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This last decade has been marked by significant advances in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies. C&G therapies offer transformative benefits to patients but present a challenge to current health technology decision-making systems because they are typically reviewed when clinical efficacy data are very limited and when there is uncertainty about the long-term durability of outcomes. These challenges are not unique to C&G therapies, but they face more of these barriers, reflecting the need for adapting existing value assessment frameworks. Still, C&G therapies have the potential to be cost-effective even at very high price points. The impact on healthcare budgets will depend on the success rate of pipeline assets and on the extent to which C&G therapies will expand to wider pathologies beyond rare or ultra-rare diseases. Getting pricing and reimbursement models right is important for incentivising research and development investment while not jeopardising the sustainability of healthcare systems. Payers and manufacturers therefore need to acknowledge each other’s constraints—limitations in the evidence generation on the manufacturer side, budget considerations on the payer side—and embrace innovative thinking and approaches to ensure timely delivery of therapies to patients. Several experts in health technology assessment and clinical experts have worked together to produce this publication and identify methodological and policy options to improve the assessment of C&G therapies, and make it happen better, faster and sustainably in the coming years.

Background A significant proportion (up to 37%) of patients (pts) with chronic phase (CP) CML experience either resistance or intolerance to first-line tyrosine kinase inhibitor (TKI) therapy within 5 years and transition to other treatment options(Heeg B, et al. 2021). There are limited data regarding experiences and unmet needs of pts receiving second and later TKI therapy for CML, particularly with respect to quality of life and emotional wellbeingand concerns around disease response and long-term outcome. The CML survey on unmet needs (SUN) research aims to further characterize in depth these unmet needs from the perspective of both the pts and treating physicians. Methods CML SUN consists of two sequential phases. Phase 1 entailed of 60-minute in-depth telephone qualitative interviews with adult pts on second line or later TKI treatment and physicians treating CML in France, Germany, Japan and the USA. The insights from this initial phase will inform the design of the second phase, a larger quantitative survey. This abstract reports the results of the Phase 1 qualitative research. Pts were recruited through physician referral, pt panels, pt advocacy groups and social networking service sources, while physicians treating CML were recruited via panels/databases. Key inclusion criteria can be found in Table 1. A semi-structured interview guide covering the same topics for both pts and physicians was used to conduct the interviews. The guide consisted of pre-determined but open-ended questions on the topics of perception of the disease, burden of disease, treatment journey, the impact of treatment change, adverse events (AEs), ideal treatment and needs which have not been previously identified or met. For each of the recorded interviews, a detailed content analysis was conducted to organize the collected data by themes and topics. This content analysis was used to compare responses between individuals within and across countries, and used to identify insights and trends. Results A total of 21 pts and 24 physicians were interviewed (Table 2: demographics). Physician: While physicians were mostly the ones to initiate treatment switch discussions with their pts, they typically did not provide pts with different treatment options and/or clear treatment goals when selecting 2 nd line or subsequent therapy. Most believed that pts & caregivers have minimal or no role in this matter. Therapy switch tended to happen faster if due to lack of treatment response rather than side effects which, unless severe or very frequent, were first treated symptomatically or with dose modification. Physicians felt that pts have minimal or no unmet needs requiring support when it comes to switching therapies. Patients: An almost equal proportion switched treatments due to resistance vs due to AEs. The need to switch TKI therapy induces anxiety and concerns for pts. Some pts downplayed their AEs either because of fear of triggering a therapy switch or trying to avoid being a burden. Most pts expressed the need to receive additional information on the necessity of therapy switch and on the potential therapy side effects. While physicians and pts had similar goals for the new treatment: to be effective and generally tolerable; physicians placed a higher priority to the efficacy of the treatment, whilst pts placed a higher priority to reducing the burden of AEs. Conclusions This first phase of this research has identified different perspectives and needs between CML pts and physicians in relation to change of