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An Integrated Development Plan in Rare & Orphan Medicinal Products (OMPs)
AUGUST 2020
© 2020 Syneos Health®. All rights reserved.
SIGNIFICANTLY ACCELERATING TIME TO APPROVAL AND STRENGTHENING ASSET VALUE FOR ALL KEY INDUSTRY STAKEHOLDERS
By Maryna Kolochavina, PharmD, PMP (Syneos One)
Syneos One®, a specialist group at Syneos Health, act as a single “go-to”
partner focused solely on helping biotech companies develop and execute
the most efficient integrated product development strategies in order to
maximize value and de-risk the delivery of critical patient therapies.
Our Syneos One team – a seasoned group of product development asset
strategists – provides customers with insights-driven product development
methodologies to speed time to market. Specific to rare and orphan drug
development where first mover advantage matters, Syneos One has
developed a proprietary diagnostic tool designed to identify the most
efficient clinical and regulatory path to approval while developing strong
value propositions for market access and market adoption.
* Based on the evaluation of a cohort of 218 assets with 436 orphan drug designations granted in 40 therapeutic classes that have been launched since 2016. Detailed analysis is included on pages 10 and 11 of this paper.
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)2
Companies looking to develop medicines for rare and orphan diseases face a range of challenging issues:
Leveraging our extensive insight into these issues in the rare and orphan space, Syneos One has developed the ADAPT (Accelerate, Drive, Anticipate, Partner, be Transparent) diagnostic tool—our integrated evidence-based tool* that provides a best-practice benchmark, specific to each customers’ opportunity, to maximize value for all stakeholders along the product development journey. The ADAPT diagnostic tool enables companies to:
• Clinical and regulatory strategy must be
developed and executed within a complex
and changeable regulatory framework,
competitive trial setting and politically
sensitive environment.
• Accelerated regulatory approval pathways
means companies face early
commercialization challenges that must be
addressed in a compressed time frame.
• Identify opportunities to accelerate patient
access to new therapies
• De-risk development and generate
necessary levels of evidence
• Optimize synergies between multifunctional
teams and regulatory and financial incentives
• Development programs can pose significant
risks when knowledge about the disease
state and rare patient population is limited,
leading to many associated research and
development (R&D) challenges.
• Companies require exceptional relationships
with geographically dispersed patients, their
families and the healthcare professionals
(HCPs) that comprise the disease community.
• Facilitate early patient engagement and long-
term partnership with all key stakeholders
• Provide support, advice and insights that build
trust within in the rare disease community
Contents
Introduction 4Abbreviations / Definitions 4
Overview of the Rare Disease Pharmaceutical Market 5
The “Rare” Reality 7What Does It Mean to Live with Rare and Orphan Conditions? 7
Key Stakeholder Perspectives and Expectations 8
Effectively Demonstrating Value within the Challenging Rare & Orphan Landscape 10R-W-W (Real-Win-Worth it) Screening Approach 13
Integrated Asset Planning and Delivery Is Essential to De-Risk Path Forward 15ADAPT-Based Integrated Solution Improves Asset Value 15
A Defined Partnership Model to Create, Capture and Maximize OMP Value 16
Conclusion 17
References 19
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)3
Regulations, multiple international initiatives and incentives have resulted in a marked increase in rare disease research funding and development effort,14 giving rise to great optimism about the future. However, challenges remain for the rare disease community:
• Despite technological and medical advances in recent years, misdiagnosis and a lack of knowledge remain12,13,41
• A rare disease has a significant impact on not only the person with the condition but that person’s family and primary caregiver4-5,7
• Drug development processes are not keeping pace with improved regulatory and clinical trial efficiencies2,13
• Clinical development is often hampered by an incomplete understanding of underlying disease mechanisms and relevant clinical endpoints, as well as limitations associated with identifying a large enough sample of comparable patients for clinical trials17,18,38
• High prices associated with rare and orphan drugs are a frequent barrier to market access43
Introduction Rare and orphan diseases are complex and represent significant unmet need, with over 7,000 such conditions known and 250 to 280 new diseases described annually; as many as 20 percent of these disorders are ultra-rare.2,6 Their impact, both in terms of disease burden and product development opportunity, is significant.
