Global Summit on Emerging Orphan Drugs and Drug Abuse

About Conference

Session/Tracks

Currently the orphan drug spending in the United States is nearly $15.9 billion in 2013 and over $30 billion in 2016, an increase from 4.8 percent of total pharmaceutical spending to 8.9 percent. With the exponential growing market of orphan drugs and successful past conferences in Europe and USA we are organizing the Global Summit on Emerging Orphan Drugs and Drug Abuse during August 23-24, 2017 at San Francisco, USA.

Orphan Drugs 2017 will comprehensively discuss latest developments and market trends in orphan drugs and rare diseases over the following tracks:

Track 1: Market Forecasts for Orphan Drugs

With 30 million Americans suffering from 7000 rare diseases the market for Orphan drugs is growing exponentially. Moreover as per the defined laws orphan drugs enjoy a 50% tax credit on on R&D costs with $30m grants for each fiscal year to carry out Phase I to Phase III clinical trials. The rise of orphan drug numbers in USA is by 12% registering 291 numbers of orphan drugs. However, the growth is far more accelerated in Europe with an unbelievable rise by 62% registering 201 orphan drugs. Globally orphan drugs sales forecast expects a $178bn market share with over 11.7% market share and 20.2% worldwide prescription share by 2020.

Related Conference:

3rd Annual Congress on Rare Diseases and Orphan Drugs, October 30-November 1, 2017 San Antonio, USA; World Orphan Drug Congress USA, April 19-21, 2017, Washington DC, USA; Drug Discovery 2017, March 27–28 2017, London, United Kingdom; International Conference on Rare Diseases & Orphan Drugs, October 19-22, 2017, Cape Town, South Africa; European Biopharma Congress, November 16-17, 2017 Vienna, Austria

European Organisation for Rare Diseases, European Union Committee of Experts on Rare Diseases, International Rare Disease Research Consortium, The National Organization for Rare Disorders (NORD), Asia-Pacific Alliance of Rare Disease Organisations (APARDO)

Track 2: Orphan Drugs Pricing and Reimbursement

Pricing and reimbursement of orphan drugs is of utmost importance to policy makers, health care professionals, legislators, industry leaders, payers, pharma analytical market and patients. The pricing are led by factors like market exclusivity, fewer alternative health technologies, third-party payers, huge R&D costs need to be recouped from relatively less number of patients. There lies urgency for transparent and evidence-based approach towards orphan drugs pricing and reimbursements. The approach is expected to encompass relative effectiveness, cost-effectiveness, cost structure, economic viability of orphan drugs with an intention towards informing pricing and reimbursement decisions.

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Track 3: Development Strategies of Orphan Drugs

With blockbuster drugs going off patents the revenue loss to be incurred by many pharma companies is expected to be huge. Orphan Drugs are owned to be a worthwhile substitute for them to minimize the impact of revenue loss. Incentives awarded by FDA and EU commission are an added motivation and boost towards developing orphan drugs. Of late there has been noticed an increased development of orphan drugs by the pharma companies and gaining marketing approvals with market exclusivity.

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Track 4: Types of Rare Diseases

Rare diseases are such diseases or disorders which have been reported in less than 5 people per 10,000 people. Rare disease can be classified based on Arthrogryposis, Rare cancers‎, Cystic fibrosis‎, Rare infectious diseases, Intersex and medicine, Mesothelioma‎, People with caudal regression syndrome‎, People with tetra-amelia syndrome‎, Progeroid syndromes‎, Supernumerary body parts‎, Tay–Sachs disease‎. Rare Diseases are often manifested by anatomical or physiological deformities in the patients.

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Track 5: Challenges in Rare Disease Research

With strict rules implemented for rare diseases it is very difficult to engage in rare disease research on a subject without the assistance of a clinician and also restricting the clinical trials confined so as to have a hope of filling them. Mostly there is a scarcity of funds for research on rare conditions of the disorders added with a small market scope and lack of existing literature and experienced professionals are some major challenges.

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Track 6: Orphan Drugs Approvals and Regulatory Affairs

Orphan drugs not withstanding for lesser degree of liability for safety, efficacy and potency, FDA is willingly accepting to be flexible for their research and development approach not being stringent as in other cases. Since the patient disease characteristics are really very unique the stringency is done away with to benefit the patient outcomes. The regulatory obstacles are often tackled with strategic planning for patient recruitments and through the identification of outcome measures that reflect efficacious variations in disease manifestations. Orphan Drugs usually keep to the same developmental paths as other pharmaceutical products where focus lies on testing PK/PD factors, dosing, stability, safety, efficacy and potency. However, certain relaxations are provided in terms of statistical documentations like clinical trial of drug candidate on 1000 subjects during phase III clinical trials. Pertaining to market scope limitations the government interventions are an added momentum towards developing orphan drugs. Orphan drugs regulations enjoy benefits as tax incentives, patent protections, market exclusivity, and clinical research subsidies.

With 354 drug candidates agreed to be awarded orphan drug status from among the 472 requests submitted, the FDA awarding of rose by 22% from 2014. From 1983 with the Orphan Drug Act coming into effect the FDA has received around 5,100 requests for orphan drug designation, responding to it issued 3,600 designations were given and finally approved around 550 drugs as orphan drugs.

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Track 7: Clinical Studies with Orphan drugs

Biotechs and CROs that work in these rare diseases and orphan indications use resources that are typically highly educated and have the mental flexibility to use creative thought processes. Not only do they conduct extensive research on diseases that show a similar pathophysiology and response, but they also must be able to assess each situation on a case-by-case basis. The ability to deal with all kinds of diverse illnesses is perhaps more important than experience in the particular indication. Patient recruitment factors are by far the most important during conducting of clinical trials on rare diseases with orphan drugs.

