Explore the vast range of titles available.

It is increasingly clear that 50 years of international development have done little to reduce poverty in Africa. Indeed, more and more academics and practitioners are highlighting the detrimental effect of traditional development – as carried out by international agencies and NGOs – which often leads to dependency, inefficiency, waste and poor governance. Yet there is a new movement that is surging ahead in its attempt to reduce poverty and generate wealth in Africa: microfranchising. Set up by pioneering organisations such as VisionSpring and HealthStore, microfranchising is based on one of the most successful market-based models in Western economies: franchising. From McDonald’s to Coca-Cola, franchising has proven itself to be an effective and replicable way of scaling up a business rapidly in the Western context. It is only recently that members of the growing body of social entrepreneurs have turned to the franchise model as one of the responses to Africa’s endemic economic stagnation. And the results have been inspiring: instead of the dependency generated by traditional charity development projects, these new social capitalists have generated enterprise and self-sustainability in the most challenging environments of rural Africa. This long-needed book looks at the growth in microfranchising as a tool to generate wealth among poor communities in Africa. The book traces the evolution of the concept of microfranchising, from its foundation in Western models to its implementation in African countries today. It provides practical steps from the world’s leading experts on how to set up a microfranchise, from recruiting franchisees, to building a brand and a supply chain. It gives case studies of successful microfranchises, told by the enterprises themselves. It continues with a theoretical analysis of the place of microfranchising within global social entrepreneurship. It ends with a look at the future for microfranchising, with recommendations for development. Edited by the former CEO of SolarAid, which created the Sunny Money microfranchise, the book provides a ground-breaking set of case studies and analysis of microfranchising for development. It brings together academics and practitioners to provide context, analysis and practical advice. Indeed, it provides the theory, the practical advice and the case studies to guide any entrepreneur, NGO, business or government interested in setting up their own microfranchise scheme.

New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. Our unique consortium model incorporated several of the identified developments under one project, DevelopAKUre, truly bringing together academia, industry and patient organisations in clinical drug development. We found that the equal partnership between all parties in our consortium was a key success factor creating a momentum based on a strong organisational culture where all partners had high engagement and taking ownership of the entire programme. With an agreed mutual objective, this provided synergies through connecting the strengths of the individual parties. Another key success factor was the central role of the patient organisation within the management team, and their unique study participants’ advocacy role securing the understanding and meeting the needs of the clinical study participants in real-time. This resulted in an accelerated enrolment into the clinical studies with a high retention rate allowing for delivery of the programme with significantly improved timelines. Our project was partly funded through an external EU research grant, enabling our model with equal partnership. Further attention within the community should be given to establishing a functional framework where sustainable funding and risk sharing between private and public organisations allow for our model to be replicated.

Background Academic-sponsored trials for rare diseases face many challenges; the present paper identifies hurdles in the set-up of six multinational clinical trials for drug repurposing, as use cases. Methods Six academic-sponsored multinational trials aiming to generate knowledge on rare diseases drug repurposing were used as examples to identify problems in their set-up. Coordinating investigators leading these trials provided feedback on hurdles linked to study, country, and site set up, on the basis of pre-identified categories established through the analysis of previous peer-reviewed publications. Results Administrative burden and lack of harmonization for trial-site agreements were deemed as a major hurdle. Other main identified obstacles included the following: (1) complexity and restriction on the use of public funding, especially in a multinational set up, (2) drug supply, including procurement tendering rules and country-specific requirements for drug stability, and (3) lack of harmonization on regulatory requirements to get trial approvals. Conclusion A better knowledge of the non-commercial clinical research landscape and its challenges and requirements is needed to make drugs—especially those with less commercial gain—accessible to rare diseases patients. Better information about existing resources like research infrastructures, clinical research programs, and counseling mechanisms is needed to support and guide clinicians through the many challenges associated to the set-up of academic-sponsored multinational trials.

Background Alkaptonuria (AKU) is an ultrarare and multifaceted disease characterized by the absence of functional homogentisate 1,2‐dioxygenase activity, the enzyme responsible for breakdown of homogentisic acid—a tyrosine‐degradation product. The presymptomatic phase of the disease makes diagnosis difficult, with many patients unidentified or diagnosed late in life. Objective To date, no study has analyzed the perceived impact of different symptoms or the experiences of individuals through the patient journey in the context of AKU. This study aimed to examine patients' perceptions of AKU symptoms and their impact on quality of life as well as patients' experiences of being diagnosed and living with the disease. Methods Data for this study were collected using a quantitative self‐report questionnaire administered online to people with AKU. Results Data from 45 participants indicate that symptoms with the highest impact for patients are those related to pain and ruptures, disability and inability to perform normal routines, emotional/mental health issues, and heart complications. Findings also revealed significant delays in contact with healthcare services and time to diagnosis. Furthermore, patients reported difficulty in receiving information about AKU, treatment and care, and long‐term disease management support. Conclusions Time to diagnosis and care of AKU is significantly delayed. Symptoms of AKU with the highest impact on quality of life for patients are those related to pain and disability and the inability to perform normal routines. Bridging any gaps between patients with AKU and healthcare professionals through education could help improve patients' experiences with AKU through the patient journey.

