We contend that governments, intergovernmental agencies and non-governmental organizations should ensure that all patients have access to ‘orphan’ drugs for rare diseases and to personalized medicine services, not just those who can afford to pay.
Within the United States and the European Union, tax incentives accrue to companies that are engaged in orphan-drug development. Start-ups and large multinational companies, which tend to seek enhanced returns for investors, could soon emerge as specialist providers of medicines to those who can afford personalized health-care options. Less-affluent patients would then be at risk of being excluded from advances in molecular diagnostics and in genetic-modification strategies for disease avoidance.
Technologies often outpace preparations for their application in health care, including in such fields as regenerative medicine (P. Marks and S. Gottlieb N. Engl. J. Med. 378, 954–959; 2018). In this instance, too, we might have only a narrow window of opportunity to influence international cultures, regulations and investment to ensure equitable benefit for all.
Nature 559, 32 (2018)