ABBREVATIONS
RD: rare disease ; R&D: research & development; WW: worldwide; CTs: clinical trials; OMP: orphan medicinal product; Ph: phase; KOL: key opinion leader; MAA: market authorization application; P&R: price and reimbursement; R&O: rare & orphan; HTA: health technology assessment; CUP: compassionate use program; EAP: early access program; RWE: Real World Evidence; PRO: patient-reported outcomes; SME: small and medium-sized enterprises
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)4
DEFINITIONS
Rare diseases by definition are conditions that affect small percentage of the population. There is no single, wide unifying definition of a rare disease; it differs by country and region prevalence range (from 1/1,000 to 1/200,000), and included other factors, such as the existence of adequate treatments, a lack of resources or the severity of the disease.2,6
The subcategory “ultra-rare” disease was introduced by the National Institute for Health and Care Excellence (NICE) for drugs with indications for diseases that have a prevalence of <1 per 50,000 persons (not the legal definition).60
“Orphan diseases,” often used as a synonym for rare diseases, have a distinct legal meaning in the United States and the European Union. The United States’ Orphan Drug Act definition of orphan disease includes both rare diseases and any non-rare diseases “for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug.”61 The European Organization for Rare Diseases (EURORDIS) also includes both rare diseases and neglected diseases in a larger category of “orphan diseases.”62
OVERVIEW OF THE RAREDISEASE PHARMACEUTICAL MARKET
5
Growing segment
Novel development approach
High unmet need
Promising pipeline
– OMPs > 1/5 of WW
prescription sales56
– 163 percent sales
forecast vs. 146 percent
for non-orphan56
– $242B R&O market in 202456
– OMP is 4.5 times higher cost/
patient (to non-OMP 2018)57
– 73 percent approved
OMPs used at least 1
early access tool77
– 38 innovative R&D designs
for OMP65-75
– 18 early regulatory schemes46
– 61 percent positive OMP
HTA decisions with RWE78
– 42 percent approved OMPs
based on 1 CT only76
– 1 of 8 OMP approvals are
based on Ph1/Ph2 only76
– 400M people affected
WW,54 50 percent children,
30 percent of whom will
die before age 5 years2,4,5,52
– 80 percent RDs are genetic
origin 3-4 percent of births46
– 95 percent RDs no
approved treatment1-5
– less than one in 10
patients receive disease
specific treatment5
– 97 percent RDs not covered
with natural history flow19,20,48
– 85 percent RDs have no
organized group58
– 31 percent of the current
clinical trial pipeline55
– OMPs > 1/3 of R&D pipeline
sales (2024)56
– 1/3 of all MAAs in last 5 years57
since 2018 leading MAA:
16 percent in U.S.,
4 percent in EU57
Advances in scientific research over the last two decades have highlighted the emerging possibility of addressing the needs of patients and families with rare disorders in ways that would have been dismissed as science fiction only a few years ago.
Alastair Kent
Chair of Rare Disease UK, Director of Genetic Alliance UK63
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
6
According to the International Rare Diseases Research Consortium (IRDiRC), in the next 10 years treatments will become available for only approximately 600 of the 7,000 known rare diseases.2 Improving treatment coverage and bringing novel medicines to patients in need remain important objectives in this still formative area.2 Traditional linear operational models and siloed activities will not address the existing unmet need. Integrated approaches that link R&D, Real World Evidence (RWE) and commercialization—including early engagement of key stakeholders—are emerging as requirements within this evolving and uncertain environment.81
Now, when the rare and orphan community is overwhelmingly concerned and impacted by COVID-19, it is essential to adopt innovations and implement predictive approaches to accelerate the development, ensure successful approval, market access and use of orphan medicinal products.79 The pandemic has intensified the challenges faced by the rare disease community with regard to resource prioritization, financial stability and increased competition in this non-mature market.80 At the same time, cooperation and broad evidence generation is required.
To address these challenges and meet the stakeholders’ expectations, Syneos One has created an integrated process for OMP development driven by our proprietary ADAPT diagnostic tool. This tool was formed based on an evaluation of 218 orphan assets with 436 orphan drug designations granted in 40 therapeutic classes that have been launched since 2016. The Syneos One team has structured the findings from these assessments into a system of 51 levers organized by category that can be applied to any rare and orphan asset. This allows us to benchmark the approach taken by drug development companies with industry best practice, and thereby identify the ways to accelerate time to market, opportunities to de-risk the asset value proposition and estimate an impact on asset value.
Our analysis shows that using an integrated management methodology in the rare and orphan disease space can result in significant acceleration times to approval and strengthen the asset value proposition for all key industry stakeholders.
This paper takes a close look at the OMP development landscape—what we call the “rare reality”—including key stakeholders’ expectations and perspectives, market demands, barriers and OMP critical success factors. We then explore how integrated product development using the ADAPT diagnostic tool can de-risk and accelerate go-to-market timelines. Finally, we review how Syneos One is supporting our customers to build on the outputs of this work through partnerships to deliver integrated programs, bringing OMPs faster to the patients who need them.
…The patients and families who are involved in that have to be ambassadors and get out there and show the rest of the research community how it works and how it was successful and what the impact was…we’re trying to change a whole culture of how researchers think and work…if they can get concrete examples of how it’s worked in the past and how it’s been successful and how it’s enhanced the research, they may be more open to just having it proposed to them...
—Patient 315
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
In Syneos One’s experience, involving these patients early helps improve OMP development, and leads to more economically viable clinical trials, and a fruitful, longer-term relationship at launch and beyond to help optimize current and new therapies.15,16,24
Living with a rare condition
7
According to a number of studies, more than 80 percent of patients and their families experience serious impacts to their daily lives as a result of their rare diseases.7-11 These impacts include the substantial time burden of daily care management and care coordination.2
WHAT DOES IT MEAN TO LIVE WITH RARE AND ORPHAN CONDITIONS?
