CSP Exam Prep Course: Specialty Pharmacy Basics

Comprehensive vocabulary flashcards covering the key concepts, laws, clinical trial phases, health economics models, distribution networks, payor structures, adherence metrics, and pricing benchmarks from the CSP Exam Prep Course on Specialty Pharmacy Basics.

Specialty Pharmacy

A dedicated practice area focused on the distribution, clinical management, and continuous patient support of high-cost, high-complexity, and high-touch medications used for chronic, rare, or life-threatening illnesses.

Risk Evaluation and Mitigation Strategies (REMS)

Drug safety programs mandated by the FDA for medications with serious known or potential risks to ensure benefits outweigh harms, requiring specialized training, patient registries, or monitoring.

Specialty Pharmacy Accreditation

Third-party quality validation from bodies such as URAC, ACHC, or NABP evaluating quality assurance, clinical documentation, patient education, and reporting to grant access to limited distribution networks and payor panels.

Food, Drug, and Cosmetic Act (FDCA)

A foundational federal law establishing drug approval, manufacturing standards, and labeling requirements, providing the framework for biologics oversight and REMS administration.

Drug Supply Chain Security Act (DSCSA)

Federal legislation governing the electronic tracing and tracking of prescription pharmaceuticals throughout the distribution supply chain to prevent counterfeiting and secure cold chain delivery.

Controlled Substances Act (CSA)

Federal statute regulating the manufacturing, importation, possession, and distribution of controlled substances, mandating DEA registration, specific storage, and documentation.

Hatch-Waxman Act

Federal legislation that created the Abbreviated New Drug Application (ANDA) approval pathway for generic drugs while offering patent term restoration to brand innovator drugs.

Omnibus Budget Reconciliation Act of 1990 (OBRA-90)

A federal law establishing requirements for prospective Drug Utilization Review (DUR) and mandatory patient counseling offers for Medicaid prescriptions.

Medicare Modernization Act of 2003 (MMA)

Federal law that established Medicare Part D prescription drug coverage and mandated Medication Therapy Management (MTM) services for qualifying beneficiaries.

Orphan Drug Act

Federal statute providing financial incentives, tax credits, and extended market exclusivity to encourage manufacturers to develop therapies for rare diseases affecting fewer than 200,000 Americans.

Biologics Price Competition and Innovation Act (BPCIA)

A regulatory framework creating an abbreviated licensure pathway under the Public Health Service Act (PHSA) for biological products shown to be biosimilar to, or interchangeable with, an FDA-licensed reference product.

Health Insurance Portability and Accountability Act (HIPAA)

Federal legislation protecting the privacy, security, and integrity of Protected Health Information (PHI) across all clinical, administrative, and pharmacy coordination interactions.

Preclinical Phase

The drug discovery and laboratory testing stage involving in vitro and animal models to assess safety, pharmacodynamics, dosing, toxicity, and mechanism of action prior to first-in-human trials.

Phase I Clinical Trial

Initial human studies typically conducted in $20\text{--}100$ healthy volunteers or patients to establish safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and maximum tolerated dose.

Phase II Clinical Trial

Exploratory clinical studies in $100\text{--}300$ patients with the targeted condition to determine preliminary efficacy, side-effect profiles, biomarker response, and optimal therapeutic dosing.

Phase III Clinical Trial

Large-scale confirmatory trials involving $300\text{--}3{,}000+$ patients comparing the investigational drug against standard of care or placebo to substantiate clinical efficacy and monitor adverse reactions.

Phase IV Clinical Trial

Post-marketing surveillance studies conducted in real-world populations to evaluate long-term safety, rare adverse events, treatment persistence, and health economics.

Primary Outcome

The principal therapeutic endpoint predefined in a clinical study protocol used directly to evaluate the trial hypothesis and judge overall efficacy or failure.

Secondary Outcome

An additional measure of interest evaluated in a clinical trial to provide supplemental supportive evidence regarding health effects, tolerability, quality of life, or disease control.

Crossover Trial

A study design where each participant is randomized to receive two or more sequential treatments, serving as their own control after an intervening washout period.

Non-inferiority Trial

A clinical study design intended to demonstrate that a new intervention is not unacceptably less effective than an active comparator standard-of-care by a predefined margin.

ECHO Model

A multidimensional pharmacoeconomic assessment framework categorizing healthcare results into Economic Outcomes, Clinical Outcomes, and Humanistic Outcomes.

Absolute Risk Reduction (ARR)

The arithmetic difference in the probability of an outcome between unexposed and exposed trial cohorts, defined as $\text{ARR} = \text{CER} - \text{EER}$.

Number Needed to Treat (NNT)

An epidemiological metric indicating how many patients must receive a therapy over a given period to prevent one additional adverse outcome, defined as $\text{NNT} = \frac{1}{\text{ARR}}$.

Relative Risk (RR)

The ratio of the risk of an event in the experimental group compared to the control group, calculated as $\text{RR} = \frac{\text{EER}}{\text{CER}}$.

