Investigating and Analysing Research,  Patent and Funding Landscapes of  Rare Diseases in the European Union and Beyond

Akinsolu, Folahanmi Tomiwa

Investigating and Analysing Research, Patent and Funding Landscapes of Rare Diseases in the European Union and Beyond

dc.contributor.advisor	Varga, Orsolya
dc.contributor.author	Akinsolu, Folahanmi Tomiwa
dc.contributor.authorvariant	Akinsolu, Folahanmi Tomiwa
dc.contributor.department	Egészségtudományok doktori iskola	hu
dc.date.accessioned	2019-05-25T10:31:50Z
dc.date.available	2019-05-25T10:31:50Z
dc.date.created	2019	hu_HU
dc.date.defended	2019-05-31
dc.description.abstract	The demand for health services is both growing and changing in nature globally. In spite of substantial contribution of knowledge and technology to health improvements, there are still noticeable disparities in life expectancy and disease burden. For quite a number of years, rare diseases and NTDs were hardly addressed by research, and inadequate investment in R&D needed to address specific health problems is a vital contributing factor. A rare disease or ‘orphan’ disease is defined as one that affects a restricted number of people. Rare diseases are sets of genetic and chronic conditions. NTDs have been defined as a group of infections strongly associated with poverty in tropical and subtropical environments. The goal of this study are to map out research activities of rare diseases and NTDs through a landscape analysis of Rett syndrome showing the magnitude of financial support from public and private organizations the EU, determining the trends of R&D on NTDs by performing a patent landscape analysis and identifying the trends of drug resistance for 11 major NTDs and 20 drugs. Rett syndrome with OMIM Entry 312750 is a severe neuro-developmental rare disease that affects approximately 1 in 10,000 live female births. In Rett syndrome study, it was discovered that funders’ research activity was not homogeneously distributed among member states. Most projects were performed in Italy and UK. The landscape study indicates that funders’ research activity is not homogeneously distributed among European member states. Patent landscape analysis provides insight into the innovations that underlie technology and products. This study shows a long term trend with a continuous growth in the number of patent families of NTDs. This continuous growth in trends is not uniform for all the NTDs. However, global patenting trend is in sharp contrast with our findings on NTDs. In the last 20 years, the total number of global patenting applications has tripled, but NTD patent application has not increased. The analysis of this study also showed that the US is losing its position as a major priority country. This is consistent with the fact that China now drives global patent applications beginning with a new record achieved in 2015. A higher percentage of firms are assignees resident in the US in the field of NTDs compared to China. However, in China, there is a high proportion of patent families linked to univeristies or individuals which indicates high research activity. R&D does not provide answers for several observed problems within the NTDs. It is imperative to pay attention to the broad social factors affecting NTDs; parallel improvements in hygiene, sanitation and access to medical care. Antimicrobial resistance is a global public health threat, and its impacts have the potential to kill millions of people. From identifying drug resistance trends in NTDs, it was discovered that only six NTDs have information on drug resistance. There was lack of data to determine the magnitude and scope of AMR in the other reviewed NTDs. It is crucial to foster national surveillance systems and harmonize global standards that estimate the extent of AMR globally. Understanding research trends and how funders contributed to different research directions is a pillar of research policy making. It is crucial to intensify R&D efforts into rare diseases. Involving new players, such as more NGOs may help to mitigate and reduce the burden of these diseases. Strengthening the health systems, political and global health efforts will be of immense benefits to facilitate R&D of these diseases.	hu_HU
dc.description.corrector	de
dc.format.extent	134	hu_HU
dc.identifier.uri	http://hdl.handle.net/2437/268207
dc.language.iso	en	hu_HU
dc.subject	Rare diseases	hu_HU
dc.subject	Neglected Tropical Diseases
dc.subject	Rett syndrome
dc.subject	Pantent
dc.subject	Funding
dc.subject	Resistance
dc.subject.discipline	Egészségtudományok	hu
dc.subject.sciencefield	Orvostudományok	hu
dc.title	Investigating and Analysing Research, Patent and Funding Landscapes of Rare Diseases in the European Union and Beyond	hu_HU
dc.title.translated	Investigating and Analysing Research, Patent and Funding Landscapes of Rare Diseases in the European Union and Beyond	hu_HU