Day 2 Congress

• What are the key drivers and innovation that will steer the innovation and access of orphan drugs?
• Existing regulations and policies utilized by countries to enable access to orphan drugs
• Overcoming challenges in getting rare diseases’ patient access to orphan drugs
• Multi-disciplinary approach and patient centered support to improve accessibility and further advances which will impact the next generation aiding patients of the future.

Panelist:

  • Khrystal K. Davis, Patient Advocate & Writer, Hunt for a Cure, Author, Zebra Leaf Publishing
  • Anthony Gucciardo, SVP, Strategic Partnerships, National Kidney Foundation
  • Daniel Anderson, Head, Commercial Partnerships Invitae
  • Carl K Edwards, CSO, DNX Biopharmaceuticals

• Unifying stakeholders to improve access and health
outcomes
• Creating empowered patient advocates
• Texas Rare Alliance achievements in Texas
• Advocacy efforts going forward

Khrystal K. Davis, Patient Advocate & Writer, Hunt for a Cure, Author, Zebra Leaf Publishing

• The foundational value of building collaboration through trust, respect, transparency and empathy
• Not all advocacy organizations are alike: assessing the landscape
• Real world examples of innovative digital solutions driving collaboration among multiple stakeholders
• It isn’t easy so why do it? Innovative partnering yields unique benefits boosting capabilities, medicine development and commercialization

T Anthony Howell, Esq., Co-Founder & Digital Community Builder, rareLife solutions, Creators of onevoice.world

  • Identifying and evaluating research
  • Innovative funding
  • Academia vs Biotech
  • Grants vs Investment

Debra Miller, CEO, CureDuchenne

  • Patients as Key Partners in Drug Development
  • Role of Real-World Evidence in Rare Disease
  • Value of Registries in Rare Disease – pros and cons

Sonal Bhatia, Vice President, North America Medical Lead, Rare Disease, Pfizer*

  • How to improve knowledge and recognising a rare condition for patients to gain a sense of ease and trust
  • Addressing issues on practical challenges
  • Improving access and supporting patients

Reserve for Gold Spotlight Presentation

  • Patient recruitment: The need to go multinational - no country has all resources to pull it off in rare diseases. With ORDIUSA, we are building collaborative bridges between US and India to accelerate clinical research and sharing of best practices.
  • Taking clinical trials to patients’ homes - reducing burden of participation (This is a part of PFDD)
  • The end to end continuum or patient journey - awareness, education, diagnosis, treatment, research, clinical trials as standard of care.

Christine Von Raesfeld, Patient Advocate, More Than Lupus, Ambassador, CureClick
Harsha Karur Rajasimha, Co-Founder, Board Director, Organization for Rare Diseases India and USA

  • How can stakeholders work together to remove barriers to patient access?
  • How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
  • What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?
  • Effective collaboration contributing to faster and wider access of medicines to patients

Panelist:
Anthony Gucciardo, SVP, Strategic Partnerships, National Kidney Foundation
Angela Ramirez Holmes, Founder/President, Cal Rare

  • Payers are becoming more aggressive in scrutinizing price and implementing restrictions to patient access that have traditionally been reserved for competitive, high budget impact drug classes
  • Recent development in orphan drugs pricing and reimbursement and the impact of those changes
  • Addressing some of the major issues facing payers and orphan drug manufacturers, highlighting the way these have been handled by both parties.
  • Implementing measures designed to limit exposure to costs associated with orphan drug reimbursement.

Lauren Nelson, Account Director, US Market Access - Rare and Oncology, Sanofi Genzyme*

  • Patient registries from advocacy groups are not always your best source for trials and surveys - The BEST source actual patients who are involved and are self-advocates
  • Patient Centricity is claimed but not often achieved by CRO’s and they may not even know it. Find out why
  • Why patients are leery of registries and most avoid them
  • The rules of double-blind studies leave patients in the “cold”. There are strategies to keep them invested and Involved
  • You need to know what patients face in day-to-day challenges or you will lose them

Tom Lambdin, Co-Executive Director, Ben’s Friends Patient Communities

• Virtual Support, Authenticity and Safety at Ben's Friends which is an interactive network of over forty-seven online communities which provide emotional and social peer support for people whose lives have been touched by a rare disease.
• It is not a patient advocacy group but rather a peer to peer Support Group
• Building a successful interactive support community, with a positive culture, takes online tools, years of effort,and trial and error.
• Factors which have come together in creating a safe and supportive online environment for Ben's Friends members
• Infrastructure Ground rules Human investment and intervention
• Monitoring Network cohesion
• Be prepared to ask lots of questions!

Clasina Field, Co-Executive Director, Ben’s Friends Patient Communities

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