International experience in drug assessment for rare diseases: flexibility of approaches
Relevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to allocate resources to the development of drug...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Article |
| Language: | Russian |
| Published: |
Publishing House OKI
2024-09-01
|
| Series: | Реальная клиническая практика: данные и доказательства |
| Subjects: | |
| Online Access: | https://www.myrwd.ru/jour/article/view/67 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849697468579577856 |
|---|---|
| author | K. S. Radaeva A. S. Kolbin |
| author_facet | K. S. Radaeva A. S. Kolbin |
| author_sort | K. S. Radaeva |
| collection | DOAJ |
| description | Relevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to allocate resources to the development of drugs for diseases that might otherwise be overlooked due to limited commercial appeal. Experts from Cogentia Healthcare Consulting Ltd. analyzed trends in the assessment of orphan drugs in the UK.Objective. The aim of this work was to analyze and present the main trends in the optimization of decision-making regarding orphan drugs based on the data of an analysis conducted by the experts of Cogentia Healthcare Consulting Ltd.Results. Regulatory bodies have shown some flexibility in the assessment of orphan drugs and have used approaches designed to address uncertainties in the evidence, accepting, in some cases, low-quality evidence, expert opinion and evidence from real-world clinical practice and applying more flexible pharmacoeconomic approaches. However, despite the existing assumptions, the evaluation process for orphan drugs often takes longer than that for non-orphan drugs.Conclusions. Although different approaches to market regulation for orphan drugs have helped stimulate their development worldwide and address unmet medical needs, delays in decision-making for orphan drugs have seriously affected their availability to patients in need. |
| format | Article |
| id | doaj-art-ab1ace02f8e94248adb3b9d338d717c5 |
| institution | DOAJ |
| issn | 2782-3784 |
| language | Russian |
| publishDate | 2024-09-01 |
| publisher | Publishing House OKI |
| record_format | Article |
| series | Реальная клиническая практика: данные и доказательства |
| spelling | doaj-art-ab1ace02f8e94248adb3b9d338d717c52025-08-20T03:19:12ZrusPublishing House OKIРеальная клиническая практика: данные и доказательства2782-37842024-09-014231210.37489/2782-3784-myrwd-5256International experience in drug assessment for rare diseases: flexibility of approachesK. S. Radaeva0A. S. Kolbin1First St. Petersburg State Medical University named after I. P. PavlovFirst St. Petersburg State Medical University named after I. P. PavlovRelevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to allocate resources to the development of drugs for diseases that might otherwise be overlooked due to limited commercial appeal. Experts from Cogentia Healthcare Consulting Ltd. analyzed trends in the assessment of orphan drugs in the UK.Objective. The aim of this work was to analyze and present the main trends in the optimization of decision-making regarding orphan drugs based on the data of an analysis conducted by the experts of Cogentia Healthcare Consulting Ltd.Results. Regulatory bodies have shown some flexibility in the assessment of orphan drugs and have used approaches designed to address uncertainties in the evidence, accepting, in some cases, low-quality evidence, expert opinion and evidence from real-world clinical practice and applying more flexible pharmacoeconomic approaches. However, despite the existing assumptions, the evaluation process for orphan drugs often takes longer than that for non-orphan drugs.Conclusions. Although different approaches to market regulation for orphan drugs have helped stimulate their development worldwide and address unmet medical needs, delays in decision-making for orphan drugs have seriously affected their availability to patients in need.https://www.myrwd.ru/jour/article/view/67orphan drugsrare diseasesdecision makingrecommendationsflexibilityclinical pharmacology |
| spellingShingle | K. S. Radaeva A. S. Kolbin International experience in drug assessment for rare diseases: flexibility of approaches Реальная клиническая практика: данные и доказательства orphan drugs rare diseases decision making recommendations flexibility clinical pharmacology |
| title | International experience in drug assessment for rare diseases: flexibility of approaches |
| title_full | International experience in drug assessment for rare diseases: flexibility of approaches |
| title_fullStr | International experience in drug assessment for rare diseases: flexibility of approaches |
| title_full_unstemmed | International experience in drug assessment for rare diseases: flexibility of approaches |
| title_short | International experience in drug assessment for rare diseases: flexibility of approaches |
| title_sort | international experience in drug assessment for rare diseases flexibility of approaches |
| topic | orphan drugs rare diseases decision making recommendations flexibility clinical pharmacology |
| url | https://www.myrwd.ru/jour/article/view/67 |
| work_keys_str_mv | AT ksradaeva internationalexperienceindrugassessmentforrarediseasesflexibilityofapproaches AT askolbin internationalexperienceindrugassessmentforrarediseasesflexibilityofapproaches |