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PHRM 100 – PPHC > PPHC 12: Drugs – How do drugs go from molecule to market? > Flashcards

PPHC 12: Drugs – How do drugs go from molecule to market? Flashcards

1
Q

What are the 4 hurdles of drug approval?

A
  • quality – phase I-III trials
  • safety – phase I-III trials
  • efficacy – phase I-III trials
  • value – phase IV trials
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2
Q

Regulatory Approval Process

How is value assessed?

A

phase IV trials

  • effectiveness in real world, cost-effectiveness
  • health technology assessment – consider: epidemiology (evidence-effectiveness), health economics (cost-effectiveness), other considerations
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3
Q

List the different parts of the regulatory approval process.

A

pre-market:

  • drug discovery
  • pre-clinical – find something new
  • clinical trials – phase I (safety and dosage), phase II (efficacy and dosing), phase III (confirm efficacy and safety)
  • approval – can it be sold in Canada

post-market:

  • CDA submission – does it look good value in Canada (CDR/DEC review, HTA)
  • provincial submission – should we provide it (HTA)
  • formulary listing – can we get a better price (pCPA negotiation), can we avoid being exploited (PMPRB review)
  • phase IV/post-market surveillance clinical trial – long-term safety monitoring

(outcome: patent expiry, generic/biosimilar entry)

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4
Q

Regulatory Approval Process – Pre-market

Drug Discovery

A

at this stage, want to protect these discoveries

  • file patents (grants exclusivity)
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5
Q

Regulatory Approval Process – Pre-market

Clinical Trials

A

phase I-III

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6
Q

Regulatory Approval Process – Pre-market

What is the phase I clinical trial?

A

safety and dosage – is it safe

  • first in human – ~20-100 volunteers who do not necessarily have the disease/condition
  • open label, uncontrolled
  • safety – potential risks, side effects, safe dosing range
  • tolerability/dosing range – balancing intended effects with minimal side effects
  • pharmacokinetics and pharmacodynamics – how drug is absorbed, distributed, metabolized, and excreted into the body, biological effects
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7
Q

Regulatory Approval Process – Pre-market

What is the phase II clinical trial?

A

efficacy and dosing – does it work

  • very strict inclusion criteria for participants – ~30-300 patients who have the disease or condition
  • further evidence on efficacy, optimal dosing and safety
  • efficacy – does it work on important outcomes compared with placebo or another standard treatment
  • dose optimization – balancing efficacy and tolerability (inform design on phase III trials)
  • safety – looking for side effects in a larger group of people (identify rarer side effects)
  • therapeutic exploratory – evidence of efficacy for a particular indication
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8
Q

Regulatory Approval Process – Pre-market

What is the phase III clinical trial?

A

confirm efficacy, safety – is it better than what we have

  • larger number and diversity of participants – ~300+ patients, often in multiple sites/countries
  • compare safety and efficacy against the current standard of treatment
  • confirming efficacy – drug works in this population, statistically significant benefits
  • comparative analysis – is it better than existing treatment or placebo
  • likely to have randomization and blinding
  • expensive and time-consuming – last step before applying to regulatory agencies (ie. Health Canada)
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9
Q

Regulatory Approval Process – Pre-market

Approval

A

Health Canada submission

  • result is 1 drug with NOC
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10
Q

Regulatory Approval Process – Post-market

CDA Submission

A

CDR/CDEC review

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11
Q

Regulatory Approval Process – Post-market

Provincial Submission

A

Drug Benefit Council (DBC)

  • review after evaluation by CDA/CADTH CDR – supported by the CDA reviews/recommendation
  • possibly include individual components (ie. ethics, environment)
  • recommendations (to the Leadership Council in the Provincial Ministry of Health): list, list but not at listed price, do not list
  • consistency across hospitals – General Hospital Formulary
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12
Q

Regulatory Approval Process – Post-market

Formulary Listing

A

pCPA negotiation, PMPRB review

  • list of prescription drugs covered by a health insurance plan
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13
Q

Regulatory Approval Process – Post-market

What is the phase IV / post-market surveillance clinical trial?

