Rare diseases have increasingly been discussed in the media over the past several years as the number of new treatments has introduced. Most of these diseases are life-threatening or life-limiting, and while some conditions can now be treated and managed as chronic conditions, for the first-time new curative treatments are bringing the prospect of a better life to patients. This report updates an analysis of the Orphan Drug market published by the Institute in October 2018, the FDA approved 80 orphan indications and in 2018, it approved 57 in the first eight months of the year. and provides a historical perspective on the characteristics of rare diseases, their treatments and the role of the Orphan Drug Act of 1983 in advancing rare disease medicines. It defines the characteristics of orphan drug expenditure, capacities and prices, placing orphan drugs in the context of specialty drug trends and overall medicine spending levels and growth. The combination of scientific advances, along with a growing commitment by policy makers to advance precision medicine, is fueling the increased number of orphan therapies.

The field of rare disease products is continuously growing, expecting to reach $180 billion by 2020, with a CGR of 10.5% just for orphan drugs – this is twice the growth rate of the overall prescription drug market. Explanations for this include the arising interest for pharmaceutical companies, primarily for underlying benefits, including the potential discovery of mechanisms for more common diseases, and the involvement of many smaller biotechnology startup companies who are more focused on Rare Diseases.

Following this, We are proud to announce our Rare Diseases & Orphan Drugs 2019 conference, aiming  to bring together solution providers, biotechnology companies, clinical researchers, Orphan Drug & Rare Diseases Organizations, regulatory professionals and charity leaders together to discuss possible ways to accelerative orphan drug development and access to rare disease patients, including the introduction of recent technologies and products to help aid the access of orphan drugs.

Over View
We are proud to present our Orphan Drugs and Rare Diseases 2019 Conference in Rome. This conference will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs & Rare Diseases.
In European Union countries, any disease affecting fewer than 1 in 2,000 people is considered rare. That number may seem small, but it translates into around 246 000 people throughout the EU’s 28 member countries. Most patients suffer from even rarer diseases affecting 1 person in 1 Lakh  or more.
The congress will provide a unique platform for the convergence of investors with the orphan medicines business to discuss and network with prime level government, hospitals, pharmaceuticals, biopharmaceuticals, non-profit organizations, orphan medicines developers as well as regional and local manufacturers. We are putting together a program that address the driving political economy factors, policies and issues that will steer the development of orphan drugs globally including commercialization, policies, reimbursement, pricing and more.
The two-day Congress will provide a collaborative, comprehensive discussion and networking format led by the experts with intimate knowledge in the industry. Gain sensible methods and best practices on challenges, innovations, technologies and concepts surrounding orphan medicines and rare diseases.
We look forward to welcoming you to the conference this October.
Learning Objectives

1. Discuss current and evolving imaging options for Advanced therapies, treatments and monitoring rare Diseases.
2. Incorporate current guidelines and evolving evidence on Orphan Drugs, including new approaches in the development of Orphan medicines, into the management of patients with rare diseases.
3. Integrate biomarkers, molecular assays, and other risk assessment tools into treatment decision-making algorithms to optimize outcomes for patients with rare diseases.
4. Review rare diseases biology, staging, and predictive markers, in order to better individualize therapy and counsel patients regarding conventional and investigational treatment options.
5. Evaluate emerging clinical data and evolving strategies for the treatment for rare diseases, and apply clinical trial data appropriately in order to optimize the outcome of individual patients.
6. Utilize supportive care strategies for preventing and managing treatment-related toxicities in patients receiving treatment for rare diseases.
7. Facilitate awareness of clinical trial opportunities based on knowledge of emerging agents and variances in care in the field of rare diseases and orphan drug devolvement management.