CML Adherence Study: Lessons Learned

Transcription

1 From anecdote to evidence CML Adherence Study: Lessons Learned Jan Geissler Co-founder, CML Advocates Network, Chair, LeukaNET e.v. Director European Patients Academy (EUPATI)

2 Why patient-generated evidence? Example Chronic Myeloid Leukemia Untreated Chronic Myeloid Leukemia (CML) kills in 2-3 years. Well-treated CML allows a normal lifespan. This is cancer! Of course MY patients take their medicines! nonsense! Non-adherence is a key problem in CML (ADAGIO 2008, Hammersmith) Clinical impact of adherence is proven Adherence <90%! molecular remission (Bazeos 2009)

3 Patient-driven research on adherence in CML: Research objectives Our research objectives Understand patient behaviours associated with adherence Help identify the true issues behind non-adherence Explore cultural influence Support development of physician and patient tools to improve patient outcomes

4 Patient-driven research on adherence in CML: Process Our Process Pilot study Scientific study Publication

5 Why patient-generated evidence? Example Chronic Myeloid Leukemia Pilot study Scientific study Publication Pilot study to test hypothesis and questionnaire Build workgroup (8 advocates, 3 experts) Low effort, little budget 10 countries, 150 patients, recruited via members Published in 2013 Scientific study with validated adherence scale Multi-sponsored project supported by logistics agency 79 countries, 2549 patients, recruited online and offline in 4 months Presented at EHA and ASH in 2013 Publication to be submitted to Blood in 2016

6 Patient-led adherence research in CML: 12 languages, 2546 patients, 79 countries, validated tool Global reach Countries with >30 respondents Countries with base <30 respondents Sample: Total of 2546 respondents Online 2151 Paper 395 Methodology Online - Recruited by patient associations online & via other methods Paper & Pen (France, Germany, Italy) Recruited by physicians at consultations CML patients over 18 years old, currently taking oral medication for CML Fieldwork Started on CML World Day, 22/ th January 2013

7 Adherence is generally poor, in a cancer where disease control is clearly linked to adherence Low adherent: 21% Medium adherent: 47% Highly adherent: 33% Sharf et al., Haematol 2013; 98(s1), EHA-Abstract [1104]; Geissler et al., Blood 2013;112:4023. ASH-Abstract [4023]

8 Can a single-country study provide global answers? Certainly not. % Missed on Purpose in last year Titolo del grafico Global Average 33, , , , ,522, , , , , , , , ,178615, , , , ,1783 Above global average for missed doses 34,9593 9, ,5714 Below global average for missed 5, doses Global Average % Missed Accidentally / Due to Circumstance in last year C2a / base=all respondents (n=2546) - Patients sometimes are not able to take their medication as prescribed. In the last month, have you missed a dose accidentally or due to circumstances that were outside of your control? C2c / base=all respondents (n=2546) In the last year, have you missed a dose accidentally or due to circumstances that were outside of your control? C4a / base=all respondents (n=2546) - Patients sometimes make a conscious decision to miss a dose of medication. In the last month, have you decided to miss a dose? C4b / n= In the last year, have you decided to miss a dose?

9 Imatinib seems to be linked with higher adherence, Nilotinib more prevalent in low adherence group (on Morisky Scale, so all motivations for non-adherence regarded!) Current medication vs. levels of adherence % + Nilotinib 36% 25% 33% + Nilotinib 46% 48% 46% High Medium Low 19% 27% 21% Imatinib Nilotinib Dasatinib B3a / base-all respondents (n=2546) Low (n=528), Medium (n=1185), High (n=833) - Which medication do you take for your CML currently?

10 Forgetting & routine interruption are primary reasons for accidental, (gastro) SE for intentional non-adherence Reason for missing accidentally % (n=1283) Reason for deciding to miss % (n=491) Forgot 41 Not feeling well 35 Interrupted routine Travelling Reduce side effects Attending special occasion Wanted to socialise Too ill Fell asleep Ran out of medication Medication not ready at pharmacy Interfered with travel Feeling down Dr said could miss CML under control Interfered with work Side effects aiming to reduce: Gastro (79%) Dermatological (17%) Reminder failed Couldn't swallow 4 1 Didn't want to be reminded Feeling better To save money Mental (21%) Dosing schedule complicated 1 Friend/partner said could miss 0 C2d / n= Which circumstances led to a missed dose of your medication? C5 / n=491 - Why did you decide to miss a dose of your medication? C6 / n=126 - Which side effect(s) were you hoping to avoid by intentionally missing one or more doses of your medication?

