Juliet Moritz premiere research crdn summit 2017

The Powerful and Evolving Role of
Patient Advocacy Groups in Orphan
Drug Development
Juliet Moritz, M.P.H.
Executive Director, Patient Engagement and
Strategic Drug Development, Rare Diseases,
Premier Research

Historical Barriers to Orphan Drug Research
3
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

FDA Orphan Drug Act (1983)
4
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

EMA Orphan Drug Designation
5
Protocol
Assistance
Fee
Reduction
Market
Exclusivity for
10 years
Community
Marketing
Authorization
+ 2 additional years for
pediatric indication

6
Kakkis EveryLifeFoundation (www.everylifefoundation.org)

FDA and Patient Engagement
7
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
Ongoing…
FDA Patient
Network created
with over 200
patient
representatives
participating

EMA Commitment to Patient Engagement
8
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
http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000317.jsp&
Ongoing…

EMA Patient Engagement
9
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
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

Goals of Industry – Patient Advocacy Group Collaboration
11
 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
Goals
Industry
Patient Advocacy Groups
Involvement
Partnership
Input
Access
Data
Respect

Industry Needs from Patient Advocacy Groups
12
 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
13
 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

“
”– Rare Disease Patient Advocacy
Organization Representative

European Federation of Pharmaceutical Industries & Associations
15
“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
16
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
 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.

Clinical Trials Transformation Initiative
18
CTTI RECOMMENDATIONS:
EFFECTIVE ENGAGEMENT WITH PATIENT
GROUPS AROUND CLINICAL TRIALS
https://www.ctti-clinicaltrials.org/files/pgctrecs.pdf
 Public-private partnership of 60+ stakeholders
focused on streamlining clinical research in the US
 Industry
 Patient Groups
 Academia
 Generated recommendations for effective patient
engagement around clinical trials based on best
practices gathered via a survey, meetings
 Includes recommendations for all stakeholders and
well as specific stakeholder relationships (e.g.,
recommendations for patient groups when
interacting with industry, etc.)

EU Frameworks for Interactions
19
Revised Framework for Interaction Between the
European Medicines Agency and Patients and
Consumers and Their Organisations
16 October 2014
EMA/637573/2014 Adopted
European Medicines Agency (EMA) Stakeholder
Relations Management Framework
16 June 2016
EMA/48651/2016

US: 21st Century Cures Act
20
 Several provisions aimed at encouraging drug sponsors and FDA to incorporate “patient experience data”
into the drug development and review process.
 “Patient experience data” is defined as data that
 (1) are collected by any persons (including patients, family members and caregivers of patients,
patient advocacy organizations, disease research foundations, researchers, and drug manufacturers);
and
 (2) are intended to provide information about patients’ experiences with a disease or condition,
including—
 (A) the impact of such disease or condition, or a related therapy, on patients’ lives; and
 (B) patient preferences with respect to treatment of such disease or condition.” 1
1 https://www.congress.gov/bill/114th-congress/house-bill/34/text#toc-H3EA1C23E2F0A4028B58F2FE4068359A9

US: 21st Century Cures Act and PDUFA VI
21
 Within six months of enactment, FDA must develop a plan to issue draft and final versions of one or more
guidance documents over a period of five years regarding the collection of patient experience data and the
use of such data in drug development.
 Specifically, FDA is required to issue guidance addressing methodologies for collecting and assessing patient
experience data as well as how the FDA intends to use patient experience data in regulatory decisions.
 FDA is already engaged in patient-focused drug development activities, including meetings with patient
advocacy groups, but the Act’s provisions are intended to force FDA to develop broadly applicable policy on
patient experience data.
 NOTE: FDA will no longer coordinate and host Patient Focused Drug Development meetings starting at the
end of fiscal year 2017 but will continue to collaborate with patient groups that desire to plan and host such
externally-led meetings to inform the FDA about the patient experience and patient concerns regarding the
development of therapeutics for specific conditions or groups of conditions

”
“
“
”
– Guido Rasi, EMA Executive Director

Questions?

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Juliet Moritz premiere research crdn summit 2017

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