Benefit versus risks: from test tube to patient: improving health through human drugs

Under current law, all new drugs need proof that they are effective and safe before they can be approved for marketing. No drug is absolutely safe . there is always some risk of an adverse reaction. However, when a proposed drug’s benefits outweigh known risks, the FDA’s Center for Drug Evaluation and Research (CDER) considers has a benefit. In the first large clinical 42 days. A 12 percent decline in U.S.
large-scale controlled clinical trials.
a p p roval rule, the Center can rely as a basis for drug approval on a re a s o n a b l e s u rrogate endpoint, that is, a positive e ffect of a drug on a marker of the dis- ments to physicians and pharm a c i e s .
the actual clinical benefit of the drug. P romising Experimental Drugs
re s e a rch subjects, the agency ro u t i n e l y inspects the boards every five years.
may inspect the facilities more often.
Reviewing NDAs
especially its safety, and provide treat- out,’” Temple says. “The sponsor has final action on new molecular enti-ties, switches from prescription toOTC status, and other important actions, such as a major new use of adrug. Other approval decisions aremade at the division level.
nearly in half, while the number of Final Actions
drugs approved in a year have doubled. sion whether to approve a new drugfor marketing boils down to twoquestions: • Do the results of well-controlled studies provide substantial evidenceof effectiveness? P r i o r i t i e s
ity on the basis of the drug’s chemical disease are considered priority dru g s .
I n d u s t r y and consumer re s p o n s e division of oncology drug pro d u c t s ; P D U FA led, in part, to Congre s s ’ s n a t u r a l l y, go to the office of generic Outside Advice for Close Calls,” p.
a p p roved decision is a close call.
“ C u rre n t l y, CDER is re v i e w i n g A Special System for OTC Dru g s
Federal Register and requested public FDA’s final monograph, the third phase, identifies those active ingredients that are generally recognized as safe and effective the market as a result of the advisory pan- els’ OTC drug review. Among them were: graph identifies labeling claims that may • camphorated oil, a liniment often acci- long as it meets its category’s standards.
is deemed safe enough for self-use and is products can be reformulated or appropri- ately relabeled. For ingredients or claims approved solely on the basis of their safe-ty since passage of the 1938 FederalFood, Drug and Cosmetic Act. Specialattention soon focused on OTC drugs: of Sometimes an approved prescription the 512 OTC drug products evaluated, 75percent lacked substantial evidence ofeffectiveness.
drug is deemed safe enough for self- to tackle a broader review of OTCdrugs—no small job, considering thatmore than 300,000 products were on the use and is switched to OTC status. market. Those products, however,involved only about 700 active ingredi-ents. It didn’t take long for CDER plannersto decide on a strategy: classify thedrugs by treatment category (antacids, ingredients. So, rather than review thou- sands of, say, individual antacid products, • zirconium, still safe in most forms of include additional ingredients or to modify CDER evaluated the far fewer active ingre- including products used to treat problems review by publishing final rules within the for each therapeutic class of drugs under consideration. The first phase was accom- recognized as safe and effective for self- During the second phase, FDA pre s e n t e d The Evolution of U.S. Drug Law
FDA acts as a public health pro t e c t o r • Food and Drugs Act (1906): This
first drug law re q u i red only that drugs meet standards of strength and purity.
• Federal Food, Drug and Cosmetic
Act (1938): A bill was introduced in
The "Elixir Sulfanilamide" tragedy of 1937 ensured enact- ment the following year of the Federal Food, Drug, and Cosmetic Act. More than 100 died from using the “Elixir Sulfanilamide” to promote pas- untested, poisonous new drug formulation, but FDA had the first time, re q u i red a manufacture r legal authority to bring only a trivial charge of misbrand- ing against the manufacture r. The product was labeled an • D u r h a m - H u m p h rey Amendment
( 1 9 5 1 ): Until this law, there was no
" e l i x i r," which implied it was an alcoholic solution; actually, it was a diethylene glycol solution. If the term "solution" had been used instead, no charge of breaking the law • K e f a u v e r-Harris Drug
Amendments (1962): News re p o rt s
• Orphan Drug Act
( 1 9 8 3 ) : “Orphans” are drugs and
i n t e rest in drug re g u l a t i o n .
