Lung cancer (LC) ranks second globally in neoplasia diagnoses and exhibits the highest oncological mortality rate. Oncology has seen a marked increase in U.S. Food and Drug Administration (FDA) approvals for novel drugs, notably in the case of LC. This study aims to assess the innovativeness of recently FDA-approved LC treatments compared to existing options, spanning the 2011 to 2021 period.

The innovativeness of drugs was assessed through the Pharmaceutical Innovativeness Index (PII). The methodology considers population health needs and aggregated clinical benefits, while weighing methodological quality and the adequacy of available evidence. New drugs are analyzed considering available therapeutic alternatives. The assessment assigns an innovativeness score to the drugs, with 1.0 representing the highest degree of innovation. PII was applied to all drugs with their first FDA indication for LC treatment. The evaluation considered specific clinical indications (e.g., treatment of ALK-positive LC), and data from FDA clinical review reports and pivotal clinical trials were considered.

Eighteen new drugs were identified during the period. The drugs were evaluated with a mean PII of 0.615, ranging from 0.474 to 0.811. No discernible trends in innovativeness were observed within specific indications. Overall, the evaluations indicated that these medications were approved to address significant therapeutic needs. However, the added therapeutic value ranged from absent to moderate. Most supporting evidence for these approvals was derived from clinical studies with low risk of bias. Nevertheless, these studies often featured inadequate designs due to the absence of a comparator arm, limiting the assessment of added therapeutic value.

Pharmaceutical innovations in recent years for LC treatment have demonstrated limited clinical benefits when compared to existing alternatives, highlighting a substantial therapeutic need. The application of the PII can assist in healthcare decision-making, guide investments and research efforts, as well as inform pricing and reimbursement of new technologies, thereby contributing to the sustainability of healthcare systems.

It is vital that horizon scanning organizations can capture and disseminate intelligence on new and repurposed medicines in clinical development. To our knowledge, there are no standardized classification systems to capture this intelligence. This study aims to create a novel classification system to allow new and repurposed medicines horizon scanning intelligence to be disseminated to healthcare organizations.

A multidisciplinary working group undertook literature searching and an iterative, three-stage piloting process to build consensus on a classification system. Supplementary data collection was carried out to facilitate the implementation and validation of the system on the National Institute of Health and Care Research (NIHR) Innovation Observatory (IO)‘s horizon scanning database, the Medicines Innovation Database (MInD).

Our piloting process highlighted important issues such as the patency and regulatory approval status of individual medicines and how combination therapies interact with these characteristics. We created a classification system with six values (New Technology, Repurposed Technology (Off-patent/Generic), Repurposed Technology (On-patent/Branded), Repurposed Technology (Never commercialised), New + Repurposed Technology (Combinations-only), Repurposed Technology (Combinations-only)) that account for these characteristics to provide novel horizon scanning insights. We validated our system through application to over 20,000 technology records on the MInD.

Our system provides the opportunity to deliver concise yet informative intelligence to healthcare organizations and those studying the clinical development landscape of medicines. Inbuilt flexibility and the use of publicly available data sources ensure that it can be utilized by all, regardless of location or resource availability.

In Germany, organized cervical cancer screening with annual Papanicolaou (Pap) cytology for women age 20 to 34 years and three-yearly co-testing with human papillomavirus (HPV) and Pap for women as of age 35 years is standard. However, about 30 percent of women eligible for screening remain un/under-screened. We systematically evaluated benefits, risks, and cost-effectiveness of offering additional HPV self-sampling (HPV-SS) to non-attendees.

A validated Markov model for the German context was used to evaluate different HPV-SS screening strategies compared to standard clinician-based screening: HPV-SS for non-attendees age 25 to 65, 30 to 65 or 35 to 65 years, every five years with regular invitation, either opt-in (invitation with link to order the test), or send-to-all (test sent with invitation). German clinical, epidemiological, and economic data (index year 2022/23), along with test accuracy and HPV-SS-attendance data from international meta-analyses and trials were incorporated. Outcomes included undiscounted life years gained (LYG) compared to standard screening without HPV-SS in non-attendees, and the incremental cost-effectiveness ratio (ICER; in EUR/LYG). Comprehensive sensitivity analyses were performed.

