Specialty Pharmacy

and Biosimilars

Created by the School of Pharmacy

Relations Committee for AMCP
Updated: January 2020
 Part 1:
Specialty Pharmacy
Objectives
• Obtain an understanding of specialty pharmaceuticals
• Understand the challenges of specialty pharmaceuticals for
managed care companies
• Describe the value that specialty products bring to the market
Definition of Specialty
Pharmaceuticals
• Lack of universal definition
• “Biologic, biological, biopharmaceutical, biotech”
• Can include high cost injectable, infused product, oral agent or
inhaled medication
• May require close supervision, monitoring and handling requirements
• Medicare’s Part D definition for specialty: any drug for which the
negotiated monthly price is $600 or more
• Can be administered at home, MD office, infusion center or outpatient
hospital
Impact of Specialty Products
• Specialty drugs have been the fastest growing segment of drug spend
under the pharmacy benefit
• PBM Prime Therapeutics LLC reported that in 2013, specialty drugs
expenses amount to 33.4% of total (pharmacy and medical) drug costs. If
current trends continue, it will represent more than 50% by 2018.1
• According to PhRMA, there are currently over 7,000 new medications in
the biopharmaceutical pipeline

1
Prime Therapeutics LLC. 2013 drug trend insights. Available at: https://www.primetherapeutics.com/PDF/specialtydtr2013/index.html.
Accessed January 2, 2014.
Specialty Pharmacy Programs
• May include prior authorizations to ensure clinically appropriate
and cost-effective use of treatments when certain criteria is met
• Incorporate evidence-based guidelines, dosing and
discontinuation guidance, and monitoring
• Many specialty pharmacy providers have disease state
management programs specific for conditions that are
commonly treated with specialty pharmaceuticals
Specialty Pharmacy Providers
• Specialty pharmacy is often a subset of retail pharmacy in
managed care
• Payers often contract specifically with specialty pharmacies to provide
specialty pharmaceutical services to their members
• Many payers own their own specialty pharmacy
o Cigna / Express Scripts – Cigna Specialty Pharmacy / Accredo
o Humana – Humana Specialty Pharmacy
o Caremark / Aetna – CVS Specialty Pharmacy / Aetna Specialty Pharmacy
Components of Specialty Programs
• Refill reminders
• Delivery coordination
• Insurance verification
• Clinical interventions
• Patient education
• Adherence counseling
• Psychosocial assessment
• Patient assistance programs
• Integrated nursing staff
Commonly Targeted Disease States
• Pulmonary arterial hypertension • Blood disorders
• Multiple sclerosis • Cystic fibrosis
• Crohn’s disease
• Hepatitis C • Ulcerative Colitis
• Rheumatoid arthritis • Growth hormone
• Psoriasis deficiency
• RSV • Hemophilia
• HIV/AIDS
• Oncology
• Infertility
Challenges in MCOs
• Billing systems- integration of medical and pharmacy claims
• Utilization management
• Drug coding- NDC vs. J code
• Benefit design and patient cost share

Debbie Stern and Debi Reissman, “Specialty Pharmacy Cost Management Strategies of Private Health Care Payers,” Journal of
Managed Care Pharmacy, November/December 2006. http://www.amcp.org/data/jmcp/Nov-Dec06JMCP1.pdf
Accessed February 3, 2010
Benefit Designs
• Cause of much confusion
• Medical benefit vs. pharmacy benefit
 Each MCO can design and deliver their own product and services
• Has led to additional formulary tiers
 Tier 4, Tier 5 and Tier 6 for example
• Traditionally associated with higher co-pays or co-insurances
• Many plans are moving towards new benefit designs for
specialty drugs and therapies, including a “specialty formulary”
Impact on Patients
• Specialty expenditures per member increased by 11.1% from
2004 to 2008 (in a 8 million member commercial plan)
• Potential impact of shifting costs to patients with specialty
drugs:
• Inability or refusal to pay for medications leading to decreased
compliance
• Compliance issues may impact medical expenses and hospital costs
Monitoring for Adverse Reactions
• Prescription drug safety is key with specialty pharmaceuticals
• Problems arise when a drug offers significant therapeutic benefits but also
carries challenging health or safety risks
• FDA Mandated Risk Evaluation and Mitigation Strategy (REMS)
• Many biologics and new therapies are subject to REMS
• Includes patient registries, ongoing patient monitoring, and certification for
prescribers and pharmacies
• Specialty pharmacies have a high level of patient and physician interaction and
specially trained therapy teams to assist with meeting these requirements
Summary
• Specialty pharmacy is a growing part of the pharmacy industry
• Specialty pharmacy providers continue to develop programs
and value-added services to better manage patients
• Payers continue to develop new strategies to manage the high
costs of these drugs
• Patients are often affected by the variance in coverage and cost
shares
References
• Prime Therapeutics LLC. 2011 drug trend insights. 8/11. Available at:
http://www.primetherapeutics.com/PDF/2011PrimeDrugTrendInsights.pdf. Accessed September
14,2011.
• Debbie Stern and Debi Reissman, “Specialty Pharmacy Cost Management Strategies of Private
Health Care Payers,” Journal of Managed Care Pharmacy, November/December 2006.
http://www.amcp.org/data/jmcp/Nov-Dec06JMCP1.pdf. Accessed February 3, 2010.
• Gleason PP, Starner CI, Gunderson BW, Schafer JA, Sarran HS. Association of Prescription
Abandonment with Cost Share for High-Cost Specialty Pharmacy Medications. Journal of Managed
Care Pharmacy 2009;15(8)648-58. Specialty pharmacy's role in REMS, FDA's new drug safety
program. Formulary. 2009;44:300–308.
• FMCP Specialty Pharmacy Initiative: Phase I Discovery & U.S. Environmental Scan. October 2009.
www.fmcpnet.org. Accessed February 1, 2010.
• The Biopharmaceutical Industry: Creating Research, Progress and Hope.
http://www.phrma.org/about/biopharmaceutical_sector. Accessed February 1, 2015.
 Part 2:
Biosimilars
Biologics Price Competition and Innovation Act
(BPCIA)
Established in 2009 to create an abbreviated licensure
pathway for biological products that are demonstrated to be
“biosimilar” to or “interchangeable” with an FDA-licensed
biological product.
Biologic Marketplace

Kozlowski, S et al. NEJM. 365(5); 385-88.