TKI therapies, as well as potential divergencies between the two groups regarding their perception of unmet needs. This information will help design the second phase of the study which will further quantify the impact of these unmet needs in a larger population across a broad geography of 11 countries. This knowledge can be used to raise awareness and to develop support strategies to improve current CML management practices and the pt experience. Figure 1 Figure 1. Disclosures Pemberton-Whiteley: Acute Leukemia Advocates Network: Consultancy, Membership on an entity's Board of Directors or advisory committees; Leukaemia Care: Current Employment; AbbVie, Agios, Amgen, Astellas, Autolus, Bristol-Myers Squibb, Celgene, Daiichi Sankyo, Gilead, Incyte, Jazz, Janssen, Kite, Kyowa Kirin, Mallinckrodt, Novartis, Pfizer, Roche, Servier, Takeda: Consultancy, Other: Organizational grant funding, Speakers Bureau; WECAN (Workgroup of European Cancer Patient Advocacy Networks): Consultancy, Membership on an entity's Board of Directors or advisory committees; CML Advocates Network: Membership on an entity's Board of Directors or advisory committees. Cortes: Pfizer: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Bristol Myers Squibb, Daiichi Sankyo, Jazz Pharmaceuticals, Astellas, Novartis, Pfizer, Takeda, BioPath Holdings, Incyte: Consultancy, Research Funding; Bio-Path Holdings, Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Research Funding; Sun Pharma: Consultancy, Research Funding. Clements: Novartis Oncology: Honoraria, Other: Member of advisory board; Takeda Pharmaceuticals: Honoraria; Pfizer Pharmaceuticals: Honoraria, Other: Educational Grant; Leukemia and Lymphoma Society NPO: Membership on an entity's Board of Directors or advisory committees, Other: Patient Financial Assistance; CML Advocates Network NPO / CML Horizons Conferences: Honoraria, Membership on an entity's Board of Directors or advisory committees; The MAX Foundation NPO: Honoraria. Ruiz: Novartis: Honoraria; Pfizer: Honoraria. Rea: Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Incyte: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees. Machado: Pfizer Canada: Consultancy, Honoraria; Novartis: Consultancy, Honoraria. Lang: AstraZeneca: Consultancy, Honoraria; Incyte: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Takahashi: Novartis Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Otsuka Pharmaceutical: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Chugai: Research Funding; Eizai: Research Funding; Asahikasei: Research Funding; Kyowahakko-Kirin: Research Funding; Toyamakagaku: Research Funding; Ono: Research Funding. Deekes: Novartis: Honoraria. Moon: Novartis: Honoraria, Other: Member of advisory board. Grigg: Novartis: Honoraria, Other: Member of advisory board. Steagall: Novartis: Honoraria. Borowczak: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees. Pilipovic: Novartis: Current Employment, Current equity holder in publicly-traded company. Frank: Novartis: Current Employment, Current equity holder in publicly-traded company. Constantinescu: Novartis: Other: Cristina Constantinescu is an employee of Ipsos, who were paid consultants to Novartis. Boquimpani: Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Jansen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pinth Pharma: Speakers Bureau.

Background The Acute Leukemia Advocates Network (ALAN) conducted a multi-country survey to gather information on the experiences, quality of life (QoL) and symptoms of adults (16+) with acute leukemia [acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and acute promyelocytic leukemia (APL)]. Aims To examine which factors are most associated with differences in QoL and symptom burden (measured by HM-PRO scores), focusing here on age and patient-reported experience. Methods This survey comprised 99 items, designed from a literature review of QoL and acute leukemia followed by input from clinical and patient advocacy experts. The study material was translated (9 languages) and promoted via patient advocacy groups from March 1, 2019 to November 29, 2019. HM-PRO, an instrument designed to measure patient-reported outcomes in patients with hematological malignancies, was incorporated into the study for assessing QoL and symptoms. This consists of: Part A (impact/QoL); and Part B (signs and symptoms). A higher score in each part represents greater (negative) impact on QoL and symptom burden. Q6 of the survey asked respondents to report their year of birth. Q9 of the survey provides a measure for disease state: undergoing treatment, in remission following treatment or relapsed following treatment. It was hypothesised that patients with a worse experience in each of the following areas would report a worse overall QoL: management by healthcare professional of physical symptoms and side effects (Q13) and emotional impact (Q14); physical and mental health (Q15), information from healthcare professionals (Q16), ability to perform meaningful activities (Q17) and well-being of carers, friends and family (Q18). Spearman correlation analysis was used to determine the direction and strength of relationships between