Chapter 5: Drug Use, Access, and the Role of the Pharmaceutical Industry

Introduction
Pharmaceuticals are some of the most visible treatments in medical care.
Consumers possess a familiarity with them and choose the drug therapy they will consume or might
advocate for a drug their physician prescribes for them.
But, this simplicity and familiarity mask the true complexity of pharmaceuticals, especially their sideeffects, interactions, cost, and lengthy process of development.
Moreover, not all drugs work the same in every patient.
Patients wonder why their drugs cost so much or why their doctor has prescribed a certain medication.

DRUGS
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Pharmakon: Greek word for drug

o Three meanings: remedy, poison, and magical charm
o Remedy: alleviate symptoms or eliminate disease in patients
o Poison: cause unpleasant side effects or death in an overdose
o Magical charm: patients do not know or understand how a drug works
Definition: Substances that produce a change in cellular or physiologic functioning in humans
Manufacturers focus on a drugs therapeutic effect with the goals of:
o Optimizing biochemical activity
o Increasing specificity
o Limiting negative or adverse effects
Healthcare providers are concerned with:
o Therapeutic effects
o Treating their patients quickly and efficiently
o Choosing drugs that maximize treatment effects and minimize side effects
Consumers / Patients are concerned about
o Treatment effects
o Adverse events
o Costs

Prescription requires a prescription by an authorized licensed health care provider

Non-prescription medication available to general public without a prescription; may have restrictions
Also known as OTC
Approved Use medication or indication officially recognized by the FDA for marketing
Unapproved Use medication or indication without FDA permission for marketing

Medicalization: redefining or relabeling of a personal or social problem as a medical condition, thus

necessitating treatment in the health care system (defining or describing problems in medical terms or
treatment through medical interventions).
Conditions with physical pathologies that also have complex mental, emotional, and social processes:
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Anxiety/Depression
ADD/ADHD
Eating disorders

They often require treatment with nonpharmacologic therapy (counseling, diet/environmental changes).
Medicalization may minimize importance of nonpharmacologic treatments and prevent patient use.
Drug Use
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Medicalization of Normal Human Experience

Trivialization of Prescription Drug Use
Minimization of Healthy Living

Consumers Perspective
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Consumers make the final decision about whether or not they will use a drug
With medications, consumer beliefs are often very different from the beliefs held by health care
professionals
Perceptions of current or potential symptoms along with their social knowledge of health may
suggest whether a health problem exists
Possible actions taken for a perceived health problem:
o Do nothing
o Take action
Begin self-care
See a physician

- When beginning self-care, consumers are influenced by personal experience, advice of friends, webbased advice, direct-to-consumer advertising, and restrictions by law.
- 2/3 of consumers will leave with a prescription after visiting a doctor
- Slower rate of expenditure due to recession, fewer new drugs, and
increased safety concerns
Prescribers Perspective
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Drug therapy is a mainstay of prescribed treatments by physicians

Right or wrong, prescribing has a ritualistic component between physicians and patients
o Enhances and solidifies the interaction as a symbolic component to healing
o Provides a sense of satisfaction to the patient
- Reduces uncertainty and frustration by reinforcing the physicians power to cure
- Fulfills an ingrained need (habit) to take action against illness
o Usually requires little follow-up by the physician

Factors influencing prescribing behavior:

o
o
o
o
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Education
Training in pharmacotherapeutics and preceptors prescribing habits
Colleagues or other health care providers
Demands by consumers and society
Promotional activities by drug manufacturers
Control and regulatory mechanisms (drug laws, formularies, triplicate prescriptions)

Dispensers Perspective
- Independent pharmacies rely more on sales in filling prescriptions than chain pharmacies
- Retail pharmacists spend less time filling prescriptions due to advanced technology, rise of Internet
sales, and mail-order delivery of prescriptions; more time checking and verifying prescriptions
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As volume and regulatory pressures have mounted, pharmacists have had less time to perform
patient-centered services other than dispensing
Dissatisfaction with this situation is partly responsible for the rapid changes seen in the
profession
o Pharmacy technicians
o Robotics
o Increased clinical activities
Computerized monitoring of therapies
Patient counseling

