17
January 2019. These four molecules alone account for approximately $14 billion in spend over 12 months.5
Specialty pharmacy providers should work to encourage acceptance of biosimilars as comparable therapeutic
alternatives.

However, it increasingly appears that we will have to wait until 2023 for the biggest biosimilar opportunity within
the specialty pharmacy space. The total expenditure for adalimumab (Humira; AbbVie) eclipses the total for all
four of the biologics listed above, with an annual spend of $17 billion.5 Providers, payers and patients have been
eagerly waiting for competition for this high-cost molecule. But as a result of several recent settlements,
biosimilars for adalimumab are still about four years away.6 As of November 2018, AbbVie has reached patent
settlements with Amgen, Merck, Mylan, Sandoz and Momenta to delay launches of biosimilars, as noted in
Table 7. One supplier that has a biosimilar already approved and does not have a settlement agreement with
AbbVie, Boehringer Ingelheim, continues to engage in litigation.6

Table 7. Anticipated dates of introduction for adalimumab biosimilars

                                                                                                   Settlement-allowed
 Supplier                       Drug (brand name)               Approval date                      launch date
 Amgen                          Adalimumab-atto (Amjevita)      Sep 23, 2016                       Jan 31, 2023
 Merck/Samsung                  SB5                             Jul 2019 (estimated)               Jun 30, 2023
 Mylan                          MYL-1401A                       Not filed                          Jul 31, 2023
 Sandoz                         Adalimumab-adaz (Hyrmioz)       Oct 30, 2018                       Sep 30, 2023
 Momenta                        M923                            Not filed                          Nov. 20, 2023
 Boehringer Ingelheim           Adalimumab-adbm (Cyltezo)       Aug 5, 2017                        Ongoing litigation
                                                                    6-8
Data derived from Sandberg, IPD Analytics, and Drugs@FDA website.

The timing of pharmaceutical launches can change dramatically as a result of unanticipated legal rulings, so it is
possible that adalimumab biosimilars could become available earlier, but in light of the legal settlements that have
been made thus far, direct competition is not expected for several years.

Since biosimilar competition for the most commonly purchased drug, both in the specialty pharmacy category and
overall, is still four years away, it is critical that health systems continue to expand their specialty pharmaceutical
management strategies.

Summary
Given the existing population of specialty pharmaceuticals, the continued increase in the cost of these
medications and the dynamics of the investigational drug pipeline, a defined specialty pharmacy strategy is a
necessity, not an option, for health systems. A health system that does not address the challenges posed by
specialty pharmacy may risk loss of reimbursement in an accountable care environment, loss of patients and
revenue to competitors with established ambulatory and specialty pharmacies, and lower provider and patient
satisfaction across multiple service lines. Increasing availability of biosimilars over the next several years should
eventually result in meaningful competition, and providers should continue to promote these alternative products
where possible. In addition, providers must consider the financial impact of expensive specialty pharmaceuticals
not only on their organizations, but also on the patients they serve.

© 2019 Vizient, Inc. All rights reserved.                                 January 2019 Drug Price Forecast              18
1.   Novel drug approvals for 2018. Food and Drug Administration website.
     https://www.fda.gov/drugs/developmentapprovalprocess/druginnovation/ucm592464.htm. Accessed October 27, 2018.
2.   IQVIA Institute. Orphan Drugs in the United States: Growth Trends in Rare Disease Treatments.
     https://www.iqvia.com/institute/reports/orphan-drugs-in-the-united-states-growth-trends-in-rare-disease-treatments. Published October
     2018. Accessed October 27, 2018.
3.   Silverman E. Backlash against copay accumulators. Manag Care. 2018;27(9):15.
     https://www.managedcaremag.com/archives/2018/9/backlash-against-copay-accumulators. Accessed October 27, 2018.
4.   Pending biosimilars. The Pink Sheet. https://pink.pharmaintelligence.informa.com/PS120038/Pending-Biosimilars. Published February
     13, 2017. Accessed October 27, 2018.
5.   Schumock GT, Stubbings J, Wiest MD, et al. National trends in prescription drug expenditures and projections for 2018. Am J Health
     Syst Pharm. 2018;75(14):1023-1038.
6.   Sandburg B. AbbVie inks sixth Humira biosimilar settlement, battles Boehringer’s ‘Unclean Hands’ Claim. The Pink Sheet.
     https://pink.pharmaintelligence.informa.com/PS124226/AbbVie-Inks-Sixth-Humira-Biosimilar-Settlement-Battles-Boehringers-Unclean-
     Hands-Claim. Published November 8, 2018. Accessed November 18, 2018.
7.   IPD Analytics Biologics/Biosimilars database [database online]. Bay Harbor Island, FL: IPD Analytics; 2018. http://www.ipdanalytics.com.
     Accessed October 27, 2018.
8.   FDA approved drug products. Drugs@FDA website. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm. Accessed November 1,
     2018.

