Search

ObjectivesThe pan-Canadian Oncology Drug Review (pCODR) evaluates new cancer drugs for public funding recommendations. While pCODR's deliberative framework evaluates overall clinical benefit and includes considerations for exceptional circumstances, rarity of indication is not explicitly addressed. Given the high unmet need that typically accompanies these indications, we explored the impact of rarity on oncology HTA recommendations and funding decisions.MethodsWe examined pCODR submissions with final recommendations from 2012 to 2017. Incidence rates were calculated using pCODR recommendation reports and statistics from the Canadian Cancer Society. Indications were classified as rare if the incidence rate was lower than 1/100,000 diagnoses, a definition referenced by the Canadian Agency for Drugs and Technologies in Health. Each pCODR final report was examined for the funding recommendation/justification, level of supporting evidence (presence of a randomized control trial [RCT]), and time to funding (if applicable).ResultsOf the ninety-six pCODR reviews examined, 16.6 percent were classified as rare indications per above criteria. While the frequency of positive funding recommendations were similar between rare and nonrare indication (78.6 vs. 75 percent), rare indications were less likely to be presented with evidence from RCT (50 vs. 90 percent). The average time to funding did not differ significantly across provinces.ConclusionRare indications appear to be associated with weaker clinical evidence. There appears to be no association between rarity, positive funding recommendations, and time to funding. Further work will evaluate factors associated with positive recommendations and the real-world utilization of funded treatments for rare indications.

INTRODUCTION:As of July 2016, funding from England's Cancer Drugs Fund (CDF) is dispensed based on the results of National Institute for Health and Care Excellence (NICE) technology appraisal guidances instead of independent CDF appraisals (1). As part of this transition, NICE is reconsidering drugs previously funded through the CDF (2). This analysis examines CDF reconsiderations conducted between the inception of the new process in July and the end of 2016 to identify any possible trends.METHODS:We collected all NICE final technology appraisal guidances (3) completed before the end of 2016 and noted whether each drug was a CDF reconsideration, what the final decision was, and which factors impacted the decisions.RESULTS:We identified twenty-one NICE oncology reviews competed between July 2016 and the end of 2016. Of these reviews, eight were reconsiderations of drugs previously funded through the old CDF; the rest were new reviews. Only one drug evaluated in the reconsiderations received a negative decision. All the reconsiderations included confidential manufacturer discounts and all noted updated clinical data. End of life (EOL) criteria expanded the acceptable incremental cost-effectiveness ratio (ICER) range for some of the CDF reconsiderations.CONCLUSIONS:All the reconsiderations included updated clinical data and analyses, though it does not appear that updated clinical data were sufficient to bring ICERs to acceptable levels. This is to be expected as the old CDF process served as an alternate funding source for many drugs that did not or were unlikely to fare well under NICE's evaluations. The updated clinical data may have at least increased NICE's confidence in the accuracy of the ICERs. All of the reconsiderations included confidential manufacturer discounts to reach acceptable ICER ranges. The results of this first round of reconsiderations suggest that manufacturers prefer offering their drugs at lower prices to potentially losing National Health Service (NHS) reimbursement entirely.

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.

Introduction:High-cost innovative therapies are considered as high-risk investments in the reality of limited health care budgets. Health technology assessment (HTA) facilitates evidence-based decision making that relies on robust assessment of the clinical cost-effectiveness of the technology that is often not available for expensive cancer therapies (CT).Methods:The objective of the study is to undertake a systematic comparison between HTA agencies worldwide in order to identify similarities and differences in the methods and processes in HTA of CT. A cross-sectional web-based survey was conducted between September 2013 and May 2015. We received responses from 161 HTA organizations based in thirty-nine countries.Results:HTA of CT is mainly performed by agencies in South America (38.46 percent), Australia (37.05 percent) and Europe (36.07 percent), followed by agencies in North America (20.00 percent) and Asia (16.67 percent). Logically, the agencies in high income countries produce more assessments of CT (40.23 percent), which in 34.43 percent they determine as innovative technologies compared with 10.00 percent of the units based in middle income countries and active in CT assessment (11.11 percent). We prove association (p < 0.05) between (i) the type of HTA and income per capita; the level at which the organization operates; its main activity; and the level of recommendation dissemination; (ii) the main target group and consumers of the final HTA product; the stage of evolution of the technology, on which it is likely to be assessed; and approaches to identify innovative technologies. The most active in the preparation of HTA reports are biomedical companies (50.00 percent), government agencies (42.11 percent) and professional organizations (40.00 percent). HTA bodies that assess CT distribute recommendations (37.50 percent) nationally and they are mainly addressed to private health care providers (66.67 percent).Conclusions:Making coverage decisions based on HTA recommendations control the technologies introduction into the healthcare system that is why it's very important this tool to be properly adjusted to the specific needs of CT assessment.

