Empirical essays on global pharmaceutical innovation

The economic impact of the pharmaceutical industry is incontestable. The total drug bill across OECD countries has continuously risen over the last decades (OECD, 2011), and, consequently, healthcare payers are increasingly implementing stricter policies that promote access to cheaper treatments. These policies, however, may hinder incentives to innovate, especially in diseases of substantial public health importance for which there is either under-investment or where innovation is difficult. Therefore, it is fundamental that health systems design policies that strike the right balance between promoting the development of affordable drugs, and allowing sufficient rents to innovators in order to incentivise R&D investment. The debate on this balance has never been more pertinent, with a slowdown in the number of drugs in the pipeline for potential market launch. Around 90% of drug candidates do not successfully complete the mid-stage of drug discovery (Paul et al., 2010; Mestre-Ferrandiz et al., 2012), contributing to the escalation of R&D costs with potentially significant social welfare consequences. The aim of this thesis is to contribute to this debate by exploring the nature of the R&D process, and assessing the factors associated with decreased productivity across disease areas and its equity implications. We survey the literature on the determinants of pharmaceutical innovation and critically appraise the evidence on factors that influence innovation of new therapies. We identify gaps and contribute conceptually to the understanding of the determinants of innovation. We depart from the existing literature in three significant ways. The major contribution is the analysis of failure for all stages of the R&D process, using a unique global panel dataset built by merging data on the lifecycle of industry innovation processes with global health data (Chapter 4). Secondly, we have used methodological approaches that model the dynamic nature of R&D decisions to forecast drug availability in the coming decades (Chapter 5). Thirdly, we are the first to assess the global impact of innovation on equity of access to new therapies (Chapter 6). Acknowledging the implications of data limitations (Chapter 3) we produce insight that contributes to understanding the determinants of failure in innovation and its implications for future availability of new therapies. Results suggest those determinants differ across the R&D stages. Furthermore, market competition may intensify the level of failure if too many young drugs are competing in the market, whilst collaboration between firms has an unclear effect on innovation. Moreover, the distribution of the R&D activity and disease burden have not changed significantly over the last two decades with a concentration of innovation in more commercially attractive disease areas associated with high mortality in the richest countries. Finally, that those equity concerns are likely to persist unless new policy interventions are designed to address inequalities in R&D and access to new therapies.Open Acces

Measuring health outcomes and experiences from a patient’s perspective

The primary objective of health systems is to improve people's health and well-being. Yet, health systems today face significant challenges, ranging from delivering high-quality care to tackling inequalities in health outcomes, getting rid of ineffective care, and investing more in health systems' resilience. Faced with those challenges, the ability to assess whether health systems are successful in delivering good health outcomes and experiences for the people they serve is critical. Health systems are awash with data. There are good measures of what providers can do and the resources this takes – think of the many administrative data about healthcare activities, costs, inputs, outcomes measuring mortality, and incidence and prevalence of diseases. Yet far too little is known about how healthcare impacts the lives of people. This makes it difficult to gain insight into the effectiveness of the healthcare and assess how policy change contributes to improvement in health outcomes.info:eu-repo/semantics/publishedVersio

Health care costs of domestic violence against women – evidence from Portugal

Our main objective is to estimate the additional health care costs to the Portuguese National Health Service (NHS) due to domestic violence against women. We collected information through a survey addressed to health care centres’ female users. Both victims and non-victims of violence were inquired. We estimate costs according to five different groups – consultation costs, health care treatment and therapeutic costs, costs of complementary and diagnostic exams, drugs costs and transport costs. The estimations have been split into two perspectives – the NHS perspective (public perspective) and private perspective of inquired women (out of pocket payments). The timeframe of our calculations is one year, referring to all costs generated by domestic violence situations in the last twelve months. Essentially costs were estimated through the product of total number of episodes by the average estimated price per episode. Additionally, for the private costs, we also considered the costs originated by income losses, the opportunity cost of time spent on health care treatments and the work inability caused by sickness. The results suggest that the victims of domestic violence’s additional demand for health care is valued €140 per annum, that is about 22% higher than health care costs of non-victims. These results match those of similar studies for the United States, taking account of per capita differences in health care spending. A large proportion (90%) of the additional costs associated with domestic violence is supported by the NHS, where consultations and drugs are the most important contributors of such costs. Health consequences of domestic violence result from losses in quality of life and worst health status of victims and correspond to additional permanent economic costs of domestic violence episodes

Patient-reported outcomes and experiences assessment in women with breast cancer: Portuguese case study

In 2020, female breast cancer was the most commonly diagnosed cancer worldwide, representing the type of cancer with the highest incidence among women and the second most common cause of cancer death among women in all OECD countries. The conventional measures addressing the burden of breast cancer by measuring mortality, incidence, and survival do not entirely reflect the quality of life and patients' experience when receiving breast cancer care. The main objective of this study is to capture patient-reported outcomes and experiences in women with breast cancer in Portugal using methods developed for international benchmarking purposes, such as the OECD Patient-reported Indicators Surveys. The study included 378 women with breast cancer, with the age distribution being 19.8% aged 15 to 49 years and 80.2% aged 50 years and over. The data collection procedure and analysis followed the "OECD Breast Cancer Patient Reported Outcomes Working Group" protocol, allowing subsequent comparability with data from other OECD member countries. Most women were satisfied with the treatment outcome regarding the shape of their lumpectomy breasts when wearing a bra (96.1%) and with the equal size of both breasts (78.3%). Findings on the WHO QOL-BREF showed that women manifest a lower score in well-being when compared with the general population or populations living with chronic diseases. This study shows the feasibility of implementing and using patient-reported metrics (PROM and PREM) in breast cancer services in Portugal. Measuring PROMs and PREMs from Portuguese women receiving breast cancer care provides insightful evidence of the quality and value of cancer care.info:eu-repo/semantics/publishedVersio

