Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need

Progress in understanding human diseases is continuous: by providing new insights it has been a major factor in developing and optimizing the use of old and new drugs. As a result, the numbers of diseases that can be cured or at least significantly modified are increasing continuously. This progress in quality and in quantity has not been matched by progress in the universality of access to care. Vast inequalities in health and in health services exist and persist (see Closing the gap in a generation: health equity through action on the social determinants of health. Geneva, World Health Organization, 2008; and references therein). In the past, for many countries in Africa, the main limiting factor in medical services was failure of diagnosis; nowadays, many of those countries have made efforts to train medical and other health personnel who, having made a diagnosis, are confronted with the need to prescribe drugs that are simply too expensive in relation to the patients' resources. The Orphan Drug Act of 1983 has been a powerful stimulus to the development of new drugs for rare diseases, but the incentives provided by the Act have not been reflected in price structure: as a result, for a majority of patients these diseases have remained orphans. Indeed, inequalities in access have become both greater and more glaring: however, in some cases, there have been initiatives aiming to counter this injustice. The Global Fund, founded in 2002 with the Bill and Melinda Gates being the first and main donors, spends US$4 billion a year, largely in Africa, with the specific objective to defeat HIV, tuberculosis (TB), and malaria. Here we make a call for three concrete steps that would correct at least some of the inequalities in the area of hematology. 1. Adding Sickle Cell Disease (SCD) to the triad of conditions for which the cost of basic treatment is borne by the Global Fund. Under such a plan hydroxyurea (HU) should be made available free of charge to all patients with SCD. This call is similar to one addressed recently to the President's Emergency Plan for AIDS Relief (PEPFAR: focused until now on HIV-AIDS); and since a multi-center trial has provided evidence that HU is highly beneficial in Africa there have been initiatives to provide HU, but they have been local and limited The original rationale whereby the Global Fund decided to target the triad of HIV, TB and malaria was that, because they are transmissible diseases, they could be eliminated; however, we seem to be still some way from elimination. SCD is also transmissible, through inheritance rather than through a micro-organism: and malaria has been the selective agent responsible for the high frequency of SCD in Africa. As a public health problem, SCD is at least as serious as the other three diseases and, given its long-term clinical course, the burden in Africa may be even greater, as highlighted by WHO/Afro (see https://www.afro. who.int/news/african-health-ministers-launch-drive-curb-sickle-celldisease-toll). 2. BMT solidarity program: for every BMT (HSCT) procedure in Europe/US, 0.1% of the expense is to be deposited into a fund to support BMT in accredited centers in Africa. This is under the guise of a tithe. Since the cost of a BMT procedure in an LMIC is about one-tenth of that in high-income countries, a tithe of 0.1% would fund 1 BMT in Africa for every 100 BMT procedures in Europe/North America. Since in Europe alone there are more than 30 000 per year, this approach would fund at least 300 BMT procedures in Africa (probably more than the present infrastructure can support). A Network of African BMT Centers could be responsible for accreditation; the European Bone Marrow Transplantation group and the American Society of Hematology could help organizing and administering this plan. 3. Rare Disease treatment matching program: for every 10 patients treated with a super-expensive drug (e.g., eculizumab) reimbursed by a National Health Service or by Health Insurance, the manufacturer is to offer the drug at a symbolic price to one patient with the same disease in Africa. The rationale for this proposal has been stated previously. In essence, the pharmaceutical industry has benefited greatly from the Orphan Drug legislation: it seems reasonable that they undertake a coordinated effort to show generosity in return. The challenges associated with health care in Africa are a major and very demanding responsibility of the Governments of African countries in the first place. We do not have the presumption to offer a recipe: we merely propose a practical approach to relieving at least some of the inequalities, at least in hematology.