NICE a HUSUKS EXPERIENCE Navigating the Highly Specialised - - PowerPoint PPT Presentation
NICE a HUSUKS 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
Navigating the Highly Specialised Technologies Programme FindacureWorkshop 29 January 2016
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
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.”
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
Septembe r-October 2011
February 2012
March – May 2012
interviews
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
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. Girl, 5 years Father Receiving dialysis at home, awaiting transplant, receiving Eculizumab. Girl, 7 years Mother Receiving plasma exchange and on long-term dialysis Boy, 13 years Mother In remission. Young female adult Patient and sibling (2 interviews) Has end stage renal failure and is receiving dialysis at home. Transplant failed. Working. Young female adult Patient and husband (3 interviews) Has end stage renal failure and is receiving dialysis at home. Transplant failed. Retired due to ill-health. Young female adult Patient/husband/daughter (3 interviews) Has end stage renal failure and is receiving dialysis in hospital. Transplant failed. Retired due to ill-health. Adult late onset – female Patient In remission after treatment with Eculizumab. Adult late onset - female Patient Has end stage renal failure and is receiving dialysis at home. Transplant failed.
Table 1: Characteristics of the interviewees
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
Septembe r-October 2011
February 2012
March – May 2012
interviews
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
June 2012
January 2013
“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”
March 2013
professional
April 2013
July 2013
NICE
July – September 2013
November
2013
March 2014
resource needed and its impact on other National Specialised Services.
May 2014
June-July 2014
specialised therapies
August 2014
September 2014
October – November 2014
January – April 2015
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!