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OP127 Sugar And Spice And All Things NICE - Managed Access Agreements
- Adam Hall, Lok Wan Liu, Richard Macaulay, Sean Walsh
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, pp. 28-29
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Introduction
The National Institute for Health and Care Excellence (NICE) has increasingly agreed to reimburse innovative products with high levels of uncertainty as part of managed access agreements (MAAs) while additional data are collected, through the new Cancer Drugs Fund (CDF) or highly specialized technology (HST) pathways. This research aimed to review the data collection stipulations of current MAAs.
MethodsWe reviewed all current MAAs entered into between NHS England and manufacturers as of 29 October 2018 and key data were extracted.
ResultsTwenty-two MAAs were identified (19 through the CDF; three through HST). All MAAs involved an observational data collection component. The source of observational data collection was existing NHS databases (19/22 MAAs: 86.5 percent), existing independent registries (one MAA: 4.5 percent [ataluren]); bespoke MAA registry maintained by manufacturer (1/22 MAA: 4.5 percent [asfotase alfa]), and registries developed as a part of regulatory approval and maintained by the manufacturer (1/22 MAA: 4.5 percent [elosulfase alfa]). Only eight MAAs (asfotase alfa, ataluren, elosulfase alfa, brentuximab vedotin, venetoclax, ibrutinib, daratumumab, and pembrolizumab) had observational data collection as the primary method of data collection. Additionally, 17/22 MAAs (77 percent; all from the CDF) also required ongoing data collection from clinical trials as a key component of the data collection arrangement.
ConclusionsThis research identified observational data collection as a requirement in all MAAs, which is primarily through existing registries (except ataluren, which required development of a bespoke registry), while ongoing trial data collection was limited to the CDF. The relatively low cost of using existing registries to fulfil data requirements, with the ability to achieve reimbursement whilst still collecting data from ongoing RCTs, make MAAs an attractive proposition for manufacturers. NICE reportedly plan to increase use of MAAs, with ongoing NICE consultation for changes in the appraisal process potentially allowing expansion to include all indications, which would mean increased opportunities to explore innovative MAAs to support access in the future.
PP86 Reimbursement of Combination Oncology Products: Can Two (Companies) Tango?
- Erika Turkstra, Lok Wan Liu, Andrea Berardi, Richard Macaulay
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 54
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Introduction
A range of innovative, targeted anti-cancer therapies have been developed over the past 20 years. More recently, companies have been developing combinations of these drugs. While this promises substantial efficacy benefits, dual-brand oncology therapy combinations may potentially create substantial economic burden. Obtaining a positive health technology assessment (HTA) recommendation and public reimbursement can be a major challenge, and may be more difficult when each constituent monotherapy is marketed by a different company. We evaluated whether dual-brand oncology therapies developed by a single manufacturer had faster or better outcomes than those developed by two separate manufacturers.
MethodsRecent combination oncology drug products were screened in November 2018 to identify whether one or two manufacturers were involved. The websites of various HTA organizations were screened and the relevant data extracted.
ResultsA total of 78 recommendations for dual-brand oncology treatments were identified across the HTA agencies screened: 26 of these were for combinations by the same manufacturer and 52 were for combinations with two manufacturers. Dual-brand therapies developed by a single manufacturer were more likely to receive full or optimized/conditional recommendations (58% “recommended” and 12% “optimized/conditional”) than those marketed by two separate manufacturers (42% “recommended” and 8% “optimized/conditional”). Dual-brand therapies with two manufacturers were more likely to receive negative HTA recommendations than those marketed by a single manufacturer (50% versus 31%). However, the median time from marketing authorization to recommendation in European countries was the same (6 months), regardless of whether each constituent monotherapy was marketed by one or two manufacturers.
ConclusionsHTA agencies were more likely to issue negative recommendations for dual-brand oncology treatments marketed by two separate companies, compared with those marketed by a single company. A single company may have more flexibility in price setting, which may facilitate more positive HTA recommendations.
