SDG indicator 3.b.3 - an analysis of its robustness and challenges for measuring access to medicines for children.

BACKGROUND: Sustainable Development Goal (SDG) indicator 3.b.3 monitors progress in medicines' accessibility for adults and has significant limitations when applying to medicines for children. An adapted indicator methodology was developed to fill this gap, but no proof of its robustness exists. We provide this evidence through sensitivity analyses. METHODS: Data on availability and prices of child medicines from ten historical datasets were combined to create datasets for analysis: Dataset 1 (medicines selected at random) and Dataset 2 (preference given to available medicines, to better capture affordability of medicines). A base case scenario and univariate sensitivity analyses were performed to test critical components of the methodology, including the new variable of number of units needed for treatment (NUNT), disease burden (DB) weighting, and the National Poverty Line (NPL) limits. Additional analyses were run on a continuously smaller basket of medicines to explore the minimum number of medicines required. Mean facility scores for access were calculated and compared. RESULTS: The mean facility score for Dataset 1 and Dataset 2 within the base case scenario was 35.5% (range 8.0-58.8%) and 76.3% (range 57.2-90.6%). Different NUNT scenarios led to limited variations in mean facility scores of + 0.1% and -0.2%, or differences of + 4.4% and -2.1% at the more critical NPL of $5.50 (Dataset 1). For Dataset 2, variations to the NUNT generated differences of + 0.0% and -0.6%, at an NPL of $5.50 the differences were + 5.0 and -2.0%. Different approaches for weighting for DB induced considerable fluctuations of 9.0% and 11.2% respectively. Stable outcomes with less than 5% change in mean facility score were observed for a medicine basket down to 12 medicines. For smaller baskets, scores increased more rapidly with a widening range. CONCLUSION: This study has confirmed that the proposed adaptations to make SDG indicator 3.b.3 appropriate for children are robust, indicating that they could be an important addition to the official Global Indicator Framework. At least 12 child-appropriate medicines should be surveyed to obtain meaningful outcomes. General concerns that remain about the weighting of medicines for DB and the NPL should be considered at the 2025 planned review of this framework.

Pharmaceutical Scientists' Perspectives on Capacity Building in Pharmaceutical Sciences.

With the anticipated health challenges brought by demographic and technological changes, ensuring capacity in underlying workforce in place is essential for addressing patients' needs. Therefore, a timely identification of important drivers facilitating capacity building is important for strategic decisions and workforce planning. In 2020, internationally renowned pharmaceutical scientists (N = 92), largely from the academia and pharmaceutical industry, with mostly pharmacy and pharmaceutical sciences educational background were approached (through a questionnaire) for their considerations on influencing drivers to facilitate meeting current capacity in pharmaceutical sciences research. From a global view, based on the results of the questionnaire, the top drivers were better alignment with patient needs as well as strengthening education - both through continuous learning and deeper specialisation. The study also showed that capacity building is more than simply increasing the influx of graduates. Pharmaceutical sciences are being influenced by other disciplines, and we can expect more diversity in scientific background and training. Capacity building of pharmaceutical scientists should allow flexibility for rapid change driven by the clinic and need for specialised science and it should be underpinned by lifelong learning.

Selection of essential medicines for the prevention and treatment of cardiovascular diseases in low and middle income countries.

BACKGROUND: The incidence and mortality of cardiovascular diseases (CVDs) in low and middle income countries (LMICs) have been increasing, while access to CVDs medicines is suboptimal. We assessed selection of essential medicines for the prevention and treatment of CVDs on national essential medicines lists (NEMLs) of LMICs and potential determinants for selection. METHODS: Only operational NEMLs were considered eligible for this study. A selection of medicines listed under "cardiovascular medicines" or "blood products and plasma substitutes" in the NEMLs were included if they were present on international guidelines for the prevention and treatment of CVDs (hyperlipidemia, hypertension, platelet inhibition, ischemic stroke, stable ischemic heart disease, acute coronary syndromes, heart failure, atrial fibrillation, peripheral arterial disease and acute limb ischemia). The number and diversity of essential medicines selected for CVDs were studied. Moreover, determinants of selection of essential medicines for CVDs at a national level were explored. Data analysis was done using univariate linear regression and non-parametric tests. RESULTS: All medicine groups listed by the international guidelines were selected by the majority of the 34 countries studied with the exception of adenosine diphosphate receptor inhibitors which appeared on less than half of the NEMLs studied (41% of countries). The total number of essential medicines for the prevention and treatment of cardiovascular diseases (median 24 (range 16-50)) differed significantly across income levels (median range: 19.5-25, p = 0.014) and across regions (median range: 20-32, p = 0.049). When recommendations of the international guidelines were considered, over 75% of the NEMLs contained essential medicines for the majority of CVDs. CONCLUSION: The main medicine classes for the management of CVDs were represented on NEMLs. Consequently, for the majority of CVDs, evidence-based guideline-recommended treatment is possible as far as selection of essential medicines is concerned. Selection will therefore not be the limiting step in access to medicines for cardiovascular diseases.

Risk management plans as a tool for proactive pharmacovigilance: a cohort study of newly approved drugs in Europe.

