Call for Abstract
Scientific Program
3rd Annual Congress on Rare Diseases and Orphan Drugs, will be organized around the theme “Platform to share new treatments and research for Rare Diseases”
Rare Diseases 2017 is comprised of 13 tracks and 52 sessions designed to offer comprehensive sessions that address current issues in Rare Diseases 2017.
Submit your abstract to any of the mentioned tracks. All related abstracts are accepted.
Register now for the conference by choosing an appropriate package suitable to you.
Rare Disease is technically defined as a disease that is found in fewer than 5 people per every 10,000 people. While we’ve all heard of diseases, seen someone with a disease, and had a disease personally, it’s unlikely that we’ve encountered a rare disease. In the non-medical world, people use and interchange disease to mean infection, sickness, illness, or something similar. In the medical world, a disease is an abnormal condition that impairs bodily functions and is often associated with certain signs and symptoms. Also called an orphan disease, most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear.
- Track 1-1Alkaptonuria
- Track 1-2Rare Infectious Diseases
- Track 1-3Zellweger syndrome
- Track 1-4Progeria
- Track 1-5Niemann-Pick disease
- Track 1-6Marfan syndrome
- Track 1-7Krabbe leukodystrophy
- Track 1-8Joubert syndrome
- Track 1-9Huntington disease
- Track 1-10Gaucher disease
- Track 1-11Fabry disease
- Track 1-12Duchenne muscular dystrophy
- Track 1-13Dravet syndrome
- Track 1-14Charcot-Marie-Tooth disease
- Track 1-15Barth syndrome
- Track 1-16Ataxia-telangiectasia
- Track 1-17Alexander disease
- Track 1-18Hypophosphatasia
Clinical research is a branch of healthcare science. The first step in tackling this challenge is regularly getting the public to think about participating in clinical research. People need to consider how they can help advance the prevention, diagnosis, and treatment of disease. It is never too early to consider participation whether or not someone ultimately chooses to join a study.
- Track 2-1Rare Disease and Orphan Drug/Medicinal Product
- Track 2-2Orphan Drug Status Various Probabilities
- Track 2-3Challenges in Access to and Affordability of Medicines for Rare Diseases
- Track 2-4Regulation of drugs and biologics for rare diseases
- Track 2-5Opportunities to accelerate discovery research
- Track 2-6Development of new drugs and biologics for rare diseases
Diagnostic error in medicine is common. For example, a study from an intensive care unit demonstrated nearly 20% discordance between the clinically-defined cause of death and findings at post-mortem examination. Not surprisingly; therefore, the diagnosis of rare diseases is often delayed. Rare diseases affect an estimated 2-4 percent of the population. These diseases often have a genetic basis, either as uncommon recessive conditions or as the result of “de novo” genetic mutations not present in either parent. They can take varied and often debilitating form.
- Track 3-1Nonspecific symptoms
- Track 3-2Methodological Issues
- Track 3-3Research funding gaps
- Track 3-4Small patient pool
- Track 3-5Rarity of the condition or disease
There are approximately 7,000 rare diseases, which from a regulatory perspective are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than five in 10,000 of the general population in the EU. "Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.
- Track 4-1Different types of rare diseases
- Track 4-2Misperceptions about rare diseases
- Track 4-3Role of Patient Registries
- Track 4-4Regulatory Approaches and Alternative Methodologies
- Track 4-5Rising Patient Costs
- Track 4-6Reimbursement Strategies
Worldwide, about one-third of human deaths are attributable to infections. In addition, the so-called non-infectious causes of death often have a cryptic infectious etiology. Many Rare diseases caused by infectious agents rather than genetic or environmental factors.
Rare cancers tend to be caused by simple genetic mutations, and common cancers tend to be caused by a complex set of genetic and epigenetic aberrations that continually increase in number as the tumor develops. All cancers that can be cured when in an advanced clinical stage are rare cancers. Rare cancer syndromes typically produce various types of cancers, including rare cancers and common cancers.
Aging is a collection of degenerative changes that occur in organisms that lack the ability to perpetually regenerate. Age is a major risk factor for most common neurodegenerative diseases. Dementia becomes more common with age. The spectrum includes mild cognitive impairment, Alzheimer's disease, cerebrovascular disease, Parkinson's disease and Lou Gehrig's disease. Rare diseases provide much insight into the cellular processes that hasten the aging process.
The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will lose their exclusivity in next 5 years. Therefore, the current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies from the essential medicines to the new business model — niche busters, also called orphan drugs.
- Track 8-1Cost of drug development
- Track 8-2Evidence-based policy making & commissioning
- Track 8-3Orphan drug status- Various Probabilities
- Track 8-4Challenges in assessing clinical relevance and cost effectiveness
- Track 8-5Developments in Regulating Orphan Drug Approval
A clinical trial is a medical study conducted to test the effects of a new or already existing drug, of a biological treatment or of a medical device that might treat or curb a disease already identified. The main goal of a clinical trial is to compare two or several groups of subjects, by using two or several treatments in order to determine the efficacy of a drug or of a biological treatment.Clinical trials are carefully and ethically conducted in order to protect patients against unwanted adverse reactions and to allow collection and accurate analysis of the information concerning the disease.
- Track 9-1Orphan Drug Act
- Track 9-2Clinical Development Challenges
- Track 9-3Current Legislation/Regulations
This track outlines the moral dilemma of funding orphan drug research and development. To date, ethical aspects of priority setting for research funding have not been an issue of discussion in the bioethics debate. Conflicting moral obligations of beneficence and distributive justice appear to demand very different levels of funding for orphan drug research. The two types of orphan disease, rare diseases and tropical diseases, however, present very different ethical challenges to questions about allocation of research funds. The dilemma is analysed considering utilitarian and rights based theories of justice and moral obligations of non-abandonment and a professional obligation to advance medical science. The limitations of standard economic evaluation tools and other priority setting tools used to inform health policy decision makers on research funding decisions are outlined.
- Track 10-1Orphan Medicinal Products Regulation
- Track 10-2Economic considerations
- Track 10-3Public Private Partnerships
- Track 10-4Advance Market Commitments
Work over the past 25 years has resulted in the identification of genes responsible for ~50% of the estimated 7,000 rare monogenic diseases and it is predicted that most of the remaining disease-causing genes will be identified by the year 2020 and probably sooner. This marked acceleration is the result of dramatic improvements in DNA-sequencing technologies and the associated analyses. We examine the rapid maturation of rare-disease genetic analysis and successful strategies for gene identification. We highlight the impact of discovering rare-disease-causing genes, from clinical diagnostics to insights gained into biological mechanisms and common diseases. Last, we explore the increasing therapeutic opportunities and challenges that the resulting expansion of the 'atlas' of human genetic pathology will bring.
- Track 11-1Early detection of rare diseases
- Track 11-2Genome or Exome Sequencing
- Track 11-3Precision Medicine
- Track 11-4Future detection of rare genetic diseases
- Track 11-5Genome Sequencing- Revolutionary Breakthrough for Rare Diseases
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