Proceed with caution: an economic perspective on the UK's value based pricing proposals

The shift from the Pharmaceutical Pricing Regulation Scheme to Value Based Pricing (VBP) is an important change in the way that medicines will be priced, and consequently, reimbursed in the United Kingdom. Whilst the opportunity to purchase new medicines based on value to society is one that should be welcomed, we should proceed with caution. We highlight ten issues that should be considered relating to innovation, the role and meaning of funding threshold and the adjustments to reflect burden of illness, therapeutic innovation and improvement and wider societal factors. Most importantly, the assessment of value should continue to be based on the characteristics of the displaced activities (e.g. the health produced). To a large extent, all that is changing under VBP are the characteristics being considered; weighted health rather than unweighted health. In addition, we should not totally abandon a cost-utility framework for appraisal just because its current formulation does not match the wider perspective now desired by government

Methodologies for Assessing the Acceptability of Oral Formulations among children and older adults: A Systematic Review

This is an open access article distributed under the terms and conditions of the Creative Commons Attribution NonCommercial-NoDerivatives 4.0 International CC-BY-NC-ND 4.0 license http://creativecommons.org/licenses/by-nc-nd/4.0/Acceptability of medicinal products in children and older populations is pivotal in ensuring adherence and therapeutic outcomes. This review systematically identifies studies reporting on formulation aspects of oral medications that affect their acceptability in these patient groups. Particular emphasis is placed on the evaluation of the methodologies employed in the studies. Sixty-eight studies were included for analysis, with 51 (75%) in children and 17 (25%) in older populations. The studies evaluated a range of oral formulations; however, the methodologies used differ considerably in participants’ characteristics, study settings, tools, acceptability definitions and criteria. It is evident that there is a lack of standardisation in study design as well as the assessment methods used in assessing acceptability of medicines in children and older populations. This review presents a systematic analysis on methods employed for assessing acceptability of oral medicines in children and older adults, to provide insights and recommendations regarding the design of reliable instruments in future studies.Peer reviewe

Acceptability of oral solid medicines in older adults with and without dysphagia : a nested pilot validation questionnaire based observational study

This document is the accepted manuscript version of the following article: Fang Liu, Ambreen Ghaffur, Jackreet Bains, and Shaheen Hamdy, “Acceptability of oral solid medicines in older adults with and without dysphagia: a nested pilot validation questionnaire based observational study”, International Journal of Pharmaceutics, Vol 512 (2): 374-381, March 2016. DOI: 10.1016/j.ijpharm.2016.03.007 This Manuscript version is distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs https://creativecommons.org/licenses/by-nc-nd/3.0/Older patients (aged 65 years and over) are the major consumers of medicines and many barriers affect their ability in taking medicines orally, especially swallowing difficulties. Moreover, the characteristics of differing medicine formulations might have an impact on their acceptability in older patients. The aims of this study were to validate a Medicines Acceptability Questionnaire (MAQ) and to assess acceptability of oral solid medicines in older ambulatory patients with and without dysphagia. One hundred and fifty six older patients attending community pharmacies were recruited and attended face to face interviews. Two questionnaires were administered during the interviews, the validated Sydney Swallow Questionnaire (SSQ) assessing oral and pharyngeal swallowing function and the newly developed Medicines Acceptability Questionnaire (MAQ) evaluating patient acceptability of oral solid medicines. Seventeen (11%) participants displayed symptoms compatible with swallowing difficulties identified by the SSQ. Participants with swallowing difficulties were considered themselves more likely to have problems in swallowing tablets and capsules of large sizes (11mm and 13mm tablets and size #00 capsules) compared to participants without dysphagia. Dispersible/effervescent tablets and orally disintegrating tablets were considered to be the most acceptable in this cohort, followed by mini-tablets. Chewable tablets and granules were the least favoured. Consistently higher acceptability scores were seen in the dysphagic population than in the non-dysphagic population for all of the dosage forms that were easier to swallow than tablets and capsules. The development of these formulations will assist in medication taking in older patients with dysphagia and potentially their adherence to drug treatments.Peer reviewedFinal Accepted Versio

Facing an Epidemic: An Analysis of HIV/AIDS, Antiretroviral Drug, and International Response to the AIDS Pandemic

