Scottish Medicines Consortium Research Articles

Clinical benefit, reimbursement outcomes, and prices of FDA-approved cancer drugs reviewed through Project Orbis in the USA, Canada, England, and Scotland: a retrospective, comparative analysis

Project Orbis is a global initiative that aims to streamline regulatory review processes across international regulators in the USA, Canada, Australia, UK, Israel, Brazil, Singapore, and Switzerland to bring promising cancer drugs to patients earlier. We explored the clinical benefit, time to regulatory approval and health technology assessment recommendations, reimbursement outcomes, and monthly treatment prices of cancer drugs reviewed through this initiative. For this retrospective, comparative analysis, we identified cancer drug approvals reviewed through Project Orbis in the USA, Canada, and the UK between May 1, 2019, and Nov 1, 2023. Approvals of cancer drugs reviewed Project Orbis were extracted from the Food and Drug Administration (FDA) Oncology Centre of Excellence and all other FDA approvals from the Drugs@FDA database. The co-primary outcomes were time of regulatory review, time from regulatory approval to health technology assessment recommendation (England, Scotland, and Canada), reimbursement outcomes, clinical benefit (defined as median gains in progression-free survival and overall survival) between cancer drug approvals reviewed by Project Orbis and other FDA approval processes, and monthly treatment prices. The Wilcoxon rank-sum and Fisher's Exact tests were used to examine statistical significance between approvals reviewed through Project Orbis and other FDA approvals during the same period. Between May 1, 2019 and Nov 1, 2023, 81 (33%) of 244 cancer drugs approved by the FDA were reviewed through Project Orbis. The median overall survival gains were 4·1 months (IQR 3·3-5·1) compared with 2·7 months (2·1-3·9) for other FDA approvals. Similarly, progression-free survival gains were 2·6 months (IQR 1·7-4·9) for Project Orbis compared with 2·6 months (0·6-5·1) for other FDA approvals. Neither overall survival (p=0·11) nor progression-free survival (p=0·44) gains were significantly different between the two cohorts of approvals. Of the 14 UK Medicines and Healthcare products Regulatory Agency (MHRA) approvals reviewed by the Scottish Medicines Consortium (SMC), the agency gave positive recommendations for all 14 (100%). Of the 15 MHRA approvals reviewed by the National Institute for Health and Care Excellence (NICE), the agency gave positive recommendations for six (40%). Of the 49 approvals reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH), the agency conditionally recommended 44 (90%). The time between regulatory approval to NICE recommendation increased from a median of 137 days (IQR 102-172) in 2021 to 302 days (184-483) in 2023, SMC recommendation increased from 185 days (in 2021 for one drug only) to 368 days (IQR 313-476) in 2023, and CADTH decision increased from 97 days (in 2020 for one drug only) to 202 days (IQR 153-304) in 2023. The median monthly price of approvals reviewed through Project Orbis was US$20 000 per month (IQR 13 000-37 000). Clinical outcomes of Project Orbis were no different than other FDA approvals during the same time, and access, after a successful health technology assessment, was considerably delayed or absent, raising questions about whether Project Orbis participation translates into faster patient access to medicines with high clinical benefit and sustainable costs. Although future challenges might benefit from regulatory harmonisation, the advantages are currently unclear. None.