the measures. Wilcoxon rank-sum, Chi-squared and Kruskal-Wallis rank test were used to examine the differences between groups. Multivariable linear regression, with interaction terms was used to investigate the relationships between the questions and the tumour groups. Results There were 552 respondents. Patients self-reported their acute leukemia diagnosis - 332 AML, 139 ALL and 81 APL. There was no overall significant difference between Part A (p=0.55) and Part B (0.23) HM-PRO scores for different acute leukemia types. Of these, 158 were aged 16-40, 240 were 41-60, 150 were 61-87 and 4 did not answer this question. The results suggest a significant difference across the age groups in both Part A (p=0.005) and Part B (p=0.03) of the HM-PRO scores and signs and symptoms in with younger patients reporting a worse QoL. The results also confirmed our hypothesis that those with worse scores for Q13-Q18 have a worse QoL (higher HM-PRO score). The correlations (negative) were all statistically significant (p < 0.05), suggesting that good experiences are associated with low HM-PRO scores and vice versa (Table 1). The predictive value of Q17 and Q18 on overall QoL (HM-PRO scores) varied by tumour type, with a weaker relationship shown for the ALL group. This was confirmed as significant by the multivariable analysis. Q13-Q16 factors mostly appeared to have similar strengths of relationship with the QoL scores between the different acute leukemia types. Responses to Q14 differed significantly between disease types (p=0.01) with median 6 for APL vs 7 for ALL and 8 for AML. Similar differences were observed for Q13. Conclusions The results confirm there are significant differences (as measured by HM-PRO) in both Part A (impact/QoL) and Part B (signs and symptoms) across age groups, with younger acute leukemia patients reporting a worse QoL. Patients with worse reported experience (Q13-18) have worse HM-PRO scores, suggesting that improving support in these areas may enhance overall QoL. Disclosures Pemberton-Whiteley: Celgene: Consultancy, Other; Leukaemia Care: Current Employment; Acute Leukemia Advocates Network: Consultancy, Membership on an entity's Board of Directors or advisory committees; CML Advocates Network: Membership on an entity's Board of Directors or advisory committees; Blood Cancer Alliance: Membership on an entity's Board of Directors or advisory committees; WECAN (Workgroup of European Cancer Patient Advocacy Networks): Consultancy, Membership on an entity's Board of Directors or advisory committees; European Cancer Organisation - Patient Advisory Committee: Membership on an entity's Board of Directors or advisory committees; Patient Evidence: Membership on an entity's Board of Directors or advisory committees; Abbvie: Consultancy, Other: Organisational Grant Funding; Agios: Other: Organisational Grant Funding; Amgen: Consultancy, Other: Organisational Grant Funding; Astellas: Other; Bristol-Myers Squibb: Consultancy, Other, Speakers Bureau; Daiichi Sankyo: Other; Gilead: Other; Incyte: Consultancy, Other; Jazz: Other, Speakers Bureau; Janssen: Consultancy, Other; Kyowa Kirin: Other; Novartis: Consultancy, Other, Speakers Bureau; Pfizer: Consultancy, Other, Speakers Bureau; Servier: Consultancy, Other; Takeda: Other. Nier:Acute Leukemia Advocates Network: Consultancy; Servier: Consultancy. Geissler:BMS: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding, Speakers Bureau; Incyte: Research Funding; Takeda: Research Funding; Gilead: Consultancy, Speakers Bureau; Biomarin: Consultancy; Janssen: Consultancy, Speakers Bureau; Roche: Consultancy; Servier: Consultancy; UCB: Consultancy, Speakers Bureau; Novartis: Consultancy, Research Funding, Speakers Bureau. Wintrich:Takeda: Consultancy, Other: Project funding in MDS; Incyte: Consultancy, Other: Project funding in MDS; Novartis: Consultancy, Other: Project funding in MDS; Janssen: Consultancy, Other: Project funding in MDS; Celgene: Consultancy, Other: Project funding in MDS; Silence Therapeutics: Consultancy, Other: Project funding in MDS. Verhoeven:Novartis: Consultancy; Janssen: Consultancy. Christensen:Autisme 4700 DK: Consultancy, Membership on an entity's Board of Directors or advisory committees; Abbvie: Other: Organisational Grant Funding; Bristol Myers Squib: Consultancy; Gilead: Other: Organisational Grant Funding; Incyte: Consultancy, Other: Organisational Grant Funding; Janssen: Consultancy, Other: Organisational Grant Funding; Novartis Nordic: Consultancy, Other: Organisational Grant Funding; Pfizer: Other: Organisational Grant Funding; Roche: Consultancy, Other: Organisational Grant Funding; Takeda: Consultancy, Other; LYLE: Consultancy, Membership on an entity's Board of Directors or advisory committees. Salek:Merck: Consultancy; Agios: Consultancy, Honoraria; Pfizer: Honoraria, Speakers Bureau. Oliva:Amgen: Consultancy; Abbvie: Consultancy; Apellis: Consultancy; Alexion: Consultancy; Novartis: Consultancy; BMS: Consultancy, Honoraria, Patents & Royalties, Speakers Bureau. Ionova:Takeda: Research Funding; BMS: Research Funding. Bradley:Quality Health Ltd: Current Employment. Tate:Quality Health Ltd: Current Employment.