eases. They may offer little or no pro f- about thre e - q u a rters of the cost of • Drug Price Competition and
The Review Team
A Drug Review Glossary
Abbreviated New Drug
Patient Term Restoration Act
simultaneously apply their special techni- Application, or ANDA: A simpli-
( 1 9 8 4 ) : This law expands the number
of drugs suitable for an abbre v i a t e d to ensure the identity, strength, quality, refers to the 17 years of legal pro t e c- • Pharmacologists evaluate the effects of tion given a firm for each drug patent.
• Physicians evaluate the results of the • Generic Drug Enforcement Act
( 1 9 9 2 ) : This law imposes debarment
Accelerated Approval: A highly spe-
• Pharmacokineticists evaluate the rate • P rescription Drug User Fee Act
( 1 9 9 2 ) : In this law, manufacture r s
• Statisticians evaluate the designs for actual clinical benefit of the drug.
and conclusions of safety and eff e c t i v e- Action Letter: An official communi-
• M i c robiologists with others evaluate the data on anti-infectives (antibodies, antivi- • FDA Modernization Act (1997):
rals, and antifungals). These drugs diff e r f rom others in that they affect the work- ings of microbes instead of patients.
n e e d e d to evaluate the drug’s eff e c t i v e- for the first application for small busi- Adverse Event: Unwanted effects
F D A’s accelerated approval re g u l a- track policies and pro c e d u res. In addi-tion, the agency must issue guidance Advisory Committee: A panel of
outside experts convened periodicallyto advise CDER on safety and effica-cy issues about drugs. CDER is notbound to take committee recom-mendations but usually does.
Amendment to an NDA: A submis-
Drug Products: The finished dosage
Investigational New Drug
Application, or IND: An applica-
Bioavailability: Rate and extent to
Drug Substance: The active ingredi-
Bioequivalence: Scientific basis on
New Drug: A drug first investigated
Effectiveness: The desired measure
New Drug Application, or NDA:
Clinical Trials: Human studies
Good Laboratory Practices, or
GLP: FDA guidelines governing the
and, for anti-infectives, microbiology.
New Molecular Entity, or NME: A
Incidence Rate: The rate at which
Compound: A chemical synthesized
of time in a given population at risk.
Parallel Track Mechanism: Policy
is evaluated for its biological activities d rugs for AIDS and other HIV- re l a t e d “parallel track” protocols, while the Dosage Form: The delivery system
Informed Consent: The voluntary
Dose: The amount of drug adminis-
Pharmacology: The science that
Priority Drugs: A drug that appears
Supplement: A marketing applica-
uct that already has an approvedNDA. CDER must approve all Phase 1: The first trials in humans
Raw Data: Researcher’s records of
the data at the researcher’s office.
Phase 2: Pilot studies to define effi-
Surrogate Endpoint: A laboratory
Risk: The probability of an event
likely to predict therapeutic benefit.
duration of effect during this phase.
the severity of harm that may occur.
Treatment IND: A mechanism that
Phase 3: Expanded clinical trials
Safety: No drug is completely safe or
lacking the potential for side effects.
ditions of use in the proposed labeling.
Phase 4: Studies performed after a
Safety Update Reports: Reports
User Fees: Charges to drug firms for
Postmarketing Surveillance: FDA’s
Side Effect: Any effect other than
from drug or nondrug treatment orintervention. Side effects may be Preclinical studies: Studies that test
man test systems. Since animals havea much shorter lifespan than humans, Stability: The drug product’s resis-
over an animal’s life cycle and on itsoffspring.


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