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Clinical Trials

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Session Chairs

  • Barbara C. Tilley
  • J. Donald Easton
Contact: John R. Marler

Start time 7:30 a.m.
End time 10:00 a.m.

Participants

Joseph P. Broderick
Stephen George
Philip V. Gorelick
Robert Hart
S. Claiborne Johnston
David Levy
David Nilasena
Marc Walton
Janet Wilterdink
Phillip A. Wolf

Agenda

Preamble

Overview of the Problem
What is clinical trials research trying to accomplish?

  • Our goal is long, healthy lives of high quality
  • We all have to die of something and if we keep everyone alive long enough they will surely will have a stroke, MI or cancer
  • Fatal strokes at age ≥ 85 are okay, but strokes cause premature death and that isn't okay
  • Disabling strokes cause morbidity and destroy quality of life. We must consider the cost of:
    • suffering & loss of dignity
    • lost careers
    • altered lives
  • Hospital care for strokes and cost of disabled people is ~$45 billion in the U.S. yearly

Overview of the Solution (to disability and lost quality of life)

  • We have many of the treatments at hand already but available they are not being utilized.
    • Reduce BP, cholesterol, tobacco and diabetes (weight)- together they account for ~85% of the excess morbidity &: mortality of stroke
    • Thrombolysis
    • Intervention (surgery/angioplasty or no surgery/angioplasty)
  • Why can't we implement them?
  • Do we need more research on how to succeed?
  • Should this receive high funding priority?

Thoughts

Clinical trials are the path from basic science to healthier people
Dollars spent on clinical trials:
-improve health
-are an investment in clinicians who are scientifically thinking and trained and who then provide better health care.
Focus on where the most benefit can accrue (not orphan diseases?, alternative therapies)
Focus on multidisciplinary efforts (NHLBI, NIA, HCFA, etc.)
Let's try to be clear that we know where we are going (let's go where the pharmaceutical industry won't go- e.g. EC-IC bypass surgery, carotid endarterectomy, anticoagulation v. antiplatelet therapy for stroke prevention, etc)

The greatest values of clinical trials are ??????? (list your thoughts)
How do we value basic research ("truth for its own sake") vis-à-vis clinical trials (treatment oriented; the path from basic science to healthier people)?

Problems with clinical trials
They are expensive to NINDS (but cheap to society if the result is healthcare cost saving and longer, high quality lives)
This raises the issue of innovative funding mechanisms (NIH succeeds, HCFA/Blue Cross/etc benefit) (Traystman & Hallenbeck)?
They often are not adequately science-based (most of the neuroprotection trials were not properly prepared/designed- perhaps this is a drug company issue)

Status of the Field of Stroke Treatment
The cause of stroke often is poorly understood and heterogeneous (does this suggest an opportunity?)
Precise pathophysiologic is poorly understood, even when the basic cause is known, e.g., atherothrombosis (endothelium, athero plaque, platelets, thrombus, etc.)
Prevention is substantially attainable- i.e. lifestyle behaviors, drugs, etc. ----but-----
Acute treatment is inadequate:
-recanalization (used inappropriately, dangerous,)
-prevention of re-thrombosis
-neuroprotection
Benefit/Risk-Cost is often uncertain/unstudied

Big Questions

  1. How to value clinical trials?
    • NIH Epidemiologic studies have been very valuable (morbidity & mortality are down)
    • NIH Aspirin Trials, EC/IC Bypass Study, NASCET, SPAF, BAATAF, NINDS-tPA were all worth their cost-let's not apologize for "large expensive trials"
  2. How to minimize expense (surrogates, co-funding, etc)?
  3. How to enter most eligible patients into clinical trials?
  4. How to prioritize funding (clinical trials vs. basic science RO1's; size of potential benefit to society)?

Big Problems/Challenges

  1. How will clinical trials be conducted in the future (Is research on this subject necessary/a high priority)?
  2. Should there be criteria for clinical trials?
  3. What about case control studies?

Scientific Priorities

  1. basic biology
  2. epidemiology
  3. detection & diagnosis
  4. treatment

Resource Priorities & Needs

  1. training for research investigators (basic and clinical)

Some questions that could be discussed:
What are the "ripe" areas for development for new treatments or new populations to treat (small vessel disease-intracerebral hemorrhage; cause and treatment, vascular dementia-what is it)?
What primary outcome measures should be collected in stroke clinical trials?
What should be the paradigms for development of a new treatment (Phase I, II, III, etc.)
How can epidemiology inform and be integrated with therapeutics?

Some issues that could be discussed:
Fostering new concepts in treatment
Choosing (is this our job?) the treatments to progress to Phase III trials from among many proposed new therapies including a discussion of Phase I and II trials as conducted in the field of cancer. (Ginsberg is doing a traditional Phase I, Broderick a traditional Phase II)
Choosing (is this our job?) among outcome measures including the question --- are there surrogate outcomes applicable to stroke research (Colin Begg could address both cancer Phase I-II trials as well as surrogate outcomes)
Studying "orphan" causes of stroke
Funding for post-trial analyses to inform future stroke research
New approaches to clinical trial design

Last updated February 09, 2005