Review of lidocaine plaster prescribing and monitoring in primary care

Abstract Introduction Lidocaine medicated plasters (LMPs) are accepted by the Scottish Medicines Consortium (SMC) for restricted use in post-herpetic neuralgia where first-line therapies are ineffective or intolerable.[1] Subsequently, LMPs are restricted for use in post-herpetic neuralgia within a large NHS board with Ralvo as formulary brand due to cost-effectiveness. Despite this, the spend on LMPs is increasing with the cost per 1000 list size above the national average.[2] Aim To review and improve LMP prescribing and monitoring in a general practice. Methods An audit within a GP practice was undertaken by conducting an EMIS search to extract all patients prescribed LMPs within the previous 6 months from December 2022. A baseline audit was completed to determine compliance against the criteria (standards set as 100%), shown in Table 1. Data were analysed using Microsoft Excel. In the interest of improving audit results, a pharmacist then conducted telephone reviews with a view to stopping LMP if appropriate, reducing prescription quantities and/or switching to Ralvo. Post review, criteria were re-audited to determine the impact. Following this, an education session was conducted. Results The results against the standards are shown in Table 1. The LMP reviews resulted in: therapy stopped (n=2; 8%), therapy reduced (n=7; 26%), therapy continued (n=11; 40%), and therapy removed from repeat (n=7; 26%). Reductions were achieved by reducing the number of patches, cutting patches or increasing the patch-free interval. Some patients continued therapy despite off-license indication due to a significant reduction in pain score and benefit in physical function. Following the audit and review of LMP, an informal general practitioner education session was conducted to promote adherence to formulary and SMC recommendations, aiming to prevent LMP initiation for off-license indications. Impact was not studied. Conclusion There is a lack of compliance with SMC and formulary recommendations with all patients in this study being prescribed LMPs for off-licensed indications. Pharmacy involvement in regular reviews could ensure patients are prescribed the most cost-effective brand and ongoing treatment appropriateness assessed, particularly for off-license indications. The study was implemented in one general practice; therefore, there is scope for extrapolation across the National Health Service board with aims to reduce LMP prescription costs and medication burden. Further work is also required to achieve a change in prescribing habits.

Appraisal of Novel Oncological Therapies by the Scottish Medicines Consortium and the National Institute for Health and Care Excellence: A Comparative Study of Six Years of Data.

Background and aims Pharmacoeconomic assessment of novel oncological therapies is an increasingly important factor in determining patient access to therapies. Organisations such as the National Institute for Health and Care Excellence (NICE) in England and the Scottish Medicines Consortium (SMC) in Scotland assess medications for their cost-effectiveness through health technology assessments(HTA) and provide guidance on whether the public health service should fund a therapy. We assessed six years of data to determine if there were any differences in timescales and decisions between NICE and SMC for new oncological therapies. Methods and results Time (days) from marketing authorisation (MA) to publication of final HTAguidance was calculated for single technology appraisals published by NICE and SMC between January 1, 2017, and December 31, 2022, for oncological therapies. We assessed 161 HTAs by NICE and 148 HTAs by SMC published in the study period. The median time from MA to publication of HTA guidance was 291 days (IQR 222-406) for SMC and 257 days (IQR 167-448) for NICE (p=0.054). For solid organ cancer therapies, NICE was significantly faster in publishing guidance, with a median of 231.5 days (IQR 148-392.25), compared to SMC, which took 273 days (IQR 202-378) (p=0.039). Overall recommendation of technologies was similar between the SMC and NICE (90.5% and 89.4%, respectively), with discordance in a minority of cases (12.6%). Conclusions Recommendation rates for single HTAs are similar between NICE and SMC for oncological therapies with discordance in a minority of cases. The time from MA to publication of HTA guidance was similar overall, but NICE was faster in publishing HTA guidance for solid organ cancer indications. Differences in methodology and process between the two organisations, in particular the presence of the Cancer Drugs Fund in England, may explain this difference in publication times.

HTA30 The NICE Vs SMC Cost Comparison Pathways – How Similar Are They?

The National Institute for Health and Care Excellence (NICE) offers a fast-track appraisal cost-comparison pathway (FTA) for health technologies that exhibit similar or greater health benefits at a similar or reduced cost to technologies already approved for the same clinical indication. The Scottish Medicines Consortium (SMC) provides two pathways, a full submission using a cost-comparison model or an abbreviated submission requiring a simpler comparison of acquisition costs for interventions and comparators within the same therapeutic class. Recent submissions to NICE and the SMC were reviewed to investigate the use of the SMC pathways and their success rates.

