NICE – a HUSUK’S EXPERIENCE Navigating the Highly Specialised Technologies Programme FindacureWorkshop 29 January 2016
INTRODUCTION – aHUSUK Len Woodward A Founding Trustee of aHUSUK Not a patient No clinical background Professional Accountant Used to be retired 1 of 4 Trustees who do all the work of the charity
INTRODUCTION – aHUS AND ECULIZUMAB Atypical Haemolytic Uraemic Syndrome (aHUS) Uncontrolled Complement- innate immune system Excessive clotting ,anaemia and renal failure or worse Eculizumab Complement inhibitor Already approved for another condition Paroxysmal Nocturnal Haematuria Drug also centre of a very public campaign for Dense Deposit Disease use
DISCLOSURE “Of course we are delighted for every sufferer of aHUS and their families. But we also recognise something very important has just occurred. This is the first time NICE (HST) has evaluated a rare disease ultra-orphan drug. We are impressed with the committee’s approach and believe that NICE has demonstrated a vision, willingness and capability to devise a process for evaluation of other such complex technologies to address the needs of those who seek equity of access to treatments of their severe rare diseases.”
AGNSS: BACKGROUND Advisory Group for NHS Specialised Services Early 2011 - Alexion/Professor Tim Goodship sought for it to evaluate Eculizumab for aHUS June 2011 - First aHUS Patients and Families Conference in Newcastle upon Tyne Patients found out about aHUS, Eculizumab and AGNSS Need for patient input and a patient organisation to do it
AGNSS : TIMELINE(1) • aHUSUK formed to give patient voice for the evaluation Septembe • Scoping meeting 5 weeks after formation r-October 2011 • Design of patient evidence submission February • Experience Report – Spectrum of Patients 2012 • Support from consultants to undertake and record interviews • The Report- Consultants/Patient Voice Trustee March – May 2012 • aHUSUK trustees approve draft
PATIENT EVIDENCE SUBMISSION: EXPERIENCE REPORT - METHODS A piece of qualitative research about what it is like to live with aHUS What the aHUS experience is like from on set to treatments ( living with dialysis) Conducted by trustee for Patient Voice, Toucan Associates , aHUS patients , carers and families Devised questions , recorded interviews , transcription and analysis 16 interviewees; spread of age , treatments , outcomes for patients 3 case studies of Eculizumab use
Table 1: Characteristics of the interviewees Age/ gender Interviewee Patient/family experience Girl, 9 months Mother In remission after treatment with Eculizumab. Child Mother 4 children who died as a result of aHUS, 3 as babies, 1 at age 7. Boy, 2 years Grandmother In remission after plasma exchange. Grandmother lost 2 of her own children to aHUS as babies. Father Receiving dialysis at home, awaiting transplant, receiving Eculizumab. Girl, 5 years Girl, 7 years Mother Receiving plasma exchange and on long-term dialysis Boy, 13 years Mother In remission. Young female adult Patient and sibling Has end stage renal failure and is receiving dialysis at home. Transplant failed. (2 interviews) Working. Young female adult Patient and husband Has end stage renal failure and is receiving dialysis at home. Transplant failed. (3 interviews) Retired due to ill-health. Young female adult Patient/husband/daughter Has end stage renal failure and is receiving dialysis in hospital. Transplant failed. (3 interviews) Retired due to ill-health. Adult late onset Patient In remission after treatment with Eculizumab. – female Adult late onset - Patient Has end stage renal failure and is receiving dialysis at home. Transplant failed. female
PATIENT EVIDENCE SUBMISSION: EXPERIENCE REPORT - METHODS A piece of qualitative research about what it is like to live with aHUS What the aHUS experience is like from on set to treatments ( living with dialysis) Conducted by trustee for Patient Voice, Toucan Associates , aHUS patients , carers and families Devised questions , recorded interviews , transcription and analysis 16 interviewees; spread of age , treatments , outcomes for patients 3 case studies of Eculizumab use Identify common themes, general conclusions and relevant supporting quotations from interviewees 30 pages of evidence
