Value in Health最新文献

Objectives: Value-based contracts, also called outcomes-based agreements, tie reimbursement to treatment performance, usually operationalized as one or more patient outcomes within a given timeframe. These agreements offer an appealing risk-sharing mechanism between manufacturers and payers. Despite this, uptake of value-based contracts has been limited likely due, in part, to contract complexity. This study explores how probabilistic simulations can facilitate planning of value-based contracts while characterizing the net financial benefits based on expected real-world treatment performance, rebate percentage, and the number of patients included in the value-based contract.

Methods: We simulated single milestone value-based contracts that would cover treatment of 15 or 100 patients based on a binary outcome of treatment response with 10,000 iterations for each scenario. We considered treatment response rates of 50%, 75% and 90%, obtained from clinical trials of 30, 100 or 500 patients. We estimated the equivalent discount rates (95% confidence interval) that could be achieved from value-based contracts and descriptively compared the simulation results.

Results: Our simulation found that the range of likely value-based contract outcomes increases as the size of the informative trial decreases. We also found that the range of possible rebate amounts is primarily driven by the number of patients treated, not uncertainty in the clinical evidence.

Conclusions: Our results suggest differential risk sharing between a payer and a manufacturer, and that manufacturers may benefit from a risk pooling effect by having multiple value-based contracts with multiple payers. Value-based contracts often involve complex trade-offs and multiple sources of uncertainty. Adopting simulation-guided contract planning during the negotiation process can help quantify the financial benefit offered by a value-based contracts.

Objectives: The Orphan Regulation, introduced in 2000, offers a ten-year exclusivity to recoup investments on orphan medicinal products (OMPs). Despite expectations of price drops, many OMPs seem to lack competition post-exclusivity. This study examines OMP price trends and the entry of generics/biosimilars in the Netherlands.

Methods: OMPs authorized before January 1, 2010, were included unless they had active substance patents or were withdrawn before the end of market exclusivity. Data on European generic/biosimilar authorization were gathered from the EMA and Dutch Medicines Evaluation Board. List price data were collected from the Dutch national database.

Results: Twenty-eight small molecules and nine biologicals were included in the primary analysis, all authorized between 14.0-22.3 years. The median of their most recent prices in the Netherlands was 81% of the initial price (range 11-104%). Eleven small molecule (39%) and eight biological (89%) OMPs did not have a generic/biosimilar registered. If generic/biosimilar entry did occur, this was after a median of 14.2 years (range 11.5-20.3 years). Median prices of OMPs with generic/biosimilar competition were lower than those without (to 66% versus 88% of the initial price, respectively, p < 0.01).

Conclusions: Post-exclusivity competition and subsequent price decrease is limited for OMPs. First, generics/biosimilars for OMPs enter later than non-OMPs. Second, for some OMPs (especially biologicals) there is no competition at all. Third, prices of OMPs with generic/biosimilar competition decreased only minimally compared to non-OMPs. New policies are needed to correct this apparent market failure.

Objectives: To promote uniform and high quality economic evaluations, several national Health Technology Assessment (HTA) bodies have developed guidelines. Due to ongoing (methodological) developments within the field of HTA and economic evaluations, the Dutch health economic guideline needed a revision. This article briefly discusses the process of the latest revision, highlights most important changes, and presents a research agenda with topics for which more research is desired.

Methods: An independent committee of eight Dutch academic HTA experts was installed to advise the National Health Care Institute on this revision. A survey was sent to all relevant stakeholders in order to obtain input on adjustments needed. The committee discussed the results from the survey, and during four meetings formulated its advice accordingly.

Results: The most important revisions are a lowered discount rate for costs, additional guidance concerning expert opinion and expert elicitation, the inclusion of health-related quality of life of informal caregivers, performing probabilistic analysis for the main results, indirect medical costs in life years gained, additional guidance on empirical economic evaluations and the inclusion of value of information analyses. Furthermore, the costing manual has been updated as well, including updated reference prices and additional price categories related to educational and judicial costs.

Conclusion: The revised Dutch guideline provides up-to-date guidance for conducting economic evaluations in the Netherlands that can inform health policy decisions from a broad societal perspective.

Objectives: This study examines the role of mental health in consumer healthcare choices, using a discrete choice experiment to analyse choices regarding routine primary care visits in Australia. It captures mental health through three variables: self-reported current mental health condition and clinically validated measures of depression and anxiety symptoms, the Patient Health Questionnaire 9 (PHQ-9) capturing depression, and the Generalized Anxiety Disorder 7 (GAD-7) capturing anxiety.

Methods: Data were collected during November-December 2021 from a sample (N=568) representative of the Australian population in age, gender, and location. Participants made hypothetical choices between in-person and telehealth alternatives or a 'no visit' alternative. Alternatives were described in terms of GP familiarity, out-of-pocket cost, wait time, waiting area size, mask requirements, and modality of telehealth.

