Pharmaceutical Development and Scientific Skepticism

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What may be necessary when a proposed new medicine's mechanism is not covered by published regulatory guidance?

Conducting additional clinical trials

Working interactively with regulatory officials (correct)

Amending the manufacturing process

Submitting a new regulatory filing

What is critical in planning a development program?

Determining whether proposed studies will satisfy regulatory requirements (correct)

Determining the target market for the new medicine

Finalizing the label for the new medicine

Conducting clinical and nonclinical studies

What is crucial for approved medicines after regulatory approval?

Labeling and packaging

Prolonged clinical trials

Conducting post-marketing surveillance

Manufacturing according to high standards of purity and stability (correct)

Why may manufacturing be complex and expensive for some medicines?

Particularly for biological products (NBEs) Signup and view all the answers

What is essential for medicines developed for Alzheimer's disease?

Regulatory guidance on new medicines in this area Signup and view all the answers

When planning a development program, what should be determined?

Whether the proposed studies will satisfy regulatory requirements Signup and view all the answers

What is a common issue with drug development approaches?

Focusing too exclusively on obtaining evidence of drug efficacy Signup and view all the answers

What is the benefit of evaluating measures associated with drug mechanism/hypothesis?

It can open new avenues of research as well as close them Signup and view all the answers

What is the purpose of a 'check list' in drug development?

To optimize the translation of a drug candidate through the nonclinical and clinical stages Signup and view all the answers

Why is it essential to publish both positive and negative drug development studies and clinical trials?

So that investigators can learn from earlier failures and improve methods and practices Signup and view all the answers

What is the result of not publishing negative drug development studies and clinical trials?

The predictive value of preclinical models cannot be evaluated Signup and view all the answers

What is the goal of a successful drug discovery and development program?

To maximize the potential for success and translate discoveries into clinical practice benefits Signup and view all the answers

What is the main reason drug developers are skeptical of new scientific findings?

Because of the high investment required to pursue a new biological target Signup and view all the answers

What percentage of findings reported in reputable journals cannot be reproduced?

At least 90% Signup and view all the answers

What is the main concern for investigators proposing a new biological target?

That the findings may not be reproducible Signup and view all the answers

What is required for a biological target to be useful for drug development?

That it can be affected by molecules in a therapeutically useful way Signup and view all the answers

What properties must molecules have to be considered a medicine?

That they can act like a medicine when given to people Signup and view all the answers

Why do industry investigators attempt to replicate reported findings?

To ensure the findings are reliable Signup and view all the answers

What is the approximate percentage of compounds entering clinical development that eventually gets approved for marketing in the pharmaceutical industry?

8% Signup and view all the answers

What is the success rate of compounds that entered clinical development for AD between 2002 and 2012?

0.4% Signup and view all the answers

Why do companies continue to invest in AD therapeutics despite the high failure rate?

Because the unmet medical need is great and there are scientifically plausible targets Signup and view all the answers

What percentage of clinical trials for AD between 2002 and 2012 were supported by the pharmaceutical industry?

78% Signup and view all the answers

What type of drug is memantine, which is approved for AD?

Symptomatic drug Signup and view all the answers

Why is it beneficial for failures to occur early in development rather than late?

Because it reduces the high cost of failures Signup and view all the answers

What is a crucial factor in determining the financial viability of investment in a specific biological target?

The complexity of manufacturing Signup and view all the answers

What is the primary goal of 'personalized medicine'?

To identify the right patients for a specific medicine Signup and view all the answers

What may be necessary when developing a new medicine for a patient population not identified by current diagnostic practices?

Efforts to enable clinicians to identify the right patients Signup and view all the answers

What is the main purpose of the discovery phase in the drug discovery and development process?

To consider multiple molecules to yield a single approved medicine Signup and view all the answers

What is the outcome of the drug discovery and development process?

A single approved medicine Signup and view all the answers

What is a key challenge in developing new medicines for prodromal and preclinical Alzheimer's disease?

Developing new diagnostic approaches Signup and view all the answers