Overview of Health Technology Assessment Processes for Time-Limited Recommendations


 Several regulatory agencies have implemented processes to facilitate timely access to innovative therapies for patients with rare or life-threatening diseases with high unmet medical needs, typically with additional data-generation requirements to address important uncertainties in the existing evidence base at the time of regulatory approval. Many health technology assessment (HTA) agencies have adapted their processes to include time-limited recommendations (TLRs) for such therapies, which allows patients access to these medications while additional data are collected to verify the clinical efficacy, real-world effectiveness, or economic value of the new therapeutic. The objective of this Environmental Scan is to identify what HTA agencies have processes in place for TLRs and to describe these processes, if applicable.
 HTA agencies in North America, Europe, Australia, and New Zealand were considered for this report based on commonalities to the Canadian context in terms of geography and relevant regulatory or HTA and reimbursement processes. Seven HTA agencies (the National Institute for Health and Care Excellence, the Scottish Medicines Consortium, France’s Haute Autorité de Santé, the Dutch National Health Care Institute, the Italian Medicines Agency, the Belgium National Institute for Health and Disability Insurance, and the Australian Pharmaceutical Benefits Advisory Committee) that have implemented formal TLR processes, through managed entry agreements, special access funds, or other related programs were identified. Most TLR programs are referred to as managed access or entry agreements, but other types include interim acceptance, conditional inclusion, assessment contingent on additional evidence development, and additional specialized programs or pathways.
 TLRs are primarily used for promising (and sometimes high-cost) medicines intended to treat a condition with an important unmet medical need; some specify that the disease must be serious and life threatening (regardless of prevalence), others indicate that the process is used for orphan drugs intended to treat rare conditions, and others consider multiple criteria or do not have prerequisite criteria for eligible indications.
 Evidence generation requirements for TLRs vary across HTA agencies and are often dependent on the nature of the clinical and economic uncertainties identified in the initial assessment; sources include later follow-up from ongoing clinical trials, new clinical trials, real-world data, or a combination of both clinical and real-world studies. Additionally, most TLR processes have explicit maximum durations for the time-limited period, which range from 2 to 7 years across the included HTA agencies (although some allow for time adjustments or renewals); however, the French and Scottish HTA agencies determine the duration of the time-limited period on a case-by-case basis.
 Overall, this report provides an overview of HTA processes for TLRs across HTA agencies in Europe and Australia, many of which may be useful to inform the implementation of future TLR processes in Canada, as well as to refine existing ones.

OA31 Real life experience of tocilizumab treatment for giant cell arteritis

Abstract Background/Aims Giant cell arteritis (GCA) has been revolutionised by fast track pathways that ensure early secure diagnosis. The GiACTA study provided evidence for the beneficial role of tocilizumab in the treatment of GCA and encouraged us all to reconsider the role of corticosteroids. These data relate to real life experience of the use of tocilizumab, including current status of patients after completing the initial year of treatment. In Scotland, tocilizumab was initially used for patients with relapsing disease or steroid complications. However, since 2018, the Scottish Medicines Consortium has supported use from disease onset and it has become increasingly common to consider adding at diagnosis. Methods The case notes of 23 patients treated with tocilizumab were reviewed back to 2017. Patient demographics were collated including whether cranial (C) or large vessel vasculitis (LVV) variant. The diagnosis of GCA had to be confirmed by biopsy or imaging with GCA rheumatology clinic review. Any patients who discontinued tocilizumab prior to completing one year had the reason recorded. Duration on tocilizumab was intended to be one year. Data were reviewed to capture outcomes after treatment was withdrawn. Results 2 patients with non-relapsing disease discontinued tocilizumab within the first month due to early relative neutropenia (1.4). 5 of 21 patients who completed treatment have since had additional tocilizumab treatment. 1 patient with particularly severe relapsing disease and steroid complications remains on long term tapered tocilizumab monotherapy without attempt to stop (C and LVV). 4 patients (LVV=C) flared within 1-9 months (median 6 months) and resumed with steroids and subsequent tapers; all had commenced for relapsing disease. 1 patient (C), commenced on tocilizumab at diagnosis, opted for MTX when flared. 15 remaining patients are on no steroid, methotrexate or tocilizumab and are free of disease activity as yet, but undergoing monitoring for flare (12C 3 LVV variant). Conclusion Tocilizumab is effective, limits steroid exposure and is well-tolerated by patients. The most common reason for discontinuing tocilizumab was relative neutropenia (neutrophils 1-1.4). The majority of patients discontinued tocilizumab and have not yet flared off all treatment. Cranial variant was less associated with relapse after tocilizumab treatment versus LVV. New diagnoses appear less prone to relapse off tocilizumab than patients already known to have relapsing disease prior to using tocilizumab. Patients discontinuing tocilizumab benefit from ongoing monitoring to recognise the early stages of returning disease; this would need to be provided for at least one year. Patients discontinuing treatment, their GPs and carers need advice on routes to escalate concerns about returning symptoms. Fast track services need to provide a flare service in addition to a diagnosis pathway if there is to be safety, confidence and expert support for this patient group and primary care colleagues. Disclosure L.M.M. Hutton: Other; 2022 Roche educational grant to attend BSR.

