Submission of Patient Evidence

Transcription

1 Ministry of Health and Long-Term Care Ontario Public Drug Programs 5700 Yonge Street, 3 rd Floor Toronto ON M2M 4K5 Submission of Patient Evidence Please send completed submission and/or any additional relevant information to the Ontario Public Drug Programs, Patient Evidence Submission, 5700 Yonge Street, 3 rd Floor, Toronto ON M2M 4K5, fax to or to PatientSubmission.OPDP@ontario.ca. Section I Author Information Date (yyyy/mm/dd) 2015/10/05 Drug and Indication Jardiance (empagliflozin), type 2 diabetes Author Jane Tsai Patient Advocacy Group Canadian Diabetes Association Unit No. Street No. 45 Street Name Montreal Road City/Town Ottawa Telephone No. (incl. area code) (613) address Jane.tsai@diabetes.ca Province ON PO Box Postal Code K1L 6E8 Section II Conflict of Interest Declaration The Author and the patient group must declare any potential conflicts of interest that may influence or have the appearance of influencing the information submitted. Examples of conflicts of interest include, but are not limited to, financial support from the pharmaceutical industry (such as educational/research grants, honoraria, gifts, and salary), as well as affiliations or personal/commercial relationships with drug manufacturers or other interest groups. The Canadian Diabetes Association (the CDA) solicits and receives unrestricted grants from multiple manufacturers/vendors of pharmaceuticals, supplies and devices for diabetes and its complications. These funds help the CDA to support community programs and services for people with diabetes, fund research and advocacy, across Canada. Sponsors were not involved in developing this submission. The CDA did not have any conflicts of interest in the preparation of this submission. Section III Impact of the Disease/Condition What symptoms and problems do patients have as a result of the disease/condition? How does the condition affect dayto-day life? For example, are there activities that patients are not able to do as a result of the condition? Information presented in this submission is derived from two surveys conducted by the Canadian Diabetes Association (the CDA), distributed through social media and blast. The first survey, conducted in November 2014, gathered information from 320 Ontarians with type 2 diabetes and their caregivers about the reality of living with or caring for someone with type 2 diabetes. The second survey, conducted in April 2015, gathered information about the use of empagliflozin (Jardiance) and dapagliflozin (Forxiga), to inform a patient input submission to the CDR. Questions from these surveys included current drug therapy, previous experience with drug therapy and experience with empagliflozin and dapagliflozin. A total of 173 Ontarians completed the survey, including 138 people with diabetes and 27 caregivers residing in Ontario. Impact of diabetes E (2010/04) Page 1 of 5

2 Type 2 diabetes is a chronic (progressive) condition that occurs when the pancreas does not produce enough insulin or when the body does not effectively use the insulin that is produced. Insulin is a hormone that controls the amount of glucose in the blood. Diabetes is a disease that requires considerable self-management, including healthy eating, regular physical activity, healthy body weight, taking diabetes medications (oral and/or injection) as prescribed, monitoring blood glucose and stress management. The majority of patients surveyed indicated that daily fluctuations in blood sugar were the most important aspect of diabetes to control during the day and overnight. The fluctuations impact the ability to work, interactions with friends and family, causes stress and worry as well as ability to participate in normal activities of daily living. Uncontrolled diabetes and the stigma associated with the disease can result in reduced quality of life. Maintaining control of diabetes has potential to reduce anxiety and avoid or delay complications as well as improve overall quality of life. Below are some direct quotes from people with diabetes regarding the impact of their disease: Having diabetes makes me useless. I have no energy or strength to enjoy life anymore. I can't do partial jobs around house. I can't enjoy sports anymore. Diabetes has instill[sic] a fear in me. It is a life altering disease that impacts every aspect of life. There is constant blood monitoring, diet, level of activity, cost of expensive supplies and medication. Managing diabetes is very stressful because you take lots of medication and in spite of your best efforts, maintaining target range is difficult. The most distressing side effect of all of the diabetes drugs is they make you gain weight or prevent weight loss. It is annoying to be told to lose weight then handed a drug that prevents weight loss. problems are mostly trying to lose weight..took 5 Metformin pills (1 a day for 5 days ) and put on 2 pounds a day. Took over a month to loose[sic] that 10 pounds. Basically it's an awful experience, experience highs and lows. Exercising can make my sugars low so I have to always have a snack with me. Eating anywhere besides home is a challenge as you don't know how things are made. I'm constantly checking my blood and I take 13 pills a day. Treatment outcomes that matter most to patients What are the most important aspects of the condition that patients would like to see addressed by treatments? In terms of treatment efficacy and side effects, what are patients getting from the existing treatments and what would patients like new treatments to do differently? Are there other practical implications to be considered in determining the value of a treatment? For example, how do treatments impact patients or caregivers daily routine or lifestyle? In addition to the drug cost, are there other financial implications to patients or caregivers (e.g. traveling cost, time away from work, drug disposal issues, drug administration supplies)? A large proportion of people with type 2 diabetes fail to achieve optimal glycemic control, which places patients at risk for both acute and chronic diabetes complications. Initial therapy is most often with metformin, but over time, most patients will require the addition of a second or third agent to reach glycemic targets. Many of the currently available second-line therapies cause significant weight gain while their ability to achieve optimal glycemic control may be limited by hypoglycemia. Experience with current treatment: E (2010/04) Page 2 of 5

