|
|
Multiple Myeloma Support Group |
|
|
Saturday, March 11, 2007 MEETING Lori welcomed everyone to the meeting. John Duffy, a group member, belongs to The Steeltown Toastmasters. They give Inspirational speeches. John entertained us on the topic of "Cheaper by the half dozen". John gave an anecdotal account of some of his life experiences including his arrival to Canada in 1950, his marriage in 1956, and the subsequent arrival of his six children. Raising six children was apparently cheaper than decorating! He ended with a thought of the week which came from Yogi Beara - When you find yourself travelling along life's highway and you come to a fork in the road, take it! Lori thanked John and introduced Dr. Marcellus. The topic was Clinical Trials. What is a clinical trial? Where does it fit in? Different types of trials. Examples of trials. Participation What - The observation/ intervention of any research study where human volunteers are involved in answering a specific question. Observation - asks a question about health issues in large groups of people - nothing specific is being to done to individuals Intervention - new approaches to treatments - something specific happens to individuals Lab work - research takes place to develop new drugs and find new benefits for existing drugs. Clinical Observation - interest must be developed in order to generate research - "Protocol" is written - a final product is submitted to IRB/REB (Institutional Review Board / Research Ethics Board) Theses committees are made up of doctors, researchers, and lay people. They determine whether the submission is ethical and safe. It then goes for initial approval and periodic review, where adverse event reports are reviewed. Safety - Ethical and Legal codes are established - all are reviewed by IRB/REB Funding - a funding source is established - a team is put together - potential participants are approached - enrollment begins Who may participate? Process of obtaining informed consent. How many can participate? Scheduling Length of study. Who - Inclusion /Exclusion Criteria - Inclusion involves diagnosis, stage of disease, prior treatment, age, and gender - Exclusion involves other medical problems Informed Consent - The process of learning key facts about the trial - The consent form outlines the purpose of the study, what's involved, risks, potential benefits, who to contact with questions, and the right to withdraw at any time. Number of Participants - This depends on the question being asked. - How frequent an event might occur in both groups and how big a difference would be needed between groups. This is a "sample size" Schedule - When and what the tests do - Exact treatments - When visits are required - How long the individual should be followed - What information is gained Goals - Treatment Trials - test new drugs - Prevention - better ways to prevent diseases - Diagnostic - screening trials - search for better tests to be used in diagnosing for a particular disease - quality of life - explore ways to improve an individual's comfort PHASE I Trial study Testing drug Metabolism and pharmacokinetics Evaluate safety - dose and side effects Early evidence of effectiveness Small numbers of patients treated PHASE II Larger group Test to gauge effectiveness Gathers more information about the safety of new drug(s) PHASE III Larger numbers Comparing new to previous drugs or placebos Confirming effectiveness to previous drugs Gathering more information on the side effects and safety Placebo = an inactive pill, liquid, powder that has no treatment value Placebo effect = a physical or emotional change that happens just because the patient is taking something Randomized Trial - an individual is chosen by chance for one group or another - provides a balance in the groups RANDOMIZATION The participant is assigned to treatment Differences occur by chance The differences are not due to systematic or bias BIAS "Point of view" prevents impartial judgement Bias is controlled by Randomization and Blinding BLINDING Single Blinded - One party is unaware of what drug is being taken Double Blinded - Neither participant or staff know which treatment is received OPEN LABEL TRIAL Both participant and doctor know which treatment is received STATISTICAL SIGNIFICANCE If the difference could have occurred by chance alone in less than 1 time in 20 CLINICAL SIGNIFICANCE This has little to do with statistics. It is a matter of judgement and answers the question "is the difference between groups large enough to be worth achieving"? Studies can be statistically significant yet clinically insignificant. PIVOTAL TRIAL Controlled trial used by a drug company to apply for the drug to be approved for use. FDA - United States HPB - Canada PHASE IV Specific Design - what happens after the drug is on the market - more information - risks, benefits and best use of the drug APPROVAL PROCESS Pre-Clinical - lab and animal studies Phase I - III - human volunteers Filing - New Drug Application by manufacturers with FDA/HPB Review Application Approval or Rejection of Application EXPANDED ACCESS PROTOCOL Allows regulated use of an unapproved drug Patients may benefit but don't fit into a clinical trial PAMIDRONATE - detailed discussion regarding a 10 year old study took place TRANSPLANT - changed as a consequence of 1 study - changed in an amazing way REVLAMID/THALIDAMIDE - good example as to why clinical studies are imperative - no animal studies were used with Thalidamide - went straight to human volunteers - labs worked to keep the good parts of Thalidamide and get rid of the bad - resulted in Revlamid and more studies - very tight control today PARTICIPATING IN CLINICAL TRIALS Opportunity Detailed Information Weighing Benefits and Risks Informed Consent DECIDING Benefits - gaining access to new drugs not otherwise available - more active in ones own health - helping to advance medicine Risks - side effects - not effective - more tests, visits, paperwork QUESTION PERIOD Lori thanked Dr. Marcellus for joining in the group today. Cancer Rehab - Jodi returns next month from maternity leave. There is a Rehab facility in Caledonia called Haldimand Physio Centre. ASH CONFERENCE Hematologists - Doctors are seeing a better survival rate with the median being 7.3 years as opposed to the prior 3 - 5 years. Studies over the next five years will confirm this finding. Celgene - very confident about Revlamid and Thalidamide being approved in the next few years - communication with a group who denounce it's value is going very well. NEXT MEETING Saturday May, 12, 2007
|