Economic Evaluation of Tirbanibulin for the Treatment of Actinic Keratosis in Scotland.

Tirbanibulin 1% ointment is a new treatment for actinic keratosis (AK) on the face or scalp. A health economic model was developed as part of a submission to the Scottish Medicines Consortium to evaluate the cost-effectiveness of tirbanibulin compared to the most frequently prescribed treatments. A decision tree approach was used to calculate the costs and benefits of different treatment strategies for AK on the face or scalp over aone-year time horizon. Data on the relative efficacy of treatments, which were based on the probability of complete clearance of AK, were obtained from a network meta-analysis. Sensitivity and scenario analyses were performed to determine the robustness of the model results. Tirbanibulin is estimated to be cost saving versus diclofenac sodium 3%, imiquimod 5% and fluorouracil 5%. Tirbanibulin remains cost saving when inputs are varied in sensitivity and scenario analyses. While the complete clearance rates are deemed similar across comparators, tirbanibulin is associated with a lower rate of severe local skin reactions, and a shorter treatment duration, which may improve treatment adherence. Tirbanibulin is a costsaving intervention for the treatment of AK from the perspective of the Scottish Healthcare System.

Real-world evidence in lung and hematologic oncology health technology appraisals: a review of six assessment agencies

Aim: To assess the use and acceptability of real-world evidence (RWE) in lung and hematologic cancer appraisals. Materials & methods: A review of appraisals published by National Institute for Health and Care Excellence (NICE) in the UK was conducted. A total of 20casestudies employing RWE were identified and compared across five additional health technology assessmentagencies: Scottish Medicines Consortium (SMC) (Scotland), CADTH (Canada), INESSS (Quebec), HAS (France)and IQWiG (Germany). Results: Of 80 RWE references from 20 case studies from NICE, 67 were identified in the respective CADTH submissions, 46 in IQWiG, 37 in INESSS, 37 in HAS, and 33 in SMC. NICE had the highest RWE acceptance rate (90%), followed by HAS (88%), SMC (82%), INESSS (73%), IQWiG (68%) and CADTH (67%). Conclusion: RWE was generally accepted by respective committees, allowing improved access to innovative treatments.

Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines.

The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine's added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines. A survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members. PACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n=20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future. In general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.

OP63 Patient and Public Perspectives On The Scottish Medicines Consortium Detailed Advice Document

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines, the primary output of which is a document referred to as the Detailed Advice Document (DAD). This comprises an overview of all data considered on the clinical and cost-effectiveness of the medicine, as well as the input from patient groups (PGs), patients, and carers. In 2020, SMC commenced a stakeholder evaluation of the DAD including a workshop with PGs and public partners (members of the public who volunteer with SMC) to explore the potential for using the DAD more widely.MethodsPGs and public partners, all having significant experience of engaging with SMC, participated in the workshop. Feedback was gathered using virtual post-it notes, collated and analysed for key themes. We also gathered oral feedback from participants. Sample DADs were distributed for two medicines recently appraised, one of which included a Patient and Clinician Engagement (PACE) meeting. These were chosen because they reflect different aspects of public and patient involvement at SMC, including how this is presented in the DAD.ResultsOverall, the workshop participants (n=7) recognised the DAD was a useful document for the clinicians who are its primary audience. Its language was perceived to be challenging, including complex information that is not accessible to a wide audience and may only be fully understood by those with a good understanding and knowledge of HTA. This was a key barrier to using the DAD more widely, in particular the health economics information. Suggestions for broadening the audience of the DAD included summaries of key points and an introductory section clarifying the purpose of the DAD and its intended audience, along with signposting to the plain language summary produced by SMC. These will be implemented where possible.ConclusionsImproving how SMC communicates decisions to patients and the public, by working in partnership with these stakeholders, will help strengthen public involvement throughout the HTA process.

