This document discusses rational drug use, essential drugs, fixed dose combinations (FDCs), over-the-counter (OTC) drugs, orphan drugs, and fast-track drug designation. It defines these terms and outlines their advantages and disadvantages. Rational drug use refers to using the appropriate drug, dose, form, route and cost for the right patient and diagnosis. Essential drugs are those necessary to address a population's health needs. Orphan drugs are treatments for rare diseases affecting less than 1% of the population. OTC drugs can be used safely without a prescription for minor conditions.

1. RATIONAL DRUG, ESSENTIAL DRUGS
FDC, OTC, ORPHAN DRUGS USE
presented By
Dr. Manoj Kumar
Assistant Professor
Department of Pharmacology
Adesh Medical College & Hospital Ambala Can’t

2. BY THE END OF THE SDL SESSION
You would be able to
 Know about Rational drug use
 Essential drugs
 FDC
 OTC
 ORPHAN DRUGS
 Fast-track drug designation
2

3. RATIONAL DRUGS USE (RDU)
 Use of the drugs for the best possible effect in the
shortest possible period and at a reasonable cost
 Right drug in
 Right dose in the
 Right dosage form by the
 Right route at the
 Right cost to the
 Right patient for the
 Right diagnosis 3

4. ESSENTIAL DRUGS
 Satisfy the priority health care needs of the population
 Must be available at all times, in adequate supply
 Available with assured quality, at a price the individual/
community can afford
 Intended to be flexible & adaptable to different situations
 Exactly which medicines are regarded as essential remains a
national responsibility
4

5. ESSENTIAL DRUGS
Selection criteria: Selected with due regard to
 Disease prevalence
 Evidence on efficacy & safety
 Comparative cost-effectiveness
5

6. ESSENTIAL MEDICATION LIST (EML)
 Minimum number of rational drugs and their dosage
forms which are necessary to cover preventive,
symptomatic and curative health needs of a majority of
population of that geographical area.
WHO
 1st in 1977: 208 drugs
 2019: 21st edition: 460 drugs
 Core list & complementary list 6

7. 8

8. NLEM (2015)
 1st in 1996
 376 medicines listed in NLEM 2015
 3 category included
 P→ Primary
 S → Secondary
 T → Tertiary
9

9. CRITERIA FOR INCLUSION OF A DRUG IN
NLEM
 Approved/licensed in India.
 Useful in disease which is a public health problem in
India.
 Have proven efficacy & safety profile based on valid
scientific evidence.
 Cost effective.
 Aligned with the current treatment guidelines for the
disease.
 Stable under the storage conditions in India.
 When more than one medicine are available from the
same therapeutic class, preferably one prototype/
medically best suited medicine & careful evaluation of
their relative safety, efficacy, cost-effectiveness. 10

10. .
 Price of total treatment to be considered and not the unit price of
a medicine.
 Fixed Dose Combinations (FDCs) are generally not included
unless the combination has unequivocally proven advantage
 Over individual ingredients administered separately, in terms of
increasing efficacy, reducing adverse effects and/or improving
compliance.
 The listing of medicine in NLEM is based according to the level
of health care, i.e. Primary (P), Secondary (S) and Tertiary (T)
because the treatment facilities, training, experience and
availability of health care personnel differ at these levels.
11

11. PURPOSE OF THE NLEM
 Guide safe & effective treatment of priority disease
conditions of a population
 Promote the rational use of medicines
 Optimize the available health resources of a country
12

12. ALSO BE A GUIDING DOCUMENT FOR:
 State governments to prepare their list of essential
medicines
 Procurement & supply of medicines in the public sector
 Reimbursement of cost of medicines by organizations to
its employees
 Reimbursement by insurance companies
 Identifying the ‘MUST KNOW’ domain for the teaching &
training of health care professionals
13

13. 14

14. EDL: HARYANA
15

15. FIXED DOSE COMBINATIONS (FDC)
 Product containing two or more active ingredients used
for a particular indication
 Combination of two different drugs in a single
pharmaceutical formulation
 Should have approximately equal t1/2 & the ratio of doses of
each component should depend on aVd & plasma
concentration
16

16. EXAMPLES
 Levodopa (t1/2: 1.7 hr) & Carbidopa (t1/2: 2 hr)
 Sulfadoxine (t1/2: 160 hr) & Pyrimethamine (t1/2: 112 hr)
 Amoxycillin (t1/2: 1-2 hr, aVd: 0.21 L/kg) & Clavulanic acid (t1/2: 1-
1.5 hr, aVd: 0.2 L/kg)
 Amoxycillin 500mg + Clavulanic acid 125mg can be administered as 1
to 2 tab 8hrly
 Sulfamethoxazole (t1/2: 11 h, aVd: 0.2 L/kg) & Trimethoprim
(t1/2: 10 h, aVd: 1-2 L/kg)
 Can be combined in their standard dose regimen
17