Vertical and horizontal equity of funding for malaria control: A global multisource funding analysis for 2006-2010

Background: International and domestic funding for malaria is critically important to achieve the Sustainable Development Goals. Its equitable distribution is key in ensuring that the available, scarce, resources are deployed efficiently for improved progress and a sustained Response that enables eradication.Methods. We used concentration curves and concentration indices to assess inequalities in Malaria funding by different donors across countries, measuring both horizontal and vertical equity. Horizontal equity assesses whether funding is distributed in proportion to health needs, whereas vertical equity examines whether unequal economic needs are addressed by appropriately unequal funding. We computed the Health Inequity Index and the Kakwani Index to assess the former and the latter, respectively. We used data from the World Bank, Global Fund, Unicef, President’s Malaria Initiative and the Malaria Atlas Project to assess the distribution of funding against need for 94 countries. National gross domestic product per capita was used as a proxy for economic need and ‘population-at-risk’ for health need

Custos Económicos com a Saúde Resultantes da Violência Doméstica contra Mulheres em Portugal

UID/SOC/04647/2013; SFRH/BPD/112462/2015publishersversionpublishe

Inequities in cancer drug development in terms of unmet medical need

International audienceThis study measures inequality and inequity in the distribution of clinical trials on cancer drug development between 1996 and 2016, comparing the number of clinical trials with cancer need, proxied by prevalence, incidence, or survival rates for both rare and non-rare cancers. We leverage a unique global database of clinical trials activity and costs between 1996 and 2016, constructed for 227 different cancer types to measure for rare and non-rare cancers: i) inequalities and inequity of clinical trial activity, considering all trials as well as split by R&D stage; ii) inequalities and inequity in R&D investment proxied by trial enrollment and duration; iii) evolution of inequity over time. Inequalities are measured with concentration curves and indices and inequities measured with the health inequity index. We find four important results. First, we show pro-low need inequity across cancer types for both rare and non-rare cancers, for all need proxies. Second, we show inequity differs across R&D stages and between rare and non-rare cancers. The distribution of clinical trials for non-rare cancers disproportionately favors low-need non-rare cancers from earlier to later stages of R&D, whilst for rare cancers this only occurs in Phase 2 trials. Third, inequity analyses in R&D investment show that only trial enrollment for rare cancers and trial duration for non-rare cancers are disproportionately concentrated among low-need cancers. Finally, while pro-low need inequity has persisted between 1996 and 2016 for non-rare cancers, it has faded for rare cancers post-EU orphan drugs’ legislation

Patient-Reported Outcomes and Experiences Assessment in Women with Breast Cancer: Portuguese Case Study

In 2020, female breast cancer was the most commonly diagnosed cancer worldwide, representing the type of cancer with the highest incidence among women and the second most common cause of cancer death among women in all OECD countries. The conventional measures addressing the burden of breast cancer by measuring mortality, incidence, and survival do not entirely reflect the quality of life and patients experience when receiving breast cancer care. The main objective of this study is to capture patient-reported outcomes and experiences in women with breast cancer in Portugal using methods developed for international benchmarking purposes, such as the OECD Patient-reported Indicators Surveys. The study included 378 women with breast cancer, with the age distribution being 19.8% aged 15 to 49 years and 80.2% aged 50 years and over. The data collection procedure and analysis followed the “OECD Breast Cancer Patient Reported Outcomes Working Group” protocol, allowing subsequent comparability with data from other OECD member countries. Most women were satisfied with the treatment outcome regarding the shape of their lumpectomy breast when wearing a bra (96.1%) and with the equal size of both breasts (78.3%). Findings on the WHO QOL-BREF showed that women manifest a lower score in well-being when compared with the general population or populations living with chronic diseases. This study shows the feasibility of implementing and using patient-reported metrics (PROM and PREM) in breast cancer services in Portugal. Measuring PROMs and PREMs from Portuguese women receiving breast cancer care provides insightful evidence into the quality and value of cancer care

Patient-Reported Outcomes and Experiences Assessment in Women with Breast Cancer: Portuguese Case Study

In 2020, female breast cancer was the most commonly diagnosed cancer worldwide, representing the type of cancer with the highest incidence among women and the second most common cause of cancer death among women in all OECD countries. The conventional measures addressing the burden of breast cancer by measuring mortality, incidence, and survival do not entirely reflect the quality of life and patients experience when receiving breast cancer care. The main objective of this study is to capture patient-reported outcomes and experiences in women with breast cancer in Portugal using methods developed for international benchmarking purposes, such as the OECD Patient-reported Indicators Surveys. The study included 378 women with breast cancer, with the age distribution being 19.8% aged 15 to 49 years and 80.2% aged 50 years and over. The data collection procedure and analysis followed the "OECD Breast Cancer Patient Reported Outcomes Working Group" protocol, allowing subsequent comparability with data from other OECD member countries. Most women were satisfied with the treatment outcome regarding the shape of their lumpectomy breast when wearing a bra (96.1%) and with the equal size of both breasts (78.3%). Findings on the WHO QOL-BREF showed that women manifest a lower score in well-being when compared with the general population or populations living with chronic diseases. This study shows the feasibility of implementing and using patient-reported metrics (PROM and PREM) in breast cancer services in Portugal. Measuring PROMs and PREMs from Portuguese women receiving breast cancer care provides insightful evidence into the quality and value of cancer care