Incremental undiscounted effectiveness (compared to standard screening without HPV-SS) and discounted ICERs (compared to next effective) for non-dominated HPV-SS screening strategies were 0.00090 LYG (EUR22,700/LYG) for offering with five-yearly screening invitation an HPV-SS (opt-in) to non-attendees age 35 to 65, 0.00166 LYG (EUR25,900/LYG) for HPV-SS (send-to-all) age 35 to 65, 0.00167 (EUR726,000/LYG) for HPV-SS (send-to-all) age 30 to 65, and 0.00167 LYG (EUR1.78 million/LYG) for HPV-SS (send-to-all) age 25 to 65 years. Other opt-in strategies were dominated. Results were robust over a wide range of parameter variations.

Offering HPV-SS (send-to-all) to non-attendees every five years as an additional strategy within the organized cervical cancer screening program is effective and cost effective in the German context. The results can be used to inform decision-makers and clinical guideline developers regarding incorporation of the specific version of HPV self-sampling into the established organized cervical cancer screening program in Germany.

The China Rural Hypertension Control Project (CRHCP) is a nonphysician-led community-based hypertension intervention program that has demonstrated clear benefits in improving blood pressure (BP) control and reducing the incidence of cardiovascular disease events among hypertensive patients in rural areas of China. However, it is currently unclear whether the benefits of the CRHCP outweigh its costs, and whether promoting this project in China is justifiable from a perspective of healthcare system.

We employed a Markov model to forecast the anticipated 20-year costs and effectiveness of the CRHCP trial. Cost data for this study was gathered from public records or published papers, whereas clinical data was extracted from the CRHCP trial. Our primary outcome measure was the incremental cost-effectiveness ratio, expressed in Chinese Yuan (CNY) per quality-adjusted life-year (QALY), representing the additional cost per additional QALY gained.

Over a span of 20 years, the cost for a rural hypertensive individual in China who received intensive BP intervention by a nonphysician community healthcare provider would amount to 25,129 CNY, yielding an effectiveness of 8.19 QALY. In contrast, if usual care was provided, the cost would be 26,709 CNY with an effectiveness of 7.94 QALY. The CRHCP program demonstrated lower costs and greater effectiveness for rural hypertensive individuals in China.

Our study indicates that the implementation of the CRHCP program among rural hypertensive individuals in China resulted in increased effectiveness and reduced costs. From the perspective of Chinese healthcare system, the CRHCP program proves to be cost-saving within the current healthcare landscape.

The value of health technologies may not be fully reflected in existing generic preference-based measures (GPBMs). The nine-dimension EQ Health and Wellbeing Short (EQ-HWB-S) is a new GPBM developed to assess health, informal carer, and social-care-related quality of life. The objective was to compare standard and new approaches used to generate preferences for the EQ-HWB-S.

Three feasibility studies that included qualitative work have valued the EQ-HWB-S with members of the public in the United Kingdom: (i) videoconferencing interviews using time trade-off (TTO) and discrete choice experiments (DCEs) from the EuroQol Valuation Technology (EQ-VT) protocol (n=600); (ii) online DCEs using the Potentially All Pairwise RanKings of all possible Alternatives (PAPRIKA) method (n=300), an adaptive DCE with a binary search to locate “dead”; and (iii) Online elicitation of Personal Utility Functions (OPUF) (n=300), which is a compositional method with dimension weighting, response level rating and anchoring on dead. Participant/interviewer feedback, data quality, and the weights were compared.

Self/interviewer reported understanding was high (>70%) across all studies. Qualitative findings indicated misunderstanding for some OPUF steps (e.g., the anchoring on dead). Inconsistencies or illogical answers were small in the EQ-VT study (7%) and OPUF (16%). The PAPRIKA study had a priori exclusions criteria (e.g., time taken) that resulted in 44 percent exclusions. Pain, activity, mobility, and sadness/depression were the most important in all the studies. The value of the worst state was −0.384, −0.51, and −0.15 for EQ-PVT, PAPRIKA, and OPUF, respectively, and there were differences in dimension weights (e.g., PAPRIKA gave less weight to mobility but more to cognition).

EQ-HWB-S is a GPBM that provides an innovative approach to measuring and valuing outcomes. Standard and new approaches to eliciting preferences are feasible, but there are differences in the resultant weights. PAPRIKA and OPUF may improve attribute attendance and be more cost effective as they are administered online, but there is scope for improvement to ensure understanding and engagement.

Research with members of health technology assessment (HTA) bodies has uncovered key barriers to integrating patient preference (PP) data into HTA, including concerns about resources/time constraints and a lack of clarity around who is responsible for data generation. We sought to develop a roadmap that addresses these issues, outlining the roles and responsibilities of different stakeholders to foster more sustainable PP data generation.

