Mlyuka Hamu J, Kilonzi Manase, Mutagonda Ritah F, Chirande Lulu, Mikomangwa Wigilya P, Myemba David T, Sambayi Godfrey, Mwakawanga Dorkasi L, Ndunguru Joyce, Jonathan Agnes, Makani Julie, Ruggajo Paschal, Minja Irene K, Balandya Emmanuel, Kamuhabwa Appolinary A R
Department of Clinical Pharmacy and Pharmacology, School of Pharmacy, Muhimbili University of Health and Allied Sciences, Dar es Salaam P.O. Box 65013, Tanzania.
Sickle Pan African Research Consortium (SPARCO), Dar es Salaam P.O. Box 65001, Tanzania.
Healthcare (Basel). 2022 Nov 7;10(11):2223. doi: 10.3390/healthcare10112223.
Despite three decades of proven safety and effectiveness of hydroxyurea in modifying sickle cell disease (SCD), its accessibility is limited in Sub-Saharan Africa, which shares 75% of the world's SCD burden. Therefore, it is time to explore the barriers and facilitators for manufacturing and importation of hydroxyurea for SCD in Tanzania. This was qualitative research that employed a case study approach. Purposive sampling followed by an in-depth interview (IDI) using a semi-structured questionnaire aspired by data saturation enabled us to gather data from 10 participants. The study participants were people with more than three years of experience in pharmaceuticals importation, manufacturing, and regulation. The audio-recorded data were verbatim transcribed and analyzed using thematic analysis. Two themes were generated. The first comprised barriers for importation and manufacturing of hydroxyurea with sub-themes such as inadequate awareness of SCD and hydroxyurea, limited market, and investment viability. The second comprised opportunities for importation and manufacturing of hydroxyurea with sub-themes such as awareness of activities performed by medicines regulatory authority and basic knowledge on SCD and hydroxyurea. Inadequate understanding of SCD, hydroxyurea, and orphan drug regulation are major issues that aggravate the concern for limited market and investment viability. Existing opportunities are a starting point towards increasing the availability of hydroxyurea.
尽管羟基脲在改善镰状细胞病（SCD）方面已被证实具有三十年的安全性和有效性，但在撒哈拉以南非洲地区，其可及性仍然有限，而该地区承担着全球75%的镰状细胞病负担。因此，现在是时候探索坦桑尼亚生产和进口用于治疗镰状细胞病的羟基脲的障碍和促进因素了。这是一项采用案例研究方法的定性研究。通过目的抽样，然后使用半结构化问卷进行深入访谈（IDI），并以数据饱和为目标，使我们能够从10名参与者那里收集数据。研究参与者是在药品进口、生产和监管方面有三年以上经验的人员。对录音数据进行逐字转录，并使用主题分析进行分析。生成了两个主题。第一个主题包括羟基脲进口和生产的障碍，其子主题如对镰状细胞病和羟基脲的认识不足、市场有限以及投资可行性。第二个主题包括羟基脲进口和生产的机会，其子主题如对药品监管机构开展活动的认识以及对镰状细胞病和羟基脲的基本知识。对镰状细胞病、羟基脲和孤儿药监管的理解不足是加剧对市场有限和投资可行性担忧的主要问题。现有的机会是提高羟基脲可及性的起点。
