The Powerful and Evolving Role ofPatient Advocacy Groups in Orphan Drug Development

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The Powerful and Evolving Role of
Patient 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

Background

Historical Barriers to Orphan Drug Research
4
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

US and EU Orphan Drug Legislation
5
USA: 1983
Orphan Drug Act
EU: 2000
EC No. 141/2000

POLLING QUESTION # 1
6

FDA Orphan Drug Act (1983)
7
http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/OrphanDrugAct/
 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

EMA Orphan Drug Designation
8
Protocol
Assistance
Fee
Reduction
Market
Exclusivity for
10 years
Community
Marketing
Authorization
+ 2 additional years for
pediatric indication

Orphan Drug Legislation: Rest of World
9
Japan, 1985 Singapore, 1991 Australia, 1998
South Korea, 1998 Taiwan, 2000

FDA Orphan Drug Designation Requests Since 1983
10
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

FDA Orphan Drug Approvals Since 1983
11
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

12
Kakkis EveryLife Foundation (www.everylifefoundation.org)

FDA and Patient Engagement

FDA and Patient Engagement
14
1988 20121996
1991 2001
FDA 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

FDA Safety and Innovation Act of 2012
15
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

FDASIA Patient Participation in Medical Product Discussions
Report on Stakeholder Views
16
Food and Drug Administration Safety and Innovation Act (FDASIA)
Section 1137: Patient Participation in Medical Product Discussions
Report on Stakeholder Views
5Major themes
identified
Clarification of
FDA Policies
Systematic Patient
Engagement
Transparency and
Communication
Clinical
Investigations
Workshops and
Partnerships

Stakeholder Report Summary
17
 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 Patient
Engagement
Section 1137: Patient Participation in Medical Product Discussions Report on Stakeholder Views

18
 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

19
 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

20
 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
Clinical Investigations

An Example of Collaboration
21
 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

Pending US Legislation Impacting Rare Diseases
22
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

EMA and Patient Engagement

EMA Commitment to Patient Engagement
24
1995 20062003
1996 2005
EMA 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 framework
Ongoing…
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&

EMA Patient Engagement
25
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&

EMA and Patient Engagement
26
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

”“
“
”

Rare Disease Research
Considerations

Orphan Product Development Challenges
29
Small Population
How to find patients?
Geographically
Widespread
How to access
patients?
Research Naïve Sites
How 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?

”
“
The Importance of Natural History Studies in Rare Disease
30
…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.

Patient Registry ≠ Natural History Study ≠ Randomized Clinical Trial
31
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

Patient Registry ≠ Natural History Study ≠ Randomized Clinical Trial
32
Natural 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

Working Together:
Patient Advocacy Organizations
and Industry

Goals of Industry – Patient Advocacy Group Collaboration
34
 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

Industry Needs from Patient Advocacy Groups
35
 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

Patient Advocacy Groups Needs from Industry
36
 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

Patient Advocacy Organizations and Rare Disease Research
37
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?

“
”

POLLING QUESTION # 2
39

European Federation of Pharmaceutical Industries & Associations
40
“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.“

EFPIA Code of Practice
41
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.

EFPIA Code Overview
42
 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.

Patient Advocacy Organizations - Looking Ahead
43
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

Summary
44
 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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