3 A total of 165 Ontarians with type 2 diabetes and caregivers indicated experience with diabetes medications. About 70% of respondents expressed satisfaction with current treatments, 15% were neither satisfied nor dissatisfied, and 17% are dissatisfied with current treatments. While the majority reported the current therapies helped improve their blood glucose levels, meet A1C target, and avoid hypoglycemia, a significant proportion of respondents did not note much difference or even worse outcomes in blood glucose levels the same, worse or much worse for 26% (fasting), 28% (postprandial), and 32% (upon waking). In terms of meeting target A1C levels, 25% of respondents reported no difference or worse outcomes, and 38% did not observe improvement in avoiding hypoglycemia. The majority did not observe improvement in weight management and side effects: 55% of respondents noted the same, worse or much worse for weight gain, 59% observed no improvement in GI effects; 57% and 56% indicating same, worse or much worse for dehydration and urinary tract/yeast infection, respectively. Overall, respondents were more satisfied than dissatisfied with their medications in terms of the ability to manage their blood sugar levels. However, there were considerable issues with side effects. Most important aspects to address: Surveyed patients were also asked to rate the importance of the following benefits/side effects when choosing diabetes medications, using a five-point scale from not at all important to very important. Over 90% of respondents indicated quite important or very important regarding the following benefits of therapy: - blood sugars kept at satisfactory levels in the morning/after fasting (95%), - blood sugars kept at satisfactory levels during the day/after meals (95%), - avoiding weight gain (94%), - avoiding low blood sugar during the day/overnight (91%). The following aspects are also considered important by the vast majority: - reducing high blood pressure (87%), - avoiding fluid retention (86%), - avoiding GI effects (83%), - avoiding urinary tract infection (83%). One respondent simply wanted drugs to provide a life without concerns about complications because of diabetes. Expectation of new therapies: Close to 100 Ontarians with type 2 diabetes/caregivers would like to see improved A1C and blood glucose control (including less hypoglycemia), without serious side effects or long-term damage to health. Many hoped that the new therapies would eliminate the need for multiple insulin injections or medications. Cost is a concern for many, including seniors. The following summarizes the list of outcomes that they hoped for: - drugs are safe and effective - improved A1C - improved and effective blood glucose control ( Manage blood sugar level in the target range throughout the day and night without experiencing low or high sugar levels, i.e., managing levels becomes less stressful. ; I am hoping that a new drug will regulate blood sugar so that there would be no highs and lows. ) - reduced hypoglycemia events - no side effects/free of serious side effects such as fluid retention, increased risk of renal damage - free of diabetes complications ( reduce number of patients on dialysis, losing limbs, heart problems, organ failures ) - reduction of other diabetes medications ( Reduce the amount of pills I take every day...which is 6 for type 2 diabetes only ; I hope that it would be a one tablet or injection instead of multiple medications to treat type 2 diabetes. ) E (2010/04) Page 3 of 5

