summary report - benefit-risk roundtable

8/6/2019 Summary Report - Benefit-Risk Roundtable

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On Friday, March 5, 2010, over

thirty Canadian health

stakeholders (representing

patients, providers, government,

academics and industry)participated in a roundtable

meeting to discuss the new

paradigm of benefit-risk

introduced in Bill C-51.

The day featured a rich

discussion (moderated by Dr.

Stuart MacLeod) that effectively

framed the issues in benefit-risk

evaluation and set the stage for

a continuing dialogue.

Prior to the Roundtable, surveys

were conducted with

stakeholders from diverse

backgrounds and areas of

interest to identify the best

format to enable an open,

honest discussion and ensure

that key issues would beaddressed.

An Advisory Committee wasformed to further guide the

Roundtable planning, ensure a

balanced approach, and

review background materials.

Health Canada was engaged

and informed throughout theprocess.

Canada’s Drug Regulation Renewal:Are Improved Access and Safety in our Sights?

A Roundtable Exploring the New Paradigm of Benefit-Risk Evaluation

Summary Report March 5, 2010

Format Key Themes

While many issues were raised during the vigorous

discussions, five key themes emerged from the Roundtable

meeting:

1. Consideration of quality of life should be central to the newapproach to evaluation of benefit from innovative therapies.

The evaluation will require a combination of quantitativeand qualitative methods.

2. Enhanced patient engagement (including the relationship

with stakeholder advocacy groups) is certain to be a key

feature of any future process.

3. There should be a change in emphasis to benefit-risk from

risk-benefit. This shift is appropriate and significant as it leads

to a more patient-centered approach where costs aren’t

the only deciding factor.

4. Change in the drug regulatory system (and by extension in

the review of drug reimbursement/access decisions) is

inevitable. The active process of change in which we are

now engaged is driven by our vastly improved

understanding of the social and biological determinants of

health and these will affect the assessment of drug benefits

and drug safety.

5. New approaches and new methods are needed for the

assessment of both benefit and risk. Importantly,

methodological innovation is required and will be facilitatedby the legislative renewal process as described by David Lee

and Robyn Lim from the Office of Legislative and Regulatory

Modernization. Canada’s forthcoming renewed legislation

describing a life-cycle approach to the evaluation of new

therapies will be a driver of therapeutic innovation and will

facilitate a more patient-centered approach to treatment,

which should correspond to improved access to new drug

treatments.



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Roundtable Summary Report: Canada’s Drug Regulation Renewal March 5, 2010

Overview

Russell Williams, President of Rx&D,

welcomed a diverse group of over thirtyCanadian health stakeholders to the

roundtable meeting and encouraged a

robust discussion on the new paradigmof benefit-risk introduced in Bill C-51.

Bill C-51 represents an attempt to

modernize Canada’s Food and Drugs

Act for the first time in 50 years and contains an increased emphasis on the concepts of benefit

and risk. The proposed Bill has the potential to significantly impact the manner in which drugs areregulated, monitored, and delivered to patients.

It is everyone’s role: governments, approval agencies and innovators, to ensure that this benefit-risk ratio is appropriate. We must also ensure that we do not become risk-averse to the point

where we potentially delay or deny access to medicines that could provide significant benefits to

patients.

The day featured a rich discussion that effectively framed the issues in benefit risk evaluation and

set the stage for a continuing dialogue. From the viewpoints heard at the symposium it can be

concluded that the current system does not meet the standards of “accountability for

reasonableness” as laid out by Daniels and Sabin in the their landmark position paper of 1998

(Health Affairs;17:50-64). Specifically, the current approach to drug regulation does not meet the

desired standard for openness and transparency, including the wide dissemination of the basis for

decision-making.

Neither does the current system, in spite of some recent improvements, meet the objectives of

diverse stakeholder involvement in the decision making process with regard to either regulation or

reimbursement recommendations. It was pointed out during the day’s discussions that proposed

improvements in the regulatory system without corresponding progress on decision-making around

access would be futile. Unless provincial authorities start to consistently offer Health Canada

approved medicines for reimbursement, we’re only dealing with half the issue and are taking

healthcare decisions out of the hands of patients and physicians.

Consideration of quality of life should be central to the new approach to

evaluation of benefit from innovative therapies. The evaluation will require a combination of quantitative and qualitative methods.

It was clear from the day’s discussion that many international regulatory agencies, especially in

Europe and the United States, have made progress with new evaluation systems and Canada will

need to learn from them and mesh with international trends. The development of better systems

for qualitative research in therapeutic evaluation will require a major focus on patient-reportedoutcomes.



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The active process of change in which we are now engaged is driven by our vastly improved

understanding of the social and biological determinants of health and these will affect the

assessment of drug benefits and drug safety.

We have greatly expanded our knowledge of biology as it relates to drug action and drug risk. This

improved understanding is leading us inevitably toward a system in which drug therapy will be

tailored much more closely to the characteristics of individual patients. We are on the threshold of

an era of personalized or individualized treatment that will in many cases supplant the more

general, population approaches to evaluation of therapy that have been taken in the past.

Change in the drug regulatory system (and by extension in the review of drug

reimbursement/access decisions) is inevitable.

This shift is appropriate and significant as it leads to a more patient-centered approach where

costs aren’t the only deciding factor.

