Patient Input: What is asked for and what is helpful in a patient submission
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This document summarizes a presentation given by Maureen Smith on patient involvement in drug coverage reviews in Ontario. It outlines how patient groups can submit evidence to be considered by the Committee to Evaluate Drugs, including registering as a patient group, using the submission template, and meeting submission deadlines. It also provides suggestions for making submissions more impactful, such as prioritizing the most important impacts of a disease and treatment outcomes. The goal is to systematically incorporate the patient perspective into drug review and funding decisions in Ontario.
Patient Input: What is asked for and what is helpful in a patient submission
- 1. Patient Input: What is asked for and what is helpful in a patient submission Cathy Evanochko Co-Chair Tuberous Sclerosis Canada Sclérose Tubéreuse; CORD, Board member Maureen Smith Patient Member Committee to Evaluate Drugs Ontario Ministry of Health & Long-Term Care March 7, 2015
- 2. Tuberous sclerosis complex (TSC) tumours develop in multiple organ systems including brain, heart, kidneys, lungs, skin, eyes complications can include: epilepsy, kidney failure, heart arrhythmia, lung failure, cognitive disability and mental illness issues, facial disfiguration Treatment for symptoms seizures – medication, surgery tumours – surgery nothing How did we get here? May 7, 2015
- 3. drug re-purpose – Everolimus (RAD001) - an inhibitor of mammalian target of rapamycin (mTOR) tumour shrinkage clinical trials Cincinnati, here we come May 7, 2015 New adventure
- 4. FDA approval for treating kidney AML – August, 2012 clinical trial ended August, 2013 Health Canada approval for treating kidney AML – October, 2012 Patient input – Canadian Agency for Drugs and Technologies in Health (CADTH) – Common Drug Review (CDR) negative recommendation – September, 2013 May 7, 2015 Now what?
- 5. We had 6 weeks from notification to submit input patient survey information specific to renal AMLs compilation of survey results, what is meaningful? challenges few individuals with any knowledge of the drug progression of disease – wide range of affectations – not everyone has kidney tumours responses limited to responding to questions – online process May 7, 2015 Patient submission
- 6. and then we waited… May 7, 2015 April - September
- 7. CDEC FINAL RECOMMENDATION EVEROLIMUS (Afinitor — Novartis Pharmaceuticals Canada) Indication: Renal Angiomyolipoma Associated with Tuberous Sclerosis Complex Recommendation: The Canadian Drug Expert Committee (CDEC) recommends that everolimus not be listed. Reason for the Recommendation: In one double-blind, randomized controlled trial (RCT), everolimus was shown to reduce the size of angiomyolipomas (AML) in 42% of treated patients. However, it has not been definitively established that a reduction in AML size is correlated with a reduction in bleeding complications, avoidance of surgery, or long-term preservation of renal function. Of Note: CDEC considered subpopulations of patients for whom everolimus could be recommended (e.g., those who are experiencing AML growth and who are not candidates for surgery); however, there was no evidence for making such recommendations. May 7, 2015 Who knew?
- 8. Patient input CADTH – SEGA Ontario, BC – AML, SEGA Ontario EAP recommendation March, 2014 - access criteria – November 7, 2014 Special access – Alberta, Manitoba, Quebec What next? May 7, 2015 The journey continues
- 9. Patient Involvement in Drug Coverage Review Ontario Public Drug Programs Patient Evidence Submissions Maureen Smith Patient Member Committee to Evaluate Drugs Ontario Ministry of Health and Long-Term Care March 7, 2015
- 10. Ontario Drug Benefit (ODB) Program Approximately 3,400 drugs listed in the Drug Benefit Formulary/Comparative Drug Index (Formulary), and an additional 850 through the Exceptional Access Program. Provides drug benefits for Ontarians who are: 65 years of age or older; Residents of long-term care homes and homes for special care; Recipients of professional home care services; Recipients of social assistance, including Ontario Works and Ontario Disability Support Program; Recipients of the Trillium Drug Program Ontario Public Drug Programs (OPDP)
- 11. Ontario’s Drug Review & Funding Process Health Canada issues market authorization Final funding decision made by Executive Officer Common Drug Review (CDR) CDEC recommendation to drug plans Manufacturer submits clinical & economic evidence Ontario’s CED conducts review in the Ontario context. CED provides recommendation to Executive Officer. New Drugs / Drugs with new indications (non-cancer) CDEC = Canadian Drug Expert Committee CED = Committee to Evaluate Drugs pCODR OthersCancer drugs pCODR = pan-Canadian Oncology Drug Review
- 12. An expert advisory group that makes recommendations to the Executive Officer on drug funding and related issues. Membership includes: Physicians Pharmacists Health economists 2 patient members (since June 2007) Key considerations for CED drug reviews: Clinical efficacy and safety of the drug product relative to available alternatives Cost-effectiveness (i.e. evidence of value for money) of the drug product relative to alternative treatments Patient impact Impact on other health care services Committee to Evaluate Drugs (CED)
- 13. Makes final drug funding decisions taking into consideration: CED recommendation Advice from other advisory bodies, e.g. Citizens’ Council Patient and societal impact, public interest Product listing agreements with manufacturers Drug program budgets Other factors, e.g. government priorities Executive Officer