Based on a forthcoming article to be published in The Patient, this roadmap consists of a step-by-step approach for PP evidence generation. Real-world case studies and literature will be used to illustrate each stage, from identifying priority treatment areas and evidence gaps, forming a steering committee and engaging HTA members, to securing syndicated funding and disseminating results with full transparency.

In contrast to standard approaches to data generation, this roadmap focuses on proactive data collection, collaborating with those who will ultimately use the data (HTA), and pooling resources to mitigate costs and the risk of bias. The roadmap can be applied to all preference-sensitive treatment areas and across health systems/countries. It is designed to be a continuous process, whereby preferences are regularly updated to align with changes to the treatment landscape. A graphic summary of the roadmap is available for viewing at this link: https://cappre.info/images/HTAprocess.pdf

Patient preference data has the potential to make healthcare decision-making more informed, socially legitimate, transparent, and accountable to the patient community. However, current approaches to capturing PP data can be resource intensive with narrow applicability in their findings. The present roadmap offers an alternative, sustainable solution.

Health technology assessment (HTA) guidelines are intended to support the successful implementation of HTA by enhancing consistency and transparency in concepts, methods, processes, and use, thereby enhancing the legitimacy of the decision-making process. This report lays out good practices and practical recommendations for developing or updating HTA guidelines to ensure successful implementation.

The task force was established in 2022 and comprised experts and academics from various geographical regions, each with substantial experience in developing HTA guidelines for national health policy making. Literature reviews and key informant interviews were conducted to inform these good practices. Stakeholder consultations, open peer reviews, and expert opinions validated the recommendations. A series of teleconferences among task force members was held to iteratively refine the report.

The recommendations cover six key aspects throughout the guideline development cycle: (1) setting objectives, scope, and principles of the guideline, (2) building a team for a quality guideline, (3) defining a stakeholder engagement plan, (4) developing content and utilizing available resources, (5) putting in place appropriate institutional arrangements, and (6) monitoring and evaluating guideline success.

This report presents a set of resources and context-appropriate practices for developing or updating HTA guidelines. Across all contexts, the recommendations emphasize transparency, building trust among stakeholders, and fostering a culture of ongoing learning and improvement. The report recommends timing development and revision of guidelines according to the HTA landscape and pace of HTA institutionalization. Because HTA is increasingly used to inform different kinds of decision making in a variety of country contexts, it will be important to continue to monitor lessons learned to ensure the recommendations remain relevant and effective.

Treatment for heart valve diseases (HVD) typically involves surgery, but less invasive procedures are becoming more common. Although the two procedures have similar outcomes, the risk–benefit profiles differ, indicating patients should be included in treatment decisions so they align with their values/preferences. This study aimed to determine patients’ preferences for HVD procedures, and the relative importance of treatment attributes.

An online survey with discrete choice experiment (DCE) was disseminated to patients with aortic stenosis, mitral valve regurgitation, and tricuspid valve regurgitation. Participants were presented with several choice sets, each comprising two hypothetical treatment procedures (labeled “invasive procedure” and “minimally invasive procedure”) as well as an opt-out. DCE attributes were selected based on a literature review, qualitative interviews with patients and specialist doctors, and steering committee consultation (patients, patient organization representatives, and cardiac physicians). Responses were collected via healthcare recruiters, online panels, and patient organizations. DCE data from 143 Australian patients was analyzed using a mixed multinomial logit (MMNL) model.

Results indicate an “experience effect” whereby patients preferred the same type of treatment they had undergone previously. For example, patients who had undergone a transcatheter procedure were more likely to choose the minimally invasive procedure in the experiment and vice versa for those who had undergone invasive procedures like open-heart surgery. Patients were willing to switch procedures based on its risk–benefit profile, and most patients preferred the minimally invasive procedure when it reflected the profile of transcatheter aortic valve replacement (TAVI), even if they had previous invasive procedures experience. Key attributes driving choice were valve durability and regaining independence.

There is a great deal of heterogeneity in HVD patient preferences, even when treatment outcomes appear similar. Patients preferred a minimally invasive procedure over an invasive procedure, irrespective of prior treatment experience with valve durability and independence driving choice. These results can inform healthcare decision-makers about what features of HVD procedures patients value most, taking into consideration patients’ prior experiences.

