CRP 112 Lecture 10

Question 1

AllTrials campaign

Answer 1

have all clinical trials registered and all results reported
-support data transparency
AllTrials calls for all past and present
clinical trials to be registered and their full methods and summary results reported

Question 2

Clinical Trial Reporting
4 levels of information

Answer 2

1. Knowledge that a trial has been
   conducted, from a clinical trials register
2. A brief summary of the trial’s results
3. Full details about the trial’s methods and results
4. Individual patient data from the trial
   -All Trials wants 1st 3 Levels

Question 3

Why is registration important?

Answer 3

Considered a scientific, ethical and moral responsibility
-part of the Declaration of Helsinki, along with reporting results

Question 4

Food and Drug Administration Amendments Act of 2007 (FDAAA)

Answer 4

all such trials will have summary results
information posted publicly in clinicaltrials.gov.
-includes AE info, full protocol and statistical analysis plan published on the website
FDAAA 2007 compliance is poor!
 Lack of enforcement by FDA
Industry sponsors were more compliant vs non-industry
-Sponsors with large # trials more compliant
-US Government-run studies least compliant

Question 5

Vanessa’s Law

Answer 5

Protecting Canadians from Unsafe Drugs Act
- Gov’t has power to initiate mandatory recalls for unsafe drugs & demand reports from health care institutions on
ADRs
-Tough new penalties for unsafe products – jail time & fines up to $5 Mill/day
-Authorizing creation of regulations designating when confidential business information ceases to be confidential and disclosing such information
-health care institutions to report serious adverse reactions or medical device incidents
- authorizing creation of regulations governing health care institution reporting

Question 6

Right To Try Federal US Bill

Answer 6

Gives terminally ill patients right to seek drug treatments that remain in clinical trials
 Drugs must have passed Phase I
 No application to gov’t required
 Work directly with MD & drug company
-obtain Informed Consent – no guidance – no MD liability
-statutory requirement for sponsors and manufacturers to provide an annual summary to FDA

Question 7

US – Expanded Access Pathway

Answer 7

 Patient’s condition is life threatening
 No comparable/satisfactory alternative
 Patient not eligible for clinical trial or no clinical trial available
 Potential benefit justifies potential risks
 Will not interfere with CTs that support a product’s development or marketing approval
 Use outside of a clinical trial of an
investigational medical product (i.e., one that has not been approved by FDA).
-Form 1571 or 3926 – provide FDA with
information on use, monitoring plans.
-MD must report ADRs and obtain IRB approval
-requirement that potential benefit
outweighs the risks AND must not interfere with CTs OR marketing approval

Question 8

Canada: Special Access Programme
(SAP)

Answer 8

 Health Canada considers requests for access to drugs that are unavailable for sale
 Practitioners make the request
 For patients with serious or life-threatening conditions when conventional treatments have
failed or are unsuitable or unavailable
-No restricted drugs allowed
-Drugs for research are requested via other programs (NOT for research)
-Part C, Division 8 (New Drugs) sections 10 and 11

Question 9

Part C Division 8.010 & 8.011

Answer 9

Sale of New Drug for Emergency Treatment
 C.08.010:
 Director issues letter of authorization for sale
 Practitioner gives specific patient information and agrees to report results of use to HC
 C.08.011:
 Manufacturer may sell to a practitioner named in a letter of authorization only quantity specified in
letter

Question 10

SAP Requests/Decisions

Answer 10

-Authorization: Information provided meets the criteria of
the Programme.
 Incomplete: The SAR form is incomplete, missing
information or is illegible. The SAR is returned with an
incomplete notification to the practitioner.
 Cancellation: Due to external or logistical factors, the
requested drug is not accessed via the SAP. For example, the
manufacturer is unable to provide access via the SAP.
 Withdrawal: The SAR is withdrawn by the requesting practitioner.
 Denial: The information provided by the practitioner does not meet the criteria of the Programme, or the request was returned as incomplete numerous times and information is still
lacking.

Question 11

SAP Common Reasons for Denial

Answer 11

 Availability of marketed alternatives
 Patient not having a serious or life-
threatening condition
 Insufficient data to support the specified use
 Drug being early in development with
limited information on its safety, use or
benefit available.

Question 12

Manufacturers and SAP

Answer 12

 Manufacturers have final word whether drug will be supplied
 Manufacturers can impose restrictions and conditions
 Often issues with IP availability
 Most provide drug free
 If they charge, cost is covered by patient, hospital or insurance

Question 13

USA Single Patient IND

Answer 13

 Licensed physician makes individual
expanded access submission (Form 3926)
 Request Letter of Authorization (LOA)
from manufacturer and send to FDA
 Obtain approval from IRB