Editors: Bernard Ravina, MD; Jeffrey Cummngs, MD; Michael McDermott, PhD; and R. Michael Poole, MD
Publisher:  Cambridge University Press – 369 pages
Book Review by: Sonu Chandiram

Next year (2020) a quarter of the workforce in the United States is expected to be 55 years old or older. This has been described by demographers as The Silver Tsunami that began after the end of World War II.

This age group is more commonly referred to as the Baby Boom Generation. They are expected to number almost 98 million people and comprise nearly 29 percent of the entire US population, a rise from around 25 percent in 2010. Studies have shown that brain-related ailments, diseases and disorders are more prevalent among older people, and the demand for health care professionals who care for the elderly has been growing.

This book focuses on the analysis of clinical trials needed to understand neurological disease and their progression. Its aim is “to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases.”

What in essence, is a clinical trial?

The editors define it as “an experiment in humans that is designed to test a medical, surgical, biological, or other type of intervention.” They point out that a clinical trial does not presuppose a particular design, type of control group, or analysis plan.

So when you want design a clinical trial, use this book as your guide.

Forty-six specialists in neurology from all over the United States (including the four editors name above) and one from Canada authored the 29 chapters of this book that are organized around seven Sections. We name them below as an overview for you, of the contents:

  1. Section I – The role of clinical trials in therapy development
  2. The impact of clinical trials in neurology
  3. Sequence of clinical development
  4. Unique challenges in the development of therapies for neurological disorders
  5. Section II – Concepts in biostatistics and clinical measurement
  6. Fundamentals of biostatistics
  7. Bias and random error
  8. Approaches to data analysis
  9. Selecting outcome measures
  10. Section III – Special study designs and methods for data monitoring
  11. Selection and futility designs
  12. Adaptive designs across stages of therapeutic development
  13. Crossover designs
  14. Two-period designs for evaluation of disease-modifying treatments
  15. Enrichment designs
  16. Non-inferiority trials
  17. Monitoring of clinical trials: interim monitoring, data monitoring committees, and group sequential methods
  18. Clinical approaches to post-marketing drug safety approaches
  19. Section IV – Ethical issues
  20. Ethics in clinical trials involving the central nervous system: Risk, benefit, justice, and integrity
  21. The informed consent process: Compliance and beyond
  22. Section V – Regulatory Perspectives
  23. Evidentiary standards for neurological drugs and biologics approval
  24. Premarket review of neurological devices
  25. Section VI – Clinical trials in common neurological trials
  26. Parkinson’s disease
  27. Alzheimer’s disease
  28. Acute ischemic stroke
  29. Multiple sclerosis
  30. Amyotrophic lateral sclerosis
  31. Epilepsy
  32. Insomnia
  33. Section VII – Clinical trial planning and implementation
  34. Clinical trial planning : An academic and industry perspective
  35. Clinical trial implementation, analysis, and reporting: An academic and industry perspective
  36. Academic-industry collaborations and compliance issues

It is important for readers to know, as the editors write in the Preface and we quote them here: “There is no one trial design that meets all that meets all objectives for a particular phase of development. Rather there are parameters that need to be optimized for each intervention, question, and study.”

This work introduces you to the key concepts underlying trials in the neurosciences, especially if you are a clinical researcher in the pharmaceutical industry, neurologist, neuroscientist, neurosurgeon, psychiatrist or statistician.

This volume:

  • Tackles the challenges of developing therapies for neurological disorders, such as: Measurement of agents in the nervous system
  • Progression of clinical signs and symptoms
  • Illustrating specific study designs and their applications to different therapeutic areas
  • Covers key issues in phase I, II, and III clinical trials as well as post-marketing safety surveillance
  • Discusses topics such as:
    • Regulatory and implementation issues
    • Outcome measures, and
    • Common problems in drug development

In short, this is an excellent book on clinical trials particularly useful for the study of neurological disorders and diseases and their causes. So as we wrote earlier, when you want design a clinical trial, use this book as your guide.

 

Editors:

Bernard Ravina, MD is Medical Director of Translational Neurology at Biogen Idec in Boston, Massachusetts.

Jeffrey Cummings, MD is Director of the Mayo Clinic Lou Ruvo Center for Brain Health in Nevada, Ohio, and Florida.

Michael McDermott, PhD is Professor of Biostatistics and Professor of Neurology at Rochester University School of Medicine in Rochester, New York.

Michael Poole, MD is Head of CNS and Pain Innovation Medicine Unit at Astra Zeneca in Waltham, Massachusetts.