Multiple Myeloma Support Group
Hamilton & District
Hamilton, Ontario, Canada


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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