More than 33 million people are living with HIV/AIDS around the globe with 68% of all cases occurring in sub-Saharan Africa. The global prevalence rate is shocking considering that the disease was relatively unknown just 30 years ago. After reviewing medical, health policy, and health statistical journals, I will argue in this paper that international aid to nations struggling with AIDS needs to be redirected and refocused on supplying antiretroviral therapy to afflicted nations because ARV has been proven to be effective in managing the disease in countries that can afford the costs of treatment. International aid to countries that are ravished by the epidemic, and the United States is one of the top contributors to such efforts with its “President’s Emergency Plan For AIDS Relief (PEPFAR). The U.S. has realized the potential economic benefits of helping out such as becoming primary trade partners with the nations who have plenty of valuable natural resources despite their AIDS issues. In the U.S.’s efforts to combat terrorism, the nation has an interest in “stabilizing” certain countries, which are typically in Africa, so that they can resist potential terrorist threats or military coups. PEPFAR’s goal in fighting AIDS is part of the stabilization effort on the continent of Africa. Fortunately, major pharmaceutical companies have discovered compounds that are effective in attacking the HIV virus, and this has led to the production of “antiretroviral” drugs. Anti-retroviral therapy has proven to be a useful tool used by those suffering from HIV/AIDS to manage their disease better and obtain a higher quality life. Such medications are widely available in wealthy nations, but poor countries that have the highest HIV/AIDS prevalence have a harder time affording such therapy or managing the drug supplies. Potential solutions to this problem include selling antiretroviral drugs at a lower cost to developing nations or using generic versions of such drugs

Two Decades of Publishing Excellence in Pharmaceutical Biotechnology

Recombinant biological products have revolutionized modern medicine by providing both remarkably effective vaccines to prevent disease and therapeutic drugs to treat a wide variety of unmet medical needs. Since the early 1980s, dozens of new therapeutic protein drugs and macromolecular vaccines have been commercialized, which have benefitted millions of patients worldwide. The pharmaceutical development of these biological products presented many scientific and technical challenges, some of which continue today with newer candidates including recombinant protein-based vaccines with novel adjuvants, peptide and RNA-based drugs, and stem cellular therapies. Compared with small molecule drugs, the characterization, stabilization, formulation, and delivery of biomolecules share common hurdles as well as unique challenges. This area of drug development research has been referred to as “pharmaceutical biotechnology”, in recognition of the critical role that recombinant DNA technology plays in the design and production of most of these biological products. Current research focus areas in this field include (i) determination of structural integrity of the primary sequence, post-translational modifications, and higher-order three dimensional shapes, (ii) assessment of physicochemical degradation pathways and their effects on biological activity and potency, (iii) formulation design and development to optimize stability and delivery, (iv) evaluating and optimizing process development steps including lyophilization and fill-finish, (v) analytical method development and applications of new instruments and data visualization tools, (vi) design and development of drug delivery approaches, and (vii) studies of biological effects including pharmacokinetics, pharmacodynamics, and adverse immunogenicity. During the early days of pharmaceutical biotechnology research, there were numerous scientific challenges because the analytical characterization approaches needed for development of recombinant biological molecules in “real world” pharmaceutical dosage forms were essentially unknown. Furthermore, understanding critical drug product manufacturing issues (e.g., stability of biological compounds during processing, storage, and shipping as well as reproducibility of fill-finish production technologies) and behavior during and after patient administration was often achieved by “on-the-job” training. Fortunately, the pioneers in the field regularly presented research at key conferences and started publishing early in pharmaceutical sciences journals such as Journal of Pharmaceutical Sciences. Recognizing this critically important new field, the then Editor of the journal, Professor Bill Higuchi, instituted a new “pharmaceutical biotechnology” category for research papers. This insightful move was coupled with an equally wise decision to recruit Dr. C. Russell Middaugh as the new Associate Editor for the new research category. As will be detailed below, under Dr. Middaugh’s diligent and expert guidance, pharmaceutical biotechnology papers have grown in number, scope, and impact over the past 20 years, and these days, the Journal of Pharmaceutical Sciences is viewed by scientific leaders in the field as the “go to” place for publication of the most important results and descriptions of innovations in pharmaceutical biotechnology