11 Successes First patient group ever to present data in oral scientific sessions at European Hematology Association (EHA) congress Focus: overall data Poster presented at American Society of Hematology Meeting (ASH) in Focus: Risk factors of patients on intentional non-adherence Mobile iphone/android App launched in 2015 Publication just about to be submitted to Blood Journal

12 Lessons learned Run a pilot to test your hypothesis and survey/research design Anticipate potential criticism! Generate solid data e.g. by using validated tools. Use a reliable agency and medical writers (costs $$, but worth it) to support collection, run first analysis, write the papers volunteering is cheaper but takes ages! Publish the data to get acceptance in HCPs! (And get reliable help for this)

13 Patient-generated evidence is great! Jan Geissler

14 WE CAN Informal Workgroup of European CAncer Patient Networks Initiated at CC, 28 Sept 2015

15 18 European Cancer Patient Networks 1. EuropaColon - Colon Cancer (Jola Gore- Booth PAC) 2. Europa Donna - European Breast Cancer Coalition (Sema Erdem PAC, Susan Knox CEG-CC) 3. Europa Uomo - European Prostate Cancer Coalition (Ken Mastris PAC) 4. ECPC (Vlad V PAWG, Jana Pelouchova PAC, Kathi A PAWG, FdL CEG-CC) 5. EMHF - European Men's Health Forum / Prostate (Ian Banks, PAC, PAWG) 6. ENGAGe - European Network of Gynaecological Cancer Advocacy Groups (Isabel Mortara, CEG-CC) 7. EURORDIS - Rare Cancers / Rare Diseases (Ariane Weinman, CEG-RD) 8. IBTA - International Brain Tumour Alliance (Kathy Oliver PAC PAWG CEG-CC) 9. IKCC - Kidney Cancer (Markus Wartenberg, ESMO PAWG, RCE) 10. Childhood Cancer International (CCI) (Gerlind Bode PAC) 11. Leukemia Patient Advocates Foundation - Leukemias (Jan Geissler, PAC, PAWG, CEG-CC, CEG-RD) 12. LuCE Lung Cancer Europe (Stefania Vallone) 13. Myeloma Patients Europe (Sarper Diler PAC) 14. MPNE - Melanoma Patient Network Europe (Bettina Ryll, ESMO PAWG) 15. SPAEN - Sarcomas (Markus Wartenberg, ESMO PAWG, RCE) 16. EWMnetwork - European Morbus Waldenström Network (Marlies Ooom) 17. MDS Alliance - Myelodysplastic Syndrome (Sophie Wintrich) 18. Lymphoma Coalition Europe (Charlotte Roffiaen)

16 Our challenges Lack of coordination: No regular opportunity for the leaders of pan- European cancer patient umbrella organisations to get together to discuss ideas and build consensus. Leading organisations acting in isolation, with best intentions, but little alignment or mutual understanding. Inefficiency: Umbrella organisations are fragmenting their resources between different committees like PAC, PAWG, EU Expert Groups, Advisory Boards. There is currently no forum to discuss how to share workloads and involve additional advocates. Diversity and representativeness: None of these committees will ever have a representative of all key indications. Little space for new advocates Opacity: The leaders of the umbrella organisations are not reporting back to the community about their work in the committees, losing the opportunity for better coordination, mutual learning, effective sharing, more capacity building.

17 Objectives and tasks Share workload for meetings, but maybe send only 1-2, in close alignment, consultation and feedback structure with wider workgroup Coordinate and align our representation and community input into advisory boards, expert groups and cancer initiatives Ensure communication and reporting mechanisms between leaders. Form a "think tank" to develop ideas on how to cross bridges between advocacy Share resources and concepts on joint patient-driven evidence Map the current European scene on initiatives/political actions/ organisations