The Powerful and Evolving Role ofPatient Advocacy Groups in Orphan Drug Development
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Transcript of The Powerful and Evolving Role ofPatient Advocacy Groups in Orphan Drug Development
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The Powerful and Evolving Role ofPatient Advocacy Groups in Orphan Drug Development
CEO, Connexion Healthcare Member, Board of Directors, Global Genes
Susan Stein, M.P.H. Juliet Moritz, M.P.H. Executive Director, Strategic Drug Development –
Rare Diseases, Premier Research
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Background
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Historical Barriers to Orphan Drug Research
Limited funding Lack of researcher interest
Few patient groups / Limited patient involvement
High cost of development vs expected return on
investment
Only 34 drugs developed for orphan
diseases between 1973 and 1983 in the
United States
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US and EU Orphan Drug Legislation
USA: 1983Orphan Drug Act
EU: 2000EC No. 141/2000
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POLLING QUESTION # 1
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FDA Orphan Drug Act (1983)
http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/OrphanDrugAct/
Original Purpose of
Orphan Drug Act
Goals of Orphan Drug
Act Legislation
To provide incentives for the development of therapeutics for the treatment of rare diseases that would normally be unprofitable or unpatentable
Stimulate investment in orphan drug development by sponsors
Benefit rare disease patient community
Speed time to approval for rare disease therapeutics
Sponsors qualify for reduced regulatory fees
Sponsors qualify for tax credit of up to 50% of clinical development costs
Sponsors qualify for seven years of market exclusivity in the approved indication
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EMA Orphan Drug Designation
Protocol Assistance
Fee Reduction
Market Exclusivity
for 10 years
Community Marketing
Authorization
+ 2 additional years for pediatric indication
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Orphan Drug Legislation: Rest of World
Japan, 1985 Singapore, 1991 Australia, 1998
South Korea, 1998 Taiwan, 2000
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FDA Orphan Drug Designation Requests Since 1983
http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2016/02/orphan-drug-approvals-dipped-in-2015-while-designations-and-designation-requests-continue-upward-tre.html
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FDA Orphan Drug Approvals Since 1983
http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2016/02/orphan-drug-approvals-dipped-in-2015-while-designations-and-designation-requests-continue-upward-tre.html
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12Kakkis EveryLife Foundation (www.everylifefoundation.org)
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FDA and Patient Engagement
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FDA and Patient Engagement
1988 20121996
1991 2001FDA begins working with HIV/AIDS patient advocates
First patient advocate recruited into FDA Patient Representative Program
Patient Representatives become voting members on advisory committees
Role of Patient Representative expanded to provide input on early development of medicinal products
FDA Patient Network created with over 200 patient representatives participating
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FDA Safety and Innovation Act of 2012
The Federal Drug Administration Safety and Innovation Act (FDASIA) was signed into law 09 July 2012. Among other components of the law it included specific language requiring that the FDA develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions, including:
Fostering participation of a patient representative who may serve as a special government employee in appropriate Agency meetings with medical product sponsors and investigators; and,
Exploring means to provide for identification of Patient Representatives who do not have any, or have minimal, financial interest in the medical products industry
U.S. Public Law 112-144
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FDASIA Patient Participation in Medical Product DiscussionsReport on Stakeholder Views
Food and Drug Administration Safety and Innovation Act (FDASIA)Section 1137: Patient Participation in Medical Product DiscussionsReport on Stakeholder Views
5Major themes
identified
Clarification ofFDA Policies
Systematic PatientEngagement
Transparency and Communication
ClinicalInvestigations
Workshops andPartnerships
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Stakeholder Report Summary
FDA should have a prominent and centralized office to advise the FDA Commissioner on patient engagement activities and systematically implement a coordinated function
Works across all divisions
Expand the role of the Office of Health and Constituent’s Affairs to better establish internal and external patient engagement processes
FDA should establish an external advisory group to provide ongoing counsel about input to and monitoring of patient participation in regulatory processes and policy development
The advisory body could offer perspectives on how to foster greater inclusion of patient engagement in regulatory decision-making for product review, post-market requirements, direct-to-consumer promotion, risk communication, and safety surveillance
