An Educational Webcast for Patients

PresentersCatherine Gregor, MBA, CCRP, CCRCProject ManagerResearchMatch

Leslie R. Boone, MPHTranslational Research Coordinator Vanderbilt University Medical Center

Today’s Objectives• Discuss the role of clinical trials in advancing

research• Review the methods and process of a trial,

including the phases of a study• Review common features of trials for inflammatory

bowel diseases• Discuss resources for more information

What is a clinical trial?• Medical research studies that look for:

– New drugs or new combinations of drugs – New procedures or devices – New ways to use existing treatments – Types of care, such as improving the quality of life for

people with chronic illnesses• Also known as interventional study or clinical study• Determine safety and effectiveness

The process from the start of basic research to developing new drugs, treatments, or finding cures

takes an average of 13-20 years

2015… …2032

Lab Clinical Trials

Patient Care

10,000compounds

250 Compounds

5 Compounds1 drug approval

Drug Discovery Pre-Clinical Clinical Trials FDA Review Clinic

6.5 years 1.5 years6 years

What is the hold up?

What is the hold up?• 50% of clinical research sites enroll one or no

patients in their studies.4

• Eighty percent of total trials are delayed at least one month because of unfulfilled enrollment.2

• Each day a drug is delayed from market, sponsors lose up to $8 million.5

All statistics from The Center for Information & Study on Clinical Research Participation: http://www.ciscrp.org1. Beasely, "Recruiting". 2008 2. Lamberti, "State of Clinical Trials Industry", 292 3. Pierre, "Recruitment and Retention". 2006 4. Beasely, "Recruiting". 2008

Imagine if the whole process took closer to 5 years

2015… …2020?That’s much better!

Lab Clinical Trials

Patient Care

Insert Poll• Have you participated (or currently participating) in

a clinical trial? Yes No

Insert Poll 2• If you answered no, what would you say is the main

reason? No study in my area I do not meet criteria for a study I’m afraid of the risks involved I do not feel knowledgeable about clinical trials I am content with my treatments Other

Why Don’t People Participate?• Lack of opportunity• Lack of knowledge• Lack of comfort

Purpose Compensation, treatment for injury Description Whom to contact Procedures Right to refuse or withdraw Risks Confidentiality Benefits Clinicaltrials.gov (if applicable) Alternatives

Deciding Whether to Participate: Informed Consent

– Food and Drug Administration (FDA) requires that volunteers are given information before they participate

– Informed Consent form includes:

Six Additional Elements• Statement that there may be risks which are unforeseeable• Under what circumstances investigator could terminate your

participation• Additional costs to you• Consequences of your withdrawal from the study• When will research findings be disclosed to you• Approximate number of people participating in the study• You can still decide to withdraw from the study at any time

How do trials work? Study Methods

• 2 or more treatments assigned by chanceRandomized

• Active drug or placebo?Double blind

• Inactive product resembles study drug

• Continue course of standard treatment

Placebo

Phases of Clinical Trial: Pre-Clinical• In vitro (test tube or cell culture) and in

vivo (animal) experiments • Wide-ranging doses to obtain

preliminary data on effectiveness, toxicity and movement of drug within body

• Determine if potential drug has scientific merit for further development as investigational new drug (IND)

Phase 1 Phase 2

Phase 4 Phase 3

Pre-Clinical

Phase 1• Determine safe dose• How does the body work? Phase 2

Phase 4 Phase 3

Pre-Clinical

Phase 1• Determine safe dose• How does the body work?

Phase 2• Safety and Effectiveness• Any short-term side effects?• Narrow criteria selection

Phase 4 Phase 3

Pre-Clinical

Phase 1• Determine safe dose• How does the body work?

Phase 2• Safety and Effectiveness• Short-term side effects• Narrow criteria selection

Phase 4

Phase 3• Is it better than standard

treatment?• Evaluate risks/benefits• Most expensive and difficult

Pre-Clinical

Phase 1• Determine safe dose• How does the body work?

Phase 2• Safety and Effectiveness• Short-term side effects• Narrow criteria selection

Phase 4• Observation of serious

side effects• Interactions with other

drugs• Tests in groups or special

populations

Phase 3• Is it better than standard

treatment?• Evaluate risks/benefits• Most expensive and difficult

Pre-Clinical

Common Types of Clinical Trials• Treatment Trial• Prevention Trial• Diagnostic Trial• Quality of Life Trial• Compassionate Use

Clinical Research Team• Principal investigator

– Plans and leads the trial– Typically a physician

• Clinical Research Coordinator– Coordinates daily trial activities – Main contact for volunteers

and staff

Clinical Trials for Inflammatory Bowel Diseases (IBD)• Trials are appropriate for many different types

of people• Specific requirements vary by trial• The more people who take part in clinical trials,

the faster we will find better ways to treat and potentially cure IBD

Common features of IBD studies• Clinical Trials identify “Endpoints” or goals that the

medication is meant to achieve• Common Endpoints for IBD related clinical trials:

– Symptom improvement – Improve quality of life– Small bowel or colon healing– Reduction in need for other medication (ex.

Corticosteroid) – Improvement in blood or stool markers of inflammation

Special considerations of IBD studies• Pediatric clinical trials require parental consent• Financial considerations:

– Clinical trials often cover medication under investigation and any measures (i.e. lab work, clinic visits, etc.) relevant to endpoint being investigated

– Other treatments may not be covered • Some studies provide continued medication for

patients who respond after specified trial endpoint– Specific to each trial

Where can I find more information?• Clinical Trials on CCFA.org

– www.ccfa.org/research/participate-in-research/find-studies-and-clinical-trials• ResearchMatch

– www.researchmatch.org/partner/ccfa • ClinicalTrials.Gov

– www.clinicaltrials.gov • CCFA Partners

– www.ccfapartners.org • Your doctor

We all want the same thing• Working together to find answers faster!• Goes beyond the limits of financial

contributions and allows for a bigger impact made by all

• www.researchmatch.org/partner/ccfa

Questions and Answers

IBD Help Center 9 AM- 5PM EST

(888) 694 8872, via email at info@ccfa.orgOr Answerchat on our website

www.ccfa.org

Irwin M. and Suzanne R. Rosenthal IBD Resource Center (IBD Help Center) M-F, 9:00 AM-5:00 PM ET

– Phone: 1-888-694-8872 – Email: info@ccfa.org

Educational webcasts: www.ccfa.org/resources/webcasts.html

Connect with other patients– CCFA Community website: www.ccfacommunity.org – Support groups and Power of Two (peer mentors): – www.ccfa.org/chapters

GI BuddyOnline tracking tool and mobile app www.ccfa.org/gibuddy

CCFA Partners: Patient Powered Research Network www.ccfapartners.org

Local educational events, visit: www.ccfa.org

Additional Resources for IBD Patients

THANK YOU

www.researchmatch.org/partner/ccfa