Will the reformed Cancer Drugs Fund address the most common types of uncertainty? An analysis of NICE cancer drug appraisals

Abstract

BackgroundOne of the functions of the reformed Cancer Drugs Fund in England is as a managed access fund, providing conditional funding for cancer drugs where there is uncertainty in the economic case, and where that uncertainty can be addressed by data collection during two years’ use in the NHS. Our study characterises likely sources of such uncertainty, through a review of recent NICE Technology Appraisals.MethodsDiscussions of uncertainty in NICE Appraisal Committees were extracted from published Single Technology Appraisals of cancer drugs, 2014–2016, and categorised inductively. The location of the comments within the structured Appraisal document was used as a proxy for the degree of concern shown by the Committee.ResultsTwenty-nine appraisals were analysed, of which 23 (79%) were recommended for funding. Six main sources of uncertainty were identified. Immaturity of survival data, and issues relating to comparators, were common sources of uncertainty regardless of degree of concern. Uncertainties relating to quality of life, and the patient population in the trial, were discussed frequently but rarely occurred in the more uncertain appraisals. Concerns with trial design, and cost uncertainty, were less common, but a high proportion contributed to the most uncertain appraisals. Funding decisions were not driven by uncertainty in the evidence base, but by the expected cost per QALY relative to acceptance thresholds, and the resultant level of uncertainty in the decision.ConclusionsThe reformed CDF is an improvement on its predecessor. However the main types of uncertainty seen in recent cancer appraisals will not readily be resolved solely by 2 years’ RWD collection in the reformed CDF; where there are no ongoing trials to provide longer-term data, randomised trials rather than RWD may be needed to fully resolve questions of relative efficacy. Other types of uncertainty, and concerns with generalisability, may be more amenable to the RWD approach, and it is these that we expect to be the focus of data collection arrangements in the reformed CDF.

Determining lines of therapy in patients with solid cancers: a proposed new systematic and comprehensive framework

“…Furthermore, no standard format exists for expressing LoT in a way that would succinctly convey multifaceted, complex and clinically relevant information. There is, therefore, a lack of uniform methodology for enumerating LoT in patients with solid cancers and, in the absence of standard guidelines, clinicians and investigators often apply their own interpretation, resulting in variability and potential mis-classification, 13 thereby hindering both clinical care and research.…”

Does The Reformed Cancer Drug Fund Generate Evidence On Effectiveness? A Cross-sectional Analysis On Publicly Accessible Documentation

“…The 19 DCAs described a wide range of clinical uncertainties for the treatments covered. We used relevant elements of Morrell’s classification system to characterise the uncertainties described in the documents (Appendix 3) (Morrell et al ., 2018). 19 DCAs (100%) described outstanding uncertainties due to immature survival data; 9 (47%) described uncertainties due to lack of an appropriate comparator; 9 (47%) described uncertainties due to lack of relevant patient population; 7 (37%) described uncertainty around duration of use of the treatment; 3 (16%) described uncertainty around quality of life.…”

“…The CDF policy documents contain very little information on how this should be expressed or categorised, and the contents of the DCAs themselves are typically free text. Therefore we used Morrell et al’s classification of uncertainties in cancer drug technology appraisals (Morrell et al ., 2018) to provide a clearer structure. For each indication we read the DCA and recorded whether the outstanding uncertainties were caused by: immature survival data; lack of relevant comparators; problems with design of prior trials; problems with external validity of prior trials; shortage of quality of life data; reliance on observational data; lack of evidence on optimum treatment duration; lack of evidence on adverse events.…”

Достъп До Иновативни Лекарствени Терапии В Онкологията – Опитът На Фонд За Онкологични Лекарства Във Великобритания

“…При формулирането на този компонент следва да бъдат отчетено мнението на пациенти и медицински специалисти също. Търговското споразумение е конфиденциално и определя нивото на заплащане на продукта за периода на договора [5][6]. http://journal.raredis.org Искров Г, Чолакова Х, Якимов И, Стефанов Р. Достъп до иновативни лекарствени терапии в онкологията -опитът на Фонд за онкологични лекарства във Великобритания.…”

“…Този проблем е най-отчетлив при оценяване на относителната ефикасност. В случаи на липса на текущи рандомизирани клинични проучвания, данните от реалната практика трудно могат да отговорят на това предизвикателство [6]. Коментирайки несигурността, проучване на решения на фонда за период 2014-2016 г. разкрива, че формирането на достъп зависи не толкова от несигурността относно доказателствената база, колкото от постигането и вместването в определени пределни граници на ICER.…”