World Congress on Rare Diseases and Orphan Drugs

Giulio Tarro

Market Analysis

The Rare Diseases are distributed in such a w ay that forty of the cases accounted by some 350 Rare Diseases. About only 5% of rare diseases are hav ing approv ed drug treatment w ith only 326 new drugs being approv ed by the FDA and brought in to the market. Rare diseases are mostly often debilitating or ev en the life-threatening diseases or in some case the conditions w hich prev alence of 0.65%-1%, cause of rare diseases is mostly genetic in disorder and sometimes it may be as a result of infections or degenerativ e causes.

In 2019, Orphan Drug sales w ere the order of 93 billion. Orphan Drugs mostly represented by 35% of the industry ’s for new drug approv als. The genetic disorders are diagnosed by the area, Genetic w hich are going to lead the global market in the past w ill show similar traction in the nex t coming eight y ears. According to Statistics of MRC, the Global market of Orphan Drug is to estimate by $145.89 million in 2018 and ex pected to reach $265.63 million by 2022 grow ing at a CAGR of 10.5% from 2018 to 2022.

The worldwide market for rare diseases w ill possibly improv e up at a consistent pace of 6.5% from 2017 to 2025, at this pace; the market is anticipated from US$17,318.0 Mn in 2016 to US$30,314.5 MN by the end of 2025.Estimation of the report is done by Transparency Market Research.

Rare Diseases usage increases w ith an increase in the w ide spread of diseases and w ith mounting diseased population. Rare Diseases globally used for applications influencing research discov ery of new drugs by academic personalities, disease diagnosis in clinical and healthcare centers.

Diseases Diagnosis w hich is recorded to be $11.9 billion in the y ear 2013 is about to reach $13.1 billion by 2019 w ith a dev eloping CAGR of 1.6% betw een 2014 and 2019. Drug discov ery is relied upon to reach $85.8 billion by 2022 from $54.7 billion by 2017 w ith a dev elopment in CAGR up to 9.4% from 2017 to 2022

The global Rare Diseases market is driv en by ease of transportation of slides, surge in prev alence of chronic diseases & aging population, and increase in the laboratory w orkflow efficiency . How ev er, there is a restriction in the market grow th due to lack of reimbursement and dearth of skilled personnel. Furthermore, unmet pathological needs in the emerging economies are ex pected to prov ide numerous opportunities for the market grow th during the forecast period.

Key Benefits

• Along w ith current trends and future estimations, an indepth analy sis of the market has been studied to elucidate the imminent inv estment pockets

• To enable the stakeholders to capitalize on the prev ailing market opportunities, It offers a quantitativ e analy sis of the industry from 2016 to 2023 to enable the stakeholders to capitalize on the prev ailing market opportunities.

• The use of pathological product in detecting cause, origin, and nature of diseases can be elucidated by Ex tensiv e analy sis of product

• To determine the prev ailing opportunities, Comprehensiv e analy sis of all geographical regions is prov ided

• To understand the competitiv e outlook of the global market, Key play ers are profiled and their strategies hav e been analy sed thoroughly .

Rare Diseases Market Overview

The global rare diseases market generated $17.4 billion rev enue in 2018 and is projected to adv ance at a CAGR of 5.8% during the forecast period, mainly on account of increasing occurrence of chronic diseases, rising geriatric population, and adv ancements

Rare Diseases Market Dynamics

Betw een 5,000 and 8,000 distinct rare diseases ex isted, affecting betw een 6% and 8% of the population in total – in other w ords, betw een 27 million and 36 million people in the EU. Most of the people suffer from rare diseases affecting few er than 1 in 100,000 people.80% of rare diseases hav e identified genetic origins, and affect betw een 3% and 4% of births. Rare diseases causes due to degenerativ e and proliferativ e. Sy mptoms of some rare diseases may appear at birth or in childhood, including spinal muscular atrophy , ly sosomes storage disorders, patent duct us arteriosus (PDA), familial adenomatous poly posis (FAP) and cy stic fibrosis. Most of the rare diseases appear during adulthood, such as renal-cell carcinoma and acute my eloid leukemia.

Rare Diseases and Orphan Drugs Market Competitive Landscape

On-going acquisitions and collaborations among play ers in the Rare Diseases and Orphan Drugs market are resulting in accurate diagnosis and increased market reach and share of the play ers. For instance, in December 2018, NeoGenomics Laboratories Inc. (NeoGenomics), a leading prov ider of cancer-focused genetic testing and information serv ices, acquired a U.Sbased Genoptix Inc., a clinical oncology laboratory , specialized in haematology and solid tumour testing. Also, in February 2019, Quest Diagnostics Incorporated, a U.S.-based prov ider of diagnostic information serv ices, partnered w ith Houston Healthcare, a community hospital based in the U.S., to enhance the quality and v alue of diagnostic serv ices through an agreement under w hich Quest Diagnostics Incorporated w ill prov ide full laboratory management serv ices. The accretion is ex pected to ex pand NeoGenomics’ reach into oncology practices, and significantly accelerate the company ’s progress tow ard key scale and grow th objectives.