HPR37 Comparing Reimbursement of New Drugs During the COVID-19 Pandemic in Ireland and Scotland

Following the onset of the COVID-19 pandemic, the Scottish Medicines Consortium (SMC) suspended monthly meetings from April to August 2020, to support the National Health Service (NHS) Scotland in maintaining essential work. Following the return of meetings, a fast-track process was introduced to expedite reimbursement for certain medicines. Conversely, the Irish health technology assessment (HTA) agency, the National Centre for Pharmacoeconomics (NCPE), continued to assess new reimbursement applications throughout this period, using the two-stage process of a rapid review (RR), followed by full HTA for some medicines. This research explores how these different approaches impacted reimbursement submissions and recommendations during this time. A database was developed using published data (from 01/03/2020 to 01/03/2022) on the NCPE, SMC and Irish Health Service Executive (HSE) websites, including; • Breakdown of submissions received • Assessment outcomes • Breakdown of reimbursement recommendations Data was extracted into Excel® and analysed using summary and descriptive statistics. SMC advice for 169 medicines were identified (n=89 full submissions; n=36 abbreviated submissions; n=28 non-submissions; n=13 resubmissions; n=3 ultra-orphan assessments). Positive reimbursement recommendations were made for 136 of these medicines (n=68 accepted for use, n=58 accepted for restricted use, n=7 accepted on interim basis, n=3 available under ultra-orphan assessment), with 48 recommendations made using the fast-track process. Contrastingly, the NCPE published 148 RR outcomes and 61 HTA outcomes, with 70 reimbursement recommendations published by the HSE (n=38 positive recommendations, n=11 restricted reimbursement, n=21 reimbursement not recommended). While a similar number of submissions were assessed by the Irish and Scottish HTA agencies during the COViD-19 period, there was a significantly higher number of positive reimbursement recommendations from the SMC, which may be explained by the SMC fast-track process. This research further explores the reimbursement recommendations for each category of submission and compares the reimbursement timelines for each jurisdiction.

PD48 Does Unmet Need Influence The Scottish Medicines Consortium Health Technology Assessment Decisions For Rare Disease Conditions?

IntroductionThe Scottish Medicines Consortium (SMC) defines end-of-life medicines as drugs used for treating conditions that usually lead to death within three years using currently available treatments. Orphan medicines are drugs used for the treatment of very rare conditions and ultra-orphan drugs are used for the treatment of extremely rare conditions. The objective of this study is to determine the influence of unmet need on SMC health technology assessment (HTA) decisions for rare disease conditions.MethodsThe reimbursement data between 2004 and 2021 from SMC for rare disease conditions were included. These data were categorized based on the presence of an unmet need, that is, drugs considered under the orphan or ultra-orphan process, or those that fulfilled SMC end-of-life criteria. A chi square test was conducted to determine an association between the presence of an unmet need and the HTA decision. HTAs without a decision were excluded.ResultsA total of 91 HTAs were included in the analysis of which, 57.1 percent (n = 52) were recommended, and 42.9 percent (n = 39) were not recommended. Out of the recommended reviews, 32.7 percent (n = 17) addressed an unmet need and 67.3 percent (n = 35) did not. Recommended drugs had positive clinical evidence and high cost-effectiveness or the submission of a patient access scheme while negative decisions were associated with lower or uncertain cost-effectiveness. The chi square test result showed no association between the presence of an unmet need and the HTA decision (p = 0.315).ConclusionsUnmet need does not influence the SMC HTA decisions for rare disease conditions. Economic elements were the driving factors in the decision-making process.

