Midterm #1 Flashcards
1
Q
Pharmaceutical Biotechnology
A
- Any technology, advanced or otherwise, used to develop and test compounds-chemical or biologics- for therapeutic/clinical application
- Essentially it is the integration of science and know-how for biomedical applications
- Relationship to biopharmaceutics?
2
Q
Historical perspective of pharmaceutical biotechnology
A
- The path to new biopharmaceuticals
- Dramatic changes in the past 10-15 years
3
Q
What are biopharmaceuticals?
A
- Hormone (protein/peptide)
- Antibody (monoclonal/antiserum/immunoglobulin)
- Peptides
- Vaccines
- Cells
- Genetic material (nucleotides)
4
Q
Small MW drugs:
A
- pharmaceuticals
- small change>huge effects
5
Q
Biopharmaceuticals: change in amino acid sequences
A
- change in amino acid sequences > incremental effects
- The variants are still known by the same name and often for the same indication
- insulin, hep B recombinant vaccine
- Same volume, different dose
- Name different but interchangeably used
- Substitutions/replacements at the end of the insulin. Used for similar indications
6
Q
Biopharmaceuticals refer to, where as pharmaceutic refer to:
A
- Biopharmaceuticals:
- proteins
- macromolecules
- peptides >16-18 amino acids
- enzymes
- antibody
- hormone
- glycan or glycopolymers
- nucleic acid polymers (DNA/RNA/Aptamers/Oligonucleotides)
- Biologics
- Biomolecules
- Pharmaceutical
- Small molecule drugs
- Chemical derrivatives
7
Q
Protein Biopharmaceuticals are:
A
- different from small molecule drugs, foods, or supplements
- Products that successfully transformed from proteins into drugs require
- biopharmaceutic technologies and processes (Many steps)
- Government and private funding of research and development (time and money)
- Regulated by FDA through evolving review and regulatory standards
- Important and growing areas of knowledge for pharmacists to best serve patients
8
Q
Large vs. Small Distinction
A
- Insulin products (MW>1000; large) versus
- Chemical drugs (i.e. acylovir and ganiciclovir; terfenadine and fexofenadine) (MW<1000; small)
9
Q
Differences between small chemical compounds and large macromolecules
A
- MW
- Chemical: <1000
- Protein Drugs: >1500
- Route of Admin
- Chemical: All Routes
- Protein Drugs: Mostly Parenteral (IV/IM/SC, etc)
- Volume
- Chemical: Depends on hydrophobicity/protein binding
- Protein Drugs: Limited to blood volume for most proteins
- Elimination
- Chemical: Biotransformation
- Protein Drugs: Protein filtration/aggregation/receptor binding
- Immunogenicity
- Chemical: usually not important
- Protein Drugs: May play a significant role
- Production:
- Chemical: Synthesis
- Protein Drugs: Cell and complex processes
- Fidelity:
- Chemical: Excellent
- Protein Drugs: Challenging
- Master Stock: (Cells that make the product day in and day out)
- Chemical: Not required
- Protein Drugs: Required
- Purity:
- Chemical: Homogeneous
- Protein Drugs: Non-homogeneous
10
Q
Differences in drug approval process
A
- Macromolecules produced by recombinant cells or tissues are unlikely to be homogenous-free of trace materials
- Therefore, FDA has two branches
- CDER: Center for Drug Evalutation and Research
- **CBER: **Center for Biologics Evaluation and Research to review new drug applications
- **New Drug Application (NDA): **Small molecules
- Biologic Liscence Application (BLA): Macromolecules and some vaccines
- Product Liscense Application (PLA): Vaccine and blood products
11
Q
“Responsible Head”
A
- Head of all processes such as contamination
- Responsible head will go to jail
12
Q
The difference in regulatory reviews are getting smaller
A
- While BLA and NDA still exist, as better preparation and detection techniques are available, some “well-characterized protein products” such as antibody molecules are now reviewed by CDER
- FDA announced it intention to consolidate the reveiw of biotech products under CDER-interferon alfa-2b (Intron A) was review by CDER
- Even with well-characterized proteins, the cost of production and detection of purity and contaminants remains challenging
- It is not only “what’s in the bottle” but more importantly, what not detectable in trace elements continues to challenge us
13
Q
Consequence of biopharmaceutics on pharmacy practice
A
- FDA approval based on risk/benefit consideration-who is the risk manager?
- Public expectation on “absolute” safety is not always consistant with FDA’s position
- Some of the added safegaurds may increase clinical development time; thus it adds to the overall medication cost
14
Q
Epogen vs. Eprex-recombinant Epoetin-alpha
A
- A change in manufacturing and packaging procedures causes production of antibody against both the endogenous erythropoietin as well as the recombinant drug
- As a result, erythropoietin made by patients is neutralized and they no longer can make RBCs—requiring life-long transfusion
15
Q
Summary of Distinction between biopharmaceutical and pharmaceuticals
A
- There are significant differences between chemical compounds and large macromolecules
- Unique properties of proteins and macromolecules require appropriate pharmaceutics principles and tools for development, evaluation, approval, and safety monitoring
- Evolving public expectation and regulatory standards may have impacts on the drug approval, dispensing, management and overall costs
19
Q
New innovation in therapeutics are products of public and private collaboration and investments
A
