Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?

Abstract

BackgroundMore than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs.MethodsStudy data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of orphan drugs.ResultsIn the period 1983–2015, the FDA granted 3,647 orphan drug designations and 554 orphan drug approvals. The orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of orphan drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of orphan drugs.ConclusionOrphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved orphan drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients’ access to treatment. Thus, aligning pharmaceutical companies’ incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to orphan drugs need to be agreed upon.

Innovation and competition in advanced therapy medicinal products

“…Aligning the necessary incentives for research and development with health care budgetary constraints is an important challenge for policymakers and regulatory agencies (Rodriguez-Monguio et al, 2017). Aligning the necessary incentives for research and development with health care budgetary constraints is an important challenge for policymakers and regulatory agencies (Rodriguez-Monguio et al, 2017).…”

Öffentliche Lenkung und Preisbegrenzung für Orphan-Drugs

“…vs. UD$ 638 Mio., [11,24] Da die OD-Entwicklung ökonomi-schen Gesetzen folgt und noch keine öffentliche Lenkung erfährt, ergab sich eine ungleiche Verteilung auf das Spektrum der SK: Onkologie (32 %), Infektiologie und "human immunodeficiency virus" (HIV; 12 %), Hämophilie (6 %), Kleinwuchs (2 %), pulmonale Hypertonie (2 %), Transplantation (2 %) und Sonstige 44 % [32]. So gibt es für ein Drittel der mit OD behandelbaren SK mehr als eine OD und für fast 8000 keine.…”

Doenças Raras, Medicamentos Órfãos: reflexões sobre o Dark Side das organizações da indústria farmacêutica

“…A pessoa com doença rara precisa de um cuidado intenso de um ou mais membros da família, o qual terá menos tempo hábil para trabalhar (ANNEMANS et al, 2017). Tanto o paciente, quanto sua família enfrentam barreiras econômicas e de saúde no diagnóstico, na prevenção, no acesso a tratamentos, nos cuidados, nos recursos educacionais e sobre o entendimento do que é a doença rara na sociedade (RODRIGUEZ-MONGUIO et al, 2017).…”

“…De maneira geral, há uma dificuldade em diagnosticar as doenças raras precocemente e há poucas opções terapêuticas e pesquisas nesse campo (RODRIGUEZ-MONGUIO et al, 2017). Alguns sintomas podem ser desencadeados no nascimento ou durante a infância, como a atrofia muscular espinhal (AME), as desordens de armazenamento lisossômico, a persistência do canal arterial (PCA), a polipose adenomatosa familiar (PAF) e a fibrose cística (FC).…”

“…Os esforços em pesquisa e desenvolvimento (P&D) em doenças raras se voltam apenas para aquelas que afetam um grande número de pacientes, que resultam no desenvolvimento e na aprovação de medicamentos órfãos para melhorar a qualidade de vida e a saúde dos pacientes, porém, além de abranger uma pequena fração frente aos diferentes tipos de doenças raras existentes, o custo limita o acesso dos mesmos ao tratamento (RODRIGUEZ-MONGUIO et al, 2017). Os medicamentos órfãos têm um custo de desenvolvimento muito elevado, e a maioria deles não são aprovados, assim, as empresas farmacêuticas não têm disposição de gastar grandes recursos para a obtenção de uma baixa recompensa em troca, por isso os países, junto à Organizações Governamentais e Não-Governamentais, nacionais e internacionais, lutam em busca de incentivos para o desenvolvimento desses fármacos (MARAIS et al, 2012).…”