Doc number: A37

Make Poverty History was a major episode of protest that took place in 2005 in the UK, with the aim of influencing the G8 meeting in July of that year in Edinburgh. This book follows the campaign throughout its lifetime and unpacks the tensions that developed around the branding of Make Poverty History, particularly the conflict between campaigners and marketers over the content of messages. Looking at how attitudes towards government and political opportunities influenced the negotiation of communications, it analyses how these communications were understood by the campaign's three audience segments: the mass public, the interested and the activists. It looks at public stereotypes on global poverty and whether the campaign managed to modify them. It maps out Make Poverty History's frame on economic justice and confronts it with audience reactions. The book ends by looking at the campaign's troubled relationship with celebrities.

Background Outcomes from studies employing nitisinone 10 mg and 2 mg in alkaptonuria were compared. Patients and methods Sixty‐nine patients in each of the nitisinone (10 mg daily) and controls of suitability of nitisinone in alkaptonuria 2 (SONIA 2), as well as 37 and 23 in nitisinone (2 mg daily) and control cohorts at the National Alkaptonuria Centre (NAC), respectively, were followed up for 4 years. Severity of alkaptonuria (AKU) was assessed by the AKU Severity Score Index (AKUSSI). 24‐h urine homogentisic acid (uHGA24), serum HGA (sHGA), serum tyrosine (sTYR) and serum nitisinone (sNIT) were also analysed at each time point. Dietetic support was used in the NAC, but not in SONIA 2. Safety outcomes were also compared. All statistical analyses were post hoc. Results The slope of the AKUSSI was 0.55, 0.19, 0.30, and 0.06 per month in the control NAC, nitisinone NAC, control SONIA 2, and nitisinone SONIA 2 cohorts, respectively. The intersection of the slopes on the x‐axis was −132, −411, −295, and − 1460 months, respectively. The control and nitisinone slope comparisons were statistically significant both in the NAC (p < 0.001) and the SONIA 2 (p < 0.001). Corneal keratopathy occurred in 3 and 10 patients in the NAC and SONIA 2, respectively. Discussion The nitisinone 10 mg dose decreased disease progression more than the 2 mg dose although the incidence of corneal keratopathy was 14.5% and 4.9%, respectively. Conclusion Nitisinone 10 mg decreased urine and serum HGA, increased serum tyrosine, and decreased disease progression more than 2 mg. Low‐protein dietetic support may be needed to mitigate tyrosinaemia following nitisinone. Highlights Nitisinone 10 mg apparently slows alkaptonuria disease progression more than 2 mg in adults. Corneal keratopathy during nitisinone therapy was more common in men. Serum nitisinone concentrations increased significantly over time. Nitisinone may inhibit cytochrome P450 self catabolism.

BackgroundAlkaptonuria (AKU) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for AKU. Nitisinone decreases homogentisic acid (HGA) in AKU but the dose-response relationship has not been previously studied.MethodsSuitability Of Nitisinone In Alkaptonuria 1 (SONIA 1) was an international, multicentre, randomised, open-label, no-treatment controlled, parallel-group, dose-response study. The primary objective was to investigate the effect of different doses of nitisinone once daily on 24-h urinary HGA excretion (u-HGA24) in patients with AKU after 4 weeks of treatment. Forty patients were randomised into five groups of eight patients each, with groups receiving no treatment or 1 mg, 2 mg, 4 mg and 8 mg of nitisinone.FindingsA clear dose-response relationship was observed between nitisinone and the urinary excretion of HGA. At 4 weeks, the adjusted geometric mean u-HGA24 was 31.53 mmol, 3.26 mmol, 1.44 mmol, 0.57 mmol and 0.15 mmol for the no treatment or 1 mg, 2 mg, 4 mg and 8 mg doses, respectively. For the most efficacious dose, 8 mg daily, this corresponds to a mean reduction of u-HGA24 of 98.8% compared with baseline. An increase in tyrosine levels was seen at all doses but the dose-response relationship was less clear than the effect on HGA. Despite tyrosinaemia, there were no safety concerns and no serious adverse events were reported over the 4 weeks of nitisinone therapy.ConclusionsIn this study in patients with AKU, nitisinone therapy decreased urinary HGA excretion to low levels in a dose-dependent manner and was well tolerated within the studied dose range.Trial registration numberEudraCT number: 2012-005340-24. Registered at ClinicalTrials.gov: NCTO1828463.