Risk Management Plans (RMPs) have become a cornerstone in the pharmacovigilance of new drugs in Europe. The RMP was introduced in 2005 to support a proactive approach in gaining knowledge on safety concerns through early planning of pharmacovigilance activities. However, the rate at which uncertainties in the safety profile are resolved through this proactive approach is unknown. We therefore examined the evolution of safety concerns in the RMP after initial approval for a selected cohort of 48 drugs, to provide insight into the knowledge gain over time. We found that 20.7% of the uncertainties existing at approval had been resolved 5 years after approval. Because new uncertainties were included in the RMP at a similar rate, the overall number of uncertainties remained approximately equal. The relatively modest accrual of knowledge, as demonstrated in this study through resolution of uncertainties, suggests that opportunities for optimization exist while ensuring feasible and risk-proportionate pharmacovigilance planning.

Selection of oncology medicines in low- and middle-income countries.

BACKGROUND: High cancer mortality rates in low- and middle-income countries (LMICs) have raised concerns regarding access to oncology medicines. Essential medicines are those which satisfy the primary health care needs and provide a basis for public procurement or reimbursement decisions in LMICs. We explored selection of oncology medicines in LMICs through investigating national essential medicines lists (NEMLs) for cancer treatments. METHODS: Recently updated NEMLs were retrieved for 76 countries. Oncology medicines were classified based on therapeutic categories. Countries were clustered based on geographic regions, income levels and burden of cancer (mortality and morbidity). Indicators of frequency (number of medicines), diversity (number of therapeutic (sub)categories) and more importantly absence were measured and compared across countries using parametric and nonparametric tests. RESULTS: The overall median number of oncology medicines on NEMLs was 16 (interquartile range = 23) chosen predominantly from subcategories of 'antineoplastic agents', with substantial variation across regions and income groups. Five countries did not select any oncology medicine and 68% did not have any 'hormones and related agents' on their NEMLs. Newer technologies like targeted therapies were infrequently incorporated. The cluster of countries suffering most from the burden of cancer selected more essential oncology medicines and diversified further. CONCLUSION: The observed selection of oncology essential medicines can reflect insufficiencies and inequalities in access to cancer treatments at least in the public sector of LMICs. Further resources need to be allocated from governments and international organizations to tackle the problem of access to oncology medicines in these countries.

Personalised medicine as a challenge for public pricing and reimbursement authorities ­ A survey among 27 European countries on the example of trastuzumab.

OBJECTIVES: To survey possible funding models and pricing practices as well as prices for the treatment package of trastuzumab and its accompanying diagnostic test in European countries, as an example of personalised medicines. METHODS: Qualitative descriptive data on national pharmaceutical pricing and funding policies applied to trastuzumab and its accompanying diagnostic test were obtained from a survey among competent authorities from 27 European countries as of August 2011. Further, price data (for the years 2005-2013) of trastuzumab in the respective European countries were surveyed and analysed. RESULTS: In 2011, testing and treatment mainly took place in hospitals or in specific day-care ambulatory clinics. In the European countries either both trastuzumab and the accompanying diagnostic test were funded from hospital budgets (n = 13) or only medicines were funded from the third party payers such social insurances and the test from hospital budgets (n = 14). Neither combined funding of both medicine and diagnostic test by third party payers was identified in the surveyed countries nor did the respondents from the competent authorities identify any managed entry agreements. National pricing procedures are different for trastuzumab versus its diagnostic test, as most countries apply price control policies for trastuzumab but have free pricing for the diagnostic test. The ex-factory price is, on average, €609 per 150 mg vial with powder in 2013; in nine countries the price of trastuzumab went down from 2005 till 2013. CONCLUSION: The example of trastuzumab and its accompanying diagnostic test highlights some problems of the interface between different funding streams (out-patient and hospital) but also with regard to the interface between the medicine applied in combination with a medical device. The findings suggest a need for further developing and refining policy options to address the identified interface issues.

Differences in external price referencing in Europe: a descriptive overview.

OBJECTIVE: This study aimed to provide an up-to-date description as well as comparative analysis of the national characteristics of pharmaceutical external price referencing (EPR) in Europe. METHODS: Review of the country-specific PPRI (Pharmaceutical Pricing and Reimbursement Information) Pharma Profiles written by representatives of the PPRI Network. The Profiles were analysed according to predefined criteria. RESULTS: Of 28 analysed European countries 24 applied EPR in 2010. The majority of countries have statutory rules to implement EPR. Most countries had less than 10 countries in their reference baskets. Higher income countries tend to include higher income countries in their basket, whereas lower income countries refer to lower income countries. Taking the average price of all countries in the basket as the basis to calculate the national price was the most common strategy (n=8). The methodology of EPR has changed in most European countries over the past 10 years (n=19). CONCLUSIONS: EPR is a widely used pricing policy in Europe and is still actively used as well as adjusted by national authorities. However, we still see room for improvement by implementing more detailed legislations in terms of the revision of prices and by identifying alternative countries in case a product is not on the market. We also see the need for formal information sharing (e.g. congresses dedicated to pricing strategies and systems) with other public pricing authorities to learn about the different EPR methodologies as well as the national experiences. These congresses might also give room to better understand national pricing methods including discussions on possible limitations of these pricing methods.