Introduction:The Cancer Drugs Fund (CDF) was set up in 2011 in England to enable patients to access oncology therapies that are not routinely publicly funded. In April 2016, the CDF became a temporary reimbursement fund under the remit of the National Institute for Health and Care Excellence (NICE) with the aim of collecting observational data to inform subsequent technology appraisals. This study aims to evaluate how the reformed CDF has been utilized in the 18 months since this reform.Methods:NICE Final Appraisal Determinations for Single Technology Appraisals of oncology drugs from (29 July 2016 to 24 November 2017) were identified and key data extracted.Results:Seventy-four oncology drug:indication appraisals were identified, 54 (73 percent) were recommended/optimized, 10 (14 percent) were not recommended and 10 drug:indication pairings (14 percent: osimertinib, brentuximab vedotin, pembrolizumab, olaratumab, obinutuzumab, venetoclax, nivolumab [3 indications], and ibrutinib) were referred to the CDF. For most, the greatest uncertainty in their cost-effectiveness analyses related to their survival benefits, intended to primarily be resolved through subsequent clinical trial readouts. However, for venetoclax, ibrutinib and brentuximab, the main areas of uncertainty (relating to comparative survival benefit, pre-progression mortality, and rate of subsequent stem cell transplants, respectively) are expected to be resolved primarily through observational data collected under the CDF.Conclusions:The newly reformed CDF has been utilized in a minority of cases. Typically, the CDF acts as a temporary access mechanism for treatments that receive market authorization based on early/single-arm trial data until longer-term and/or Phase III data are available. However, venetoclax, brentuximab, and ibrutinib demonstrate how the CDF may address significant areas of uncertainty through the collection of uncontrolled observational data. For venetoclax, with only single-arm supportive clinical trial data, observational data of this intervention and appropriate comparator are to be collected, providing a potential case study of how to appropriately manage reimbursement in the face of significant clinical uncertainty.

INTRODUCTION:As of July 2016, funding from England's Cancer Drugs Fund (CDF) is dispensed based on the results of National Institute for Health and Care Excellence (NICE) technology appraisal guidances instead of independent CDF appraisals (1). NICE can recommend providing temporary CDF funding for drugs that have potential to demonstrate cost-effectiveness after further data collection (2). This analysis examines drugs considered for temporary CDF funding since the start of this new process in July 2016.METHODS:We collected all final NICE oncology technology appraisal guidances completed before the end of 2016 and noted whether each drug was considered for or awarded CDF funding and which factors impacted the decision to give or withhold CDF funding (3).RESULTS:We identified twenty-one NICE oncology reviews completed between July 2016 and the end of 2016. Of these reviews, only one was recommended for temporary funding under the CDF because further data collection had the potential to significantly reduce incremental cost-effectiveness ratios (ICERs). Three further reviews were considered for temporary CDF funding but ultimately received negative decisions. In all three cases, NICE found no potential for further data collection to sufficiently improve ICERs. The evaluations also noted that the manufacturers either did not intend or did not have sufficient time to prepare a case for CDF funding.CONCLUSIONS:NICE focused strongly on evidence maturity in making CDF funding decisions. The only drug recommended for CDF funding had immature trial data with uncertainties that could be resolved by further data collection. The three drugs that did not receive CDF funding had relatively mature evidence that would not be improved through further data collection. Timing was also an issue: two reviews specifically noted that the manufacturers had insufficient time to prepare strong cases for CDF funding. The CDF historically had significant budget issues, so NICE may be trying to be more judicious in allocating CDF funding.

Italy is one of the few countries that have matured substantial experience of risk-sharing agreements so far. The first performance-based arrangement was agreed in July 2006, and as of October 2010, eighteen contracts have been in force.The complex management of discount schemes is entirely based on Web registries run by AIFA, the Italian drug agency. The system validates each prescription and automatically requests the hospital pharmacy by e-mail to release the drug. If a patient meets nonresponder criteria, the hospital pharmacist should apply for pay-back to the manufacturer.There are still some important question marks to address. First of all, nonresponders have to be documented by health authorities, otherwise any undocumented nonresponder will be paid as a success. Another question concerns pre-set timing. Although the scientific rationale of the nonresponder criteria for each drug has not been made public, time frames appear too short to allow a reliable assessment. Another question is whether regions, which are financially accountable in Italy for pharmaceutical expenditure, are really able to claw back refunds from manufacturers. Unfortunately here again there are no official figures, and regions do not seem yet able to quantify the amount of pay-back matured in the 4 previous years. The delayed and incomplete availability of pay-back procedures may be one explanation.

Background: Using multiples of India's per capita gross domestic product (GDP) as the threshold for economic value as suggested by the World Health Organization (WHO), decision analysis modeling was used to estimate a more affordable monthly cost in India for a hypothetical new cancer drug that provides a 3-month survival benefit to Indian patients with metastatic colorectal cancer (mCRC).Methods: A decision model was developed to simulate progression-free and overall survival in mCRC patients receiving chemotherapy with and without the new drug. Costs for chemotherapy and side-effects management were obtained from both public and private hospitals in India. Utility estimates measured as quality-adjusted life-years (QALY) were determined by interviewing twenty-four oncology nurses using the Time Trade-Off technique. The monthly cost of the new drug was then estimated using a target threshold of US$9,300 per QALY gained, which is three times the Indian per capita GDP.Results: The base-case analysis suggested that a price of US$98.00 per dose would be considered cost-effective from the Indian public healthcare perspective. If the drug were able to improve patient quality of life above the standard of care or survival from 3 to 6 months, the price per dose could increase to US$170 and US$253 and offer the same value.Conclusions: The use of the WHO criteria for estimating the cost of a new drug based on economic value for a developing country like India is feasible and can be used to estimate a more affordable cost based on societal value thresholds.