Constrained resources under universal health coverage (UHC) necessitate a balance between medication costs and essential health system requirements. Policymakers practice priority-setting, as either implicit or explicit rationing, embedded in evidence-informed decision-making processes to guide funding decisions. Health technology assessment (HTA) is a method that may assist explicit evidence-informed priority setting. South Africa developed an official HTA methods guide in 2022, however before this, commissioning and performing economic evaluations was not standardized.

We conducted a descriptive collective case study to explore the impact of economic analyses on the selection of, and access to, essential medicines in South Africa. Four cases were purposefully selected, and both official information and secondary data, including media reports, were reviewed. Data elements were extracted and organized in a matrix. Cases were reported narratively with a positivist epistemological approach, presenting the authors’ reflections.

We found economic analyses that reflected methodologies described in the HTA guide: international reference pricing, cost-minimization, cost-effectiveness, cost-utility, and budget impact analyses. Economic analyses informing the ‘resource-use’ domain in the GRADE evidence-to-decision framework supported decision-making, influenced market-shaping with price reductions of interventions through benchmarking (fosfomycin, flucytosine), improved equitable access nationally (flucytosine), and prioritized a defined patient group in a justifiable and transparent manner (bortezomib).

A standardized HTA evaluation process guided by a nationally accepted framework is necessary for evidence-informed decision-making. Economic analyses (cost-effectiveness, affordability, and resource use) should be consistently included when making decisions on new interventions.

There are significant delays in the funded access to medicines. Studies indicate that in many countries it takes more than a year for patients to have funded access to medicines after market authorization. This study aimed to understand the disparities in timelines for funded access to medicines across different countries and to identify underlying reasons for this access gap.

We conducted a scoping review to examine the nature of health technology assessment (HTA) processes, current methods, and policies for medicines in ten jurisdictions. The jurisdictions included in this study are Australia, Canada, France, Germany, South Korea, the Netherlands, United Kingdom (divided into England, Scotland and Wales), and United States of America. The information was extracted from the websites of International Network of Agencies for Health Technology Assessment (INAHTA) member agencies in the selected jurisdictions, grey literature from governments’ websites, and peer-reviewed literature.

Overall median time from submission of the evidence dossier to HTA recommendations for most jurisdictions is 22 weeks. Although there are similarities in the time taken to reach a funding decision, there are considerable variations in the time taken for patients to have funded access to medicines after HTA recommendations. Only a few countries mentioned a specific timeline within which medicines approved for funding should be listed. Time taken for price negotiations and other arrangements (i.e., risk-sharing agreements) may contribute to varying timelines for listing medicines for funding. Mostly, such negotiations are confidential and may not be time limited.

There was surprising consistency, globally, in the time it takes for funding decisions after medicines registration. The causes of delays in the medicines’ listing decisions are multifactorial and mostly occur after HTA recommendations. The parallel regulatory-assessment process and prioritization tend to reduce the time to a funding decision. However, transparency is needed in the listing process to improve overall timeliness.

The research included a rapid review of current literature to describe epidemiology, management, and system impact of heart valve disease (HVD) in adult populations. Key issues were identified in consultation with expert focus groups and were framed across the continuum of care and systemic policy issues. The groupings were adapted and adjusted during deliberations.

A rapid literature review was conducted on HVD key interventions and evidence of effectiveness along with expert interviews to identify high-level themes for reform. This served as the evidentiary backgrounder. Two virtual policy engagements with clinical leaders, patients, and health system managers were conducted. The focus was their collective drafting of recommendations. These workshops identified nine thematic areas and developed associated recommendations for action under each theme. The success of the process is evident as the report has been taken up as a roadmap for ongoing research and policy work.

A comprehensive grouping of recommendations for improving HVD detection, management, and treatment in Canada was produced. It was designed to be comprehensive to then allow more targeted work to proceed under an evidence-informed and clinically endorsed agenda.

Heart valve conditions are increasingly treatable, especially if detected early. Innovation in treatments as well as detection and management to address gaps in care were identified as the most urgent priorities. The key result was formation of formal working groups with a professional society to explore spoke–hub-and node models for care delivery and to launch awareness programs for early detection.

Digital health technologies have been enhancing the capacity of healthcare providers and, thereby, the delivery of targeted health services. The Southeast Asia Region (SEAR) has invested in strengthening digital public health. Many digital health interventions have been implemented in public health settings but are rarely assessed using the holistic health technology assessment (HTA) approach.

