The Canadian Agency for Drugs and Technologies (CADTH) is the powerful arm of the federal government responsible for making recommendations to the provinces and territories about medicines accepted for public drug plans. This process is known as Health Technology Assessment (HTA for short). CADTH holds an annual Symposium showcasing its major achievements, trends in drug development and pricing and other important themes impacting HTA.
The official theme of the 2016 CADTH Symposium, held April 10-12 in Ottawa, was “Better Evidence for Everyone,” but if CCSN had to summarize the unofficial theme in a few words, it would be “Skepticism about the extent to which Patient Input counts as Evidence.”
Symposium attendees included researchers, clinicians, other health care providers, health economists, politicians and bureaucrats from across Canada, pharmaceutical industry representatives and representatives from insurance companies and other private plan providers. Last but not least, patients and patient organization representatives attended and kept track of what was going on with research and health technology assessment for disease and disability communities.
Why is HTA important to Patients?
Health Technology Assessment (HTA) is a complicated and highly influential process that the provincial/territorial governments use as a tool to make recommendations to these governments about whether or not to reimburse particular drugs in each province/territory.
Since public drug plans have limited budgets, governments make decisions about how to spend dollars on from a societal health perspective. To do this they ask themselves, “what drugs do we fund and which ones don’t we fund so that the greatest benefit is afforded to the greatest number of people?” In answering this question, governments use the HTA process, which compares the positive impact a drug will have on patients versus the cost of the drug. The drug is also compared to existing therapies and against the “lost opportunity costs” of paying for a drug for one disease versus the cost of paying for the drug for another disease. This is called an Economic Evaluation, where various aspects of patient health are allocated various dollar values based on all kinds of considerations, including Quality Adjusted Life Years (QALYs) gained by the drug. In addition, a clinical analysis of the drug is done and patient submissions are gathered about the real world impact of the drug on the lives of patients.
HTA for oncology drugs is done through the pan-Canadian Oncology Review (pCODR), and other drugs through Common Drug Review (CDR). Each of these programs and processes work, to an extent (many in oncology consider the pCODR process more effective), but much more could be done to make the process take factors that are important to patients into account. This is where patient input is very important. Patient groups that put in submissions about a drug or biologic under review by HTA need your help to give this real world evidence about the good, bad and ugly about the treatment under review.
The better the patient submission, the better the chances are that it will impact the HTA recommendation for public funding. This is why the work that patient groups do with HTA is important if patients are to get access to drugs they need.
At a workshop entitled “Introduction to Health Technology Assessment,” economist Don Husereau explained that, “economics is a scary business.” For patients and patient groups, the role that economics plays in HTA is all the more scary because we heard the usual complaints that patient groups take money from the pharmaceutical industry to obtain help in preparing their submissions and/or in general. The submissions are thus considered too professional, not grassroots enough. When we do them ourselves with our limited resources, they are not professional enough.
There are codes of ethics in place that prohibit pharmaceutical companies from helping to prepare patient submissions; the Innovative Medicines Canada Code of Ethical Practices – Guidelines for Transparency in Stakeholder Funding clearly state that “projects, events or activities undertaken with stakeholders should not be used to promote specific medicines.”
Recommendations for CADTH on Supporting Patient Submissions
1. Set up a fund that patient groups can access to do HTA submissions.
2. Develop tools and training that will help patients groups understand what HTA is looking for in a “model” patient submission and how to develop such a submission.
3. Provide advice to patient groups developing patient submissions.
4. Ensure that all HTA staff working with patients and particularly Canadian Drug Expert Committee (CDEC) members have a mandatory preceptorship with patient groups as part of their orientation to understand patient group challenges and positive values of patient group input.
What is an Economic Evaluation?
Husereau also highlighted the fundamental purpose of Economic Evaluations: it is a tool, he said, that meets policy needs. How that tool is constructed depends on what one wants to achieve; HTA is fit for a particular purpose, he explained. Put differently, HTA is not a science; it is a formula, and there are many different formulae for health technology assessments. The formula you choose depends on what you think is important in the value proposition to decide what to reimburse.
The HTA process relies on three different kinds of evidence:
1) clinical trial outcomes
3) ethical, legal, social, cultural and other forms of evidence, such as the patient experience
Themes from the CADTH Symposium of Interest to Patients
Patient Engagement in HTA Process
The HTA tool used depends on how one weighs these three types of evidence. All HTA tools use these, but they weigh each differently. Decision-makers are responsible for determining which types of evidence will be prioritized over others. The current tool that CADTH uses is heavily weighted on clinical trial outcomes followed by costs, and very little weight is given to the third criteria. It seems that it is only when we as patients raise our voices that decision-makers start to listen to our demand to be heard.
