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When the Scottish government recently launched a £21 million fund to provide patients with rare diseases access to treatment, Alex Neill, the Scottish Health Secretary, stated that 'it was only right that Scottish patients with rare conditions had access to innovative medicines which were clinically justified, and that they were not disadvantaged due to the very high cost of these treatments'. This statement raises questions about the criteria that play a role in reimbursement decisions on orphan drugs. This editorial examines the criteria that were used in the decisions about the reimbursement of an orphan drug for Pompe disease and explores methods to improve the transparency and consistency of reimbursement decisions for orphan drugs in general.
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http://dx.doi.org/10.1586/14737167.2015.1045882 | DOI Listing |
Pharmacoeconomics
September 2025
Center for Innovation and Value Research, Alexandria, VA, USA.
Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.
View Article and Find Full Text PDFJ Pharm Pharmacol
September 2025
Facultad de Farmacia y Bioquímica, Departamento de Tecnología Farmacéutica, Cátedra de Tecnología Farmacéutica II, Universidad de Buenos Aires, C1113AAD Buenos Aires, Argentina.
Objectives: To develop the orphan drug benznidazole (BNZ) in orally disintegrating tablets, for the neglected disease American Trypanosomiasis (Chagas disease) therapy. Although children are highly affected by this disease, there are no specific commercial pharmaceutical preparations for this age group in Argentina and in many other countries.
Methods: In the production process, co-milling in a ball mill was applied to enhance dissolution rates, followed by direct compression.
Health Econ Policy Law
September 2025
Department of Health Policy, London School of Economics and Political Science, London, UK.
While a substantial amount of evidence exists on factors associated with positive health technology assessment (HTA) outcomes, the evidence on the same regarding rejections is scarce. Using a proprietary dataset of HTA outcomes in seven Organisation for Economic Co-operation and Development (OECD) countries, we empirically examine the factors associated with HTA rejections and study the magnitude of inter-agency differences in technology appraisals. Data were extracted from HTA reports between 2009 and 2020.
View Article and Find Full Text PDFChin Clin Oncol
August 2025
Newcastle University Centre for Cancer, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK; Newcastle Hospitals and NHS Trust, Newcastle upon Tyne, UK.
Background And Objective: Most of newly diagnosed prostate carcinomas (PCas) present as non-metastatic disease, with approximately 15% of them presenting with characteristics predicting for a high-risk of relapse. Hence, specific focus has to be placed on patients affected by localised or locally advanced disease, whose chances of cure are notably higher than those of patients in advanced settings. With androgen receptor pathway inhibitors (ARPIs) and docetaxel chemotherapy improving treatment efficacy outcomes when compared to traditional androgen deprivation therapy (ADT) in the metastatic disease, clinical trials are currently investigating the activity of ARPI for clinical management of localised or locally advanced prostate patients, with the aim of preventing the cancer from spreading systemically.
View Article and Find Full Text PDFDrug Discov Today
August 2025
European Medicines Agency, Amsterdam, the Netherlands.
Despite progress in rare disease treatment, many conditions still lack therapeutic options. In addition to specific legislation promoting research and investment, regulators have supported early dialogs with stakeholders, optimized processes and expedited the approval of medicines in areas with unmet medical needs, such as rare diseases. However, several challenges persist, particularly in generating robust evidence.
View Article and Find Full Text PDF