scholarly journals Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

2017 ◽  
Vol Volume 9 ◽  
pp. 327-342 ◽  
Author(s):  
Elizabeth Adkins ◽  
Lindsay Nicholson ◽  
David Floyd ◽  
Mark Ratcliffe ◽  
Helene Chevrou-Severac
Keyword(s):  
2013 ◽  
Author(s):  
Stephen Tueller ◽  
Laurie Cluff ◽  
Deborah Galvin ◽  
Heather Healy ◽  
Georgia Karuntzos ◽  
...  

2018 ◽  
Vol 18 (18) ◽  
pp. 1550-1558
Author(s):  
Muhammad Aamir ◽  
Asma Sadaf ◽  
Sehroon Khan ◽  
Shagufta Perveen ◽  
Afsar Khan

Background: Many of the tropical diseases are neglected by the researchers and medicinal companies due to lack of profit and other interests. The Drugs for Neglected Diseases initiative (DNDi) is established to overcome the problems associated with these neglected diseases. According to a report published by the WHO, leprosy (Hansen's disease) is also a neglected infectious disease. Methods: A negligible amount of advancements has been made in last few decades which includes the tools of diagnosis, causes, treatment, and genetic studies of the bacterium (Mycobacterium leprae) that causes leprosy. The diagnosis of leprosy at earlier stages is important for its effective treatment. Recent studies on vitamin D and its receptors make leprosy diagnosis easier at earlier stages. Skin biopsies and qPCR are the other tools to identify the disease at its initial stages. Results: Until now a specific drug for the treatment of leprosy is not available, therefore, Multi-Drug Therapy (MDT) is used, which is hazardous to health. Besides Mycobacterium leprae, recently a new bacterium Mycobacterium lepromatosis was also identified as a cause of leprosy. During the last few years the genetic studies of Mycobacterium leprae, the role of vitamin D and vitamin D receptors (VDR), and the skin biopsies made the treatment and diagnosis of leprosy easier at early stages. The studies of micro RNAs (miRNAs) made it easy to differentiate leprosy from other diseases especially from tuberculosis. Conclusion: Leprosy can be distinguished from sarcoidosis by quantitative study of reticulin fibers present in skin. The treatment used until now for leprosy is multi-drug treatment. The complete genome identification of Mycobacterium leprae makes the research easy to develop target specified drugs for leprosy. Rifampicin, identified as a potent drug, along with other drugs in uniform multi-drug treatment, has a significant effect when given to leprosy patients at initial stages. These are effective treatments but a specific drug for leprosy is still needed to be identified. The current review highlights the use of modern methods for the identification of leprosy at its earlier stages and the effective use of drugs alone as well as in combination.


2020 ◽  
Vol 20 (11) ◽  
pp. 1288-1299
Author(s):  
Paromita Kundu ◽  
Deepika Singh ◽  
Abhalaxmi Singh ◽  
Sanjeeb K. Sahoo

The panorama of cancer treatment has taken a considerable leap over the last decade with the advancement in the upcoming novel therapies combined with modern diagnostics. Nanotheranostics is an emerging science that holds tremendous potential as a contrivance by integrating therapy and imaging in a single probe for cancer diagnosis and treatment thus offering the advantage like tumor-specific drug delivery and at the same time reduced side effects to normal tissues. The recent surge in nanomedicine research has also paved the way for multimodal theranostic nanoprobe towards personalized therapy through interaction with a specific biological system. This review presents an overview of the nano theranostics approach in cancer management and a series of different nanomaterials used in theranostics and the possible challenges with future directions.


2020 ◽  
Vol 10 ◽  
Author(s):  
Rupali Singh ◽  
Rishabha Malviya

Background: The chronotherapy concept attains considerable focus towards itself due to its pulsatile fashion rather than continuous delivery. The delivery of the right amount of drug to the target organ at the most appropriate time is fulfilled by using the chronotherapeutic dosage form. Aim: The present study aims to develop and evaluate a chronotherapeutic drug delivery system by using natural polymer for time specific drug delivery at the target site. Material and Method: Tamarind seed polysaccharide was extracted and used in the preparation of core tablets. Nine formulations of core tablets were prepared with nifedipine at 5 tonnes of pressure on 6 mm punch. The core tablets were prepared by using the compression coating method. The three batches F1, F2 and F3 were prepared by using tamarind gum in different concentration i.e. 45%, 22.5% and 67.5% respectively and compressed at 8 tonnes of pressure on 12 mm of punch. The finally compressed tablet was coated with different concentrations of ethyl cellulose in which isopropyl alcohol used as a solvent. In a controlled medium, a stability study was performed to evaluate the physical appearance, drug content and release of the prepared core tablet. Result: All the nine formulations of tablets were prepared successfully and the evaluation studies (thickness, weight variation, hardness, friability etc.) revealed that all the formulations were within the official range. The release study of the drug revealed that the formulation F7 containing 67.5% of tamarind polymer, coated with 2%, 4% and 5% of ethyl cellulose solution released 59.68±1.03% (Q50%) drug within 5 h whereas, 87.09±2.08% (Q80%) within 6 h and within 12 h 97.74±2.19% of the drug was released. The formulation F7 was found to be more effective as it released the maximum amount of drug in a short period as compared with other formulations. Conclusion: The coating of core tablets allowed to prepare pharmaceutical dosage form for time specific drug delivery. These chronotherapeutic core tablets can be used for the treatment of angina pectoris and hypertension etc.


Small ◽  
2021 ◽  
pp. 2100753
Author(s):  
Ya‐Xuan Zhu ◽  
Hao‐Ran Jia ◽  
Yuxin Guo ◽  
Xiaoyang Liu ◽  
Ningxuan Zhou ◽  
...  

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