17. ADVANTAGES
 Convenient, Improves patient compliance
 Reduction in numberof pills
 Simplifies dosing
 Enhanced effect
 Allows for synergistic combinations
 Cotrimoxazole, Levodopa + carbidopa
 Reduced dose & adverse effects
 Prevents and/or slows attainment of antimicrobial
resistance
 Treatment of chronicinfectious diseases.eg- treatment ofTB,HIV
 Less expensive 18

18. DISADVANTAGE
 Dosing is inflexible & cannot be regulated to patient’s needs
 May showmore adverse effects
 Nimesulide + Paracetamol, Ramipril + Losartan
 It becomes difficult to identify one particular drug which is
causing harmful/beneficial effects
 FDC must be stopped & replaced by separate tablets
 More expensive
 May require bio-availability testing
 Potential quality problems, especially with rifampicin in FDCs for
TB
19

19. DISADVANTAGE CONT..
 Incompatible pharmacokinetics is irrational
 Different elimination t½ of individual components
 Incompatible pharmacodynamics
 Combination of antihistaminic & antidiarrhoeal is dangerous as
antihistaminic action may mask other symptoms
 Reaction of one of the components (e.g., a rash to
sulfamethoxazole in cotrimoxazole) may result in patient
avoiding the “innocent” trimethoprim in the future
 Drug interactions may lead to alteration of the therapeutic
effect 20

20. DISADVANTAGE CONT..
 One of the drugs in the combination maybe wasteful
 Vitamin + iron
 There will be increase in price if unnecessary drugs are
included.
 Ibuprofen + paracetamol + caffeine
 The dose of any component cannot be adjusted
independently
 Individual medicines may not be present in adequate
amounts e.g-multivitamins 21

21. OTC DRUGS
Drugs that are safe & effective for use by the general
public without a prescription
 Considered as minimally effective & safe
 Do not require approval by the physician
 Prescription drugs considered more potent &
frequently dangerous.

22. OTCS
 Indicated for
 Cough
 Diarrhoea
 Influenza
 Vomiting
 Allergy
 Gastric hyper-acidity
23

23. MOST COMMONLY USED FOR
• Aches & Pain (78%)
• Cough, cold (52%)
• Heartburn (45%)
• Constipation, diarrhea (37%)
• Skin care (10%)
• Multivitamins
24

24. OTCS
 Adverse effects: Generally mild
 Nausea, vomiting, diarrhea, dizziness, drowsiness
ADVANTAGES
• Allows the patient to have greater access to a
variety of drugs available in the market to treat
some medicinal conditions e.g.
 Cough
 Allergy
 Gastric hyper-acidity
 Pain 25

25. ADVANTAGES OF OTC DRUGS
 Benefits outweigh risks
 Low misuse & abuse potential
 Consumers are able to
 Self diagnose
 Self treat
 Self manage
 health practitioners are not needed.
 adequately labeled 26

26. ADEQUATELY LABELED
 Active ingredient
 Inactive ingredients
 Uses "indications"
 Warnings
 Directions
 Other information
 Questions or Comments
 Expiry date
27

27. DISADVANTAGES
o Poorer compliance
o More difficult to study drug effects
o Misdiagnosis may occur
o Risk of abuse
o Reduced opportunities to receive counseling about
possible lifestyle therapies (e.g. exercise & diet),
28

28. ORPHAN DISEASES
 All pathological conditions, affecting 0.65-1% out of
every 1000 population.
 Usually not studied for their patho-physiology or for
newer therapeutic options, as these are not
economically viable
 Orphan Drug Act, passed in 1983 by USA, to stimulate
the research, development, & approval of those
products that treat orphan diseases
29

29. ORPHAN DRUGS
Incentives for ODS
 Tax incentives for clinical research,
 Study design assistance from FDA ,
 Rebate from application-filing fees,
 Grant for Phase I and II clinical trials, and
 Seven years of marketing only after the approval of
the drug or biological product
30

30. USE ORPHAN DRUGS
31

31. ESSENTIAL VS ORPHAN DRUGS
.
32

32. FAST-TRACK DRUG DESIGNATION
 No therapy must exist for that specific condition,
 The new therapy must demonstrate an improved effect,
 Affect alternative outcomes other than the current therapy,
 Benefit patients unresponsive to current therapy,
 Avoid serious toxicities associated with current therapy, or
 Offer improved compliance and convenience compared to
current therapy.
33

33. 34