PP74 Taking A Lifecycle Approach To Scottish Medicines Consortium Budget Impact Analysis

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment of new medicines in Scotland. While budget impact is not a factor in decisions on reimbursement, budget impact information is provided initially through horizon scanning reports for high impact medicines (estimated net budget impact >GBP 500,000 [EUR 585,710] per annum) to aid financial planning and implementation of advice at the local level, and later through budget impact templates from the submitting company issued alongside SMC advice. This research aimed to understand how the information is used and to evaluate the benefits of a lifecycle approach to budget impact analysis.MethodsHealth Board users of the budget impact templates were surveyed to explore the degree of utilization and identify areas for improvement, including the need to cross-validate the horizon scanning estimates with those of the submitting company. Responses were analyzed quantitatively and qualitatively, with comments coded in Nvivo (QSR International) and themes established through thematic analysis.ResultsThe initial responses received (n=17) provided representation from 57 percent of Health Boards (i.e., payers) covering 79 percent of the population. Preliminary results showed that while the budget impact templates were valued, 69 percent of respondents found them ‘somewhat useful’, suggesting scope for improvement. Almost half (48%) of the respondents used the templates for high impact medicines, with only 30 percent using them for all medicines. The majority (76%) of those surveyed thought there would be value in linking budget impact information throughout the SMC process. An emerging theme was that some users found the templates complex and inflexible, and that a simpler, more adaptable tool to aid the planning process would be welcomed. Priorities identified for improving template included adapting them to the local population and adjusting medicine prices to reflect confidential discounts.ConclusionsThis research suggests that budget impact information is valued by Health Boards and that there is strong support for linking budget impact estimates and engaging stakeholders throughout a medicine’s lifecycle. Simplifying the templates, increasing their adaptability, and providing guidance and training in their use will be key steps in improving this important part of SMC process.

HTA95 Analysis of Neurological Conditions Submissions to the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) From 2001 to 2021

HTA95 Analysis of Neurological Conditions Submissions to the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) From 2001 to 2021

HTA154 Comparing Non-Oncological Rare Condition Submissions to the National Institute for Health and Care Excellence (NICE) Versus Scottish Medicines Consortium (SMC) From 2001 to 2021

HTA154 Comparing Non-Oncological Rare Condition Submissions to the National Institute for Health and Care Excellence (NICE) Versus Scottish Medicines Consortium (SMC) From 2001 to 2021

PP42 Impact Of The COVID-19 Pandemic On Scottish Medicine Consortium Submission Characteristics, Acceptance Rates, And Time To Advice

IntroductionScottish Medicine Consortium (SMC) meetings were suspended in March 2020 in response to the coronavirus disease 2019 (COVID-19) pandemic. This led to a high number of submissions awaiting appraisal, prompting interim process changes to ensure minimal disadvantages to patient access. We expanded the eligibility criteria for the shorter (abbreviated) submissions process and expedited advice for submissions the New Drugs Committee (NDC) intended to accept. This study aimed to evaluate the impact of the COVID-19 pandemic and these interim process changes on the characteristics of submissions received, acceptance rates, and time to advice publication.MethodsData for all submissions received between January 2015 and November 2021 (n=720) were extracted from an organizational database. Characteristics of and acceptance rates for submissions received before and after the start of the pandemic were compared using chi-squared and one-proportion Z-tests, respectively. Additional analyses explored the number of submissions received per month and the time from receipt of submission to NDC and SMC decision.ResultsThe numbers of full and abbreviated submissions increased from March 2020 (6% in each case), with a corresponding decrease in the number of medicine-indication pairs (e.g., pembrolizumab for breast cancer) for which companies did not submit (8%; p=0.01). An increase in the SMC acceptance rate was also observed (62 to 72%; p=0.03). Fewer submissions were received in 2020 (n=65), compared with the pre-pandemic average (mean=79.6), whereas the total in 2021 to date was higher than average (n=92). Time series analysis suggested an increasing trend in monthly submissions (from approximately 6 to 9), which is the likely reason for the increase in average time to decision (146 versus 170 days).ConclusionsProcess changes in response to the pandemic have been effective in expediting advice for submissions with sufficiently robust evidence. This demonstrates agility and efficiencies for submitting companies and patient groups, with no perceived impact on process rigor. The average number of submissions has increased since March 2020, and further work is warranted to understand the influence of process improvements on reducing time to advice.