VP39 The Alphabet Lottery? How NICE Outcomes Vary By Appraisal Committee
- Richard Macaulay, Lok Wan Liu, Cornelia Roibu, Andrea Berardi
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 86
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Introduction
NICE (National Institute for Health and Care Excellence) makes recommendations on the public reimbursement of medicines based on their clinical- and cost-effectiveness. The recommendation is made by an Appraisal Committee (comprising a multi-disciplinary group of independent experts) as part of a technology appraisal. There are four Appraisal Committees (A,B,C,D); this research investigates whether appraisal outcomes vary by committee.
MethodsAll publicly-available Final Appraisal Determinations from NICE Single Technology Appraisals (STA) were screened (01/10/2009-14/11/2018) and key data were extracted. Homogeneity in rates of acceptance or rejection across the committees was assessed using Chi-squared tests.
ResultsThe Appraisal Committee was identified for 298 technologies, 56% (168/298) of which were ‘recommended’. The number of technologies assessed by each committee was similar (A:79, B:62, C:91, D:66). However, STAs conducted by Committee D were significantly less likely to receive ‘recommended’ outcomes (A:68% [54/79], B:65% [40/62], C:53% [48/91], D:39% [26/66]; p < 0.01). STAs for oncology indications had higher ’not recommended’ outcomes than those for non-oncology indications (25% vs. 9%). The lower ‘recommendation’ rates for committee D persisted across oncology (A:60%, B:83%, C:50%, D:38%; p = 0.01) and non-oncology indications (A:73%, B:53%, C:55%, D:40%; p < 0.01). However, STAs conducted by Committee D were significantly more likely to receive ‘optimized’ recommendations (A:16%, B:21%, C:33%, D: 36%; p < 0.01) and when considering the rates of ‘recommended’ and ‘optimized’ outcomes compared to ‘only in research’ and ‘not recommended’ outcomes, no significant differences were found (A:85%, B: 85%, C:86%, D:76%; p = 0.27).
ConclusionsSTAs undertaken by NICE Appraisal Committee D was associated with a significantly lower rate of ‘recommended’ outcomes but tended to an ‘optimized’ recommendation significantly more than the other committees. Further research is needed to determine if this reflects any deviation in uniform implementation of NICE methodology between Committees.
OP19 Does The HST Represent A Best Practice Model For Ultra-Orphan HTA?
- Richard Macaulay, Lok Wan Liu, Erika Turkstra
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, pp. 3-4
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Introduction
Ultra-orphan therapies (prevalence: <1:50,000) can have trouble meeting Health Technology Assessment (HTA) clinical- and cost-effectiveness criteria, set by HTA bodies to inform reimbursement decision-making, due to low patient numbers limiting the supporting clinical evidence generated and high per-patient prices. Since 2013, National Institute of Health and Care Excellence (NICE) appraise Highly Specialised Technologies (HST) (“for use in the provision of services for rare and very rare conditions”) using a distinct appraisal framework. This research compares NICE HST appraisal outcomes with corresponding guidance by other HTA bodies.
MethodsAll NICE HST technology guidance was screened (1 January 2013–6 November 2018) alongside corresponding guidance by Gemeinsamer Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), Scottish Medicines Consortium (SMC), and National Centre for Pharmacoeconomics (NCPE).
ResultsNICE have published eight HST guidances all with positive recommendations after a median of 21 months (range: 7–38) after European Marketing Authorization (MA). An additional eight HST have guidance in-development despite having European MA for a median of 12 months (range: 2–46) with 5/8 having draft guidance issued, all being “not recommended”. Of the 18 HSTs with NICE guidance published/in-development, 29 percent (2/7), and 33 percent (2/6) have been assessed with positive outcomes (definition: “recommended”/”accepted”/“conditional”/”restricted”) by SMC, and NCPE, respectively vs. 100 percent (9/9) by G-BA (definition: any additional benefit), and 50 percent (5/10) by HAS (definition: ASMR I-III). Median delays between European MA and positive appraisal outcomes were seven (G-BA), nine (HAS), 12 (NCPE), and 19.5 months (SMC).
