OP02 A Managed Access Approach To Appraising New Cancer Drugs In England

2017 ◽  
Vol 33 (S1) ◽  
pp. 1-2
Author(s):  
Linda Landells ◽  
Martyn Burke ◽  
Meindert Boysen
Keyword(s):  
Cost Effective ◽  
Integrated Approach ◽  
Evidence Base ◽  
Phase Iii ◽  
Appraisal Committee ◽  
Cancer Drugs ◽  
Cancer Treatments ◽  
Considerable Uncertainty ◽  
Health Technologies ◽  
Current Price

INTRODUCTION:The changing regulatory landscape brings new challenges to Health Technology Assessment (HTA). Marketing authorizations are being granted as the evidence base evolves to facilitate timely patient access to promising health technologies. Consequently, some products come to HTA bodies sooner in their development cycles with less evidence, which ultimately leads to greater uncertainty in decision making. A key challenge for payer and HTA bodies is providing access to promising medicines while the evidence is still emerging, in a financially sustainable way.METHODS:Changes to the Cancer Drugs Fund (CDF) have resulted in a managed access fund for cancer medicines in England. The National Institute for Health and Care Excellence (NICE) can now recommend a treatment for use within the CDF if there is plausible potential to satisfy the criteria for routine use in the National Health Service (NHS) at its current price, but the evidence is not robust enough and associated with significant uncertainty. Further evidence is then generated in clinical trials, through observational data collection, or a combination of the two, while the drug's price reflects the decision uncertainty. At the end of the managed access period, NICE reviews the guidance to determine if the treatment can be recommended for routine commissioning.RESULTS:The first treatment recommended for use within the new CDF was osimertinib for non-small cell lung cancer (1). At the time of NICE appraisal, there was considerable uncertainty in osimertinib's clinical and cost effectiveness because only short-term phase II trial results were available. NICE's independent appraisal committee considered there was plausible potential for osimertinib to be cost effective and identified that an ongoing phase III trial would provide longer-term data addressing the key uncertainties.CONCLUSIONS:An integrated approach between payer and HTA decision-maker has significantly changed how cancer treatments in England are appraised. This collaborative way of working heralds a more sustainable approach to introducing promising cancer treatments.

scholarly journals Statins as Potential Chemoprevention or Therapeutic Agents in Cancer: a Model for Evaluating Repurposed Drugs

2021 ◽  
Vol 23 (3) ◽  
Author(s):  
Nalinie Joharatnam-Hogan ◽  
Leo Alexandre ◽  
James Yarmolinsky ◽  
Blossom Lake ◽  
Nigel Capps ◽  
...  
Keyword(s):  
Drug Development ◽  
Controlled Trial ◽  
Randomised Trial ◽  
Drug Repurposing ◽  
Cancer Chemoprevention ◽  
Cost Effective ◽  
Evidence Base ◽  
Phase Iii ◽  
Epidemiological Research ◽  
Repurposed Drugs

Abstract Purpose of Review Repurposing established medicines for a new therapeutic indication potentially has important global and societal impact. The high costs and slow pace of new drug development have increased interest in more cost-effective repurposed drugs, particularly in the cancer arena. The conventional drug development pathway and evidence framework are not designed for drug repurposing and there is currently no consensus on establishing the evidence base before embarking on a large, resource intensive, potential practice changing phase III randomised controlled trial (RCT). Numerous observational studies have suggested a potential role for statins as a repurposed drug for cancer chemoprevention and therapy, and we review the strength of the cumulative evidence here. Recent Findings In the setting of cancer, a potential repurposed drug, like statins, typically goes through a cyclical history, with initial use for several years in another disease setting, prior to epidemiological research identifying a possible chemo-protective effect. However, further information is required, including review of RCT data in the initial disease setting with exploration of cancer outcomes. Additionally, more contemporary methods should be considered, such as Mendelian randomization and pharmaco-epidemiological research with “target” trial design emulation using electronic health records. Pre-clinical and traditional observational data potentially support the role of statins in the treatment of cancer; however, randomised trial evidence is not supportive. Evaluation of contemporary methods provides little added support for the use of statin therapy in cancer. Summary We provide complementary evidence of alternative study designs to enable a robust critical appraisal from a number of sources of the go/no-go decision for a prospective phase III RCT of statins in the treatment of cancer.