Results: The results suggest that symptoms of depression and anxiety could affect healthcare choices with opposite direction of effect on uptake and distinct from the presence of a mental health condition.

Conclusions: These findings support the need for more careful consideration of the role of mental health in the analysis of DCEs, particularly in healthcare, including better understanding of the mechanisms and time-varying nature of any effect.

Objectives: Research into methods for eliciting adolescents' health state preferences has mostly avoided tasks for identifying health states worse than dead, which is required for calculating quality-adjusted life years for economic evaluations. This study investigated the feasibility of eliciting the health state preferences of older adolescents, including for states worse than dead, using the EQ-5D-Y-5L (Y-5L) and EQ-5D-5L (5L), and compared participants' preferences across the two instruments.

Methods: Two online surveys were created for the Y-5L and 5L respectively using the Potentially All Pairwise RanKings of all Possible Alternatives (PAPRIKA) method, a type of adaptive discrete choice experiment, and a binary search algorithm for identifying health states worse than dead. The surveys were completed by 24 adolescents aged 16-19 years in two think-aloud sessions, with semi-structured interviews at the end of each session. Dimension preference weights and rankings for the Y-5L and 5L were compared using intraclass correlation coefficients, Bland-Altman plots and paired t-tests.

Results: The adolescents were capable of valuing health states and identifying states worse than dead. There is no evidence of a difference in mean preference weights between the Y-5L and 5L, and the rankings of dimensions are similar.

Conclusions: Eliciting the health state preferences of older adolescents, including for states worse than dead, is feasible and acceptable. The similarity in Y-5L and 5L mean preference weights suggests their corresponding value sets, if obtained using the methods used in this study, would be similar. Data quality was enhanced by the surveys being administered in a supportive environment.

Objectives: Amyloid-targeting therapies for Alzheimer's disease (AD), such as lecanemab, target and reduce brain β-amyloid plaques. To initiate therapy accurately, confirming the presence of brain β-amyloid plaques is necessary. This research investigated the added value and cost-effectiveness of ¹⁸F-flutemetamol positron emission tomography (PET) in the United States.

Methods: A cost-effectiveness model was developed to evaluate the differential diagnostic accuracy for AD and associated clinical and economic outcomes between ¹⁸F-flutemetamol PET imaging and cerebrospinal fluid (CSF) testing. The model simulates the impact of the diagnostic modality choice in a hypothetical patient cohort with mild cognitive impairment or suspected AD and aged 40 to 79 years for eligibility to receive Amyloid-targeting therapy.

Results: The use of ¹⁸F-flutemetamol PET to diagnose AD results in an additional 14% of patients correctly identified versus CSF testing. Furthermore, the use of ¹⁸F-flutemetamol PET leads to fewer patients expected to progress to moderate/severe AD (6.83% vs 6.91%) and is associated with decreased mortality (36.88% vs 37.06%). Over a patient's lifetime, ¹⁸F-flutemetamol PET contributes an additional 0.02 discounted quality-adjusted life years compared with CSF (4.91 vs 4.89) with an incremental discounted cost of $1405. This translates to a cost of $73 872 per quality-adjusted life year gained.

Conclusions: Compared with CSF testing, ¹⁸F-flutemetamol PET imaging is less invasive, leading to more patients being correctly diagnosed and targeted for appropriate therapy, which has a downstream impact on disease progression and mortality. The model suggests that ¹⁸F-flutemetamol PET is a cost-effective diagnostic modality for US payers.

Objectives: To review health technology assessment reassessments (HTARs) and characterize the use of real-world evidence (RWE) in HTARs.

Methods: Six agencies were chosen for inclusion in this targeted review: Canadian Agency for Drugs and Technologies in Health, National Institute for Health and Care Excellence, Haute Autorité de Santé, Gemeinsamer Bundesausschuss/Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Zorginstituut Nederland, and Pharmaceutical Benefits Advisory Committee. Each agency's assessment was screened to identify their 8 most recent HTARs, which were evaluated to determine if they used RWE. If for a given agency less than half of the screened HTARs used RWE, we identified an additional 4 HTARs for evaluation. For each reassessment, we extracted drug characteristics, HTAR details, initial assessment details, and if/how the RWE was used. Narrative synthesis in conjunction with descriptive statistics were used to characterize the findings.

Results: We identified 40 HTARs across the agencies. Over half of the HTARs were for oncology therapies. Additionally, 55% used RWE; these reassessments tended to be for orphan therapies. RWE was submitted to address at least 1 clinical uncertainty, with the most common being related to the primary/secondary endpoints. The majority of RWE studies came from registry data (57.1%). Moreover, the proportion of HTARs resulting in no change in patient access was similar between HTARs that did and did not use RWE. Lastly, no de novo RWE comparative effectiveness studies were identified.

Conclusions: Our findings imply that RWE can play a role in addressing uncertainties identified at launch, especially in addition to clinical trial evidence; agencies and sponsors should collaborate/align on evidence needs and study feasibility to ensure RWE can be effectively used in reassessments.