We are not alone in this demand. Presenter Dr. Fiona Miller (shown in photo), from the University of Toronto, gives us hope that there are experts who recognize the need for more meaningful incorporation of patient feedback in HTA. In the workshop entitled “Taking Account of What Can’t Be Counted: The Place of Qualitative Evidence in Health Technology Assessment Drug Reviews,” she said that in order to improve the HTA process we need to bring the patient, caregiver, and community experience into the process in a much more integral way. She explained that those who differentiate between empirical evidence and “patient insight” are making no useful distinction. Instead of asking how patient insight differs from empirical evidence, we ought to be asking, “is the patient experience relevant? Is it rigorous?” When we ask these questions, taking patient feedback as evidence becomes possible.
Miller pointed out that no evidence generation system for this kind qualitative evidence exists. In addition, she argued that we need to think about the systems that produce high quality evidence. If we need evidence from patients, caregiver and communities, she explained, we need systems to support its generation and use. It doesn’t make sense to call for more patient submissions, and then complain about the quality of patient submissions. “We don’t provide the resources to generate rigorous evidence by or about patients, and then we complain about the lack of rigour. This doesn’t make sense,” she explained.
So often throughout the conference, suggestions to decision-makers that patient input be considered more seriously in Health Technology Assessment were met with suspicion and dismissal. That the motives of patient advocates were tainted by the funding that they receive from pharmaceutical organizations implicitly pervaded the conference. Proposals to include patient input as a fundamental part of the HTA process were dismissed as being too difficult; an ideal that could not be translated into reality. It seems as though patient advocates are deluded to imagine a world in which patient input could be respected and meaningfully intertwined into the HTA process.
It is not outrageous to consider that patient input is important in this context, but this view is, surprisingly, not so strongly shared by those who have the power to make these important decisions. They want patient group input but not input “tainted “ by groups that take industry funding since it will be “biased”. What groups does that actually leave standing?
We patient advocates have not taken leave of our senses: we are mindful of the courage of patients and of our members, as well as the courage on which this organization is based. We resolve to continue to urge policy- and decision-makers to include patient input meaningfully, be taken seriously, and that it be made an essential part of HTA.
There will come a day when we realize this vision within the process of Health Technology Assessment, but today is not that day. In the meantime, we press on.
Enhancing Ethical Considerations in the HTA Process
CADTH has started incorporating formal, systematic ethics evaluations into the HTA process. By identifying and analyzing ethical issues, the HTA process can support better-informed, value-sensitive decisions. However, there is no established process for these evaluations. In contrast to other parts of HTA, such as systematic reviews of outcomes and safety and economic analysis, there is little agreement on how to incorporate ethics into HTA.
The major barrier we observe preventing this from happening is simply a lack of resources to perform ethical analysis. Additionally, there are differences in culture between HTA experts and ethicists. Ideally, the process should use a practical framework for integrating ethical issues and professional ethicists into the HTA, and should include guidelines to communicate the process to non-experts involved in the HTA process. This will improve interaction between different groups, enhance transparency and consistency of the evaluation process and allow for accountability in evaluation.
Given that this year’s theme was “better evidence for everyone,” it was unsurprising that there was much talk about evidence and evidence-gathering. While worthy goals, we believe it is important to question whether more evidence is a good in and of itself. What kind of evidence will be gathered? What precedence will different kinds of evidence be given? We remain hopeful that bringing medical ethicists into the HTA process in a more concerted way will help to address patient concerns.
Approximately 16% of total costs of the federal health care budget are allocated to drugs. We constantly heard that drugs are too expensive and create the biggest problem in increasing the overall health care budget. However, in a session entitled “Economic Costing for the Health Care System,” the speakers conceded that for the other 84% of the health care budget including hospitals, doctors, and administration, we do not have any national agreement to define the value for money that patients get for those services.
Put differently, different parts of the health budget are evaluated inconsistently. In our view, there is a strict standard of evidence applied to the drug budget that is not applied nearly as consistently to other parts of the health budget. We do not know of any institution that is measuring in any consistent way whether patients are getting value for money in these other areas. Maybe we do not necessarily need a bigger overall health budget, but instead better value for money across health budget silos.
We affirm that the evaluation of new drugs and health technologies should be a rigorous and evidence-based process. It is not that policy makers should stop applying a strict standard to drugs and medications, but that the rest of the health care system should be scrutinized to the same standards of evidence.
Saving Money in Drug Budgets
There were certain themes that were repeated at CADTH in different sessions throughout the Symposium including:
Disinvestment and Reassessment
There were a number of presentations about reassessing drugs that are already publicly covered and “disinvestment“ (also called “delisting”) of drugs already publicly reimbursed to make room to pay for newer better drugs.
While patients certainly do not want clinicians to prescribe drugs that are no longer scientifically and clinically efficacious for any patients, it is important that decisions made about delisting are based on a nuanced, analytical approach that involves information from clinicians and patients to ensure that drugs are not delisted that are needed by any patient populations or individuals. It may be true that a drug may be clinically less effective in trials but in the real world they may be safer for some patients than the newer drug.
The reality is that if one is taking the older drug for any of these reasons, the patient is not also taking the newer drug so it is hard to comprehend why we would want to take them off listing since it should be a wash in terms of cost to the drug budget if not in fact a cost savings.