Pediatrics
Pediatric inflation rate: 4.14%

Recent approvals
In 2018, approvals of drugs for the pediatric population are focused in three categories: specialty drugs, gene
therapy and chimeric antigen receptor T-cell treatments. All three of these treatments raise budgetary and
revenue capture obstacles. To alleviate the burden they impose, additional evaluations must be made before
including these medications for rare diseases in hospital formularies.

The approval of elapegademase-lvlr (Revcovi; Leadiant Biosciences) in October added another enzyme
replacement therapy option for both pediatric and adult patients with adenosine deaminase severe combined
immune deficiency (ADA-SCID). The disorder mainly affects infants and young children. ADA-SCID is an inherited
genetic disorder in which deficiency of the ADA enzyme renders the immune system nonfunctional, resulting in
SCID.1 Elapegademase-lvlr is a recombinant product providing an alternative to pegadmease bovine (Adagen;
Leadiant Biosciences).

In August 2018 three drugs received approval or expansion of indications to include patients under 18 years of
age. Lanadelumab (Takhzyro; Shire) is the first monoclonal antibody approved for hereditary angioedema (HAE),
a rare genetic disorder in which patients have poorly functioning C1-INH protein, resulting in random episodes of
angioedema.2

Patients with Dravet syndrome, which requires combination therapy because of the potential for drug-resistant
increase in seizures, had two big wins in 2018. The approval of stiripentol (Diacomit; Biocodex) as adjunctive
therapy to clobazam (Onfi; Lundbeck) in patients 2 years of age and older3 adds a new therapeutic option. It will
also add an average of $14,000, depending on dosage, to the current annual average of $100,000 in health care
costs associated with Dravet syndrome, including multidrug therapy, hospitalizations and in-home medical care
visits.4

© 2019 Vizient, Inc. All rights reserved.                                  January 2019 Drug Price Forecast                                  19
In June the long-awaited epilepsy medication cannabidiol (Epidiolex; GW Pharmaceuticals) was approved on the
basis of improvements in quality of life and reductions in seizure frequency in patients with refractory epilepsy,
including those with Lennox-Gastaut and Dravet syndromes.5 Cannabis-based therapy is derived from cannabidiol
(CBD), the nonpsychoactive components of the marijuana plant. Release and availability of the product was
delayed awaiting the DEA schedule classification. In late September, cannabidiol medications were given a
Schedule 5 designation, allowing GW to manufacture its product. It is expected to cost about $32,500 per year.6

In patients with intestinal-failure–associated liver disease (IFALD) and parenteral nutrition–associated cholestasis
(PNAC), maintaining nutritional status with minimal risk to the liver requires reduction in intravenous lipid
emulsion. For several years, such patients in the U.S. used alternative fatty acid sources under compassionate-
use protocols. In August, an omega-3 fatty acid fish oil triglycerides emulsion, (Omegaven, Fresenius Kabi), was
approved as a source of fatty acids in patients with PNAC.7

Cost increases driven by specialty drugs, outpatient care
As in other areas, pediatric pharmaceutical costs are rising as a result of the targeting of rare diseases that
require specialty or orphan drugs.

