Rare Collective

MME and LapidusData to Lead Workshop at WODC
2019-04-10 13:00 - 17:00 UTC | Gaylord National Harbor Hotel, 201 Waterfront St, National Harbor, MD, 20745, United States
On Wednesday, April 10, 2019, MME and LapidusData will host a workshop on Predicting and Influencing the Market Potential of Orphan Drugs and Gene Therapies as part of the World Orphan Drug Congress.
Market forecasting presents special challenges for orphan drugs and gene therapies. This workshop will provide real-world examples of how epidemiology, pricing, and early clinical decisions can inform your forecast, and more importantly, how you can influence these factors to achieve commercial success. Our unparalleled experience with rare diseases allows us to share cases of winning—and losing—strategies for market sizing, pricing/reimbursement, trial design, and even product formulation. Topics will include:

Market potential: Defining the treatable population
Key drivers: Assessing prevalence, diagnosis rates, and disease severity. What resources can provide reliable information, and how can you generate new data when faced with unknowns?
Predicting performance: Leveraging historical product analogs, prioritizing multiple indications, and understanding the value of early product formulation
Market share: How to think about unmet need and competition for orphan drugs
Price: How to assess and capture the value offered by your product. What novel reimbursement mechanisms are viable?
Q&A

The World Orphan Drug Congress will place April 10–12, 2019 at the Gaylord National Harbor Hotel in Oxon Hill, MD.

Engage Health to Appear at the World Orphan Drug Congress USA 2019
2019-04-10 09:00 - 12:00 | Gaylord National Harbor Hotel, 201 Waterfront St, National Harbor, MD, 20745, United States
On Wednesday, April 10, 2019, Engage Health will host a workshop on Identifying and Engaging Patients and Clinicians in the Rare Disease Space as part of the World Orphan Drug Congress. Anyone in the rare disease space who has ever needed to find patients with rare diseases or the clinicians who care for them, will benefit from attending!
A complete description of the workshop is below. In order to attend our workshop, you must purchase the three day pass (conference and workshop day) when you register for the World Orphan Drug Congress.
Identifying and Engaging Patients and Clinicians in the Rare Disease Space
Has the experience of searching for patients with rare diseases, or for the health care providers who care for them, caused you to feel like you were looking for the proverbial needle in a haystack? If so, then this workshop is for you!
Advancing therapies for rare diseases requires a strong community comprised of patients, patient organizations, pharmaceutical companies and health care providers. Introducing new patients to the rare disease community is challenging. To begin with, the path to diagnosis is difficult, somethings taking as long as 9 years, and often involving an average of 7.3 specialists1. Once diagnosed, some families join a patient organization. Others become advocates at the state and national level. Some turn their pain into action by founding patient organizations for diseases that have none. Patients and their families who engage in these activities become part of the small but mighty rare disease community.
When a new therapy or a clinical trial is available, patients who are already part of the rare disease community are relatively easy to reach. But what about the ones who do not know there is a rare disease community? What about those who know but do not seek out community involvement? What about those who are lost to expert follow up? Many patients and their families obtain a diagnosis, are told there is nothing to be done, and are sent home to be cared for by their local health care provider. How do we find these patients? How do we find the health care providers who are treating them? How do we reach them with potentially life-saving or life-improving therapies before it is too late?
Hosted by Engage Health, Inc., this three-hour workshop will introduce new tools to engage patients and clinicians at every stage of drug development. Together we can find those needles in that haystack and speed the development of life-improving and life-saving therapies. Together we can make a difference to the patients and families impacted by rare diseases.
1 Engel PA, Bagal S, Broback M, Boice N. PHYSICIAN AND PATIENT PERCEPTIONS REGARDING PHYSICIAN TRAINING IN RARE DISEASES : THE NEED FOR STRONGER EDUCATIONAL INITIATIVES FOR PHYSICIANS. J Rare Disord. 2013;1(2):1-15. http://www.journalofraredisorders.com/pub/IssuePDFs/Engel.pdf. Accessed December 13, 2018.
For additional information about the workshop click HERE. You may also contact Engage Health at 651-994-0510.
The World Orphan Drug Congress will place April 10–12, 2019 at the Gaylord National Harbor Hotel in Oxon Hill, MD.

Global Genes 2018 RARE Patient Advocacy Summit
2018-10-03 06:30 - 2018-10-04 22:00 | Hotel Irvine, 17900 Jamboree Road, Irvine, CA, 92614, United States
The RARE Patient Advocacy Summit is the can’t-miss event of the year for rare disease stakeholders. The Summit will take place October 3-4 at the Hotel Irvine in Irvine, California, and is the largest gathering of rare disease patients, advocates and thought leaders worldwide.
Take advantage of this opportunity to connect and learn from more than 100 experts in rare disease leading educational sessions in four specialized learning tracks:

Living with a Life-Altering Condition
Architecting Your Disease Community
Patients as Partners and Drivers
Science and Technology Innovation

The RARE Patient Advocacy Summit offers many unique opportunities to support the rare community by participating in the largest educational event for rare patients and advocates. Join Global Genes, partners and rare disease community members from all walks of life to become equipped with the tools needed to persevere and thrive on our paths forward!

World Orphan Drug Congress USA
2017-04-19 08:00 - 2017-04-21 17:00 | Washington, D.C., WA, United States

Genetic RX Boston
2016-12-08 07:00 - 18:30 | Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston, MA, 02115, United States
Genetic Rx will take place at Harvard Medical School and will bring together academics, key industry leaders and investors to discuss the emergence of new genetic medicines as well as the treatment of patients with rare and ultra-rare genetic diseases.

