2012-09-30 16:17:26 - Recently published research from Datamonitor, "Strategies for Comparative Efficacy Trials", is now available at Fast Market Research

Clinicians and patients want to be able to compare new drugs with those already available to inform evidence-based medicine, and reimbursement authorities require comparative data to support their decisions. This report discusses the role of comparative efficacy trials and the challenges in their design, with particular attention to the question of comparator selection.

Scope
* Understand the role that comparative efficacy plays in the evaluation of new drugs.
* Review the various types of trial design that can provide comparative data and assess the purpose for which each is most suited.
* Identify decision-making processes for guiding key choices in the design of pivotal comparative efficacy trials.
* Understand the importance of comparator selection and the consequences of later disagreements with

external authorities.
* Review the factors that need to be considered in making a comparator selection.


Full Report Details at
- www.fastmr.com/prod/464862_strategies_for_comparative_efficacy_t ..


Highlights

The choice of active control group for a comparative trial can be difficult due to variation in the standard of care across different geographic regions and changing medical practices.

Numerous recent appraisals by the UK's National Institute for Health and Clinical Excellence (NICE) and Germany's Institut fur Qualitat und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) have demonstrated the critical stance that is being readily taken by these bodies with regard to comparator selection.

Scientific advice from regulators and, where possible, from health technology assessors or reimbursement authorities can be extremely useful in guiding clinical development planning, especially in the choice of comparator.

Reasons to Get this Report
* What is driving the use of comparative efficacy trials and what role do they play in approval and reimbursement decisions?
* How should an active comparator and other features of comparative efficacy trials be chosen?
* Can the range of comparators be expected to extend beyond pharmaceutical interventions?
* Which regulatory and reimbursement authorities are willing to offer scientific advice in the design of a comparative efficacy trial?
* To what extent have drug evaluation committees shown flexibility in disagreements about the optimum comparator?

Partial Table of Contents:

EXECUTIVE SUMMARY

* Key findings

The impetus to conduct comparative efficacy trials

* Summary
* Availability of comparative data during regulatory review
* Addressing uncertainty

* FDA
* EMA
* Differences in approach in the US and EU
* Research ethics

* Efficacy or effectiveness: when should comparative trials be conducted?

* Case study: antihypertensives

Clinical trial designs

* Summary
* Introduction
* Superiority trials
* Non-inferiority trials

* FDA versus EMA guidance
* Comparative efficacy and biocreep

* Three-arm trials
* Adaptive trials
* Subgroup analysis

* Case study: subgroup analysis within a non-inferiority trial

* Placebo effects
* Biosimilars
* Pragmatic clinical trials
* Observational studies
* Network meta-analyses
* Simultaneous consideration of benefits and harms
* Conclusions

Trial optimization

* Summary
* Introduction
* Recent examples of challenges in comparator selection

* AstraZeneca's Faslodex rejected by NICE on the basis of indirect comparison
* IQWiG and NICE judge GlaxoSmithKline's Benlysta to have been inadequately compared
* Biogen Idec's Fampyra highlights challenges of comparison with non-pharmacological treatment
* IQWiG disagrees with GlaxoSmithKline regarding comparators for Trobalt
* Eisai's Halaven considered inadequately compared despite following EMA protocol
* Comparison with experimental treatments not demanded by the FDA but view of reimbursers is uncertain
* A late-arriving statin has adequate comparator data for regulatory approval but proving differentiation is another matter

* Selection of active control group for the comparative trial

* Use of existing knowledge
* Decision-making techniques
* Reflecting on the range of interventions

* Patient populations, outcomes, and other study variables
* Scientific advice
* Conclusions

Outlook

* Summary
* Introduction
* Comparative efficacy: a regulatory requirement?
* Regulators and reimbursement authorities: working together

* Progressive approvals

* Conclusion

Appendix

* Methodology

* Acknowledgements

* Glossary
* Abbreviations
* References
* Disclaimer

TABLES

* Table: New medicines with premarketing randomized active-controlled trials (1995-2005)
* Table: New medicines without premarketing randomized active-controlled trials (1995-2005)
* Table: Trials described in approval packages in the US and EU (2005-07)
* Table: Comparator information available for drugs approved in the EU (2009-10)
* Table: New medicines (1999-2005) with an improved efficacy
* Table: Therapeutic areas in which response to placebo varies widely between trials
* Table: Advantages and disadvantages of using adaptive clinical trial designs
* Table: Sample sizes for a three-arm randomized controlled trial or an adaptive Phase III/IV trial
* Table: Possible trial designs providing comparative efficacy data
*

Full Table of Contents is available at:
-- www.fastmr.com/catalog/product.aspx?productid=464862&dt=t

About Datamonitor

The Datamonitor Group is a world-leading provider of premium global business information, delivering independent data, analysis and opinion across the Automotive, Consumer Markets, Energy & Utilities, Financial Services, Logistics & Express, Pharmaceutical & Healthcare, Retail, Technology and Telecoms industries. Datamonitor's market intelligence products and services ensure that you will achieve your desired commercial goals by giving you the insight you need to best respond to your competitive environment. View more research from Datamonitor at www.fastmr.com/catalog/publishers.aspx?pubid=1002

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