Table 8 lists the top 10 drugs based on total expenditures among self-governed children’s hospitals as well as the
drug’s ranking from the July 2018 Drug Price Forecast and based on overall spend among Vizient members for
Sept. 1, 2017, through Aug. 31, 2018. Dinutuximab (Unituxin; AbbVie), indicated for high-risk neuroblastoma,
retained its previous position as the highest-spend drug for the current period. Pegaspargase (Oncaspar; Shire), a
modified enzyme used in the treatment of acute lymphoblastic leukemia, and infliximab (Remicade; Janssen
Biotech, Inc.), a monoclonal antibody used in the treatment of autoimmune diseases, have remained in the top 3
for year-over-year spend. One newcomer to the top 10 in the July 2018 forecast, eculizumab (Soliris; Alexion), a
monoclonal antibody directed against C5 complement protein, increased to fifth in spend for this group.8

Table 8. Top 10 pediatric drugs based on spenda among self-governed children’s hospitals that
participate in the Vizient group purchasing organization

    Generic drug name (brand name; manufacturer)
    Dinutuximab (Unituxin; United Therapeutics)
    Pegaspargase (Oncaspar; Baxalta)
    Infliximab (Remicade; Janssen Biotech)
    Palivizumab (Synagis; Astra Zeneca)
    Eculizumab (Solaris; Alexion)
    Sildenafil (Revatio; Pfizer)
    Asparaginase (Erwinaze; Jazz Pharmaceuticals)

    Dornase alfa (Pulmozyme, Genentech)
    Pneumococcal 13-valent conjugate vaccine (Prevnar-
    13, Pfizer)
    Pegfilgrastim (Neulasta, Amgen)
a
    340B purchases were excluded from the analysis.

© 2019 Vizient, Inc. All rights reserved.                      January 2019 Drug Price Forecast                    20
The high cost of CAR-T and gene therapy agents such as tisagenlecleucel (Kymriah) and voretigene neparvovec
(Luxturna) remains a consideration for pediatric care centers.9 The cost of a single infusion of tisagenlecleucel
was reduced in 2018 from $475,000 to $373,000.10 Discussions between Novartis and CMS about indication-
based pricing were abandoned in July 2018, leaving reimbursement under the unclassified drug/biologic J-
codes.11 Voretigene neparvovec, a gene replacement therapy for RPE65-medicated inherited retinal dystrophy,
which can cause complete blindness,12,13 continues to be priced at a total of $850,000 for treatment of both eyes.
The costs of these and other novel agents continue to weigh on pediatric pharmacy budgets.

These drugs may also have significant restrictions on handling, storage and administration. For example,
tisagenlecleucel can only be administered at preapproved centers and patients must be able to reach the center
within two hours for at least four weeks after administration in case of emergency. Institutions must therefore
review their procurement and administration policies for these therapies.

In the pipeline
The investigational pipeline in pediatrics continues to focus on treatment of targeted diseases using monoclonal
antibodies, gene therapy and orphan drugs. One gene therapy to treat type 1 spinal muscular atrophy, AVXS-101
(AveXis), is intended to be an intravenous alternative to intrathecal nusinersen (Spinraza; Biogen). An open-label,
single-arm, single-dose, multicenter trial, known as STR1VE, is being conducted to evaluate the efficacy and
safety of a one-time intravenous infusion of AVXS-101.14

Another trial of a gene therapy for the treatment of Duchenne muscular dystrophy, a genetic degenerative disease
that leads to progressive weakness of muscles, has begun to enroll human subjects. The goal of this therapy to is
replace the affected mRNA with mutations between exons 18 and 58.15

Table 9 lists additional products currently in development that, in some cases, are expected to have substantial
costs associated with their use.

© 2019 Vizient, Inc. All rights reserved.                     January 2019 Drug Price Forecast                     21
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