Patient engagement should begin early and be iterative throughout the development process, with patient’s identifying their most urgent needs to inform clinical trial design
Systematic PatientEngagement
Food and Drug Administration Safety and Innovation Act (FDASIA)Section 1137: Patient Participation in Medical Product Discussions Report on Stakeholder Views
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Stakeholder Report Summary
FDA to report regularly summarizing patient participation in the Patient Representative Program
FDA should improve transparency on how patient input is evaluated and incorporated into the Agency’s decision-making process for medical products
Recommended FDA develop a patient data collection tool that systematically organizes issues by disease area and identifies topics to facilitate communications between patient organizations
FDA should continue to develop multiple modes of communication styles and tools. Some examples were webinars, round tables and tele-town hall meetings to facilitate cross talk among researchers, clinicians, industry and patients
FDA should enhance its patient portal allowing greater two-way communication to hear from patients about their experiences with FDA-regulated products, particularly products such as biosimilars
Transparency and Communication
Food and Drug Administration Safety and Innovation Act (FDASIA)Section 1137: Patient Participation in Medical Product Discussions Report on Stakeholder Views
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Stakeholder Report Summary
FDA develop guidance on interactions between patients and manufacturers to facilitate collaboration in the early stages of research and development and across the lifecycle of product development, clinical trial design, endpoint selection, and patient reported outcomes
FDA issue guidance describing the appropriate parameters and regulatory/legal safe-harbor for sponsor engagement with patient groups during medical product development
Work towards developing methodologies and study protocols for obtaining patients views of their conditions and benefit-risk assessment. An overarching goal would be to develop a standardized, repeatable, and representative pre-competitive data collection model that could be used across FDA divisions
FDA increase public-private partnerships (PPP) and establish, provide or collaborate via forums among patients, industry, clinicians, the scientific community and FDA
Clarification of FDA Policies
Workshops and Partnerships
Food and Drug Administration Safety and Innovation Act (FDASIA)Section 1137: Patient Participation in Medical Product Discussions Report on Stakeholder Views
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Stakeholder Report Summary
FDA to expand use and adoption of measures of patient-centered patient-reported outcomes (PROs) such as physical function and quality of life, much like what the European Medicines Agency has done in its Patient-Reported Outcome (PRO) qualification process
FDA collaborate with patient organizations to determine methods for conducting shorter, hypothesis-driven, novel trials that evaluate biomarkers as surrogate endpoints to more expeditiously develop effective therapeutics
Food and Drug Administration Safety and Innovation Act (FDASIA)Section 1137: Patient Participation in Medical Product Discussions Report on Stakeholder Views
Clinical Investigations
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An Example of Collaboration
Development of this guidance was preceded by the submission to FDA of a proposed draft guidance independently prepared by a consortium of stakeholders including patients, parents and caregivers, clinicians, scientific experts, and industry representatives.
The proposed draft guidance submitted by the consortium was made available through a Federal Register notice seeking public comment. Both the independently prepared proposed draft guidance and the public comments received in response to the Federal Register notice were considered in writing this guidance.
Rare Disease Drug Development Guidance
file:///C:/Users/JMoritz/Documents/Rare%20Disease%20General/FDA%20DMD.pdf
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Pending US Legislation Impacting Rare Diseases
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21st Century Cures Act Title II Senate Bill 1597
TITLE II - PATIENT-FOCUSED DRUG DEVELOPMENT PATIENT-FOCUSED IMPACT ASSESSMENT ACT
Title II of the legislation seeks to build on FDA's recent launch of a Patient-Focused Drug Development (PFDD) program, which was launched in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), is meant to incorporate patient preferences into FDA's regulatory decision-making.
The bill would require FDA to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.
This bill amends the Federal Food, Drug, and Cosmetic Act to require the package of information published by the Food and Drug Administration (FDA) upon approval of a new drug to include documentation of efforts to assess patient engagement. This documentation must include identification of patient-focused drug development tools and an explanation of whether certain information was reviewed or examined, including patient preferences and patient-reported or caregiver-reported outcomes. The FDA must annually summarize the data collected in this documentation.
The FDA must publish guidance on collaboration between patients, advocacy organizations, and industry for the purposes of developing patient-focused drug development tools and obtaining patient perspectives on medical products under development.