In CML, treatment-free remission (TFR) refers to having a stable deep molecular response without the need for ongoing tyrosine kinase inhibitor treatment. Whilst recommendations exist about the technical management of stopping and re-starting therapy, much is still unknown about the experiences of those considering and undertaking TFR. This study sought to obtain the patient perspective, identify areas of unmet needs and create recommendations for improvements. Fifty-six percent of patients reported fear or anxiety during treatment discontinuation, whereas only 7% of patients were asked if they needed psychological support during this period. Where patients re-initiated treatment; 59% felt scared or anxious, and 56% felt depressed. Twenty-six percent of re-initiated patients received psychological and/or emotional support at this time. Sixty percent of patients experienced withdrawal symptoms whilst discontinuing treatment, however, 40% of patients who experienced withdrawal symptoms reported that they were not fully supported by their doctor in managing all the symptoms. Healthcare professionals should further consider how they monitor the psychological well-being of patients who are discontinuing or re-initiating treatment, and review what support is offered in response to identified concerns. Surveillance of withdrawal symptoms should be a priority during treatment discontinuation, along with how healthcare professionals assist in the management of these.

Introduction Due to recent changes in acute leukemia treatment, there is an urgent need for greater understanding of the factors affecting quality of life at different points in the journey of patients. The Acute Leukemia Advocates Network (ALAN) is running a multi-country survey to gather information on the experiences, quality of life and symptoms of adults (16+) with different types of acute leukemia. The study objectives were to: (1) investigate whether the HM-PRO scores differ according to disease state; (2) determine whether those with worse scores on Q13-Q18 (patient-reported experience) will also report higher HM-PRO scores (a worse quality of life); (3) examine if there are significant differences for the HM-PRO scores between acute leukemia types and gender. Methods This survey comprises 99 items and was designed based on a literature review of quality of life and acute leukemia followed by input from clinical and patient advocacy experts. HM-PRO, an instrument to measure patient-reported outcomes in patients with hematological malignancies, was incorporated into the survey for assessing quality of life and symptoms. This is a composite measure consisting of: Part A (impact/quality of life); and Part B (signs and symptoms). A higher score in each part represents more impaired outcomes. Question 9 provides a measure for disease state (undergoing treatment, in remission following treatment or relapsed following treatment). We hypothesised that patients with a worse experience in each of the following areas would report a worse overall quality of life: physical symptoms and side effects (Q13), emotional impact (Q14), physical and mental health (Q15), information from healthcare professionals (Q16), ability to perform meaningful activities (Q17) and well-being of carers, friends and family (Q18). These are itemised on an interval scale ranging from 0-10, where 0 represents a bad experience and 10, a good experience. Questions 19-99 investigated further each of the hypotheses, to identify patient issues and background demographic questions. The survey was translated into 9 languages and promoted via patient advocacy groups from 1/3/19 to 31/5/19 (ongoing). Spearman correlation analysis was used to determine the direction and strength of relationships between the measures. Two sample Wilcoxon rank-sum tests and Kruskal-Wallis rank test were applied