Authorisation & HTA Process Link

Question 1

Efficacy Def

Answer 1

Degree to which action(s) produce a clinically measurable effect under ideal conditions

Question 2

Effectiveness Def

Answer 2

Degree to which actions achieve intended health result under normal/usual circumstance

Question 3

Ideal Conditions Outline

Answer 3

Clinical trials aren’t accurate to real life. Lower risk of non-compliance (forgetfulness, business) doesn’t consider interactions/demographics

Question 4

Phase 1: New Medicine Clinical Trials

Answer 4

PK & PD data, dose range tolerated and Adverse event signals. Is there an immunogenic response

Question 5

Phase 2: New Medicine Clinical Trials

Answer 5

Dosing & efficacy in target population. Can be randomised and double blind

Question 6

Phase 3: New Medicine Clinical Trials

Answer 6

Randomised control trials, Comparative, Non-comparative (placebo controlled) and Non-inferior (evidence & efficacy eval). Ensures that results aren’t effected by other conditions (eg chance)

Question 7

Phase 4: New Medicine Clinical Trials

Answer 7

Post marketing and RMP requirements

Question 8

What are the limitations of clinical trials

Answer 8

Only monitors adverse events for as long as trial exists

Question 9

Clinical Trials Information System (CTIS, 2023)

Answer 9

Digital portal for application of investigational medicinal product clinical trial by sponsor

Question 10

Considerations of Trial Pre-Authorisation

Answer 10

Ensure trial is necessary. Not redundant, is result generation actually feasible. Can’t ask people to take unnecessary risk

Question 11

Independent Ethic Committees

Answer 11

Safeguards rights and ethics of subjects. Minimise participant risk exposure, review protocols & procedures, monitor patient info, verifies suitability

Question 12

Trial Investigator Roles & Responsibility

Answer 12

Person conducting study day-to-day; recruit patients/staff/resources in timeframe, ensure protocol compliance. Ensures appropriate medical care is received during/after trial and documenting subjects withdrawal

Question 13

Motivator for Patients To Join Clinical Trials

Answer 13

Access to promising new novel or standard of care (placebo)

Question 14

EMA Centralised Authorized medicine steps

Answer 14

R&D (EMA can compel topic), Scientific Advice (supports high quality & safe medicines), Evaluation (CHMP pre-submission guidance), authorisation, access and safety monitoring

Question 15

Common Technical Document (CTD) Includes

Answer 15

Target Patient Group (unmet need), Medicine Quality (Physical, Chemical & Biological), GMP compliance, Pharmacology, Pharmacokinetics, Benefits & AE, post authorisation info

Question 16

CHMP Evaluation Process

Answer 16

Compulsive for medical products with new API. Rapporteur MS appointed with a team of assessors (EMA assessment report)

Question 17

3 Pillars of Medicine Eval

Answer 17

Safety, Efficacy and Quality

Question 18

ICH Good Clinical Practice Guidelines

Answer 18

Ethics, risk vs trail, Participant rights, Medical Product Info, Quality Protocol, Protocol Compliance, Medical Decisions, Trial Staff, Informed Consent, Clinical Data, Confidentiality, Good Manufacturing Practice and Quality Assurance

Question 19

EMA Risk Management

Answer 19

Identified risks, Potential risks and Information missing from study

Question 20

When is liscence accelerated

Answer 20

Lack of alternatives

Question 21

Access Steps

Answer 21

CHMP responsible for; quality, safety and efficacy. Liscenced Indication SmPC. MA tracks pathway to marketing with few restrctions

Question 22

Health Technology Assessment

Answer 22

Cost vs value for money. Value derived from inervention assessed by decision makers. Health Promotions

Question 23

HTA Key Principles

Answer 23

Clinical Issue Assessment, Cost-effectiveness, budget impact, decision maker recommendations and post imbursement