OP303 Do You Get The Message? Making HTA Findings Easier For Decision-Makers To Implement

2021 ◽  
Vol 37 (S1) ◽  
pp. 11-11
Author(s):  
Jess Kandulu ◽  
Ed Clifton ◽  
Iain Robertson ◽  
Neil Smart ◽  
Karen Macpherson
Keyword(s):  
Decision Making ◽  
Technology Assessment ◽  
Health Technology ◽  
Evidence Base ◽  
Decision Makers ◽  
Appraisal Committee ◽  
Economic Modelling ◽  
Health Technologies ◽  
Patient Groups ◽  
The Impact

IntroductionOften health technology assessment (HTA) products developed by the Scottish Health Technologies Group (SHTG) did not reach clear directive conclusions because the evidence base for a technology was weak. Despite being methodologically robust, these products did not meet the needs of decision-makers and may have had negligible impact.MethodsSHTG set out to equip and empower the recommendation-making council (that is, appraisal committee) to reach clear conclusions. SHTG broadened the HTA components and types of evidence that could be considered. The increased breadth of evidence included: clinicians attending council meetings to respond to questions; patient groups making submissions and presenting at council meetings; Scotland-specific economic modelling; and consultation on draft recommendations. SHTG also restructured the council for improved deliberative decision-making.ResultsClear directive conclusions were reached in a substantially higher proportion of HTA products (eighty-eight percent in 2019 compared with eight percent in 2017). It became possible for decision-makers to implement findings. It also became feasible to assess the impact and implementation of recommendations.ConclusionsBroadening SHTG's consideration of HTA components has led to a clearer conclusion being reached and stronger messaging for decision makers. This positions SHTG to increase its influence in the use of health technologies in Scotland.

scholarly journals Cost-effectiveness of mandatory folate fortification v. other options for the prevention of neural tube defects: results from Australia and New Zealand

2009 ◽  
Vol 13 (4) ◽  
pp. 566-578 ◽  
Author(s):  
Kim Dalziel ◽  
Leonie Segal ◽  
Rachelle Katz
Keyword(s):  
New Zealand ◽  
Cost Effectiveness ◽  
Neural Tube ◽  
Neural Tube Defects ◽  
Health Economic Evaluation ◽  
Cost Effective ◽  
Evidence Base ◽  
Considerable Uncertainty ◽  
Supplement Use ◽  
Disability Adjusted Life

AbstractObjectiveTo provide input to Australian and New Zealand government decision making regarding an optimal strategy to reduce the rate of neural tube defects (NTD).DesignStandard comparative health economic evaluation techniques were employed for a set of intervention options for promoting folate/folic acid consumption in women capable of or planning a pregnancy. Evidence of effectiveness was informed by the international literature and costs were derived for Australia and New Zealand.ResultsPopulation-wide campaigns to promote supplement use and mandatory fortification were the most effective at reducing NTD, at an estimated 36 and 31 fewer cases per annum respectively for Australia and New Zealand, representing an 8 % reduction in the current annual NTD rate. Population-wide and targeted approaches to increase supplement use were cost-effective, at less than $AU 12 500 per disability-adjusted life year (DALY) averted ($US 9893, £5074), as was extending voluntary fortification. Mandatory fortification was not cost-effective for New Zealand at $AU 138 500 per DALY ($US 109 609, £56 216), with results uncertain for Australia, given widely varying cost estimates. Promoting a folate-rich diet was least cost-effective, with benefits restricted to impact on NTD.ConclusionsSeveral options for reducing NTD appear to fall well within accepted societal cost-effectiveness norms. All estimates are subject to considerable uncertainty, exacerbated by possible interactions between interventions, including impacts on currently effective strategies. The Australian and New Zealand governments have decided to proceed with mandatory fortification; it is hoped they will support a rigorous evaluation which will contribute to the evidence base.