AGNSS : TIMELINE(1) • aHUSUK formed to give patient voice for the evaluation Septembe • Scoping meeting 5 weeks after formation r-October 2011 • Design of patient evidence submission February • Experience Report – Spectrum of Patients 2012 • Support from consultants to undertake and record interviews • The Report- Consultants/Patient Voice Trustee March – May 2012 • aHUSUK trustees approve draft
PATIENT EVIDENCE SUBMISSION: EXPERIENCE REPORT: OUTCOME Not as hard on the interviewees as was expected Took up a lot of Patient Voice Trustee’s time. The transcription of the recorded interviews is hard work Finding adult male patients to participate in interviews difficult Not only learned a lot about aHUS but also the methodology http://ahusuk.org/wp-content/uploads/2011/12/aHUSUK-Experiences-Document.pdf
AGNSS : TIMELINE (2) • AGNSS MEETING • 5 minute presentation and Experience Report • 7 month wait begins to hear outcome • total unwillingness to tell us anything June 2012 • Health and Social Care Act 2012 victims • AGNSS recommended eculizumab for aHUS • Clinical and patient case made January 2013 • Minister wanted advice on “affordability” from NICE • We were not happy!
FROM AGNSS TO NICE “Affordability”, “Value for Money”, “Wise use of Resources”, “ Cost effectiveness”, “use of limited funds” - depended on who you wrote to. No one mentioned “opportunity cost” “ Excessive Profits” • Met NICE likely timeline/patient numbers/correct finance professional March 2013 • IFRs again -OK for children not for adults • Clinical Priorities Advisory Group 1 – all new onsets in scope April 2013 • Clinical Priorities Advisory Group 2 – dialysis patients in scope NICE July 2013
NICE : TIMELINE (1) • Scoping begins • Evidence submission: Experience Report with additional patients from Wales July – • Some amendment to NICE “form” September • Patient Experts Chosen 2013 • Receipt of evidence pre meeting (700 pages!) • First HST Committee Meeting November - • No presentation but patient submission accepted December • “ compelling case for a call on NHS resources for an unmet need” 2013 • Evaluation Consultation Document 1 ( delayed 1 week from original date notified) • Minded not to recommend – insufficient explanation of price and size of budget resource needed and its impact on other National Specialised Services. March 2014 • We were not happy !
NICE: TIMELINE (2) • Responded but mainly on matters finance- own financial model May • Made a better stab at it even though unable to see key data 2014 • Made efforts to get parties together to discuss price • Alexion willing they say, NICE do not • Found out about PPRS 2014 (PAS/Governance Tolerances/Rebates) June-July • Delayed by a report on financial comparisons of eculizumab with other highly 2014 specialised therapies • Second HST Committee meeting August • We were not happy 2014
NICE : TIMELINE (3) • Evaluation Consideration Document 2 recommends with conditions • aHUSUK financial model acknowledged as nearer the mark September • Doses and withdrawal introduced by aHUSUK featured as a condition 2014 • Cost per patient more affordable if not yet seen as more “affordable” • PPRS 2014 October – • Third HST Committee meeting November 2014 • Final Evaluation Determination • Final Guidance January – April 2015 • Policy introduced by NHS England
AHUSUK- CONCLUDING ADVICE Worth investing time in a qualitative research report about patient experience Use consultant services if offered and you do not have administrative resources and you could benefit from an independent advisor Presentation or no presentation at meeting – ambivalent, both work depends on you Understand and accept the process project structure with “stages” and “products” Recognise the bigger part that patient organisations can play in the quality review of those “products” including financial matters and specific clinical issues It is evidence based and positive looking- for reasons to be minded to say “yes” Use the support offered by the public involvement team it is a good relationship to have. It helps confidence though to have a proven very clinically effective therapy – we had eculizumab!
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