Clinical Trials - Three Dimensions of Success

https://biostrategics.wordpress.com/2011/03/28/clinical-trials-three-dimensions-of-
success/

Posted on March 28, 2011 by BioStrategics Consulting Ltd

Dr Michael Silverman, principal at BioStrategics Consulting Ltd., talks about the

necessity to view the success of a clinical trial from three very different
perspectives, each of which has its own unique business implications, audience and
role in resource allocation decision making.

In my previous blogs, I have talked about the importance of clinical trial end points,
both in terms of the choice of measure, and as it relates to the phase of clinical
development.

I have also talked about the commercial considerations that need to be taken into
account when designing a study. In this blog, I would like to explore three very
divergent dimensions of clinical trial success. These three dimensions of success do not
operate in isolation, but are closely interwoven throughout the course of drug
development. They are:

Strategic Success
Scientific Success
Operational Success

Strategic Success
Strategic success incorporates those contributions that a given protocol makes to the
organizations overall asset management strategy, as well as its role in advancing the
compounds clinical development strategy.

Asset Management: A biopharmaceutical companys development pipeline is, in a very

real sense, a collection of assets, even if they have not fully realized their potential. It is
the responsibility of corporate management to maximize the value of these assets,
through an asset management strategy or reasoned priority setting, targeted resource
allocation, and efficient development activities. Every compound, every development
program, competes for resources with every other program. The key to good priority
setting and allocating resources is information: the crucial information being outcomes
of data from pre clinical studies and clinical trials of products within a portfolio of drugs
and studies. Which drug candidates show the most promising data, and are there enough
drug candidates in each phase of study to support getting one through to approval? The
audience here is clearly upper management.

Clinical Development Strategy: The clinical development process is mapped out with
project milestones, decision points, go/no-go criteria, and contingency plans, and is the
domain of management and its abilities to design the most appropriate work plan to
obtain data necessary for regulatory approval and competitive data that will convince
doctors of the drugs safety and efficacy, patients of its therapeutic value, and payors of
the overall health benefits, so as to maximize sales.

Scientific Success

In the scientific dimension, we consider the biological and pharmacological principles

that provide the scientific rationale for advancing a particular compound to the clinic.
Endeavors in the scientific dimension aim to establish the underling biology of the new
drug and how it translates into measurable and meaningful clinical benefit. Once
sufficient data is available to support moving into clinical trials, then clear clinical
protocol objectives are carefully written to express the scientific questions to be asked
of the new drug in a particular clinical trial. The answers to these questions are critical
to understanding the drugs activity in patients, its potential clinical utility and its
optimum use in the target population. They also bear directly on the products eventual
commercial success.

Operational Success

The operational dimension deals with the real nitty-gritty of the clinical trial, where the
trial is initiated and implemented, where researchers collect patient data, complete and
retrieve case report forms, create databases, and analyze results. It also involves
randomization, blinding, and measurement, and also powering studies, collecting
sufficient patients, staying on the timeline and within the budget. It is these areas that
take considerable thought and careful planning as each one can make or break the
success of the clinical trial. However, the primary objective of operational excellence in
clinical research is to ensure the integrity of the trial data. Without accurate data that
reliably and unequivocally reflect the patients clinical condition and the effect of drug
upon it, the trial results are meaningless. Success in the operational dimension means
the trial is conducted in a way that neither helps nor hinders the prospects of the drug;
the product is allowed to stand or fall based on its own inherent attributes. To achieve
this goal is to answer the question. But sometimes data integrity is not enough; many
trials fail to answer the question posed, because they were not powered correctly, or
insufficient thought was given to patient randomization or drop out. So to answer the
question posed, one must also ensure that data sufficiency will be achieved to allow
appropriate statistical analysis to be performed to arrive at an answer one can trust.

In summary

Strategic success, therefore, encompasses both positive and negative results as long as
the results lead clearly to the next step in the asset management strategy, be it
proceeding, terminating or switching priorities.

Scientific success means positive results that confirm the scientific hypothesis posed.

Operational success is contingent on the accuracy and reliability of the results and
having sufficient data to perform ones analysis.

I would be delighted to hear your thoughts on these issues, your approaches, or

anecdotes about decisions you have faced in similar circumstances. To make comments
or ask questions, click on this link.

For more information: go to my website or send me an email at

msilverman@biostrategics.com

Copyright 2011 BioStrategics Consulting Ltd.