to test for differences between groups. Results There were 371 respondents: acute myeloid leukemia (213), acute lymphoblastic leukemia (86) and acute promyelocytic leukemia (72). There was no evidence of any difference in the HM-PRO score for either Part A - quality of life (p=0.9) or Part B - signs and symptoms (p=0.4) between acute leukemia types. Of these 149 were men and 219 female and 3 provided no gender. The median Part A score for males and females was 24.1 and 30.39, respectively (p=0.07). The median for Part B was 17.6 and 23.5 for males and females, respectively (p=0.01) with females reporting greater burden of signs and symptoms. The analysis suggests that there is a difference in the HM-PRO scores between disease state, with those in remission following treatment having lower HM-PRO scores (better quality of life) than those currently undergoing treatment (Figure 1&2). The results confirmed our hypotheses that those with worse scores for Q13-Q18 have a worse quality of life (higher HM-PRO score). The responses to all of these questions were either weakly or moderately related to scores of both parts of the HM-PRO (Table 1). The correlations (negative) were all statistically significant, suggesting that low HM-PRO scores are associated with good experiences and vice versa. Some individual questions were also highly correlated (rs = ≥0.7) with one another (e.g. Q14 and Q13, or Q17 and Q18), showing internal stability of the items. Conclusion The study confirms that acute leukemia patients in remission report a better quality of life than those currently undergoing treatment. However, there is no evidence of any difference in the HM-PRO scores between acute leukemia types which needs to be further explored in controlled studies. In terms of gender differences, the results indicate that female patients experience greater impact on quality of life and symptoms. Furthermore, patients with worse reported experience (Q13-18) have a lower overall quality of life, suggesting that improving support in these areas may enhance overall quality of life. Disclosures Pemberton-Whiteley: CML Advocates Network: Membership on an entity's Board of Directors or advisory committees; Patient Evidence: Equity Ownership; AbbVie: Other: Grant funding; Amgen: Consultancy, Other: Grant funding, Speakers Bureau; Bristol-Myers Squibb: Consultancy, Other: Grant funding, Speakers Bureau; Celgene: Consultancy, Other: Grant funding; Acute Leukemia Advocates Network (ALAN): Consultancy; Pfizer: Consultancy, Other: Grant Funding, Speakers Bureau; Takeda: Other: Grant funding; Shire: Other: Grant Funding; Kyowa Kirin: Other: Grant funding; Novartis: Consultancy, Other: Grant funding, Speakers Bureau; Incyte: Consultancy, Other: Grant funding; Jazz: Other: Grant funding, Speakers Bureau; Janssen: Consultancy, Other: Grant funding; Daiichi Sankyo: Other: Grant funding; Gilead: Other: Grant funding, Speakers Bureau; Leukaemia Care: Employment. Oliva:Celgene Corporation: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Apellis: Consultancy. Geissler:Incyte: Research Funding; Roche: Consultancy; Novartis: Consultancy, Research Funding, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; UCB: Consultancy, Speakers Bureau; Servier: Consultancy; Takeda: Research Funding; Pfizer: Consultancy, Research Funding, Speakers Bureau; Biomarin: Consultancy; Amgen: Consultancy; Bristol-Myers Squibb: Consultancy, Research Funding, Speakers Bureau. Wintrich:Takeda: Consultancy; Janssen: Other: Grant Funding; Celgene: Other: Grant Funding; Novartis: Consultancy, Other: Grant Funding. Salek:Pfizer: Honoraria, Speakers Bureau; Merck: Consultancy; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria. Ionova:Takeda, BMS: Other: Principal Investigator of IISR, Research Funding. Tate:Quality Health: Employment. Bradley:Quality Health: Employment.

