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Progressive Licensing Workshop

Health Canada
November 27 - 28, 2006

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Recommendations on the Theme

Deepening our understanding of Canadians' values, issues, interests and priorities regarding a new drug regulatory framework.

Book of Recommendations

Table of Contents

List of Recommendation Reports

After all of the discussion groups had finished their reports, participants were asked to group and prioritize the 38 self-identified topics. The top seven priorities were explored in further depth, with participants choosing which group to attend. Recommendations were developed by each group. These reports have not been edited by Health Canada other than for format

Priority #	Report #	Topics	Page
1		What should federal approval mean? / responsibilities, roles	3
	2	What should Federal "approval" mean?
	18	What is "safe"
	20	Notice of Compliance - what is the bar?
	29	What should HC's role be?
	32	Pre & Post Market roles and responsibilities
2		Values, planning, assumptions that would underpine the framework	5
	8	Key Values
	10	Non-industry Funding for Patient Advocacy
	21	Gender Based Analysis in drug regulation
	19	Precautionary Principle versus Risk
3		Adverse drug reaction reporting	7
	24	Increasing the quantity and quality of ADR Reporting
	30	How can we improve ADR Signals
	7	Communication
	27	Responsibilities and information flow
4		Ethics around early access	9
	13	Ethics and Early Access
	14	Does Increased Post-Market Surveillance Justify a Decrease in Pre-Market Requirements
5		Access to new indications, low volume drugs	10
	23	Key Issues in current HC regulations affecting access to new medications
	4	Access to drugs - low volume
6		Process and accountability regarding pre-market process	11
	9	Process & Accountability
7		Informing the public	13
	36	Communication of Info from the drug safety system to patients and the public
	16	Feedback Mechanism for Consumers/Prescribers

Recommendation Report

• Priority # of the priority: 1
• Title: Roles and Responsibilities/ Criteria for Approval
• Report # of the discussion from which this priority was identified: 2, 18, 20, 32
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: 38 Educating Patients/Public and Involve in Decision-making process; #2, #13, #19
• State briefly the general intention or goal that will be addressed by your recommendations:

Key Recommendations	Who	When	Resources
Maintain the standards for safety and efficacy in pre-market stage in a PLF; flexible departure would be an exception, not the rule (ensuring that outlying safety/access issues do not drive the whole process)	Shared responsibility (industry still responsible for generating initial data for efficacy and safety)
Recommend HC strengthen its capacity to evaluate safety and effectiveness - when appropriate -- (relative to risk/benefit of product) in post-market setting in collaboration with other decision-makers and ensure measurable outcomes	HC & industry. And establish independent body to generate and gather effectiveness data and do the analysis
PLF must be a flexible system-wide process that deals with context (ie: disease, severity, alternatives) across a product lifecycle and emerging information in a way that: evaluates health and safety (safety, efficacy, and quality) at all stages of the product lifecycle maintains favourable benefit/risk which contextualizes safety/efficacy/effectiveness involves review and evaluation at all stages in the process (pre-, approval, and post) improves access is transparent
Definitions for benefit/risk; safety and efficacy/effectiveness - needs to be clarified and operationalized

Recommendation Report

• Priority # 2
• Title of the priority: Values and Ethics for a Framework for the Blueprint
• Report # of the discussion from which this priority was identified : 6, 8, 10, 21
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: 3, 19
• State briefly the general intention or goal that will be addressed by your recommendations: To highlight the need for an explicit statement of values, principles and ethics in the PLF Concept Paper and within the regulatory framework for pharmaceuticals and biologics in Canada

Key Recommendations / Recommandations principales	Who	When	Resources
1. Articulate a framework at the start of the Concept Paper to clearly address the purpose, values, principles and ethical assumptions behind progressive licensing and a life cycle approach to drug regulation. This framework could include: a) key lessons learned in drug regulation in Canada historically (eg., Krever, diethylstilbestrol, insulin) b) how the PLF builds upon a population health approach and a needs-based planning model which takes population needs into account. (e.g. could cite international models and systems in which 'me-too' drugs are given a lower priority than truly innovative drugs); c) principles, such as Hippocratic oath: 'first do no harm'; d) commitment to safety, efficacy and quality, and to increased therapeutic benefit in relation to other available pharmaceuticals or other empirically validated interventions; e) commitment to transparency and accountability, including engaging civil society in the drug regulatory process (Industry-sponsored groups should have a different status in the consultation process); f) framework should apply gender- and diversity-based analysis (See Recommendation #2)	Workshop participnts were willing to help!
2. Integrate Gender-based Analysis (GBA), including diversity analysis, to Progressive Licensing Framework as a foundation in planning and decision-making. Health Canada's GBA Policy (2000) mandates application of GBA as a matter of standard practice to policies, programs, risk assessments, research, legislation and other initiatives, as appropriate. In applying sex/gender/diversity analysis, GBA reinforces an evidence-based approach to pre-market approval and post-market surveillance and needs to be considered in all aspects of a life cycle approach to drug/product regulation, including clinical trials. (See Discussion Report recommendations: #21)	HC/Health Portfolio and other GBA tools and resources; in collaboration with PLF team; Women and Health Protection Working Group
3. That assumptions about values and ethics be made explicit in the PLF Concept Paper and in the Framework. For example: a) the underlying assumption that Canadian patients are willing to trade safety for early access needs to be further explored and challenged until validated; b) the assumption that Health Canada should have a mediating role between industry and public safety should be secondary to the government's role in public health protection and to the compatibility between the public's interests and government's interests in a viable publicly-funded health care system. (See Discussion Report #8)

