Pan-Canadian Oncology Drug Review

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Lisa Pearson
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1 UPDATE Pan-Canadian Oncology Drug Review UPDATE ON PROGRESS By DAUNA CROOKS, RN, DNSC AND COLLEEN SAVAGE Far fewer oncology drugs were approved in Canada between 2003 and 2011 than in the US or Europe. Further, slower review times have led to delays in practitioner and patient access to new drugs. Marketing approvals were also noted to be slower than in the US. Worse yet, very few drugs in this period of review were covered by some degree of government insurance and variances of coverage from province to province caused considerable concern for cancer patients and cancer advocacy groups. 1 These facts are at the foundation of the attention given to drug access by Canadian cancer charities and non-profits. Concern led to shared learning, which led to participation in the processes that permit or deny the use of promising new therapies for cancer. The pan-canadian Oncology Drug Review (pcodr) is a pivotal agency in that process. pcodr was established in 2010 by the provincial and territorial ministers of health, all but Quebec. The purpose of pcodr was to assess the clinical evidence and cost effectiveness of new cancer drugs and to use this information to make evidence-based recommendations to the provinces and territories to guide their drug funding decisions. Along with the provinces and territories, with the exception of Quebec, pcodr partners are the provincial cancer agencies, the Canadian Partnership Against Cancer (CPAC) and the Canadian Agency for Drugs and Technology in Health (CADTH). As an observer of the cancer system and all ancillary services and supports, and as a critic of processes that cause unnecessary anxiety, cost or sub-optimal care to cancer patients, the Cancer Advocacy Coalition of Canada (CACC) encouraged the formation of pcodr and its predecessor, the interim Joint Oncology Drug Review (JODR). Maturation of these initiatives has been, in some eyes, very slow: JODR was announced early in 2007 and now in 2013 pcodr has just enough experience to be evaluated. JODR was a courageous initiative charged with the daunting task of creating innovation and repairing trust in the quality of cancer drug reviews. Facing somewhat rigid positions on many fronts, it must be admitted that JODR struggled with the high expectations. Grafted to the Ontario drug program, seeming to resist transparency, lecturing as often as listening to stakeholders and overall a bit embattled by provincial/clinical/economist affection for the past, JODR was no fun. The learnings were uncomfortable but perhaps necessary for the eventual formation of a distinct entity, pcodr, which immediately displayed a finely tuned interest in credibility, collaboration and stakeholder relationships. pcodr spent a year planning its work before completing the first review in December 2011; a frustrating delay for cancer groups. pcodr documented every element to be considered, the structure and flow of expert input into the pcodr expert review committee (perc), timeline commitments, checkpoints for feedback and, most importantly to cancer groups, building in formal openings for and recognition of patient input. One of the long-standing complaints about other review bodies had always been that once the expert advisors (clinical and pharmaco-economic) submit their documents to the lead committee, a mysterious discussion results in a recommendation about whether the drug is useful and affordable. If there were any rules or guidance for that discussion, nobody was going to tell a patient group. Even though it is now common practice for such review committees to have one or two patient representatives in the room, they may or may not know anything about the disease in question. Submissions from patient groups were a technicality that could not match, let alone override the experts. So how were those different inputs measured and what weights were applied to their importance? Equally unknown in these mysterious discussions was the question of whether ad hoc opinions and undocumented input (last minute bias) were permitted. The lack of clarity and transparency, even for the rules of the game, not the proprietary details, undermined trust in the process that the new pcodr decided to address quickly. The deliberative framework (released in the spring of 2012) is a guide for that critical point in the review process where the perc comes to a conclusion about the clinical merits, the cost-effectiveness, the need for and importance of the treatment. The framework provides an outline of all the elements that should be considered by perc during its review, and reinforces that no single element over-rides another, but rather that perc uses the sum of all elements to formulate a funding recommendation. The framework can be applied to all oncology drugs and situations including situations such as rare cancers or end of life care. In addition, the framework reinforces that there is no threshold that must be met for any single element in the review; rather, it is the individual drug, disease and context that determine perc s information 24 REPORT CARD ON CANCER IN CANADA,