Passed as part of 21st Century Cures Act (H.R. 6) July 2015
Reported from committee 05 April 2016
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EMA and Patient Engagement
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EMA Commitment to Patient Engagement
1995 20062003
1996 2005EMA created
Dialogue with HIV patients
Working group with patients created
Framework of interaction with patient and consumer organizations
Patients and Customers Working Party created
2014
Dedicated Patients and Healthcare Professionals Department created
Revised frameworkOngoing…
FDA Patient Network created with over 200 patient representatives participating
http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000317.jsp&
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EMA Patient Engagement
Patients and consumers are involved in a wide range of activities at the Agency, including:
As members of the Management Board
As members of scientific committees
Being consulted on disease-specific requests by the scientific committees and working parties
Taking part in discussions on the development and authorization of medicines
Reviewing written information on medicines prepared by the Agency
Being involved in the preparation of guidelines
Taking part in the Agency's conferences and workshops
http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000317.jsp&
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EMA and Patient Engagement
The EMA engages with a network of over thirty-five eligible organizations, ensuring that the needs and concerns of a wide range of patients and consumers are represented via direct contact with the Agency. These include:
Umbrella organizations encompassing a number of smaller or national organizations (e.g., EURODIS)
Organizations with a focus on a specific area (e.g., FABRY International Network)
Eligible organizations receive targeted EMA communications and consultations and frequently assist in the identification of experts for product-specific matters.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/q_and_a/q_and_a_detail_000082.jsp
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”““
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Rare Disease Research Considerations
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Orphan Product Development Challenges
Small PopulationHow to find patients?
Geographically Widespread
How to access patients?
Research Naïve SitesHow to guarantee
data integrity?
Unknown/Sparse Natural History
What controls to use?
Lack of Defined Biomarkers
How to measure activity?
Lack of Surrogate Endpoints
How to define success?
Obstacles to Patient Retention
How to get all the data?
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”
“The Importance of Natural History Studies in Rare Disease
…Because of the small numbers of patients affected, and with clinical experience dispersed among a small number of clinical referral centers, the natural history of rare diseases is often poorly described.
…FDA does not require that natural history studies be conducted, but when knowledge about the disease is insufficient to guide clinical development, a well-designed natural history study may help in designing an efficient drug development program.
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Patient Registry ≠ Natural History Study ≠ Randomized Clinical Trial
Patient Registries: An organized system for the collection, storage, retrieval, analysis, and dissemination of information on individual persons who have either a particular disease, a condition (a risk factor) that predisposes them to the occurrence of a health-related event, or prior exposure to substances known or suspected to cause adverse health effects.
Can include purpose-driven collection of demographic, epidemiological, clinical-effectiveness, cost-effectiveness, quality of care, quality of life, and care pattern data.
Patient Registries
Natural History Studies
Natural History Studies
Supporting Regulatory
Submissions
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Patient Registry ≠ Natural History Study ≠ Randomized Clinical TrialNatural History Study: Documents the natural course of a disease from time immediately prior to its inception, progressing through its presymptomatic phase and different clinical stages to the point where it has ended and the patient is either cured, chronically disabled, or dead without external intervention.
Natural History Study Supporting Regulatory Submissions: A Natural History Study that is designed to meet the current regulatory requirements to be included in a regulatory submission. These can be critically important in rare disease where populations are small and the inclusion of a control or placebo population is more likely to be limited.