Trend Towards Biologics
Biologic
• Biologic: Therapeutic product derived from a biological source
 Vaccines
 Antitoxins
 Blood products
 Proteins
 Monoclonal antibodies

www.fda.gov
Biologics Vs. Small Molecules
Biologics, including biosimilars are much more complex than small molecule
drugs
Large
biologic
Small
biologic
Small
molecule drug

COMPLEXITY
Biosimilar
• Biosimilar
 Highly similar to the U.S. licensed reference biological product
notwithstanding minor differences in clinically inactive components
 No clinically meaningful differences from the reverence product in
terms of safety, purity, and potency

www.fda.gov
Interchangeable
• Interchangeable
 Biosimilar to the U.S. licensed reference product
 Expected to produce the same clinical result to the reference product
in any given patient
 If a product is indicated for multiple administrations, then the product
must be able to be alternated with the reference product without any
loss of efficacy or change in risk of adverse events
 May be substituted at the pharmacy level without the intervention of a
healthcare provider

www.fda.gov
Biosimilar 351(k) Application
• In order to be granted biosimilar status by the FDA, the biological product
must show clinical data derived from:
 Analytical studies that demonstrate that the biological product is highly similar to the
reference product notwithstanding minor differences in clinically inactive
components;
 Animal studies (including the assessment of toxicity); and
 A clinical study or studies (including the assessment of immunogenicity and
pharmacokinetics or pharmacodynamics) that are sufficient to demonstrate safety,
purity, and potency in one or more appropriate conditions of use for which the
reference product is licensed and intended to be used and for which licensure is
sought for the biological product.

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM291128.pdf
Biosimilar 351(k) Application
• Stepwise Approach:
 Extensive structural and functional characterization of both products
 Animal data if contains pertinent information not able to be obtained
from humans (e.g. toxicity)
 Human Pharmacokinetic studies
 Human Pharmacodynamic studies
 Compare clinical immunogenicity
 If inconsistencies exist, consider clinical trial with appropriate safety
and efficacy measures

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM291128.pdf
FDA Purple Book
• First published September 9, 2014
• Lists biological products, including any biosimilar and interchangeable
biological products licensed by the FDA under the Public Health Service Act
(PHS Act)
• Includes the date a biological product was licensed under 351(a) of the PHS
Act and whether the FDA evaluated the biological product for reference
product exclusivity under section 351(k)(7)
• Shows if the FDA has determined a product to be biosimilar or
interchangeable with an already licensed FDA reference biological product
• Biosimilar and interchangeable products are listed under the reference
product
Biosimilar: Filgrastim
• First application under the new biosimilar pathway brought to an FDA
advisory committee
• On January 7th, 2015, the FDA Oncologic Drugs Advisory Committee
unanimously voted that Sandoz’s EP2006 (Zarxio) should be licensed as
biosimilar to the reference product, Neupogen
– Includes all 5 indications for which Neupogen is approved
• Zarxio (filgrastim-sndz) was approved March 6, 2015
as a biosimilar, not an interchangeable
– Route of administration, indications and side effects are the same as Neupogen
– Launched September 3, 2015
– 15 percent wholesale list price discount
Biosimilar Naming
• Nonproprietary Naming of Biological Products Guidance finalized January, 2017

• Per FDA Guidance biosimilars receive a nonproprietary name consisting of a common core plus a unique FDA-approved suffix
composed of four lowercase letters

• For example, for products sharing the core name replicamab, those proper names may be displayed as:
-replicamab-cznm
-replicamab-hixf

• The proposed suffix should:

o Be unique
o Be devoid of meaning
o Be four lowercase letters of which at least three are distinct
o Be nonproprietary
o Be attached to the core name with a hyphen
o Be free of legal barriers that would restrict its usage
www.fda.gov
Outstanding Questions
• How much will the addition of biosimilars lower costs to the
health care system?
• Will increased access to biologics lead to better health
outcomes overall?
• How will managed care organizations manage these agents?
References
• Information for Healthcare Professionals (Biosimilars).
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApp
roved/ApprovalApplications/TherapeuticBiologicApplications/Biosimilars/ucm241719.htm
. Accessed February 1st, 2015.
• "Biosimilars," Health Affairs, October 10, 2013. Accessed February 1st, 2015.
• Purple Book.
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApp
roved/ApprovalApplications/TherapeuticBiologicApplications/Biosimilars/ucm411418.htm
. Accessed February 1st, 2015.
• Kate Traynor. FDA Advisers Recommend Approval of Biosimilar Filgrastim.
http://www.ashp.org/menu/news/pharmacynews/newsarticle.aspx?id=4163. January 26th,
2015. Accessed February 1st, 2015.
Thank you to AMCP member
Jorge Farias for updating this
presentation for 2016.
Mission & Vision
To improve patient health by
ensuring access to
high-quality, cost-effective
medications and other therapies.