Project #81503 - Pharmacy/Drug Design/Clinical Trials/


Drug Design


A) The design of clinical trials is important to obtain results that are reliable but must also provide benefit, or at least minimise harm to subjects that volunteer for the study.

The following questions relate to a paper titled:

A Phase I Study of PF-04449913, an Oral Hedgehog Inhibitor, in Patients with Advanced Solid Tumors

This paper is available from the following site,

Examine and discuss the clinical trial design focussing on the following criteria

1. The aim of the Phase 1 trial


2. Reasons for the number of patients in the trial at each dose


3. The inclusion criteria for subjects in the trial


4. The exclusion criteria for subjects in the trial


5. A rationale for the dosing levels chosen by the study designers


6. Any considerations that the study designers have not addressed in their design that you believe would give further information on toxicity


B) Find a Phase 1 clinical trial using the Scopus search engine which uses a 1+1 design. Write a brief summary (250 words or less) of the trial design, the objectives of the trial, whether the endpoint was reached and any obvious problems with the clinical trial.

C) Clinical trials are required for generic medicines entering the market once patent protection expires on registered medicines.

1. What 2 key features of a generic medicine must be demonstrated using clinical trials and other tests?


2. Explain a design for a bioequivalence trials and the benefits of the design you have described?


Part B

Pharmacoeconomics incorporates pricing considerations into the treatment of populations to allow objective choices to be made between differing therapies in light of the limited budgets available for medicine usage. The TGA website contains information of pricing paid to manufacturers which can be used in cost-benefit analysis calculations.

In this section you will do cost-benefit analysis on several drugs.

1. Use the literature to find life expectancy increases (disease free survival or time to progression) for a cancer treated with two current marketed drugs. You may use combination therapies in your comparison if single study values are not available.


2. Using literature values for quality of life as reported in the review by Tengs and Wallace (cancer, breast) (, and your life expectancy increases, calculate the quality adjusted life years gained through treatment with the drugs that you are comparing. There are many values for breast cancer that have been calculated so you will need to use your judgement to select one that matches your study providing life expectancy increases.


3. Give a short description of how the quality of life estimates that you used were calculated.


4. Using the information supplied on the TGA website on the price paid to manufacturers (Price ex-Man) and the dose required to treat an individual, which is the dose that provides the life extension reported in question 1, calculate the cost per quality adjusted life year for both drugs. (show your calculations)


5. Provide several reasons with explanations why your two drugs, although they cost significantly different amounts per quality adjusted life year have been approved for the treatment of breast cancer by the TGA.


Part C

Pharmacovigilance Plan

Scenario: A form of avian flu has been identified in a number of states in Australia and a new oral medication, Fluless, shown to ameliorate symptoms and reduce transmission of the virus, has recently been approved for widespread use. It is expected that there will be very high demand for the new medication. You are asked to contribute to the development of a pharmacovigilance plan to optimise safe introduction of Fluless.

What is meant by pharmacovigilance?

Briefly describe four of the most important elements for a Fluless pharmacovigilance plan and how they might be implemented. (limit 150 words per element).

Subject Science
Due By (Pacific Time) 09/27/2015 12:00 am
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