scholarly journals A service improvement project of a legacy nurse programme to improve the retention of late career nurses

2021 ◽  
pp. 174498712110361
Author(s):  
Sue Haines ◽  
Kerry Evans ◽  
Stephen Timmons ◽  
Ellen Cutler
Keyword(s):  
Registered Nurses ◽  
Workforce Planning ◽  
Cost Effective ◽  
Integrated Approach ◽  
Nurse Retention ◽  
Evidence Base ◽  
Service Improvement ◽  
Improvement Project ◽  
Nurse Programme

Background A Nottingham Legacy Nurse Programme was developed in response to the reducing supply of new nursing registrants and an ageing workforce. The programme comprised components of focussed mentorship, knowledge transition, support and development of new learners in practice. Aims The work-based development programme aimed to improve the retention and experience of late career registered nurses. Methods The programme was informed by the evidence base and co-produced with late career registered nurses (aged 55 years or over, approaching retirement). A small pilot programme ( n = 6) was evaluated through a mixed-methods approach. Refinements and recommendations were proposed in response to findings of a scoping search of the literature, feedback from participants and stakeholder groups across the NHS Midlands and East regions ( n = 238). Results A Legacy Nurse programme has potential to address nurses’ individual career development needs, valuing and retaining them in the workforce, enabling them to share professional knowledge and skills within clinical teams and offers a cost-effective solution to improving retention of late career nurses. Conclusions Addressing the needs of late career registered nurses is required to improve retention, job satisfaction, quality-of-care provision and facilitate knowledge transfer. The programme requires evaluation in other care settings and should be considered as part of an integrated approach to nurse retention, inclusive talent management and workforce planning, alongside financial and careers advice.

Evaluation of anticancer drugs in NSCLC

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e14616-e14616
Author(s):  
H. M. Guirgis
Keyword(s):  
Anticancer Drugs ◽  
Cost Effective ◽  
Stage Ii ◽  
Phase Iii ◽  
Cancer Drugs ◽  
Scoring Model ◽  
Anti Cancer ◽  
Grading Scale ◽  
Corrected Ratio ◽  
Financial Relationships

e14616 Background: Methodology to evaluate anti-cancer drugs in GI malgnancies was previoulsy reported. It has been estimated that the society would be willing to pay $50,000-$100,000 per life-year S gain (S Negrier et, 2007). Objectives: Design scoring model methodology to evaluate and grade anti-cancer drugs in NSCLC with emphasis on the association between between S, AEs, A and C. Methodology: 0S and AEs data were abstracted from J Douillard, R Herbert, Le Chevalier, C Mangeold, A Sandler, F Shepard, R Rossell and C Thienelt (2005–2008). A was graded in points (P) from 3 oral to zero daily injections. AEs: 4 (placebo) to 0 (fatalities) using CTCAE v 3.0. S gain (days,d) was calculated in a corrected ratio of S/360 (SR); Cost R: as C/d/s/$1500, divided by (A+ AEs), corrected and expressed as C scores. C in US $ was based on treating one patient for entire course,70 kg or 1.7/ m2. Using a grading scale from A to D, Survival > 360d, SR>10; C/d/s up to $63 and CR up to 4.2 were rated as (A+) while S< 15d, SR <0.42, C/d/s> $1500 and CR>100 as (D). Results: Examples shown in Table . Adj Cis + Vin after resection in stage II and III scored the highest SR and lowest CR with A+ rating. However, there was a lesser SR using the same combination in mNSCLC. OS markedly improved with Er and BV in 2nd-line. Addition of Cetuximab (Cet) to Cis and Vin demonstrated modest S gain at a much higher CR. Bevacizumab (BV) combined with Pac + Car improved OS over Pac + Car in 1st and 2nd lines at higher CR. Combination of BV, Cis and Gemcitabine demonstrated the lowest S, highest CR and D rating. Discussion: S and C were calculated as Ratios. C scores were based on the association of S with arbitrary but relative weights assigned to AEs and A. BV and Er improved OS in 2nd line, in contrast to phase III Beta study. Adjuvant Cis+Vin seems to be cost-effective. Increased CR was not necessarily associated with improved S. Grading of drugs varied depending on S or C. Higher C/d/s, increased AEs and cumbersome A resulted in higher C scores. Conclusions: SR, CR and possibly C scores might be advantageous in evaluation, grading and comparison of anti-cancer drugs. [Table: see text] No significant financial relationships to disclose.