ConclusionsAlthough all NICE HST final guidances to date have been positive, few technologies have completed this process after substantial delays from MA. Other cost/QALY HTA bodies (i.e. excluding the G-BA and HAS clinical-assessment HTAs) have shown low appraisal and recommendation rates for these technologies; therefore, ultra-orphan technologies may require a distinct appraisal process/framework but the HST may not (yet) represent best-practice.
OP20 Has The New HST Process Improved The Recommendation Chance In England?
- Erika Turkstra, Lok Wan Liu, Silvy Mardiguian, Sangeeta Budhia
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 4
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Introduction
The National Institute for Health and Care Excellence (NICE) in England has a separate appraisal process for drugs for very rare conditions, i.e. Highly Specialised Therapies (HST). In April 2017, the HST process has been changed to incorporate a quantitative approach: automatically fund treatments with incremental cost-effectiveness ratio (ICERs) up to GBP 100,000 (EUR 113,008 based on the 2018 average GBP / EUR exchange rate) per quality-adjusted life year (QALY). For treatments with an ICER above GBP 100,000 per QALY, NICE will consider treatments that offer a substantial magnitude of improvement, with additional QALY weighting. We investigated the impact of this more quantitative approach on the likelihood of a HST receiving a positive recommendation.
MethodsAll HST appraisals and draft guidance documents were reviewed (up to November 2018) and data were extracted on ICERs, incremental QALY gain, budget impact, and recommendations. The extracted data from each HST were assessed based on the interim HST guidance.
ResultsEighteen products have been or are currently going through the NICE HST process. Of these, 8/18 (44%) have received a positive recommendation, while 5/18 (28%) have received a draft negative guidance, and for 5/18 (28%) products, no recommendations have been published. For the products with a positive outcome, 5/8 (63%) had incremental QALY gain of at least 10, qualifying these products for additional QALY weighting. For the products that received a draft negative recommendation, the negative decision was related to the cost-effectiveness estimates being higher than GBP 100,000 per QALY (5/5 reported) in all cases, while none of these products were eligible to receive a ‘QALY modifier’.
ConclusionsIt has become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 per QALY for many products.
PP189 Filling In The Blanks: Is RWE From MAAs Used In NICE Decision Making?
- Lok Wan Liu, Adam Hall, Richard Macaulay, Sean Walsh
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, pp. 72-73
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Introduction
The National Institute for Health and Care Excellence (NICE) may recommend temporary funding through managed access agreements (MAAs) for oncology drugs (via the Cancer Drugs Fund [CDF]) and highly specialized therapies for rare diseases. MAAs allow for the collection of evidence to address key areas of clinical uncertainty, while providing access of medicines to patients, prior to re-appraisal by NICE. Observational data and other real-world evidence (RWE) are crucial requirements for all MAAs and herein we examine the extent these data are being used to inform HTA decisions at re-appraisal.
MethodsExisting MAAs entered into between the National Health Service (NHS) England and manufacturers as of 30 October 2018 were identified; for drug:indication pairings with NICE re-appraisals, all information was reviewed and the key data extracted.
ResultsOf the twenty-two MAAs identified, only two drug:indication pairings have been subsequently re-appraised by NICE: BV(brentuximab vedotin):non-Hodgkin lymphoma (’recommended’) and pembrolizumab:relapsed or refractory classical Hodgkin lymphoma (’recommended’). Data from a retrospective questionnaire regarding the proportion of patients that received curative stem cell transplant (SCT) post-BV (from patients who received BV in the old CDF) were accepted to provide sufficient evidence on the post-BV SCT rate by NICE. Meanwhile, for pembrolizumab, long-term survival benefit was the key clinical uncertainty; the primary data collection source was updated phase III randomized controlled trial data. At re-appraisal no reference was made to the observational data component; more mature survival data reduced uncertainty over survival benefits and were sufficient to support a positive NICE recommendation.
ConclusionsOf the twenty-two MAAs to date, only two drugs have been re-appraised thus far, with both receiving positive NICE recommendations. Observational data were successfully used to address key clinical uncertainties regarding subsequent real-world treatment patterns for BV, but observational data were not referred to in the NICE recommendation for pembrolizumab. The re-appraisal of more drugs in the future will clarify the importance being placed on observational data collection requested by NICE for existing MAAs.