Background In Chronic Myeloid Leukemia (CML), Treatment-free Remission (TFR) refers to having a stable deep molecular response without the need for ongoing Tyrosine Kinase Inhibitor (TKI) treatment. While first recommendations exist about how to manage stopping and re-starting therapy, based on data from the EURO-SKI study, much is still unknown about the experiences of those considering and undertaking TFR. Through this study, we sought to obtain quantitative evidence of patient experience that has previously only been anecdotal and to identify areas of unmet needs. One strong theme to emerge was patients' differing views on the need for psychological support. Method A global online survey was conducted, recruiting patients through CML patient associations, via online forums, social media and other methods. The questionnaire was designed by an expert panel of eight CML patients to capture the experiences of people along all phases of the TFR journey. The different phases were classified as: Phase I - Considerations around stopping treatment; Phase II - Probation period (experiences during the first 6 months of stopping treatment); Phase IIIA -Restarting treatment (experiences where treatment had to restart due to molecular reoccurrence), and Phase IIIB - Long-term remission (experiences of being in long-term, treatment-free remission). Once the question set was agreed, the questionnaire went through two rounds of testing by eight volunteers. This exercise contributed towards refining the questionnaire into a finished version. The questionnaire was translated into eleven languages: Arabic, Danish, English, Finnish, French, German, Hebrew, Italian, Japanese, Russian and Spanish. Fieldwork lasted 20 weeks. Results A total of 1016 responses were collected from CML patients across 68 countries. Patients only answered the sections of the questionnaire that were relevant for them. All 1016 had experience of Phase I, 494 (49%) had experience of Phase II, 159 (16%) had experience of Phase IIIA, and 203 (20%) had experience of IIIB. Of the 494 patients who stopped treatment, 32% said disease reoccurred and 41% reported being in long-term remission (this includes Conclusions There are opportunities for more communication and support around psychological issues to be given through the provision of information during the decision-making stage, and/or discussions between doctors and patients during the stopping stage. A considerable proportion of patients feel fear or anxiety at some level during stopping treatment, and not all patients who want psychological and/or emotional support receive it. The psychological well-being of patients should be a consideration of healthcare professionals and addressed at all stages of the TFR journey, to ensure patients receive support at the level they want through personalised care. Figure Disclosures Sharf: Incyte: Honoraria, Other: Advocacy Advisory Board, Research Funding; Abbvie: Other: Advocacy grants funding; Roche: Other: Advocacy grants funding; BMS: Other: Advocacy grants funding, Research Funding; Takeda: Other: Advocacy grants funding; Janssen: Other: Advocacy grants funding; Novartis: Honoraria, Other: Advocacy Advisory Board, Research Funding; Pfizer: Honoraria, Other: Advocacy Advisory Board, Research Funding. Hochhaus:Novartis: Research Funding; BMS: Research Funding; Pfizer: Research Funding; MSD: Research Funding; Incyte: Research Funding. Geissler:Pfizer: Consultancy, Research Funding, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Novartis: Consultancy, Research Funding, Speakers Bureau; Bristol-Myers Squibb: Consultancy, Research Funding, Speakers Bureau; Biomarin: Consultancy; Takeda: Research Funding; Amgen: Consultancy; Incyte: Research Funding; Roche: Consultancy; Servier: Consultancy; UCB: Consultancy, Speakers Bureau. Marin:Leukemia Patient Advocates Foundation: Employment. Pemberton-Whiteley:Acute Leukemia Advocates Network (ALAN): Consultancy; CML Advocates Network: Membership on an entity's Board of Directors or advisory committees; Patient Evidence: Equity Ownership; Daiichi Sankyo: Other: Grant funding; Gilead: Other: Grant funding, Speakers Bureau; Incyte: Consultancy, Other: Grant funding; Jazz: Other: Grant funding, Speakers Bureau; Janssen: Consultancy, Other: Grant funding; Kyowa Kirin: Other: Grant funding; Novartis: Consultancy, Other: Grant funding, Speakers Bureau; Pfizer: Consultancy, Other: Grant Funding, Speakers Bureau; Takeda: Other: Grant funding; Shire: Other: Grant Funding; AbbVie: Other: Grant funding; Amgen: Consultancy, Other: Grant funding, Speakers Bureau; Bristol-Myers Squibb: Consultancy, Other: Grant funding, Speakers Bureau; Celgene: Consultancy, Other: Grant funding; Leukaemia Care: Employment. Bombaci:CLL Advocate: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: grant funding ; CML Advocate: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: Grant funding ; Incyte: Consultancy; Onlus: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: grant funding; Novartis Pharma AG: Consultancy, Other: Advisory board ; MPN Advocate: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: grant funding ; Abbive: Consultancy, Other: grading funding; Bristol- Myers: Consultancy, Other: Grant Funding; Jazz: Consultancy, Other: Grant funding ; janssen: Consultancy, Other: grant funding. Christensen:Novartis: Consultancy, Other: Advisory Board; Incyte: Consultancy, Other: Advisory Board. Gouimi:AMAL: Membership on an entity's Board of Directors or advisory committees; Novartis Pharma: Consultancy, Other: Advisory Board; Bristol-Myers: Consultancy; Novartis: Consultancy, Other: funding grant; Takeda: Consultancy; Pfizer: Consultancy. Deekes:Novartis Pharma: Honoraria. Bradley:Quality Health: Employment.