The cost-effectiveness of atezolizumab in first-line for metastatic triple negative breast cancer is heavily linked to PD-L1 level

2021 ◽  
pp. 107815522110194
Author(s):  
Jacopo Giuliani ◽  
Beatrice Mantoan ◽  
Andrea Bonetti
Keyword(s):  
Breast Cancer ◽  
Triple Negative ◽  
Controlled Trial ◽  
Metastatic Breast ◽  
Cost Effective ◽  
Phase Iii ◽  
Line Treatment ◽  
First Line ◽  
Pivotal Phase ◽  
First Line Treatment

The present analysis was conducted to assess the pharmacological costs of atezolizumab as first-line treatment in triple negative metastatic breast cancer (mBC). Pivotal phase III randomized controlled trial (RCT) was considered. Nine hundred and two patients were included. Differences in costs between the 2 arms (atezolizumab plus nabpaclitaxel versus placebo plus nab-paclitaxel) was 17 398 €, with a cost of 7564 €per month of OS-gain in the overall population and 2485 €per month of OS-gain in PD-L1-positive (≥1) population. Combining pharmacological costs of drugs with the measure of efficacy represented by the OS, atezolizumab could be considered cost-effective in first-line treatment for triple-negative mBC only in PD-L1-positive population, but a reduction of costs is mandatory.

The Mae on Project: Using Acupuncture for Symptom Relief and Improved Quality of Life for People Living with Hiv and Aids in Rural Thailand

2010 ◽  
Vol 28 (1) ◽  
pp. 37-41 ◽  
Author(s):  
Laura Louie ◽  
Nopporn Pathanapornpandh ◽  
Unchalee Pultajuk ◽  
Robert Kaplan ◽  
Ian Hodgson ◽  
...  
Keyword(s):  
Symptom Relief ◽  
Cost Effective ◽  
Integrated Approach ◽  
Hiv And Aids ◽  
People Living With Hiv ◽  
Preliminary Evaluation ◽  
Related Symptom ◽  
History Of ◽  
Living With Hiv

Acupuncture in combination with antiretroviral therapies is a potentially useful treatment for HIV-related symptom relief in resource-poor settings. Traditional Chinese medicine has a long history of being used to enhance immune function. In the setting of HIV, Chinese traditional medicine allows for symptom treatment without adding extra medications to a complex drug regime. This paper provides details of a project at Mae On Hospital in rural northern Thailand where allopathic/conventional treatments are used in tandem with acupuncture. A preliminary evaluation of the project suggests that an integrated approach to symptom relief is viewed positively by respondents receiving acupuncture, though further studies are required to confirm the association between acupuncture and symptom relief. The project also demonstrates the feasibility of developing a cost-effective acupuncture programme using local healthcare staff.

Dose-escalation strategy in refractory metastatic colorectal cancer: A change in terms of cost-effectiveness

2021 ◽  
pp. 107815522199254
Author(s):  
Jacopo Giuliani ◽  
Francesco Fiorica ◽  
Giovanni Ponturo ◽  
Maurizio Azzurro ◽  
Andrea Ruzzenente ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Cost Effectiveness ◽  
Metastatic Colorectal Cancer ◽  
Dose Escalation ◽  
Lower Cost ◽  
Cost Effective ◽  
Phase Iii ◽  
Controlled Trials ◽  
Pivotal Phase ◽  
Randomized Controlled