A

long-term safety monitoring – are there any unexpected safety effects in the real world

  • primary goal is to assess the drug’s long-term safety and effectiveness in the general population
  • long-term and rare/delayed safety signals – rare but serious adverse events, chronic toxicity
  • effectiveness in real-world setting (vs. efficacy established in phase II): use in special groups (ie. children, pregnancy, elderly), potential uses in new indications
  • open-label, non-randomized, no eligibility restrictions – not for all treatments
  • may include thousands of people for long periods of time
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14
Q

Regulatory Approval Process – Post-market

Patent Expiry
Generic/Biosimilar Entry

A

–

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15
Q

What is the order of the government/provincial bodies in the regulatory approval process?

A
  • Health Canada
  • Canada’s Drug Agency (CDA)
  • pan-Canadian pharmaceutical alliance (pCPA)
  • patented medicine prices review board (PMPRB)
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16
Q

What is the purpose of Health Canada?

A

notice of compliance (NOC)

  • can the drug legally be sold in Canada (quality, safety, efficacy)
17
Q

Health Canada

What is the goal of Health Canada in drug approval?

A

Notice of Compliance (NOC)

  • confirmation a drug meets regulatory standards of safety, efficacy, and quality
  • means that drugs can become commercially available (can legally be marketed and sold in Canada) for a specific indication (but opens up to off-label use)
  • product is entered onto the Drug Product Database
18
Q

Health Canada

What does the Therapeutics Products Directorate do?

A
  • looks at evidence of pivotal trials (usually at least 4–5)
  • need to be convinced that benefits outweigh risk
19
Q

What is the purpose of Canada’s Drug Agency (CDA)?

A

common drug review (CDR)

  • does the drug look good value for formularly listing by the provinces
20
Q

Canada’s Drug Agency (CDA)

What does the CDA do?

A

assessment of (commercially available) drugs (procedures, devices):

  • effectiveness
  • value for money (reasonable cost) – clinical, economic, patient and clinician evidence
  • equitable use
21
Q

Canada’s Drug Agency (CDA)

What is the audience?

A
  • federal/provincial/territorial drug plans
  • provincial cancer agencies
  • Canadian Blood Services
  • hospital
  • private payers
22
Q

Who conducts a common drug review (CDR)?

A

Canada’s Drug Agency (CDA)

23
Q

Canada’s Drug Agency (CDA)

What is the common drug review (CDR)?

A

purpose is to assess the effectiveness and cost-effectiveness of new drugs for public drug plans

  • triggered by manufacturer
  • pan-Canadian process for conducting objective, rigorous reviews of the clinical, cost-effectiveness, and patient evidence for drugs
  • provides formulary listing recommendations to Canada’s publicly funded drug plans (except Quebec)
24
Q

Canada’s Drug Agency (CDA)

What are the objectives of the common drug review (CDR)?

A
  • reduce duplication of reviews by jurisdictions
  • provide equal access to timely, evidence-based information and expert advice
  • consolidate the submission filing process for pharmaceutical manufacturers
25
Q

Canada’s Drug Agency (CDA)

What is the outcome of the CDA’s review?

A
  • recommendations: reimburse, reimburse with conditions, do not reimburse
  • note: recommendations are recommendations – they are non-binding
26
Q

Does Canada affect drug prices around the world?

A

NO – Canada is a relatively small market (in the world) and has little power to influence on price

  • within Canada, we effectively have 13 health systems diluting this further – inconsistency between provinces
27
Q

What is the purpose of the pan-Canadian pharmaceutical alliance (pCPA)?

A

can interested provinces group together to negotiate a better price

28
Q

pan-Canadian Pharmaceutical Alliance (pCPA)

What does the pCPA do?

A

allows provincial, territorial and federal governments to jointly negotiate for brand name and generic drugs in Canada

  • better power combined negotiating power
29
Q

pan-Canadian Pharmaceutical Alliance (pCPA)

What are pricing decisions based on?

A

pricing decisions are a precondition of having a medication covered on provincial drug formularies

  • last stage before public plans decide whether or not to list
30
Q

pan-Canadian Pharmaceutical Alliance (pCPA)

Process Diagram

A

see notes

31
Q

What is the purpose of the patented medicine prices review board (PMPRB)?

A

have prices of approved drugs remained reasonable

32
Q

Patented Medicine Prices Review Board (PMPRB)

What does the PMPRB do?

A
  • monitors the prices charged by patentees for patented drugs on an ongoing basis – patentees file information about price and sales at introduction and every 6 months for all forms of the patented drug sold in Canada
  • tries to ensure the prices of patented medicines in Canada are not excessive
  • prices relative to prices in other markets – fines to compensate for the high prices

PHRM 100 – PPHC (18 decks)

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