2 TABLE 1 PATIENT ADVOCACY GROUP ENGAGEMENT AS OF DECEMBER 31, 2012 Generic Name/ Brand Name Cancer Type Patient Patient Input Feedback Pazopanib (Votrient) Metastatic Renal Cell Carcinoma 1 1 Ipilimumab (Yervoy) Advanced Melanoma 2 1 Sunitinib (Sutent) Pancreatic Neuroendocrine Tumours 1 0 Vemurafenib (Zelboraf) Advanced Melanoma 1 1 Eribulin (Halaven) Metastatic Breast Cancer 2 Everolimus (Afinitor) Pancreatic Neuroendocrine Tumours 1 0 Crizotinib (Xalkori) Advanced Non-Small Cell Lung Cancer 1 1 Bendamustine (Treanda) Non-Hodgkins Lymphoma (NHL) 2 1 Bendamustine (Treanda) Chronic Lymphomatic Leukemia (relapsed/refractory) 2 1 Pazopanib (Votrient) Soft Tissue Sarcoma 1 0 Total (%) 14 (100%) 8 /14 (57%) needs for each element of the framework. 2 Within the documentation for the deliberative framework, the listing of all the sources and types of information to be considered, are the magic words: Alignment with Patient Values. In these few words, with a formal process for soliciting patient input (repeatedly) as information is gathered for the review, pcodr showed the respect for cancer patients that groups wanted to see. In 2011, as part of the process of review and maturation of pcodr, objectives for working with cancer advocacy groups and the public were developed. They hoped to enable: understanding of the rationale for this pan Canadian initiative and the inherent review processes; to help advocates become familiar with guiding principles that ground pcodr s work and that of the agencies and structures that support the work of pcodr; and finally to describe how pharmacists, patients, physicians and economists from across Canada could be involved. A number of groups have been particularly active with pcodr, scheduling regular meetings, collaborating on guidance documents for patient group submissions and offering tutorials. CACC chose to conduct an interim evaluation of pcodr based on its first 18 months of operation. The objective was to examine how well pcodr delivers on its mandate and whether the cancer groups who represent patients in need have developed a partnership relationship with pcodr that works. This is not a comprehensive evaluation, rather a snapshot of the key performance features that cancer groups expect to see from a government agency with significant impact on the lives of patients. This article reports on data from 22 of 30 invited participant groups, all describing themselves as interested or very interested in pcodr. pcodr Activity As can be seen in Tables 1, 2 and 3, pcodr has been active in reviews, their projected timelines are aggressive and patient advocacy groups have been regularly involved in reviews and in giving feedback on final recommendations. In the first year of operations, up to December 31, 2012 pcodr had reviewed and received patient/group input on eight separate drug submissions with two drugs being reviewed for two different purposes. They met their mandate to seek out patient/group input. Following the finalization of recommendation for these drugs only eight of 14 of the original patient/group responses were solicited about the recommendations. This particular set of drugs reviewed were primarily for metastatic disease across a wide range of cancers. It is only fair to point out that the pace of reviews continues to increase new drug submissions expected in Table 2 relates the number of submissions and type of TABLE 2 pcodr SUBMISSION STATISTICS JULY 13, 2011 TO DECEMBER 31, 2012 Number of Submissions Received Total Submissions Received 18 Under Review 7 / 18 Total Number of Submissions Completed With Notification to Implement Issued 11/18 With Final Recommendation Posted 11/18 With Initial Recommendation Posted 12/18 Patient advocacy group input 18/18 Number of pre-noc Submissions Received 10/18 Number of Early Conversions 1 / 11 Number of Oral Drug Submissions Received 8 / 18 Number of IV Drug Submissions Received 10/18 Number of Submissions with diagnostic companion test or other supplementary issue reviewed 2 / 18 REPORT CARD ON CANCER IN CANADA,