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Track 8: Bio-ethics for Orphan Drugs

International research ethics mainly focusses on protecting individual participants from potential harm during the research procedures. Ethical issues of priority setting for research funding are mostly not been an issue of discussion in the bioethics debate. The conflicting moral obligations of beneficence and distributive justice apparently demand very different levels of funding for orphan drug research. Both types of orphan diseases—rare diseases and tropical diseases—pose different ethical challenges to questions of allocating research funds. For either of them the conflict between principles of distributive justice based on utilitarian or legal rights and principles of beneficence based on social or moral obligations is at the forefront. However, a more detailed analysis reveals that very different moral issues are at stake in each case.

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Track 9: Gaming and abuse of Orphan Drugs

Drug companies often demonstrate an "increasingly common pattern of gaming the system" by slicing and dicing indications so that drugs qualify for lucrative orphan status benefits and "getting taxpayer subsidies for blockbuster drugs, says a veteran scientist in USA. Companies are intentionally exploiting the law by putting up an initial listing of only single narrow indication for an orphan drug's use, which is just enough to qualify for "orphan" drug designation. Under the terms of this act, companies can receive federal taxpayer subsidies of up to half a million dollars a year for up to four years per drug, large tax credits and waivers of marketing application fees that can cost more than $2 million. In addition, the Food and Drug Administration (FDA) can grant companies seven years of marketing exclusivity for an orphan drug to ensure they recoup the costs of research and development. When those financial incentives become sort of an investment opportunity to take advantage of the Orphan Drug Act. The review found that orphan drug spending in the United States totaled $15 billion in 2007 and $30 billion in 2013, an increase from 4.8 percent of total pharmaceutical spending to 8.9 percent. The current study projects orphan drug spending will remain fairly stable as a proportion of total drug spending. That stands in contrast to other published reports that estimate orphan drugs will account for 20 percent of worldwide spending on drugs (other than generics) by 2020.

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Past Conference Report

Rare Diseases 2016

Rare Diseases-2016: Past Conference Report

With the overwhelming success of the previous conference, Conference Series LLC hosted Annual Congress on Rare Diseases and Orphan Drugs during October 26-27, 2016 at Chicago, USA (Rare Diseases-2016). The conference was marked with the attendance of Editorial Board Members of supported Conference Series LLC journals, scientists, young and brilliant researchers, business delegates and talented student communities representing more than 20 countries, who made this conference fruitful and productive. This conference was based on the theme, “The voice of rare diseases - research and treatment” which included the following scientific tracks:

· Different Types of Rare Diseases

· Clinical Research and Public Awareness

· Mystery Diagnosis of Rare Diseases

· Challenges in Rare Diseases Treatment

· Rare Diseases in Cancer

· Rare Diseases in Aging

· Rare Infectious Diseases and Immune Deficiencies

· Clinical Immunology

· Orphan Drugs, Development Trends and Strategies

· Orphan Drugs and Ethical Issues

· Future Hereditary and Rare Diseases e Prospects of Rare Diseases

· Entrepreneurs Investment Meet

We are thankful to our below Honorable Guests for their generous support and suggestions:

· Philippe Jouvet, Sainte-Justine Hospital University of Montreal, Canada

· Stephen Shrewsbury, Fortuna Fix, USA

· Thomas C Chen, Keck School of Medicine of USC, USA

The conference proceedings were carried out through various scientific-sessions and plenary lectures, of which the following topics were highlighted as Keynote presentations:

· Title: The rare diseases clinical research network (RDCRN) program (a model for collaborative research) and resources at NCATS, NIH

Rashmi Gopal-Srivastava, NCATS, NIH, USA

· Title: Inside track to an orphan designation

Timothy Cote, Cote Orphan, USA

· Title: How patient organizations can change the game in innovation and drug marketing: Example of AFMTelethon

Serge Braun, AFMTelethon, France

Conference Sessions Chair and Co-Chair:

· Amrik Sahota, Rutgers University, USA

· Lisa Baumbach-Reardon, Translational Genomics Research Institute, USA

· Alan B Moy, The John Paul II Medical Research Institute and Cellular Engineering Technologies, USA

Conference Series LLC has taken the privilege of felicitating Rare Diseases-2016 Organizing Committee, Editorial Board Members and Keynote Speakers who supported for the success of this event. The esteemed guests, keynote speakers, well-known researchers and delegates shared their innovative research and vast experience through their fabulous presentations at the podium of grand Rare Diseases-2016. We are glad to inform that all accepted abstracts for the conference have been published in Conference Series LLC Journal of Genetic Syndromes & Gene Therapy as a special issue.

We are also obliged to various delegate experts, company representatives and other eminent personalities who supported the conference by facilitating active discussion forums. We sincerely thank the Organizing Committee Members for their gracious presence, support and assistance. With the unique feedback from the event, Conference Series LLC would like to announce the commencement of the "3rd Annual Congress on Rare Diseases and Orphan Drugs" during October 30-November 1, 2017 in San Antonio, USA.

Conference Highlights

Special Issues

All accepted abstracts will be published in respective Journals.

Abstracts will be provided with Digital Object Identifier by CrossRef

Global Summit on Emerging Orphan Drugs and Drug Abuse

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Friday, 18 November 2016

Orphan Drugs 2017

About Conference

Session/Tracks

Past Conference Report

Past Conference Report

Conference Highlights

Special Issues