Recommendation Report

• Priority # 3
• Title of the priority: Adverse Drug Reaction Reporting
• Report # of the discussion from which this priority was identified: 19, 24, 27, 30
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: See above
• State briefly the general intention or goal that will be addressed by your recommendations:

Key Recommendations	Who	When	Resources
1. Greater use of consumers as a source of ADR reporting Increased educational components/assistance to express adverse drug reactions on reports Link for online resources for various diseases/patient support groups Avoid text entry and support of codified data Identification of sentinel patient cohorts where appropriate Health Canada feedback to consumers reporting ADRs Use of community based organizations to collect ADRs	Health Canada in co-operation with consumers and patient groups	ASAP	Infrastructure, promotion = $$$$ Take advantage of pharmacies, academic centres and patient groups to facilitate implementation
2. Improved physician and pharmacist reporting Better education in universities Health Canada feedback to physicians and pharmacists reporting ADRs Cognitive service payments for ADR reporting Sentinel physicians or pharmacists to identify cohorts in their practices and report upon them (prospective surveillance)	Health Canada in cooperation with universities, physicians and pharmacists	ASAP	Input into curriculum at health professional schools, $$$$
3. Better use of electronic health record data in support of ADR reporting Global ADR databases Diagnostic coded provincial databases to identify events Real-time access for Health Canada to manufacturers product specific ADR databases Timely feedback from Health Canada to manufacturers regarding ADRs submitted	Health Canada in collaboration with global health authorities, provincial database providers	ASAP	Input from CIHI, $$$$

Recommendation Report

• Priority # 4
• Title of the priority: Challenges around early access
• Report # of the discussion from which this priority was identified: 13, 14
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: 2, 9, 11, 25
• State briefly the general intention or goal that will be addressed by your recommendations: see title

Key Recommendations	Who	When	Resources
1. PLF needs to conduct a thorough analysis of the ethical issues that would arise with early licensing e.g., equipoise, informed consent, therapeutic misconception etc.
2. The smaller the pre-market data package and identified and expected safety concerns, the greater the emphasis on post market requirements (proportionality).
3. Surrogate endpoints need to be fully validated in terms of clinical value and use to regulators in making regulatory decisions.

Recommendation Report

• Priority # 5
• Title of the priority: Regulatory issues affection access to new medication or low volume ( manufactured on site and withdrawn)__________________
• Report # of the discussion from which this priority was identified: _23-4-37__________
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: __________________
• State briefly the general intention or goal that will be addressed by your recommendations: ________________

Key Recommendations	Who	When	Resources
1. Increase communication and collaboration between Health Canada and CDR. Avoid duplication of effort. Ensure earlier engagement of CDR in the review process.	HC CDR Industry Provinces Other Stake holder	Early in the review process	Minimum increase in required resource: Legal expert, Communication expert
2. Promote innovation and establish flexible infrastructure and provisions to deal with "niche" agents (e.g radiopharmaceuticals, orphan drugs and withdrawn drugs)	HC Industry Experts Stakeholder	As part of the progressive licensing	Expert advisory panel. Increase incentive for innovations
3. Considering limited resources, take advantage of all available scientific evidence, national and international experience and incorporate it in the review process and throughout the product life cycle.	HC Peer agencies ICH International Experts	As part of the progressive licensing;	WHO ICH Peer agencies Reputable scientific literature. Legal framework. Partnerships/ and exchange.

Recommendation Report

• Priority # 6
• Title of the priority: Process and Accountability (Related to providing earlier access)
• Report # of the discussion from which this priority was identified: 9
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: __________________
• State briefly the general intention or goal that will be addressed by your recommendations:
  How can the process be built to make sure it works out the best it can. Where does the accountability lie?

Key Recommendations	Who	When	Resources
1. There should be a clearly defined process that includes accountability to describe and explain the process to stakeholders. Components:	HC/PLF	Prior to Final step in Consultation and implementation	HC, Stakeholders.
2. Develop key components of the process that considers the exceptional drivers. To develop mechanism to recognize the unmet medical need and candidate products. Involvement of early panel? Incorporate criteria for moving to a late panel? Capture decisions and rationales at each level. Possibility of patient-doctor contract for sharing of accountability.	HC/PLF	Prior to Final step in Consultation and implementation	HC, Stakeholders,
3. Develop safeguards, feedback mechanisms and other lifecycle related decision points for products that have access to market by exceptional drivers. Consider patient safety concerns related to off-label use and how to manage risk.	HC	Post-PLF	HC, Medical Community (Colleges)

Recommendation Report

• Priority # 7
• Title of the priority: INFORMING THE PUBLIC
• Report # of the discussion from which this priority was identified: ___________
• Related reports: Report # s of other Discussion Reports related to this priority as listed in plenary on flip charts: __________________
• State briefly the general intention or goal that will be addressed by your recommendations:

Key Recommendations	Who	When	Resources
1. Health Canada shouldtake a leadership role in ensuring that the public has access to appropriate information on drugs and drug safety.	Health Canada in partnership with stakeholders	Now	Lots
2. Health Canada should conduct an environmental scan of information that is already available, and a needs assessment to determine: WHAT information is needed WHO needs the information HOW to deliver the information WHO should provide the information (specific group or partnership)	HC with multi-stakeholder working group	Now	Lots
3. Health Canada should ensure that information provision is aligned with the Progressive Licensing life cycle model (e.g. clinical trials, NOC, early post-market) and incorporates feedback from the public at the appropriate stage.	Health Canada in partnership	Now	We're worth it
4. In providing this information, HC should take into account the diversity of Canada's patient population (e.g. hard-to-reach groups).	Health Canada in partnership	Now	As much as it takes.

Assumption: "General public" includes all stakeholders external to Health Canada, including patients, consumers and health professionals