Relative Risk Reduction (RRR)

The proportional percentage reduction in event risk achieved by an active intervention compared to a control, calculated as $\text{RRR} = \left(\frac{\text{ARR}}{\text{CER}}\right) \times 100$.

Odds Ratio (OR)

A measure of association comparing the odds of an outcome occurring in an exposed group to the odds of it occurring in an unexposed group, represented from a $2 \times 2$ table as $\text{OR} = \frac{A/C}{B/D}$.

Number Needed to Harm (NNH)

The number of patients who must be exposed to an intervention for one additional person to experience a specific adverse event, expressed as $\text{NNH} = \frac{1}{\text{ARI}}$.

Biosimilar

A biological agent demonstrating high structural, functional, and clinical similarity to an FDA-approved reference biologic, possessing no clinically meaningful differences in safety, purity, or potency.

351(k) Biologics License Application (BLA)

The specific regulatory submission pathway created under the BPCIA enabling licensure of biosimilar and interchangeable therapeutic proteins.

Interchangeability (Biosimilar)

An FDA regulatory determination permitting a biosimilar to be substituted for the reference product at the pharmacy counter without prescriber intervention, subject to state pharmacy practice laws.

Limited Distribution Drug (LDD)

A specialty medication made available by the manufacturer only through an exclusive or tightly restricted network of certified specialty pharmacies due to safety, clinical monitoring, or logistics requirements.

Manufacturer HUB

A centralized, manufacturer-sponsored operational center that coordinates access, reimbursement navigation, financial support, clinical onboarding, and distribution support between stakeholders.

Benefits Investigation (BI)

The structured verification of a patient's insurance policy parameters to determine drug coverage, pharmacy vs. medical benefit eligibility, copay tiers, and prior authorization needs.

Prior Authorization Support

A collaborative pharmacy or HUB workflow that assists healthcare prescribers in gathering and submitting clinical rationale to satisfy payor medical criteria.

Accumulator Adjustment Program

A payor benefit design preventing manufacturer copay card assistance from counting toward a patient's annual health plan deductible or out-of-pocket maximum.

Maximizer Program

An insurance benefit feature that adjusts monthly cost-sharing to capture the total annual maximum value of manufacturer copay coupons evenly across the benefit year.

Alternate Funding Program (AFP)

A third-party benefit model where high-cost specialty drugs are excluded from plan formularies and patients are guided to seek charitable foundations or manufacturer assistance programs.

Social Determinants of Health (SDOH)

The environmental, social, and economic conditions in which people live and work—such as housing, income, transportation, and health literacy—that influence health outcomes and therapy adherence.

Inflation Reduction Act of 2022 (IRA)

Federal legislation establishing drug inflation rebates, granting CMS negotiation powers for select high-spend drugs, and restructuring Medicare Part D with a $\$2{,}000$ annual out-of-pocket cap starting in 2025.

Proportion of Days Covered (PDC)

A standardized medication adherence calculation showing the proportion of days a patient had drug access over a treatment period: $\text{PDC} = \left(\frac{\text{Total Days Covered}}{\text{Total Days in the Measurement Period}}\right) \times 100$.

Medication Possession Ratio (MPR)

An adherence metric evaluating pharmacy claim supply over time, formulated as $\text{MPR} = \left(\frac{\text{Total Days Supply Dispensed}}{\text{Total Days in the Measurement Period}}\right) \times 100$.

Factor VIII Dosing Formula

The clinical calculation used to determine replacement therapy units in hemophilia A: $\text{Units of factor VIII needed} = \frac{\text{patient weight (kg)} \times (\text{desired factor conc} - \text{initial conc})}{2}$.

Average Wholesale Price (AWP)

A published medication reference cost or 'sticker price' used across pharmacy contracts as a baseline benchmark for reimbursement discounting, though exceeding real acquisition cost.

Wholesale Acquisition Cost (WAC)

The list price set by a drug manufacturer for commercial sale to wholesalers or direct purchasers, excluding prompt-pay discounts, volume rebates, or concessions.

Average Sales Price (ASP)

A volume-weighted average price across all commercial purchasers net of price concessions and rebates, used primarily by Medicare Part B for provider-administered drug reimbursement.

Average Manufacturer Price (AMP)

The average price paid to drug manufacturers by wholesalers and retail community pharmacies, utilized by CMS to determine statutory Medicaid drug rebate amounts.

National Average Drug Acquisition Cost (NADAC)

A federal survey-derived metric reflecting the actual invoice acquisition costs incurred by retail and specialty pharmacies to purchase prescription products.

Direct and Indirect Remuneration (DIR) Fees

Concessions, performance adjustments, and network fees collected by Pharmacy Benefit Managers (PBMs) after prescription adjudication that lower net pharmacy reimbursement.

Cold Chain Shipping

A temperature-controlled logistics and distribution infrastructure designed to maintain continuous thermal stability for temperature-sensitive biologics from manufacturer to patient delivery.