Describe different phases of clinical trials and discuss differences in the appr

Question

Describe different phases of clinical trials and discuss differences in the approach for testing a “New” and a “Re-purpose drug”

Your answer should include:

Phase [number] 0, I, II, III          

Basic description of different

Aim of the phase – e.g. toxicity or efficacy

Target participant? [healthy or diseased person]

Approximate number of participants

How long the trial would take

Benefit to the participant?

Differences in the approach for testing a “New” and “Re-purpose drug” - Regulations and GCP
Advantages of taking a repurposed drug through trials: Higher possibility of approval. Possibility of skipping phases – why? Which drugs? Why this is good? Costs involved, time savings
Phase design (e.g. fuller description of RCT with controls and reason for controls – bias & confounding. Description of gold standard RCT – advantages disadvantages).
Definition of clinical trial
Definition of repurposed drug.
Drug repurposing discovery – on-target (medium novelty); off-target (high novelty)

Explanation / Answer

The clinical trials includes the 5 phases like phase0, phase1, phase2, phase3, phase4. phase0 also called microdosing. Micodosing is the 1/1000 of dose of test substance calculated to produce the pharmacological action.

The purpose of Phase 1 is to ensure that the treatment is safe in humans and to determine how and where it distributes within the body. This testing normally takes place with a small group of healthy volunteers.At the end of Phase 1, the results are collected, analyzed, and submitted to the FDA for permission to proceed to Phase 2 Clinical Trials. The purpose of a Phase 2 Clinical Trial is to determine the right dosage and effectiveness in treating that particular disease. This testing normally takes place with a larger number of volunteers who have the disease.

However, if the results show that the treatment did not work better than the current standard of care or even caused acceleration of the disease or other unexpected serious adverse events, the FDA may not give permission to proceed to Phase 3. A Phase 3 Clinical Trial involves a much larger group of volunteers and primarily focuses on determining whether the treatment* would be safe and effective for a wide variety of people. The plan normally involves assigning participants to treatment or control groups. After approval by the FDA and manufacturing of the drug on a large scale by the sponsor, the process enters what is called Phase 4 Clinical Trial/Post-Market Surveillance/Report Adverse Events.

Drug repositioning (also known as drug repurposing or drug reprofiling) is the process of redeveloping a compound for use in a different disease. This approach capitalizes on the fact that approved drugs and many abandoned compounds have already been tested in humans and detailed information is available on their pharmacology, formulation, dose, and potential toxicity. Drug repositioning is underpinned by the fact that common molecular pathways contribute to many different diseases. Before a commercial entity will move forward with a generic drug repurposing clinical trial, there are three key issues it needs to evaluate. First, there needs to be a possibility for significant financial profit. Several factors contribute to profit potential, including patent protection (arising from modifications to the generic drug), market exclusivity, and/or manufacturing exclusivity. If patent/exclusivity is possible, then the commercial entity next gathers additional information to determine the potential return on investment (ROI) of the clinical trial. Finally, the commercial entity will determine if the specific opportunity fits into its portfolio and aligns with its business goals. If any of the decision points above is negative, then the project normally stops. A commercial entity is unlikely to fund or seek funding for a clinical trial simply to provide the support for physician off-label use.

Identify the most promising drugs for further clinical investigation;

Review drug-related data and bring it to the attention of clinical investigators;

Document how these drugs can be combined with existing therapies or other repurposed drugs;

Develop clinical trials to provide positive or negative evidence of efficacy;

Suggest areas where further preclinical work is necessary.

in some cases during the clinical trials the pharmaceutical committiees appoints some technical comittee to decide whwthwr to skip the clinical trials. many drugs like cardiovascular drugs , cancer treatment drugs, muscular dystrophy donot need the phase2 clinical trial and they can enter directly into the phase3 trials. This decreses the time and facilitates the drug availability in the market.

Approximate number of participants;Phase 0 trials are optional first-in-human trials 10-15 persons

Phase1 Often the first-in-man trials. Testing within a small group of people (20–80)

Phase2 Testing with a larger group of people (100–300)

Phase3 Testing with large groups of people (1,000–3,000)

Description of gold standard RCT – advantages disadvantages:

In an evidence-based approach to evaluation of effectiveness, the randomized controlled trial (RCT) is the acknowledged standard and is considered to generate the highest level of evidence, followed by controlled trials. Trials without controls, case series, case reports, and finally expert opinions, generate low or insignificant evidence.

advantages

Disadvantages:

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