Agenda

22-24 July 2019

ABOUT
This interactive presentation will provide you with an opportunity to learn more about how can manufacturers meet global patient demand for access to medicines prior to approval. This will also discuss some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market. R&D strategies to be implemented to accelerate market access for orphan drugs will also be explore. And how can patient, governments and orphan drug companies help support each other’s initiative to provide market access to patients?

WHO’S IT FOR
This workshop will be extremely beneficial to those individuals involve in development, commercialization and market access of orphan drugs. Project Managers and Executives in Pharma/Biotech companies, consultancy, Patient Advocacy, interested in knowing how to successfully launch an orphan drug.

AGENDA
9:00 – 10.30am - Interactive presentation
10:30 – 11:00am - Networking break
11:00 – 12:30pm - Interactive presentation and Discussion

Lunch Break: 12:30 - 13:30

ABOUT
This interactive presentation will provide you with an opportunity to learn more about how EAP’s provide pre -launch access for drugs in advance of their Marketing Authorization (MA) for patients with life-threatening conditions and no approved treatment options. Best practices in EAP planning, Set-Up and management, and provide information on current regulatory changes.

WHO’S IT FOR
This workshop will be extremely beneficial to those individuals considering an EAP as part of their drug development strategy. Project Managers and Executives in Pharma/Biotech/Advocacy interested in knowing how to set-up an Early Access Program.

AGENDA
13:30 – 15:00pm - Interactive presentation
15:00 – 15:30pm - Networking break
15:30 – 17:00pm - Interactive presentation and Discussion

• What are the growing trends in Rare Disease treatment
• How can governments and orphan drug companies help support each other’s initiative to provide access to patients?
• Stakeholders collaboration to continue fulfilling unmet needs?
• What R&D strategies can be implemented to accelerate the market access for orphan drugs?
• Challenges in bringing orphan drugs to patients after approval
• Updates on the natural history studies
• Issues for evaluating and validating biomarkers as surrogate endpoints
• Flexibility on the non-clinical aspects of the evaluation of novel drug compounds
• Consideration of safety questions and added information on changes to drug substance or drug product manufacturing process
• What can be done by healthcare providers to be proactive in monitoring side effects that can put patient safety at risk and effectively communicate results to the developer?
• Why should patients’ information about their treatment experience be collected and how can this steer the evolution of orphan drugs?
• How social media plays a role for developers and patients to communicate and how companies can utilize social media to innovate and create new drugs for patients?
• How are advance therapy fundamentally changing the way rare diseases patients are treated
• How policy makers and regulators approach Advance Therapy Medicinal Products (ATMP)
• What are the new development in Advance Therapy Medicinal Product
• Incentives Supporting Development of ATMP
• Challenges faced when embracing a completely new model of healthcare
• Technology needed to develop and deliver targeted therapeutics
• How to design clinical trials for small patient group
• Making commercialization viable by tackling high cost
• Improving clinical development by new age clinical trial design and recruitment
• Overcoming challenges in manufacturing and supply chain
• Understanding diseases thru national history
• Addressing challenges for Rare diseases therapy development
• Current strategies to advance rare diseases therapy development
• Efficient and effective models for therapy development that are scalable and sustainable
• Current state of RNAi therapeutics development
• Overcoming challenges in developmental process of RNAi treatment
• Overcoming the challenges of delivering RNAi to new cell types
• Leveraging the potential of RNAi technology
• RNAi therapies in the pipeline
• Selection of the study population
• Selecting appropriate study design for research
• Statistical analyses, the use of biomarkers, primary endpoint definition and proving drug efficacy
• Pharmacovigilance considerations for characterisation and risk management
• Timely and adequate recruitment of eligible trial participants
• Development of efficient trial designs relevant to small populations to gain the most information from the available data
• Practical conditions to be considered when conducting a clinical trial for rare diseases
• Promoting international collaboration and advance rare diseases research worldwide
• Patients as Key Partners in Drug Development
• Role of Real World Evidence in Rare Disese
• Value of Registries in Rare Disease – pros and cons
• Understanding the collective voice of the patients and represent the patients’ interest
• Patient involvement is crucial for identifying the questions to ask and the outcomes to assess
• Patient involvement is essential for achieving true translational research
• The identification and proper handling of patient needs in clinical research will yield advantages in terms of clinical and economic benefits.
• Overview of current methods for their prevention, diagnosis, and treatment.
• Strengths and limitation of current development pathways for new drugs, medical devices, and biologics for rare diseases
• Special challenges that rare diseases create for research and product regulation
• Current public policies relevant to product development for rare diseases
• 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.
• Identifying barriers for access to orphan drugs
• Addressing the challenges associated with patient access to these medicines
• High cost associated with the medicines: Is paying for expensive treatment for a few patients sustainable?
• Means of ensuring access and affordability of Orphan drugs
• Establishing a reasonable price for orphan drugs
• Patient recruitment and retention for a successful clinical trial in rare diseases
• Innovations proven to streamline the conduct of clinical studies
• Preparing healthcare providers to know about the therapy and identify the right patients for your therapy?
• 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
• 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
• What are some of the strategies for stakeholders to come together to get orphan drugs to patients especially in emerging countries?
• How can stakeholders work together to promoted international collaborations in developing orphan drugs and make it more accessible to all patients?
• Using initiative to drive market access to products for rare diseases
• Challenges surrounding funding for orphan drugs specifically for HTA
• What manufacturers should consider after successfully overcoming the hurdles to achieve market access
• Key concerns payers have about an orphan drugs
• What Set of Criteria Should be Considered for Orphan Drugs to Reach Patients with Rare Diseases in a Shorter Period of Time?
• How can reimbursement agencies deal with the uncertainty on efficacy of drugs but yet allow drugs to be available to patients as soon as possible?
• Vital strategies in maximizing the chances of success
• How can manufacturers meet global patient demand for access to medicines prior to approval?
• How can governments and orphan drug companies help support each other’s initiative to provide market access to patients?
• What are some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market?
• What R&D strategies can be implemented to accelerate the market access for orphan drugs?
• 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.
• Successful example of value-based pricing in Rare Disease
• Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
• Implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.
• Legislative incentives to encourage development of orphan rare diseases therapies
• Impact of orphan diseases treatment on healthcare payment system

To Sponsor/Exhibit

To Speak

To Register

sponsor@paradigmglobalevents.com
Tel. +44 203 567 1321

jocelynr@paradigmglobalevents.com
Tel. +44 203 567 1321

booking@paradigmglobalevents.com
Tel. +44 203 567 1321