3 drugs (oral or IV) reviewed in the period July 13, 2011 to December 31, PCODR indicated that 18 drugs were submitted for review and of these seven were in progress at that time. Final recommendations were posted on 11 of 18 with one preliminary recommendation posted as well. This is a considerable productivity for 18 months and all submissions reviewed had patient advocacy group input. Two important items are of note in this table, one drug had an early conversion to practice and two drugs were considered along with diagnostic tests. pcodr also provided information on the review timelines, as shown in Table 3, indicating projected and actual times to complete each phase of a review. The variances in range are considerable and pcodr offered the following details to explain. The longest review (in business days) was for bendamustine (Treanda) in Non-Hodgkin's Lymphoma and Chronic Lymphocytic Leukemia (relapsed/ refractory). The pre-noc submission was received by pcodr on April 24, The product received NOC on August 24, For New Oncology Drugs filed Pre-NOC or Pre-NOC/c, the Drug must have received the NOC or NOC/c before it will be placed on a perc agenda; bendamustine was delayed from the perc agenda by one month to await the NOC issuance. Submission Deemed Complete 6 perc Meeting Date 104 Initial Recommendation Issued 114 perc Re-Consideration Meeting 143 Final Recommendation Issued 153 Days from Submission to Notification of Implementation 164 Days from NOC issued to Notification of Implementation 79 The shortest review (in business days) was for ipilimumab (Yervoy) in advanced melanoma. A three person panel of perc assessed the stakeholder feedback received on the Initial Recommendation for iplimimumab (Yervoy).The panel determined the Initial Recommendation was eligible for early conversion because one of the three criteria for early conversion were met. perc s Initial Recommendation was able to become a Final Recommendation without reconsideration or re-deliberation by perc. Because of the early conversion mechanism, pcodr issued a final recommendation about 2 months earlier than had re-deliberation been required. Submission Deemed Complete 5 perc Meeting Date 69 Initial Recommendation Issued 79 perc Re-Consideration Meeting n/a Final Recommendation Issued 91 Days from Submission to Notification of Implementation 101 Days from NOC issued to Notification of Implementation 63 THE CACC SURVEY Survey Design Survey questions were designed to capture opinions on a range of topics relevant to patient advocacy groups having or potentially having input into pcodr reviews and recommendations, or drug review policies in Canada. The respondents expressed mixed ability, capacity or interest and also a mixed interest in advocacy for their membership about access to new cancer drugs. TABLE 3 pcodr REVIEW TIMELINES AS OF DECEMBER 31, 2012* Submission Deemed Complete perc Meeting Date Initial Recommendation Issued perc Re- Consideration Meeting Final Recommendation Issued Notification of Implementation Days from NOC issued to Notification of Implementation Explanation/ Notes Dates may change if submission is initially deemed incomplete (ex. Crizotinib) Dates may vary depending on where in the cycle the submission is received Will not occur if submission qualifies for early conversion submission qualifies for early conversion procedural review is received pre-noc submission or post-noc submission Target Mean Medium Range to to to to to to N/A From date of filing submission to pcodr * Calculated in pcodr Business Days ** N/A Dates are not applicable as steps have not yet taken place 26 REPORT CARD ON CANCER IN CANADA,

4 Some groups would not advocate for access to drugs but do want to participate in formal review processes and all the groups surveyed want to be certain that pcodr hears from patients. The CEO of CACC prepared a 27 item survey addressing awareness of pcodr, merits of the effort of involvement, perceptions of credibility, necessity for services like pcodr, involvement in pcodr processes, usefulness of pcodr reviews and recommendations. The working relationship with pcodr was a primary focus throughout the survey vis a vis pcodr objectives. Monitoring of drug coverage results, advocacy directed at drug plan managers, participation in educational initiatives about pcodr and in drug review generally, and the importance of patient/advocate participation in reviews were also addressed. The survey included both closed and open ended questions and was developed using Survey Monkey. Cancer charities, non-profit cancer organizations and advocacy groups known to be most active in issues surrounding access to cancer drugs, either by virtue of their public record or their consistent participation on committees, at information seminars and conferences on the subject were approached to complete the study. These agencies represent the interests of more than 800,000 Canadians currently diagnosed with many different types of cancer. 3 Perceptions of pcodr The groups responding to the survey indicated that they were very interested and active in drug reviews but indicated these activities were not always possible. Seventy three percent of respondents were familiar or very familiar with pcodr reviews. There is room for improvement nationally in education about pcodr, drug reviews in general and in purposefully seeking out patient/advocate involvement. Most (80 per cent) of respondents monitored drug coverage for their particular FIGURE 1 disease site or for all cancers and 30 per cent approached drug plan providers to advocate for drug coverage for their collective group. Those who did not pursue this activity were bound by the parameters of their organization. Regarding pcodr specifically, advocates had the best understanding of the submission process and the role of the Expert Review Committee (perc). Least understood was the role and activities of the provincial Advisory group. Of the respondent groups 59 per cent had provided a submission to pcodr for review. There was a certain lack of clarity around the task of submission, the task was complex and challenging, deadlines were short and feedback about the submission was less than satisfactory. Groups recognized their work in the final product. Of the groups who have submitted to pcodr, when describing the difficulty or complexity of the task their answers settle between challenging but manageable to difficult and frustrating with few outliers. Fifty percent of respondents felt that patient groups were permitted expression of all materials they would like to have considered in the review process, which is interpreted to mean that half the groups would like to provide more/different ACTIVITIES OF PCODR THAT GROUPS UNDERSTAND BEST Corporate structure and governance Expert Review Committee (perc) Provincial Advisory Group Clinical Guidance Panel Economic Guidance Panel The conflict of interest and code of conduct guidance documents The submission guidelines The deliberative framework of the perc 33.33% 61.9% 66.67% 80.95% FIGURE 2 CANCER GROUPS' EXPERIENCE SUBMITTING TO pcodr Was the task what you expected? Did you have enough time to deliver the quality of submission you wanted? Were you asked for more information, or explanation on any points in your submission? Did you recognize your input in the final recommendations from pcodr? Did you receive regular, timely feedback from pcodr on the status of your input or the status of the review? 30.77% 46.15% 38.46% 36.36% 46.15% % 53.85% 61.54% 63.64% Yes No REPORT CARD ON CANCER IN CANADA,

