Background Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by the collection and use of patient-reported outcomes (PROs), which can provide valuable information on the effectiveness, safety, and tolerability of health interventions from the patient perspective. This systematic review aims to examine and summarise the available PRO-specific recommendations and guidance for RWE generation. Methods and findings Medical Literature Analysis and Retrieval System Online, Excerpta Medica Database, and websites of selected organisations were systematically searched to identify relevant publications. 1,249 articles were screened of which 7 papers met the eligibility criteria and were included in the review. The included publications provided PRO-specific recommendations to facilitate the use of PROs for RWE generation and these were extracted and grouped into eight major categories. These included: (1) instrument selection, (2) participation and engagement, (3) burden to health care professionals and patients, (4) stakeholder collaboration, (5) education and training, (6) PRO implementation process, (7) data collection and management, and (8) data analysis and presentation of results. The main limitation of the study was the potential exclusion of relevant publications, due to poor indexing of the databases and websites searched. Conclusions PROs may provide valuable and crucial patient input in RWE generation. Whilst valuable insights can be gained from guidance for use of PROs in clinical care, there is a lack of international guidance specific to RWE generation in the context of use for regulatory decision-making, reimbursement, and health policy. Clear and appropriate evidence-based guidance is required to maximise the potential benefits of implementing PROs for RWE generation. Unique aspects between PRO guidance for clinical care and other purposes should be differentiated. The needs of various stakeholder groups (including patients, health care professionals, regulators, payers, and industry) should be considered when developing future guidelines.

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Introduction: Biological medicines have increased the cost of cancer treatment and has raised sustainability concerns. In Brazil, three monoclonal antibodies (MABs): bevacizumab (BEVA), cetuximab (CETUX) and panitumumab (PANIT), are indicated for the treatment of metastatic colorectal cancer (mCRC) but currently not currently funded by the Unified Health System (SUS). However, they have been funded following successful litigation cases. Objective: Evaluate the budget impact of BEVA, CETUX and PANIT MABs if they became part of standard chemotherapy to treat mCRC within the SUS of Minas Gerais (MG), in Brazil. Method: Budget impact analysis incorporating MABs as first-line treatment of mCRC in MG/Brazil was explored. The Brazilian health system -SUS perspective was adopted and a 5-year time horizon was applied. Data from the lawsuits from January 2009 to December 2016 were collected and the model was populated based national databases and published sources. Costs are expressed in USD. Results: 351 court lawsuits were granted for first-line MAB treatment for mCRC. In three alternative scenarios analyzed there was an increase in costs, which ranged from 348 to 395% compared to the reference scenario. PANIT presented a $103,360,980 budget impact compared to the reference scenario over a 5-year time horizon. BEVA and CETUX presented a $111,334,890 and $113,772,870 budget impact, respectively. Considering the restrictions on the use of MABs Anti EGFR (CETUX and PANIT) in patients with about 41% KRAS mutations, the best cost alternative adopted for incorporation should be the combination of the PANIT and BEVA antibodies, which demonstrated a cost of approximately $106 million. Conclusion: These results highlight the appreciable costs for incorporating BEVA, CETUX and PANIT into the SUS. It is likely appreciable discounts will be needed to permit incorporation.

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Background: Hemophilia B (HB) is a rare disease caused by congenital Factor IX (FIX) deficiency. HB requires life-long management to prevent or manage bleeding and associated morbidity. Although HB affects only a small portion of the population, it is associated with high overall cost and imposes a significant financial burden on individuals, payers, and society in general. Due to variation in patient clinical characteristics and treatment choice, cost and healthcare resource utilization associated with disease management can vary significantly from patient to patient. Aims: To review published direct costs and healthcare resource utilization associated with the management of HB in the US. Methods: A systematic literature review was conducted by searching electronic databases (e.g. MEDLINE, Tufts CEA registry) to identify full-text studies (March 2009–March 2019). Additionally, a manual search for abstracts from relevant conferences was performed (from 2016). Studies were included in the review using pre-defined inclusion/exclusion criteria for population, study type, language (English), and location (US). Publications consisting of budget impact analysis, cost, burden of disease, healthcare resource utilization, and economics evaluations were included. Results: Of 693 titles and abstracts screened, a total of 17 studies evaluating cost and resource utilization in patients with HB in the US were included. Data sources for these studies included: medical records (n = 5), insurance claims databases (n = 10), and surveys (n = 2). Reported cost and resource use varied across studies depending on severity of the disease, treatment regimen, and product type: extended (EHL) or standard half-life (SHL). The cost of FIX replacement therapy constitutes the majority of costs in HB management. Among patients with severe or moderate HB, reported mean annual cost of FIX ranged from $187,070 to $925,864 with an average of $560,801. Annual cost of EHLs could exceed more than twice the cost of SHLs. For example, mean annual cost of EHL FIX was $921,291 vs $478,096 for SHL FIX. Rates of healthcare resource utilization were also substantial for patients with HB and include hospitalizations, emergency room visits, and physician visits. Conclusions: This systematic literature review found significant economic burden associated with HB in the US. The substantial costs and health resources utilized by patients highlight unmet needs remaining in HB.