PP81 Real World Data: The Early Access To Medicines Scheme Catches The Worm
- Adam Hall, Lok Wan Liu, Richard Macaulay, Sean Walsh
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 53
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Introduction
The Early Access to Medicines Scheme (EAMS) aims to provide access to medicines prior to market authorization for patients with severe, life-threatening diseases who do not have adequate treatment options. An EAMS designation enables the potential collection of United Kingdom-specific real world evidence (RWE) prior to health technology assessment (HTA) by the National Institute for Health and Care Excellence (NICE). This research evaluates whether RWE is being gathered through the EAMS and utilized to support HTA submissions.
MethodsAll EAMS designations as of 7 November 2018 were identified from the Medicines and Healthcare products Regulatory Agency website. For products with final NICE guidance, all publicly-available NICE documentation was reviewed.
ResultsSixteen product and indication pairings with an EAMS designation were identified, with 12 having received final NICE guidance (11 were recommended, 3 were recommended for temporary reimbursement via the Cancer Drugs Fund, and 2 were not recommended). Of the 11 recommended products, seven had references to the number of patients or sites with product access through the EAMS, but only one (dupilumab for atopic dermatitis) had detailed data collected during the EAMS period. The manufacturer of dupilumab reported baseline demographics and disease characteristics from a cohort of 35 patients treated under the EAMS to inform the generalizability of trial populations for clinical practice. Follow-up results from this cohort demonstrated that real-world data on dupilumab effectiveness was comparable with the clinical trial data, despite a higher proportion of patients in the real-world cohort receiving immunosuppressant therapy, which makes improvements in efficacy harder to achieve. The committee also noted that the RWE presented supported the understanding of dupilumab's long-term clinical effectiveness and informed assumptions for the economic model.
ConclusionsTo date, the majority of products receiving an EAMS designation have not presented RWE at NICE reappraisal. The case of dupilumab illustrated how RWE collected through the EAMS can be used to reduce uncertainty around how clinical trial data can be translated into clinical practice. In the future, RWE may increasingly be used to help inform NICE decisions.
PP165 The Resurrection Of The Cost-Minimization Approach In England
- Erika Turkstra, George Duo Wang, Lok Wan Liu, Silvy Mardiguian
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 34 / Issue S1 / 2018
- Published online by Cambridge University Press:
- 03 January 2019, p. 131
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Introduction:
For almost 20 years (1999–2017), the National Institute for Health and Care Excellence (NICE) focused primarily on cost utility analyses (CUA) for its health technology appraisals. This changed on the 01 April 2017, when a new fast track appraisal process was introduced for technologies that offer exceptional value for money. Under this process, a cost-comparison analysis can be included for technologies that are likely to provide similar or greater health benefits at a similar or lower cost to comparator technologies already recommended by NICE. This is in contrast to other jurisdictions (e.g. Scotland and Australia) that have long accepted cost-comparison analyses such as cost-minimization analyses (CMA) when a technology has comparable efficacy to relevant comparators. This research aimed to investigate if this new approach will have an impact on future appraisals
Methods:Publically available technology appraisal documents from NICE, Scottish Medicines Consortium (SMC), and Pharmaceutical Benefits Advisory Committee (PBAC) were screened (01/01/2016-01/12/2016), and the supportive economic analyses were identified and extracted.
Results:In 2016, the proportion of CMA submissions that formed the basis of technology appraisals were 0/53 (0 percent), 17/55 (31 percent) and 25/82 (30 percent) for NICE, SMC and PBAC, respectively. The likelihood that a technology was recommended (with or without restrictions) for those technologies that were assessed using a CUA was 60 percent, 66 percent and 33 percent for NICE, SMC and PBAC, respectively, while technologies that were assessed using a CMA were associated with higher positive recommendation rates: 76 percent and 76 percent for SMC and PBAC, respectively.
Conclusions:Incorporating a cost-minimization approach may result in more technologies being recommended by NICE through the fast track appraisal process, whereby the likelihood of a technology having a positive recommendation is much greater than the standard appraisal process.