5 information and half the groups are satisfied their message is effectively submitted. In some instances groups felt they did not possess the expertise to submit or participate and others indicated that the drugs reviewed (to date) had little relevance to their reference group of cancer patients. All respondents indicated that inclusion of the patient voice was essential for adequate reviews. Seventy percent of respondents had taken time to become informed about pcodr, submissions and reviews. While about one third of respondents indicated that pcodr lived up to their expectations, many were uncertain or did not think so. This is a major area for improvement. Respondents were not convinced that pcodr recommendations were complete, fair, well-reasoned, unbiased or well documented. Most were unsure overall. One third of respondents regarded the work of pcodr as better than provincial reviews or CADTH but most were unsure in making a comparison. Thirty seven percent of respondents would like to see closer linkages with CADTH so that expert advice can be shared. Sixty-six percent indicated that it was important to have an independent body conduct clinical and economic reviews of new cancer drugs. Although not asked it was clear that pcodr was preferred over federal and provincial reviews and definitely over insurance company reviews. The two factors identified in use of pcodr data by provinces were a) that provinces weigh pcodr advice against local factors and b) the short time pcodr has been in operation. There was significant concern from 63 per cent of respondents about whether the conditional recommendations from pcodr offer a clear and satisfactory explanation that is understandable. When asked to rate their interactions with pcodr, 20 of 22 respondents ranked their experience from satisfactory to excellent on the following factors: response to concerns; explanation of the processes; engaging with patient groups and genuine effort to encourage patient participation in drug reviews. Participants were less satisfied with pcodr openness to suggestions and planned interactions with patient groups, although remaining responses were generally positive. Conclusion pcodr is a complicated place to understand. The most active and interested of patient groups express marked confusion or uncertainty about important features of the organization, while at the same time expressing confidence that the independent review is necessary. Put simply, patient groups are willing to work more closely with pcodr and agree unanimously that it is important for the patient voice to be heard in drug reviews. FIGURE 3 DOES pcodr LIVE UP TO YOUR EXPECTATIONS? 38.10% Transparency 30% Timely reviews 25% 45% Engagement with the patient voice/community Credible, thorough reviews that the provinces can depend on Removing bias or conflict of interest Effective communication with stakeholders 38.10% 14.29% 33.33% 14.29% 57.14% 40% 20% 40% Yes No Not sure FIGURE 4 CANCER GROUPS RATE THE QUALITY OF pcodr RECOMMENDATIONS Complete Fair Well reasoned Well documented Unbiased 9.52% 4.76% 9.52% 4.76% 5% 30% 61.90% 65% Yes No Not sure References 1 Rawson, N. Access to oncology drugs in Canada compared with the United States and Europe. Fraser Alert, July 2012, pcodr. perc Deliberative Framework, April 1, 2011 accessed at p= &lang=en&_afrwindowmode=0&_adf.ctrlstate=dv4ugvww4_67 3 Canadian Cancer Society s Advisory Committee on Cancer Statistics. Canadian Cancer Statistics Toronto, ON; Canadian Cancer Society, REPORT CARD ON CANCER IN CANADA,

EVIDENCE IN BRIEF OVERALL CLINICAL BENEFIT

EVIDENCE IN BRIEF OVERALL CLINICAL BENEFIT well structured and organized, provided perc with a much deeper understanding of patients experiences with relapse or refractory CLL/SLL and its treatment. perc deliberated upon the cost effectiveness