Patient Registries
Natural History Studies
Natural History Studies
Supporting Regulatory
Submissions
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Working Together:Patient Advocacy Organizations
and Industry
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Goals of Industry – Patient Advocacy Group Collaboration
Conquest of the disease
Streamlined research policies and procedures
Minimizing time to approval while maintaining high research standards
Ethical conduct of research
Maximizing patient care, quality of life, and survivorship
Reasonable risk/benefit assessments
Input
Data
Access Involvement
Partnership
Respect
Goals
Industry
Patient Advocacy Groups
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Industry Needs from Patient Advocacy Groups
Meaningful outcome measures
Assessment selection
Inclusion/Exclusion criteria validation
Burden minimization strategies
Recruitment strategies
Messaging theme and content
Qualified patients
Key opinion leaders
Appropriate sites
Referral networks
Awareness outlets
Organization-specific
Disease-specific
Social media
Prescreened patients who pre-identify as interested in clinical research trials
Natural history data
Assessment training and utilization standards
AccessInput Data
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Patient Advocacy Groups Needs from Industry
Understanding of clinical development plan
Involvement in protocol design
Clarification of regulatory interactions and impact
Burden minimization strategies
Awareness campaigns
Patient registries
Key opinion leader relationships
Support of advocacy group relationship with community
Message development
Transparent communications
Support for additional community assistance (e.g., natural history study)
Co-develop tools for research and awareness
Understand how advocacy group is structured and resourcing levels
Natural history data
Compassion and true commitment to community
Adhere to ethical principles of interaction with advocacy groups
PartnershipInvolvement Respect
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Patient Advocacy Organizations and Rare Disease Research
What do we bring? What do we already do?
Trust from our communities
Connection to our communities
Like-minded goals with pharma
Patient centric
Scientific insight into disease state
Insight into “real-life” experience with disease management issues
Fundraising for research
Academic/Translational
Therapeutic
Lobby legislative bodies to support to research, i.e., financial, legislative, regulatory
Educate patients about the importance of clinical trials
Serving on advisory boards for government groups
Adding the patient voice for clinical trial design and protocol development
Patient registries
Improve scientific knowledge and clinical trial issues to effectively communicate
Support key research issues such as trial recruitment and study awareness
Recognize the business of research and engage researchers in meaningful ways, including reporting back to funders
Put aside personal agendas to work as effectively as possible to advance research
Stay focused on core mission and meet commitments
How can we improve?
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“
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POLLING QUESTION # 2
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European Federation of Pharmaceutical Industries & Associations
“The pharmaceutical industry recognizes that it has many common interests with patient
organizations, which represent and/or support the needs of patients and/or
caregivers.“
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EFPIA Code of Practice
The EFPIA Patient Organization Code covers relationships between EFPIA corporate members including their subsidiaries and contracted third parties (e.g. agencies) and patient organizations which operate in Europe.
Patient organizations are defined as not-for-profit organizations (including the umbrella organizations to which they belong), mainly composed of patients and/or caregivers, that represent and/or support the needs of patients and/or caregivers.
Many multinational companies adhere to these guidelines
Good starting point for non-international companies as well
Model agreement template available
Annex I: Model template for written agreements between the pharmaceutical industry and patient organizations
When pharmaceutical companies provide financial support, significant indirect support and/or significant non-financial support to patient organizations, they must have in place a written agreement.
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EFPIA Code Overview
The independence of patient organizations, in terms of their political judgement, policies and activities, shall be assured.
All partnerships between patient organizations and the pharmaceutical industry shall be based on mutual respect, with the views and decisions of each partner having equal value.
The pharmaceutical industry shall not request, nor shall patient organizations undertake, the promotion of a particular prescription-only medicine.
The objectives and scope of any partnership shall be transparent. Financial and nonfinancial support provided by the pharmaceutical industry shall always be clearly acknowledged.
The pharmaceutical industry welcomes broad funding of patient organizations from multiple sources.
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Patient Advocacy Organizations - Looking Ahead
Future Trends in for Patient Advocacy Organizations
Increased infrastructure and mission sophistication
Work to provide meaningful improvement in availability, transparency and affordability of healthcare for patients
Data pooling and other collaboration with larger organizations
Collectively seek funding
Avoid overlaps and duplication
Increased Patient Centricity
Keeping patients at the heart of drug development
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Summary
Rare disease therapeutic research has increased significantly since the introduction of Orphan Drug legislation around the world
Legislative and regulatory changes will enhance the role of patient engagement and representation in the review and approval processes for therapeutics to treat rare diseases
Industry and Patient Advocacy Organizations are positioned to collaborate with each other to meet these increasing engagement expectations
Patient Advocacy Organizations have much to offer Industry in terms of the design and implementation of rare disease clinical research
Industry and Patient Advocacy Organizations should engage in ongoing dialogue to ensure that they are both working to maintain mutually beneficial and ethically sound relationships
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Questions?
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