A systematic literature review was performed to provide an overview of evaluations of digital public health interventions in the World Health Organization (WHO) SEAR. Searches were conducted on four electronic databases. Screening title abstracts and full texts was independently conducted by two reviewers, followed by data extraction. Dimensions of HTA were analyzed against the EUnetHTA Core Model 3.0. Quality assessment of included articles was conducted using the JBI Checklist for Economic Evaluation and Consolidated Health Economic Evaluation Reporting Standards 2022 checklist to assess the reporting quality. The findings are presented using systematic evidence tables and bar charts.

Of the forty-three studies screened at the full-text stage, thirteen studies conducted across six countries were included in the analysis. Telemedicine and m-health interventions were assessed in ten studies. Nine studies conducted cost-effectiveness analysis, and five assessments were conducted from a societal perspective. Four studies utilized more than one perspective for the assessment. Health problem definition and current use of technology, description and technical characteristics of the technology, clinical effectiveness, costs, economic evaluation, and organizational aspects were assessed by all the studies, whereas legal aspects were least assessed.

The lack of HTAs on digital public health interventions in the region highlights the need for capacity-building efforts.

Ghana’s reference case, developed to guide the conduct of economic evaluation as part of health technology assessment (HTA) guidelines, recommends the conduct of cost–utility analysis using outcomes such as quality-adjusted life years (QALYs). There is no national value set available for the Ghanaian population to be used in estimating QALYs. This study aimed to develop a value set for Ghana using the EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) instrument.

Face-to-face preference data were collected from 300 adults across three regions of Ghana using the adapted version of the EuroQol Valuation Technology (EQ-VT) standardized valuation protocol developed specifically for EQ-5D-5L valuation studies using composite time-trade-off (cTTO) and discrete choice experiments (DCEs). Different preference models were generated using both the cTTO and DCE data, individually or together to provide complementary results on respondents’ utility preferences. Models explored include generalized least squares, tobit, heteroskedastic, logit, and hybrid. The best-fitting model was selected for the value set based on its logical consistency, ability to account for left-censored and heteroskedasticity data, and statistical significance of parameters.

The 300 interviews provided 4,500 cTTO responses and 4,200 DCE responses. The demographic characteristics of respondents were representative of the Ghanaian population for religious background, level of education, and marital status. The preferred model chosen for the Ghana value set was hybrid tobit, random effect heteroskedastic, constrained model. The predicted value for the worst attainable health on the EQ-5D-5L (i.e., health state 55555) was −0.493 and that of the best health state (11112; except full health) was 0.969. The largest decrement was registered for level five mobility (0.369) followed by pain/discomfort (0.312), self-care (0.273), anxiety/depression (0.271), and usual activities (0.268).

This is the first Ghanaian EQ-5D-5L value set based on social preference derived for a nationally representative sample in Ghana. The value set will play a key role in the institutionalization of HTA in Ghana and the use of economic evaluation studies to inform priority setting where different health technologies can be compared. A planned findings dissemination to stakeholders is underway.

This paper reports on the process used to embark on one of the core strategies of Abu Dhabi’s Department of Health, which was to develop a roadmap for HTA implementation and institutionalization, based on the aspirations and needs of local stakeholders and making use of the evidence-informed deliberative processes framework. The paper also highlights the main features of the road map that may be expected to address some of the current challenges.

A series of activities were undertaken that informed the subsequent development of the roadmap. They comprised a situation analysis using a combination of desk research and semistructured (group) interviews with 45 stakeholders. The findings were discussed in two workshops; face-to-face with nonindustry stakeholders from Abu Dhabi, and online with industry representatives.

Guided by the EDP framework, the roadmap provides instructions how to organize stakeholder involvement, how to identify and operationalize decision criteria, and how to ensure that the decision-making process is transparent. Specific guidance is given on establishing an HTA structure with an appropriate policy framework, the formulation of an HTA program, a communication strategy, as well as building and leveraging HTA expertise.

Broad stakeholder consultation has been instrumental toward the establishment of a comprehensive HTA framework in Abu Dhabi, and the development of a road map. The interest raised during stakeholder consultations and the commitments made hold promise for the adoption and establishment of EDP principles to support HTA in Abu Dhabi that have potential to contribute to a sustainable high-quality healthcare system.

Health technology assessment (HTA) agencies and researchers recognize the necessity of evidence-based methodologies beyond quantitative data to assess feasibility, appropriateness, meaningfulness, patient values, preferences, acceptability, and equity. Despite existing guidelines for synthesizing qualitative data, the HTA framework requires clarification. This review aims to describe the frameworks, tools, and processes used to synthesize qualitative evidence and assess the quality of HTA.