Real World Evidence in HTA Decision Making
There was much discussion about the importance of real world evidence in informing the definition of value for HTA. In principle patients strongly support the importance of measuring their experience of specific drugs outside the hothouse and somewhat unnatural environment of clinical trials.
The questions are: for what purpose will the HTA process use real world data, how will it collect the data, how will it analyze data, and how will patients be involved in this process?
Patient groups support analyzing real world data to determine whether the drug actually is safe and effective; however it is important that real world data is not used to limit access to drugs that are important to specific patient populations and individuals. Collection of real world data should not be an excuse to postpone access to badly needed treatments.
Confidentiality and privacy concerns in the collection of these data are also crucial for patients.
Clinical management by General Practitioners versus Specialists
During the final panel of the Symposium, Dr. Robert Bell (shown in photo), the Ontario Deputy Minister of Health and Long-term Care, provided his view that there could be greater savings in the health care system if there was more management of some diseases by general practitioners rather than specialists. Of the two examples he used, one was the management of mood disorders, 70% of which could be managed by GPs. Some mental health patients would beg to differ.
As many speakers said in panels, medicine is an art, not just a science, and we need to rely on the practitioners who have the education and experience appropriate to the situation. While patients certainly support the management of diseases by appropriate health care providers, including GPs, decisions about which are the appropriate health care providers generally must be made by stakeholders who have expert knowledge of the disease area under consideration.
Specific Oncology HTA Issue
During one panel session, the head of the drug benefits program at Cancer Care Ontario gave a presentation about oncology trends in Ontario. He provided data showing that the incidence of cancer is growing; the cost of cancer drugs is increasing; and there are hundreds of oncology treatments in clinical trials, some of which will probably be successful and be approved for use in Canada.
He also defined sustainability for his oncology drug budget as keeping growth at the same level in the overall health care budget, which is 1.8% in Ontario this year.
Later, he was asked if he didn’t think that some of the new oncology drugs were game changers like the HIV drugs were at one time didn’t justify budgeting more in oncology to cover them. He replied that the comparison with the HIV drugs wasn’t an accurate one since those drugs created profound survival and quality of life improvements. Interestingly, the U.S. Food and Drug Administration has expressed that it considers some of the new oncology treatments based on immuno-oncology as breakthroughs.
Address by the federal Minister of Health, The Honorable Dr. Jane Philpott
In her keynote speech, The Honorable Dr. Jane Philpott endorsed the need for evidence in all aspects of healthcare decision making. We trust this will translate into more consistent standards of evidence being applied to the non-drug parts of health budgets. “Research investments are for the long term,” she explained, highlighting the federal Liberal government’s recent announcement of the investments outlined in the 2016 budget that will be dedicated to science and new research.
Minister Philpott called for more concerted efforts in transforming science into evidence and making that evidence usable. She highlighted the growing rates of chronic illness and called for more action in this area. She also called for more education and the empowering of patients. We were pleased to hear her demand that high quality mental health services be made more accessible to patients. Finally, we were also pleased to hear her recognize the importance of the social determinants of health, including education, housing, social environment, and employment.
She expressed her government’s commitment to affordable, accessible drugs and treatments, appropriately prescribed for all.
We look forward to seeing her commitments come to fruition. And we continue to advocate for meaningful patient consultation, engagement, and participation in the decisions that affect patient’s lives.
Recommendations for CADTH:
· Allocate sufficient, sustainable funding for a formal ethics process as part of the HTA process
· Involve patient groups in developing this process with other relevant health stakeholders and CADTH staff
Recommendations for CADTH and Health Ministries:
· Set up a multi-stakeholder pan-Canadian working group, including meaningful patient group involvement, to:
· Develop value metrics for each silo in the health budgets across Canada
· Develop guidelines for reassessment and disinvestment of drugs
· Ensure that clinicians are advised and educated about new scientific, clinical and real world evidence about drugs that will impact their clinical and prescribing practices
· To develop processes for collecting and analyzing real world data with confidentiality and privacy safeguards
· Set up a process to synthesize present real world data held in numerous data bases across Canada
· Develop Guidelines for first line management of disease groups by health care providers
· Consolidate scientific and clinical information about oncology drugs in development on an ongoing basis
If you support the recommendations made in this report, please write to your Minister of Health and other politicians and bureaucrats. Feel free to contact CCSN with questions and comments about this report as well.
The CCSN team at this important symposium included Louise Binder, CCSN Health Policy Consultant, Kyle Nimmrichter, CCSN Assistant Policy Analyst, and Mona Forrest, CCSN Board Member. Friends from Partner organizations included Martine Elias from Myeloma Canada, Sabrina Hanna of the Save Your Skin Foundation, Elizabeth Lye from Lymphoma Canada, and John-Peter Bradford of the Life Saving Therapies Network, all of whom contributed to this report.
We thank CADTH for support for some patients to attend the Symposium through scholarships.