4 - reduction of insulin rejections ( I am comfortable with insulin but if medications can help reduce the amounts or frequency of injections without the risk of serious side effects I would welcome that. ) - Weight loss/maintenance ( Hoping meds will not add weight. My earlier ones added about 20 pounds--not exactly ideal for a diabetic! ) - less stress ( making life living with diabetes less complications and overwhelming ) - depression ( If these meds can help you feel better it will help with the depression of diabetes. ) - coverage for cost ( presently the drug is covered through drug company for 1 year what happens then? Won t be able to afford [drug] after the one-year supply ; as a senior I would hope these drugs will be covered ; When a new drug is introduced, it is not covered by the government and the cost is extremely high putting it out of the reach of most Canadian.. As a result, only a few people will see the benefit. Many also indicated their hope for a cure for type 2 diabetes. Information from patients who have used this drug For patients who have used this drug as part of a clinical trial or from a manufacturer s compassionate supply or have purchased it through other means (private insurance or patient out of pocket). What positive and negative impacts does the drug have on the condition? Which symptoms is the drug best or worst at treating (advantages and disadvantages)? What difference does the drug make to patients long-term health and wellbeing? What are the side effects of the drug, which ones are patients prepared to put up with, and which ones do they find unacceptable? How does the drug compared with other available treatments in terms of efficacy, side effects and other practical implications (e.g. administration, time, costs)? Empagliflozin belongs to a new class of drugs to lower blood glucose through inhibition of subtype 2 sodium-glucose transport protein (SGLT2), which is responsible for at least 90% of the glucose reabsorption in the kidney. The SGLT2 inhibition also causes a reduction in blood pressure and weight loss. Information presented in this section was gathered from the survey conducted in April 2015 about the experience with SGLT2 inhibitor drugs empagliflozin and dapagliflozin (both drugs in the same class), to inform a patient input submission to the CDR. Eight respondents with type 2 diabetes/caregivers indicated they have experience with empagliflozin. Due to the small number of responses for Jardiance, we will report on patients experience using this medication as well as provide quotes regarding both surveyed drugs in the class. People who have taken empagliflozin before indicated it lowers blood sugar/keeps blood sugar in normal range, helps to lose weight, controls blood pressure, decrease side effects, i.e. diarrhea, stomach ache. Generally, the majority of people who have had experienced with this drug or those who have not used this drug would like to see empagliflozin made available as an option to treat type 2 diabetes. Some noted its potential to lower weight and blood pressure in some people with diabetes as a main reason. Others cited the wish to reduce the number of medications if empagliflozin was available: If I can take one medication rather than 3 and still be well controlled, there should be less cost and side effects. Overall, empagliflozin is thought to (have the potential to) address several aspects that are important to people with type 2 diabetes, namely, better blood glucose control, preventing weight gain, lowering blood pressure, and minimal side effects. An Ontarian with type 2 diabetes who has used empagliflozin in a past trial and now on another class of drugs expressed his wish that empagliflozin would be approved in Canada because it worked [other drugs] cause weight gain and do not work as well as empagliflozin. Direct quotes regarding the benefits of SGLT inhibitors (empagliflozin and dapagliflozin): E (2010/04) Page 4 of 5

5 his blood sugar are below 10, he hasn't been below 10 in years the other day, it was 6.3 that's amazing and his blood pressure has come down also, if he improves the Doctor may decrease some of his other diabetic meds Better blood glucose control with lower blood sugar readings smaller amounts of insulin are needed to maintain good numbers. Easier to prevent weight gain an excellent means to lower aic's[sic] (from 8+ to 7). I am completely satisfied. I would not want to give it up. For me it is the best thing ever with my combination of meds. it is working for me first time my body has been happy i can not tell you how good my body feels I have cut my insulin in half and reduced my pills in 2 months. Readings are best I have ever had. Feel great Huge benefit in reducing fasting glucose and HbA1c. Significant weight reduction (loss of > 4 Kg) evident in first 3 months. Very +ve[sic] impact on my self-confidence and feeling of "being in control" with my diabetes. Some have found the side effects a challenge: Caused repetitive yeast infections and urinary tract infections as well as unpredictable bouts of bowel incontinence. I have never felt so ill in my life. Horrible issue with balanitis peeing all the time and dehydration concerned about fall risk in my dad First week experienced vaginal yeast infection, severe constipation and a small weight loss. Subsequently no side effects, no weight loss and no significant change in blood sugar levels. weight gain and bloating very uncomfortable and mentally/physically difficult to deat[sic] with. Very depressing Diabetes requires intensive self-management. To achieve optimal blood glucose levels, individualization of therapy is essential, including selecting the drug or combination of drugs, route of administration (oral, injection, pen or pump), how frequently the patient monitors blood glucose and adjusts dosage, the benefits and risks that the patient experiences and/or tolerates, and the lifestyle changes the patient is willing or able to make. There are clear expectations that any new drugs should offer good blood glucose control to prevent hyperglycemic and hypoglycemic episodes, as well as longer term control, with minimal side effects and long-term damage to organs, and at affordable costs. Many patients also hope that new medications can help reduce the number and types of drugs taken including insulin injections. The patients who have had experience with empagliflozin or dapagliflozin described good results in terms of blood glucose and weight control. Different people living with diabetes require different options in terms of medications to help effectively manage their disease. Their clinical profile, preference and tolerance of therapy can direct physicians to the most appropriate drug therapy. To many Ontarians who answered our survey, available and affordable options are important in the treatment of type 2 diabetes. As one respondent puts it: [there should be] open-access to this new class of diabetes therapy, so that physicians and patients can have the flexibility to find the most effective and safe "mix" of drugs to maintain control of diabetes. Diabetes changes over time, and everyone needs different medications at different times of their life with diabetes. The availability of empagliflozin (Jardiance) provides an important option for people with type 2 diabetes. Confirmation of Authorship: I declare that I am the sole author of this submission and confirm that no other parties had input into the submission. Signature Date (yyyy/mm/dd) 2015/10/ E (2010/04) Page 5 of 5