Access to antiepileptic drug therapy in children in Camagüey Province, Cuba

Objective: To describe access to antiepileptic drug therapy and estimate the prevalence of epilepsy in children in Camagüey Province, Cuba. Methods: All the community pharmacies in the province were visited and information collected about the number of children receiving antiepileptic drugs in 2009. Availability and cost of each antiepileptic drug were determined. The prevalence of epilepsy was estimated by determining the number of children receiving antiepileptic drugs. Results: There were 923 children who received a total of 977 antiepileptic drugs in Camagüey Province. The estimated prevalence of epilepsy was 5.18 per thousand children which is lower than previously reported rates in other low and lowermiddle income countries. Most of the children (871, 94%) received a single antiepileptic drug. Carbamazepine and valproate were the two most frequently prescribed antiepileptic drugs. Antiepileptic drugs were available from the local pharmacy on 76% of occasions. If the antiepileptic drug was not available from the local pharmacy, the parent had to travel to another pharmacy to obtain the medicine. Conclusions: The estimated prevalence of epilepsy in children in Cuba is lower than that estimated in other lower-middle income countries. Access to drug therapy in children with epilepsy can be achieved in lower-middle income countries

Exploring innovative Leishmaniasis treatment: drug targets from pre-clinical to clinical findings

Leishmaniasis is a group of tropical diseases caused by parasitic protozoa belonging to the genus Leishmania. The disease is categorized in cutaneous leishmaniasis (CL), mucocutaneous leishmaniasis (MCL), and visceral leishmaniasis (VL). The conventional treatment is complex and can present high toxicity and therapeutic failures. Thus, there is a continuing need to develop new treatments. In this review, we focus on the novel molecules described in the literature with potential leishmanicidal activity, categorizing them in pre-clinical (invitro, invivo), drug repurposing and clinical research.This research was funded by Conselho Nacional de Desenvolvimento Científico e Tecnológico for the Scientific grants (CNPq 301964/2019-0 Chamada No. 06/2019, Chamada CNPq No. 01/2019) and Portuguese Science and Technology Foundation, Ministry of Science and Education (FCT/MEC) through the sponsorship of the project M-ERA-NET-0004/2015-PAIRED (strategic fund), co-financed by FEDER, under the Partnership Agreement PT2020. We would like to thank Tiago Branquinho Oliveira for the help provided in drawing Figure 3.info:eu-repo/semantics/publishedVersio

Medicines Reuse

This reprint examines the concept of medicines reuse, the idea that unused medication returned by one patient can be re-dispensed for use by another. Ten papers written by over 20 authors examine a range of issues related to medicines reuse including the circular economy of the pharmaceutical supply chain; the prevalence of unused medicines or medication waste within patients' homes; people's views about the causes of medication waste and the potential for medicines reuse; what might influence people to reuse medicines in the future; how sensing technologies might facilitate medicines reuse; and the effect of including sensing technologies on people's willingness to consider medicines reuse

Access to antiepileptic drug therapy in children in Camagüey Province, Cuba

Objective: To describe access to antiepileptic drug therapy and estimate the prevalence of epilepsy in children in Camagüey Province, Cuba. Methods: All the community pharmacies in the province were visited and information collected about the number of children receiving antiepileptic drugs in 2009. Availability and cost of each antiepileptic drug were determined. The prevalence of epilepsy was estimated by determining the number of children receiving antiepileptic drugs. Results: There were 923 children who received a total of 977 antiepileptic drugs in Camagüey Province. The estimated prevalence of epilepsy was 5.18 per thousand children which is lower than previously reported rates in other low and lowermiddle income countries. Most of the children (871, 94%) received a single antiepileptic drug. Carbamazepine and valproate were the two most frequently prescribed antiepileptic drugs. Antiepileptic drugs were available from the local pharmacy on 76% of occasions. If the antiepileptic drug was not available from the local pharmacy, the parent had to travel to another pharmacy to obtain the medicine. Conclusions: The estimated prevalence of epilepsy in children in Cuba is lower than that estimated in other lower-middle income countries. Access to drug therapy in children with epilepsy can be achieved in lower-middle income countries