World Orphan Drug Congress Europe
2016-11-15 08:00 - 2016-11-17 17:00 | Sheraton Brussels Airport Hotel, 1930 Brussels, Brussels, Belgium
The World Orphan Drug Congress is entering into its 7th year in November 2016.
The Congress was one of the first events to bring together the full range of stakeholders and influencers in the orphan drug and rare disease sector, from the pharma and biotechs to patient advocates and representatives from payers.
It continues to be a key meeting for the sector in pushing forward with strategy, advocacy and partnering within the rare disease space.
Building on a greatly successful event in 2015, we will be putting together a hard-hitting agenda in 2016.
We will continue to cover the issues that affect the full audience, including pricing and reimbursement, commercialisation, and improving on collaboration, as well as researching the newest areas to ensure the focussed content really reflects the future of the sector.
We will also be working hard to increase the number of representatives present from the HTA, payer and public health arenas, as it is so important to have their input throughout the discussions onsite. Additionally, we will be inviting more members of the patient advocacy community.
We believe these improvements to the programme will fulfil our aim to see 450+ attendees in 2016.

World Congress on Patient Advocacy
2016-10-27 08:00 - 2016-10-28 17:00 | Hyatt Arlington, 1325 Wilson Blvd, Arlington, VA, 22209, United States
Pharmaceutical and biotech companies all believe in representing the patient voice within the company.
The challenge is how to make patient centricity an actuality within your organization. The paradigm shift in the healthcare industry to have a greater focus on the patient voice has created a need for industry organizations to ramp up their patient advocacy efforts to more successfully engage and empower what is most important; the patient. This summit brings together manufacturer and advocacy professionals to discuss how to most effectively engage the patient in drug development, how to create successful partnerships, and discusses strategies and results from successful case examples.
Top Reasons To Attend:

2015 Attendance Pie Chart
Discuss innovative tactics for involving patients from the initial stages of drug development through study launch
Discover how to translate a volunteer network into your organization’s stakeholder groups
Understand the impact of ePatient communities on clinical trial participation
Determine how to more effectively capture the patient voice in policy
Learn how to work in partnership with global patient advocates
Show how to clarify your role to internal stakeholders when building an advocacy function
Demonstrate how data transparency can help build trust and confidence in patients participating in clinical trials
Identify how to work in partnership with global patient advocates

NORD Rare Summit
2016-10-17 - 2016-10-18 | Hyatt Regency Crystal City, 2799 Jefferson Davis Hwy, Arlington, VA, United States
Rare Diseases and Orphan Products Breakthrough Summit
NORD’s Rare Diseases and Orphan Products Breakthrough Summit is the largest and most meaningful multi-stakeholder event of its kind – historically featuring over 20 speakers from the FDA, participation from over 80 patient organizations and the Pharma/Biotech industry’s foremost experts in orphan product innovation, investment and commercialization.
The 2016 Breakthrough Summit is your opportunity to collaborate with the top leaders from the FDA, NIH, industry, patient groups, payers and research institutions to address the progress and innovations in rare disease diagnosis, treatment, patient engagement and market access of orphan products.
New for 2016
NORD’s Conference networking app helps you and your colleagues see who’s attending and schedule one-on-one meetings in advance. Plus, roundtable discussions during Lunch & Learn sessions create opportunities for robust, intimate discussions.

Rare Disease Market Access and Orphan Drug Commercialization
2016-09-29 - 2016-09-30 | Hyatt Regency Boston, 1 Ave de Lafayette, Boston, MA, 02111, United States
Join market access and commercialization executives from pharmaceutical and biotech companies with a focus on rare, genetic, and orphan diseases to explore the current commercial dynamics surrounding these unique products. This Summit evaluates market forces, their economic impact, and the implications for orphan drug market access – covering early stage commercialization efforts through pricing, reimbursement, and patient and market access considerations.
Top Reasons to Attend:

Learn strategies to enhance your patient-centered approach to product commercialization
Discuss lessons learned between industry and advocacy partnerships
Understand key pricing models and how to develop a cost effectiveness model for your product
Gain insight from payers on how they manage orphan drug pricing and reimbursement and their perceptions on value in relation to price
Dive into the mechanics of lobbying in the rare disease space
Determine how to choose the right distribution model for market success

Global Genes 5th Annual RARE Patient Advocacy Summit
2016-09-22 07:00 - 2016-09-23 17:00 | Hyatt Regency Huntington Beach Resort and Spa, 21500 Pacific Coast Highway, Huntington Beach, CA, 92648, United States
A rare diagnosis changes everything. You weren’t given a playbook on how to cope, how to take that next step, and you certainly weren’t handed a blueprint on how to build an advocacy organization and successfully bring a therapy to market. The good news is that rare disease advocates are some of the most inspiring, innovative activists on the planet, and Global Genes is works to bring the community together to share best practices, create important introductions, and help catalyze powerful collaborations.
This is why Global Genes’ would like to invite you to join The Fifth Annual RARE Patient Advocacy Summit on September 22 – 23, 2016 in Huntington Beach, California! The goal of this year’s RARE Patient Advocacy Summit is for patients, caregivers, and advocates to walk away equipped with actionable next steps whether you have been recently diagnosed, are building a disease community, thinking about funding early research, actively engaged in developing a treatment, or have been advocating in rare disease for decades.
Here’s what you’ll learn:
Practical next steps for taking action in the areas of research, legislation, fundraising, and community support. Core fundamentals and skills to help you start, grow and strengthen your nonprofit organization. Strategies for building online communities and why they are essential for rare disease awareness. Understanding the power of genetic data and patient involvement for advancing research for your disease. Tools and insights into crafting successful collaborations with researchers, biotech, pharma and the FDA. Invaluable connections with advocacy leaders that will help you define and propel your rare disease priorities forward. Be prepared – the Summit will educate you and will exhaust you, but. . . it will also inspire you.