Using the JBI methodology, the authors accessed databases such as MEDLINE, LILACS, CINAHL, Embase, Web of Science, Scopus, PsycINFO, Cochrane Library, JBI Database, and ScienceDirect. Grey literature searches included ProQuest, OpenGrey, CADTH’s Grey Matters, Google Scholar, and HTA agencies’ websites. Inclusion criteria focused on synthesizing qualitative evidence frameworks, methods for evidence synthesis, and quality rating. The review had a global scope, without specific population and time restrictions. Data, encompassing fundamental concepts, frameworks, methods, subjects, and objectives, were presented in tables and figures.

Out of 2,054 articles, 31 were included, mainly from Europe, with a predominant “guide” authored by an HTA agency and university. The majority of documents did not originate from agencies. Only three agencies developed specific documents. A surge in publications occurred in 2018/2019. Qualitative data in HTA were justified for opinions, acceptability, feasibility, and equity. SPICE was the most cited acronym; RETREAT was the preferred framework. Thematic synthesis was the most cited method, CASP for quality assessment. GRADE-CERQual graded evidence quality, and ENTREQ was cited for reporting qualitative research. The GRADE EtD framework was the sole tool mentioned for recommendations.

This review highlights a growing trend in including qualitative evidence in HTA. While various proposals suggest instruments and methods, few documents cover all necessary steps, resulting in diverse recommendations. Standardizing processes can improve decision-making by guiding the integration of qualitative evidence, potentially enhancing recommendation quality. This ensures evidence on feasibility, appropriateness, significance, patient values, preferences, acceptability, and equity are considered.

There are wide variations in the practices of patient involvement in health technology assessment (HTA) in Europe. The field is lacking a consensus on good practices, leading to divergent processes, methods, and evaluation of patient involvement. To identify potential good practice approaches and current gaps, a structured online survey was conducted among HTA stakeholders, including HTA practitioners, patient stakeholders, industry representatives, and others who had experienced patient involvement in HTA.

The questionnaire was co-created by HTA experts, patient stakeholders, and industry representatives and disseminated between 29 April and 14 September 2022.

Responses (n = 168) were submitted from thirty-two European countries by HTA practitioners (n = 33), patient stakeholders (n = 75), industry stakeholders (n = 42), providers (n = 5), academics (n = 7), and others (n = 6). The responses indicated that “allowing access to treatments that have demonstrated value”is the principle rationale for conducting HTA. In terms of the importance of patient involvement, there was consensus across stakeholder groups that “patients have insights and information [that] no other stakeholder has” and that patient involvement is important “to inform HTA which evidence is most patient-relevant”. Shortcomings were identified in the lack of systematic and transparent processes, an unsatisfactory level of information and guidance, and minimal communication and collaboration.

The diverse stakeholders who responded highlighted the need for improving specific aspects of patient involvement practices, including better guidance and information, a more consistent flow of communication between the HTA body and participating patient stakeholders, and the need to develop and implement a consensus on good practices.

Costs for pharmaceutical products are increasing. Pharmaceutical companies claim that high research and development (R&D) costs are the reason for the steep price increase of new products. However, there exists little data to support such claims; there is a lack of transparency in R&D cost reporting. This research intends to analyze and to disaggregate the costs of pharmaceutical R&D.

Studies on the costs of introducing new medications to the market can differ substantially in their methodology, their origin of data, and their results. A scoping review was conducted on costs of R&D for pharmaceutical products using Embase, PubMed, and EconLit, using a combination of the terms “drug research and development” and “costs” or “drug research and development” and “expenditure.” Additionally, semi structured interviews with 16 experts from non-governmental organizations (NGOs), pharmaceutical companies, academic researchers, and not-for-profit pharmaceutical companies were conducted to identify the main driving factors for rising costs of new drugs.

Out of 24 studies that analyzed mixed therapeutic fields, the five highest cost estimates had affiliations with industry or received funding from pharmaceutical companies. Non-affiliated researchers are unable to reproduce studies that use confidential data and therefore cannot check the validity of the results. Additionally, different definitions for R&D make analyzing costs challenging. The interviewees emphasized that driving factors influencing costs for pharmaceutical R&D are therapeutic indication, drug complexity, number of patients in clinical trials, length of the development process, and attrition rates.

Due to the diverse nature of drug development and the confidential information held by pharmaceutical companies, it is a challenge to provide an exact assessment of the average costs of pharmaceutical R&D. Transparency policies with well-defined definitions for R&D are necessary to level the information asymmetries between the private and the public at price negotiations.