When you complete your clinical trial and review the results, how do you define success? Dr Michael Silverman, principal at BioStrategics Consulting Ltd., discusses the different aspects and relevance of clinical trial success.
The study is complete. All patients have been enrolled, treated and followed for the required amount of time, all data has been collected and recorded, all Clinical Report Forms (CRFs) have been filled out and audited, the database has been locked, and the statistical analysis is finished. Did the trial succeed?
Defining the success of a clinical trial can be a subtle and complex undertaking, wholly dependent upon the criteria used to determine success and the use to which the information is to be put.
In a large Pharma or Biotech company, it is likely there is a portfolio of areas in which the company is working, and in each area of product development, there are probably several compounds in development as part of the strategy for different disease areas or product types. A hypothetical company is illustrated in the table below, which represents a large biotech company with an extensive portfolio, and representative compounds are used as examples.
Clinical Trial Success Levels & Responsibility
1. Asset Management Strategy Success – Policy & Portfolio Decisions
2. Clinical Strategy Success – Clinical Strategies for Each Compound
3. Scientific Success – Protocol Design to answer Scientific Questions
4. Operational Success – Protocol execution, data collection, management and analysis
In this model, policy, along with the portfolio that the company will pursue, is set at the level of asset management strategy and flows down to the other levels. At the level of clinical strategy, management decides how each compound should be developed to achieve the goals set by the policy makers. The next tier is responsible for ensuring the clinical trial protocols are designed to address specific scientific questions and the managers below this level then ensure operational success by overseeing the running of the study, data collection, auditing and analysis. Essentially, clinical trial results are communicated upward in the model from the operational level to the asset management level. At each level the results take on a different context and drives different decisions. In each context, clinical trial results are evaluated for their conformance to success criteria relevant to that dimension. Thus, a trial that meets one audience’s criteria for success may fail dismally in another context.
For example, in an oncology program, a given trial may be operationally successful: there is a high degree of confidence that the data represent the true clinical effects of the drug, and that the data integrity has been maintained throughout the process. However, this confidence does not ensure scientific success. If the protocol called for an inappropriate patient population or insensitive assessment techniques, the protocol still may not meet its stated objective, it may show, for example, that the drug was not able to reduce the volume of the tumor targets. If the clinical trial is operationally inadequate, there is no hope of meeting its scientific objectives because the data will be deficient.
If the trial succeeds scientifically, there is still no guarantee of success on the clinical development dimension. If a hypothetical oncology trial met its stated objective of not having an effect on a specific tumor, the product would probably not advance to the next phase of the clinical strategy. On the other hand, if a clinically important effect were shown, clinical development success would be a given. Finally, a trial that fails to meet its scientific objectives is a failure from all perspectives. If the trial provides no conclusive results, it provides no useful information toward determining clinical development or asset management strategy.
By extension, it can easily be imagined that a trial that advances the clinical development strategy also benefits the asset management strategy. At the same time, the asset management dimension occupies a unique position in that it may succeed even if one, two, or all three of the subsidiary dimensions record a failure. For example, as part of asset management strategy, it may be stated that an oncology compound must enter Phase III trials by a given date or lose its place in the portfolio to another, more promising, oncology product. Such a strategic intent can be realized even if the product fails at other levels. If the deadline is not met, the plan will proceed with a different product, satisfying asset management goals. Lastly, if a trial succeeds at the operational, scientific and clinical development strategy dimensions, it will certainly succeed as a component of asset management strategy.
Dr Michael Silverman is the principal clinical consultant at BioStrategics Consulting Ltd., a clinical development consulting company based in Marblehead, MA, with business interests around the globe. Dr Silverman would be delighted to hear your thoughts on today’s blog, your experiences, or anecdotes and of course, your questions. To make comments or ask questions, click on this link.
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