- E-prescribing is become more commonplace

- Pharmacists can spend more time reviewing, recommending, and monitoring patient therapies
Drug development and Distribution systems
- Two primary systems regulating the use of drugs
a) Various state laws and agencies that control the practice of pharmacy and professionals
b) Regulation of safety and effectiveness of drugs by the FDA
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Two acts used by the FDA to control the development and manufacturing of pharmaceuticals:
o Food, Drug, and Cosmetic Act of 1938
Small-molecule drugs and hormones
o Public Health Service Act of 1944
Biologics made up of larger and more complex molecules
- FDAs Functions
o Premarketing clearance of all new products based on purity, safety, and effectiveness
o Regulation of labeling (prescription and nonprescription) and advertising (prescription)
o Regulation of good manufacturing practices and recalls, if necessary
o Regulation of bioequivalence standards for generics and biosimilars
o Postmarketing surveillance for unanticipated problems after approval
o Monitoring of the safety of the nations blood supply
Four stages of drug development:
a) Discovery of new compounds or proteins
b) Preclinical testing stage
c) Clinical testing stage
d) Approval and postclinical testing

Drug development process:

o Preclinical research
Bench research
Identifies potentially useful compounds
o Preclinical Testing
Tests to determine whether or not a drug is safe to test in humans
Animal testing
o Investigational New Drug (IND) application
Submitted to FDAs Center for Drug Evaluation and Research (small-molecule
drugs) or the FDAs Center for Biologics Evaluation and Research (biologics)
Wait 30 days for center to review the application
o Clinical testing
Testing on humans
Three phases prior to approval
a) Phase I: determine the dosing range and method, document metabolism and
excretion of drug, and identify toxicities associated with the drug (learn how drug
works in humans; less than 100 volunteers; short term - approx. 1 year)
b) Phase II: studied in a few 100 patients diagnosed with the disease the drug is
designed to treat (determine efficacy and safety of drug in the actual patients who
will be treated; approx. 2 years)
c) Phase III: subjects are randomized into control and experimental groups and
treated with doses and in settings similar to real world situations (designed to
provide strong evidence of safety and efficacy; approx. 3 years)
o New Drug Application (NDA) submittal for approval
Includes how the drug is manufactured, how the drug acts in the body, how the
drug will be marketed, labeling information, and toxicologic information
- Approval
o Sponsor submits a NDA
FDA has 60 days to decide to review or refuse for completeness
If approved, FDAs Center for Drug Evaluation and Research (CDER) reviews;
10 months for routine, 6 for priority reviews
Inspects manufacturing facility
May deny or request more information/testing
- If approved for market, manufacturer continues to submit postmarketing information (Phase 4);
including clinical trials on subgroups
- Supplemental NDA (sNDA) if a new indication, labeling change, or new manufacturing
procedure is requested
Pharmaceutical Marketing
Pharmaceutical firms use methods to market their products:
- Meet with physicians and educate them on the merits of using their product
- Direct-to-consumer (DTC) advertising (only in U.S. and New Zealand)
- Firms can advertise products to consumers in U.S. as long as they meet FDA guidelines
Importation
- Concerns: safety and equivalence (bioequivalence), fraud, counterfeiting, patent infringement
- FDA requires inspection of supply chain for approval for legal importation of products (drugs)
regardless of country of origin (approve manufacturing process and plant where drugs produced)

FDA bans reimportation of drugs for resale through unapproved sources

Its against the law to purchase prescription drugs in another country for use in the U.S.
Personal importation of a 3-month supply is allowed provided:
o A person can produce a valid US prescription from an authorized US health provider
o The intended use of drug is unapproved and for a serious condition where effective
treatment may not be available
o The product is not considered to represent an unreasonable risk

Counterfeiting
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Prevalence: 30% of global drug market in the developing world (WHO 2008)
Consumer risk: no benefit from treatment when consuming drugs with no active ingredients; may
suffer adverse effects or death from drugs with harmful and toxic chemicals in them; delays
access to effective treatment
Pharmacists should counsel patients to only buy from reputable pharmacies to minimize risk

Intellectual Property Rights

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Intellectual property rights (IPRs): laws and regulations to protect ideas & products of innovators
o Provide incentives for firms to innovate and develop new drugs that fight diseases
o Provide power to firms to control the market for a drug and charge a higher price than
firms would be able to without intellectual property protections
o Goal: preserve the incentive to innovate for firms, not to subject consumers to high prices
IPRs for small-molecule drugs
o Regulated by the Hatch-Waxman Act of 1984 (D.E.P. = 5 years from FDA approval)
o Patent protection (patents prevent other firms from copying an innovators product)
o Copies of small-molecule drugs are considered generics
IPRs for biologics
o Regulated by the Patient Protection and Affordable Care Act of 2010 (D.E.P. = 12 years)
o Data exclusivity (a firm can rely on the innovating firms original clinical trial data to
demonstrate the safety and effectiveness of its biosimilar (generic) drug)
o Copies of biologics are considered biosimilars