The analysis was conducted to assess the pharmacological costs of regorafenib and trifluridine/tipiracil in the treatment of refractory metastatic colorectal cancer (mCRC). Pivotal phase III randomized controlled trials (RCTs) of regorafenib and trifluridine/tipiracil in the treatment of refractory mCRC were considered. We have also considered the ReDOS trial, in order to verify if the dose-escalation strategy (practice changing for regorafenib) could influences the results. Differences in OS (expressed in months) between the different arms were calculated and compared with the pharmacological costs (at the Pharmacy of our Hospital and expressed in euros (€)) needed to get one month of OS. Trifluridine/tipiracil resulted the less expensive, with 1167.50 €per month OS-gained. The ReDOS trial further reduce costs with 510.41 €per month OS-gained in favour of regorafenib with the escalation-dose strategy. Both regorafenib and trifluridine/tipiracil can be considered economically sustainable treatments for refractory mCRC, apparently with a lower cost of trifluridine/tipiracil. The adoption of a dose-escalation strategy (ReDOS trial) could reverse the situation making regorafenib more cost-effective than trifluridine/tipiracil.

Industry Watch

2007 ◽  
Vol 11 (22) ◽  
pp. 1468-1482
Keyword(s):  
Clinical Trial ◽  
Stem Cell ◽  
Tamil Nadu ◽  
Point Of Care ◽  
Opioid Therapy ◽  
Phase Iii ◽  
Cancer Drugs ◽  
Phase Iii Clinical Trial ◽  
Chinese Academy Of Sciences ◽  
Academy Of Sciences

Arana Therapeutics Completes Project with CSL. Biotech Capital to Invest $2.2 Million in Generic Health Pty Ltd. Phase III Clinical Trial for “Dual Opioid” Therapy. Beijing Med-Pharm Announces Exclusive Agreement to Market Enablex in China. Genesis Pharmaceuticals Collaborates with the Institute of Microbiology, Chinese Academy of Sciences. NPIL and Merck Collaborate on Cancer Drugs. LifeCell Launches First Stem Cell Center in Tamil Nadu. Japan's Bioventures Today — BCS, Inc. Bio-Rad Launches In2it A1C Analyzer for Point-Of-Care Diabetes Testing.

scholarly journals Self-fill oxygen technology: benefits for patients, healthcare providers and the environment

Breathe ◽  
2016 ◽  
Vol 12 (2) ◽  
pp. 113-119 ◽  
Author(s):  
Phyllis Murphie ◽  
Nick Hex ◽  
Jo Setters ◽  
Stuart Little
Keyword(s):  
Positive Impact ◽  
Healthcare Providers ◽  
Cost Savings ◽  
Cost Effective ◽  
Evidence Base ◽  
Environmental Benefits ◽  
List Type ◽  
Potential Cost ◽  
Unlimited Supply ◽  
Technology Benefits

“Non-delivery” home oxygen technologies that allow self-filling of ambulatory oxygen cylinders are emerging. They can offer a relatively unlimited supply of ambulatory oxygen in suitably assessed people who require long-term oxygen therapy (LTOT), providing they can use these systems safely and effectively. This allows users to be self-sufficient and facilitates longer periods of time away from home. The evolution and evidence base of this technology is reported with the experience of a national service review in Scotland (UK). Given that domiciliary oxygen services represent a significant cost to healthcare providers globally, these systems offer potential cost savings, are appealing to remote and rural regions due to the avoidance of cylinder delivery and have additional lower environmental impact due to reduced fossil fuel consumption and subsequently reduced carbon emissions. Evidence is emerging that self-fill/non-delivery oxygen systems can meet the ambulatory oxygen needs of many patients using LTOT and can have a positive impact on quality of life, increase time spent away from home and offer significant financial savings to healthcare providers.Educational aimsProvide update for oxygen prescribers on options for home oxygen provision.Provide update on the evidence base for available self-fill oxygen technologies.Provide and update for healthcare commissioners on the potential cost-effective and environmental benefits of increased utilisation of self-fill oxygen systems.

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