- 14. Objective: To put in place a formal framework to systematically incorporate patient evidence into the drug review and funding process. Rationale: Patients and caregivers can provide valuable information and insight about the impact of a disease and new and existing drug treatments. Patients and caregivers can identify areas of most importance to patients in the particular disease. This information can help set the context for the evaluation of clinical and economic data. Patient Evidence Submission
- 15. Patient evidence submissions are collated by the Ministry and are provided to all members of the CED. Patient evidence submissions to CADTH and pCODR are also provided to the CED. A CED patient member presents a summary of the patient evidence submissions during drug funding deliberations. Patient input is taken into consideration by the CED in its recommendation to the Executive Officer. How patient evidence submissions are provided to the CED
- 16. The CED meets face-to-face on a monthly basis The Committee follows a framework for each drug review Overview of the disease/condition treated with the product under review Presentation of societal values/patient perspective Patient members provides a summary of the patient submissions – included in the report is information on the patient groups, the impact of the disease/condition, the treatment outcomes that matter most to patients and information from patients who have used the drug Clinical review Cost-effectiveness review Deliberations on patient perspective, clinical data, and economic data Recommendation to the Executive Officer How does the CED use the patient submissions along with the other evidence?
- 17. Patient evidence submissions are accepted from registered patient groups only. Individual patients are encouraged to contact an organized patient group if they wish to make a submission. Each patient group must submit a complete registration form, either prior to or at the time of submission. A drug review schedule is posted on the Ministry’s website outlining all new drugs undergoing funding review. Patient groups are encouraged to check the drug schedule regularly and to provide their patient evidence submission on the Ministry’s template by the posted deadline. The deadline is based on the anticipated CED meeting date. A minimum of 1 month is provided from time of posting on the drug review schedule to the submission deadline. Balance: providing adequate time for groups to compile submissions while not delaying drug review and access http://www.health.gov.on.ca/english/providers/program/drugs/patien t_evidence/drugreview_schedule.htm How to make a submission
- 18. The patient evidence submission template form and the “how to guide” are posted on the Ministry’s website. http://www.health.gov.on.ca/english/providers/program/drugs/patient_evidence.html Submission template: Author information Conflict of interest declaration Impact of the disease/condition Outcomes that matter most to patients Information from patients who have used this drug How to make a submission (cont’d)
- 19. These suggestions are entirely my own and are not intended to represent the views or advice of anyone at the Ministry of Health and Long Term Care, of any other member of CED, or of the Committee as a whole. “Learnings”
- 20. Information not required: Scientific evidence (e.g. clinical trial data) regarding the efficacy and safety of the drug product and its comparators. The CED already has this information and it will be considered in the clinical section of the presentation and deliberations. If submitting to CADTH/pCODR and Ontario CED Look at your submission from an Ontario lens. Highlight what is different in the Ontario submission as compared to a submission to a national reimbursement agency Is there a higher incidence in this province? Are there unmet needs in Ontario that differ from other jurisdictions? We expect that there may be some evidence which apply to both submissions. “Learnings” to make submissions more useful and impactful
- 21. Patient input can be provided by various means and all are considered and important Surveys, , interviews, focus groups, patient stories, etc. Best advice is be transparent and describe how you gathered the information How many telephone interviews? How many patients/caregivers completed the survey, attended the focus group, etc. Include the date State the objective of your information gathering –i.e. general information on living with this condition, specific information from patients who have taken this drug, etc. Don’t worry if the numbers are small, especially for rare diseases “Learnings” – Information Gathering
- 22. Your input can be more meaningful if you present the impacts by prioritizing them from most important impact on patients or most difficult adverse events to tolerate For example, the top three symptoms that impact the day to day lives of these patients What is the effect of the symptom on your quality of life? (does it prevent you from working full-time, can you prepare meals for your family, etc.) Anecdotal statements from patients and/or caregivers can be very insightful If the same theme is repeated in anecdotal statements, choose one that is representative and state it is pervasive “Learnings” – Impact of Disease/Condition