The importance of early diagnosis and views on newborn screening in metachromatic leukodystrophy: results of a Caregiver Survey in the UK and Republic of Ireland

Metachromatic Leukodystrophy (MLD) is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency of the enzyme arylsulfatase A (ARSA). MLD causes progressive loss of motor function and severe decline in cognitive function, leading to premature death. Early diagnosis of MLD provides the opportunity to begin treatment before the disease progresses and causes severe disability. MLD is not currently included in newborn screening (NBS) in the UK.This study consisted of an online survey, and follow-up semi-structured interviews open to MLD patients or caregivers, aged 18 years and over. The aims of the study were to understand the importance of early diagnosis and to establish the views of families and caregivers of patients with MLD on NBS.A total of 24 patients took part in the survey, representing 20 families (two families had two children with MLD, one family had three children with MLD). Following on from the survey, six parents participated in the interviews. Our data showed diagnostic delay from first symptoms was between 0 and 3 years, with a median of 1 year (n = 18); during this time deterioration was rapid, especially in earlier onset MLD. In patients with late infantile MLD (n = 10), 50% were wheelchair dependent, 30% were unable to speak, and 50% were tube fed when a diagnosis of MLD was confirmed. In patients with early juvenile MLD (n = 5), over half used a wheelchair some of the time, had uncontrollable crying, and difficulty speaking (all 60%) before or at the time of diagnosis. A high degree of support was expressed for NBS among caregivers, 95% described it as very or extremely important and 86% believed detection of MLD at birth would have changed their child’s future. One parent expressed their gratitude for an early diagnosis as a result of familial MLD screening offered at birth and how it had changed their child’s future: “It did and it absolutely has I will be forever grateful for his early diagnosis thanks to his older sister.”The rapid rate of deterioration in MLD makes it an essential candidate for NBS, particularly now the first gene therapy (Libmeldy™) has been approved by the European Medicines Agency. Libmeldy™ has also been recommended as a treatment option in England and Wales by the National Institute for Health and Care Excellence (NICE) and is being made available to patients in Scotland via the Scottish Medicines Consortium’s ultra-orphan pathway.

HTA2 RAPID C-19: Achievements and Challenges of a Collaborative Response to the COVID-19 Pandemic in the UK

HTA2 RAPID C-19: Achievements and Challenges of a Collaborative Response to the COVID-19 Pandemic in the UK

HTA59 The Impact of the Patient Access Schemes on Appraisals for the Innovative Medicines in Scotland 2011-2020

The patient access scheme (PAS) has been implemented as a formal part of the pricing and reimbursement environment in the United Kingdom (UK) since 2009, which aims to improve cost-effectiveness and enable patients to gain access to high-cost drugs and treatments. This study was to investigate the impact of the patient access scheme on the appraisals for the innovative medicines in Scottish Medicines Consortium (SMC) between 2011 and 2020.

The regulatory, evaluation, pricing and reimbursement pathway for medicines in the UK: combining innovation and access

The United Kingdom has universal healthcare systems, the National Health System (NHS), in its four nations, with healthcare services provided free of charge at the point of delivery. Approximately 10.5% of the UK population has voluntary supplementary private health insurance. While the provision of inpatient medicines is free of charge, medicines provided in the outpatient setting have a dispensing fee in some of the nations, such as the case of England (co-payment). The UK marketing authorisation process is called product licensing and is overseen by the Medicines and Healthcare Products Regulatory Agency (MHRA). There are different licensing routes based on the intended market for launch. MHRA also offers early access schemes and pathways for products targeting unmet medical needs and promising technologies, that aim to accelerate and facilitate market and patient access to products in the UK. These schemes include the option for companies to engage early with regulators and other system partners such as health technology assessment (HTA) agencies. As soon as the technology is authorised, it is available at a list price. Prices for medicines are regulated in legislation and in schemes agreed between the industry association and the Department of Health and Social Care (DHSC). The prices for the NHS are negotiated between the government and the companies. Routine funding decisions in the NHS are guided by HTA evaluations informed by agencies such as the National Institute for Health and Care Excellence (NICE) in England, the Scottish Medicines Consortium (SMC) in Scotland, and the All Wales Medicines Strategy Group (AWMSG) in Wales. Many medicines and other technologies are subject to price negotiations in the NHS, sometimes with confidential price agreements. The NHS in England is legally mandated to routinely fund technologies recommended by NICE that have been evaluated by some of its programmes. The other UK nations have similar arrangements or recognise decisions made in England. The role and contribution of NICE and other HTA agencies in ensuring value for money and evidence-based decision making is well recognised worldwide.