Safety issues should certainly not be ignored but we should not lose sight of the fact that mostsevere adverse drug reactions are rare and greatly outweighed by therapeutic benefits. Drugs

with a higher potential for adverse reaction (though still rare) can be an important option in

situations where patients decide that the benefits outweigh the risks, such as cancer treatment,

antiretroviral therapy, and use of therapies such as anti-TNF biologicals.

All recipients of drug therapy have a major stake in improving our methodology for the

measurement of benefit including a more nuanced understanding of improvements in quality of

life.

Enhanced patient engagement (including therelationship with stakeholder advocacy groups) iscertain to be a key feature of any future process.

The Roundtable heard very important messages from patientadvocates in regard to the evaluation of drug benefit-risk. Patients’

viewpoints on benefit-risk will vary greatly depending on their

perspective and can be influenced by factors such as their

condition, severity of illness, who their physician is, impact on care-

givers, their view of risk and their beliefs.

Patient advocates stressed that:

• Patient groups should demand access to the evidence forming

the basis for decisions.

• Patients (lay individuals) should not be afraid of science. Science

literacy should be improved across the board.• Patients who are affected by benefit-risk evaluations should not

be reticent about involvement in the process and in taking

ownership of the eventual decision.

There should be a change in emphasis to benefit-risk from risk-benefit.

“The entire spectrum ofactivities that happen

over the course of a

drug's "life," which

includes its discovery;

the early testing done

to understand the drug;

the trials to determine its

safety and efficacy in

humans; its approval;

and its marketing and

use in larger

populations.”

-- Health Canada1

Definition:

Life-Cycle



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Importantly, methodological

innovation is required and will be

facilitated by the legislative renewal

process as described by David Lee

and Robyn Lim from the Office of

Legislative and Regulatory

Modernization. Canada’s forthcoming

renewed legislation describing a life-

cycle approach to the evaluation of

new therapies will be a driver of

therapeutic innovation and will lead

directly to improved access to new drug treatments.

Randomized controlled trials will remain a goldstandard for evidence of efficacy but there will be an

inevitable shift to decisions made on the basis of a

deliberative process that takes context into account.

Decision-making will increasingly be evidence

informed rather than based exclusively on RCTs.

Decision makers will need to take into account

observational studies particularly in relationship to

quality of life and adverse drug reactions.

Consideration of composite outcomes will drive the

final decision and colloquial evidence will be

accepted.The proposed life-cycle approach requires

government to be an active participant, and for

manufacturers to be more invested in all stages of

product life-cycle regarding benefit-risk

New approaches and new methods are needed for the assessment of both

benefit and risk.

Health Canada’s ProposedLife-Cycle Model

It is important that the new system be able to integrate with global reporting networks for adverse

drug events.

The government is mandated to not develop uniquely Canadian laws unless absolutelynecessary. Canada’s population isn’t large enough to draw out necessary health data.

The drug review and reimbursement processes shouldn’t be detached from the patient voice and

outcomes.

The lifecycle approach isn’t absolutely watching everything, 24 hours a day. It’s the ability to

intervene and assess as appropriate.

Clearly we’re all in favour of achieving the best access with minimal risk, but the devil’s in the

details.

Additional discussion

Current Drug Development and Regulatory Processin Canada



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Roundtable outcomes

The Roundtable brought together a broad cross section of

views and it is hoped that the process can be continued

through future in depth discussion of the convergence

between benefit risk evaluation and consideration of

access/reimbursement issues.

Roundtable presentations

1 http://www.hc-sc.gc.ca/dhp-mps/homologation-licensing/gloss/index-eng.php#bm_l02

Presenters Affiliation Topics

Russell Williams Canada’s Research

Based Pharmaceutical

Companies

Welcome and introductions

Dr. Stuart MacLeod Child & Family Research

Institute, University of

British Columbia

Moderator’s welcome

Balancing benefit & risk

Dr. Michael Rieder University of Western

Ontario

Impact of benefit-risk from the paediatric

perspective

Harlon Davey Patient Advocate Patient perspective on the issue of benefit-risk

Dr. Bruce Carleton University of British

Columbia

Personalized medicine and benefit-risk

David Lee Health Canada Benefit, Risk within Federal Modernization

Dr. Wendy Graham North Bay Blue Sky

Family Health Team

Innovation in technology and healthcare:

personal health records & health management

Deb Maskens Kidney Cancer Canada

Kidney Cancer: The impact of evidence ondecision-making

Robyn Lim, PhD. Health Canada International Trends in Benefit/Risk

Consideration of quality of life should be central to the new approach to evaluation of benefit

from innovative therapies.

The proposed life-cycle evaluation will require a combination of quantitative and qualitative

methods, which is a significant change and follows international trends.

Participants identified the need to ensure that public and private payers move in step with the life-

cycle model vis-à-vis reimbursement and evidence requirements. (private payers were not represented at the Roundtable)

Health stakeholders expressed a strong interest in remaining engaged on this issue and a

willingness to participate in future events (based on evaluations and verbal feedback).

Participants requested that Rx&D share a summary report from the roundtable, provide access tospeaker presentation (as appropriate) and provide updates on Bill C-51.

summary report - benefit-risk roundtable

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