- 23. Be as specific as possible in this section Most patients want choices but this is a generic statement What are some of the practical issues and how do they tie in to the impact of the condition? (subcutaneous injection vs. intramuscular, etc.) Given that the majority of treatments are not breakthrough, what value does this drug add? What will this drug do differently? (Is is the first to address a particular symptom, long-acting vs. short acting, etc.) Is there an unmet need with current therapy? What are the most important aspects of the condition that you wish to see addressed by treatments? “Learnings” – Treatment Outcomes
- 24. Often occurs that you can’t provide information for this section – just say so Can include patients’ input on the expectations for this drug If numbers are limited – say so and state number of patients Don’t base it on research from pharmaceutical companies or from doctors – keep it focused on the patient Include both positive and negative experiences Include side effects of this drug and what patients are willing to accept, what is unacceptable How does the drug compare to current therapy – ease of administration, side effects, etc. “Learnings” – Information from patients who have taken this drug
- 25. Meaningful patient information The most important aspects of the illness that patients would like the drug therapy to address. The shortcomings of existing therapies (that the new drug may or may not be able to address). Other practical aspects of the illness that should be taken into consideration (e.g. associated costs of living with the disease). If you feel that something very important was not specifically asked, you may include it Prioritize By prioritizing the most important aspects of the illness and treatment outcomes, patient evidence can help set the context for weighing the clinical and economic data and understanding the therapeutic gaps that may exist. “Learnings” in a nutshell
- 26. Contact Information: OPDP Patient Submission 5700 Yonge Street, 3rd Floor Toronto, Ontario M2M 4K5 Fax: 416-327-8123 Email: [email protected] Patient evidence submission: http://www.health.gov.on.ca/english/providers/program/drugs/patient_evidence/guidelines.html Ontario Public Drug Programs: http://www.health.gov.on.ca/english/providers/program/drugs/drugs_program_mn.html Questions & Comments
Editor's Notes
- #3: Main program is ODB ODB Program: ODB Program reimburses 3,400 drugs (either listed on ODB Formulary or reimbursed on a case-by-case basis) Eligible recipients Seniors (over 65 years of age) People on social assistance (Ontario Disability Support Program and/or Ontario Works) People residing in homes for special care and long-term care facilities People receiving professional home care services Deductibles and co-payments Exceptional Access Program (EAP) Created to handle requests for funding drugs that are not listed in the Formulary Individuals with clinical circumstances may be provided exceptional access to certain drugs Approximately 850 drugs provided on an exceptional access basis Trillium Drug Program (TDP) Intended for Ontario residents who have high prescription drug costs in relation to their net household income. Any Ontario resident that does not qualify under any of the other plans can apply for the Trillium Drug Program
- #11: Main program is ODB ODB Program: ODB Program reimburses 3,400 drugs (either listed on ODB Formulary or reimbursed on a case-by-case basis) Eligible recipients Seniors (over 65 years of age) People on social assistance (Ontario Disability Support Program and/or Ontario Works) People residing in homes for special care and long-term care facilities People receiving professional home care services Deductibles and co-payments Exceptional Access Program (EAP) Created to handle requests for funding drugs that are not listed in the Formulary Individuals with clinical circumstances may be provided exceptional access to certain drugs Approximately 850 drugs provided on an exceptional access basis Trillium Drug Program (TDP) Intended for Ontario residents who have high prescription drug costs in relation to their net household income. Any Ontario resident that does not qualify under any of the other plans can apply for the Trillium Drug Program
- #13: Patient members are Ontarians living with a chronic health condition
- #18: - CED will consider individual patient submissions under CADTH’s pilot project - it is only under very rare circumstances (has occurred just once) that CED considered an individual patient submission when there was no patient group. We want to encourage patients to continue to work through their associations/groups wherever possible. One person’s input may germinate another’s and provide a more fulsome picture.
- #22: Statements such as “connecting with other healthcare providers” or “information gathered from attending medical and educational events” are too vague
- #23: - Broad statements are generally not as helpful Give example of how anecdotal statements are used during deliberations (will often be read during the presenter’s report) Example – “I’m so frustrated because my doctor keeps telling me to lose weight to improve my condition but then gives me a drug that makes me gain weight.” Short statements that go to the heart of the matter often have most impact it is really difficult to say what will be helpful and how much is enough. There is no formula. Therefore, there may be occasions when one anecdote or information from a small number of patients would be sufficient but not for others. One anecdote may give the CED an “ah-ha” moment whereas another anecdote may not
- #24: This is where you make an effort to tie in all the different parts Other practical aspects of the illness that should be taken into consideration (e.g. associated costs of living with the disease). Administration of medication, travel, etc.
- #26: - Patient input helps inform the overall recommendation and decision – grey evidence