“I do not know about my condition”The burden for patients
suffering from any form
of a rare disease typically
starts with a late
diagnosis.4-5,7,12 Finding and
engaging these patients
early with a sophisticated,
robust, inexpensive and
easy diagnostic workflow
remains a challenge.13
“I am the only one in my country who is ultra-rare”In many cases, ultra-rare
diseases remain a mystery
to researchers, with limited
models or guidance, little
research and a poor
understanding of the natural
history of the disease.19,20
The complexity of rare
diseases can cause significant
mental and physical burdens
for patients and their families.
Most individuals will not be
cured in their lifetimes.2,45
“My physician lacks understanding about my condition”Healthcare providers often
lack knowledge of how to treat
people with rare diseases due
to their low prevalence,
meaning that patients often
have an active role and partner
with their healthcare team in
their treatment journey.14,46
“I feel I’m an expert in my own disease”The availability of cyber-based
health information and online
groups has led to the
emergence of the “expert”
patient who may, in fact, know
more about their condition than
many of the healthcare
providers they come into
contact with.4,5,7 These patients
seek a collaborative relationship
with their healthcare teams,
meaning that traditional roles
and communication
approaches have to evolve to
become more patient-centered
—and in some cases may be
patient-directed.21,25,51
“I might not have research treatment during my life”While the number of rare
disease treatments is growing
substantially, they treat only a
small fraction of rare diseases,
most of which have few or no
treatments.2,19 Most rare
diseases do not have drugs
in development to treat them,
highlighting the need for
continued incentives to address
research and treatment gaps.1-5
“I may not have approved and available treatment”Evidence uncertainties during
regulatory and reimbursement
decision-making combine to
limit access to effective
therapies.16,17 These
uncertainties arise from a lack
of robust information around
clinical benefit, value for money,
and potential adoption.18
Prescribing restrictions and drug
delivery systems also influence
the ease of patient access to
reimbursed orphan drugs.32,39
“I want to help others with my condition”Patient involvement
can improve discovery,
development and evaluation
of effective medicines by
supporting a more collaborative
understanding of unmet needs
and R&D objectives.21,22,41
“My family and I need constant psychological and physical support”Many patients with rare
diseases suffer from multi-
system dysfunction, without
effective treatment, and are
likely to require complex
care.9,11 Multiple factors lead
to additional physical and
psychological impairments
for patients and families.41
“I encounter social and emotional challenges unique to my condition”For some patients and families,
obtaining a diagnosis brings
relief; for others it can be a
crushing reality check as 95
percent of rare diseases have
no approved treatment.2,13
THE “RARE”REALITY
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
8
Key stakeholder perspectives and expectationsBefore undertaking a fully integrated approach to product development in the rare disease space, it is critical to have an in-depth understanding of the expectations and perspectives of key stakeholders in the rare and orphan disease community.
Regulators are highly willing to make decisions based on provided integrated evidence that has the characteristics needed to rely on it.— Janet Woodcock M.D.
Director, CDER, FDA25
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
Acceleration is
evidence-driven29,47
“Evidence vs.
access” conundrum34
Asset robust evidence and
clear added value (OMP being
novel / improved / different)43
Acceleration to improve
methods of diagnosis and care42
Expedited approval does not
mean “less evidence”33,59
“Live the best life possible”
understanding33
Meaningful evidence for
coverage per patient (sub)
group and population37
Better understanding of the
disease, patient’s needs and
OMP nature42
Respond to the complexity with
novel approaches to increase
R&D and RWE productivity25,31
Opportunity to collect
patient-centric data and
take advantage of fast-
evolving technologies21-23
Uncertainty associated with
realizing benefit from high-
cost treatment (downstream
health benefits, lifelong
treatment benefits)40
Address highly competitive
“lower-hanging fruits”44
Scientific short- vs. long-term
strategies45
Cooperation in generating
benefit / risk evidence27,29
Inclusion of patient voice26
Active engagement from first
experience with orphan
medicinal product24
Collaboration on OMP
economic value and agreement
with regulators on endpoints
and comparators32
Commitment to streamline
drug-to-patient with increased
efficiency of data collection
and sharing between KOLs41
Proactive steps needed
to streamline collaboration
on integrated evidence
generation plan, review and
approval process28,30
“Time Equals Lives”: Patient
knowledge, contribution at
every OMP life cycle step are
crucial to increase product
development efficiency36
React to changes in R&O
landscape: e.g., new models
of risk and incentive sharing
between public and private
partners, repurposing of existing
agents, transformative P&R
schemes and innovative MEAs44
Improve care standards with
data from disease registries49
Clinical protocol design
evidence, meaningful to
patients and KOLs39
FOR REGULATORS FOR PATIENTS FOR PAYERS & HTAs FOR PHYSICIANS
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)9
The Expectation: Transparent in Communication With All Parties Involved
The Expectation: Engage Stakeholders Early and Partner Throughout The Asset Life Cycle
The Expectation: Anticipate Risks and Complexities in Changeable Environment
The Expectation: Generate Robust Evidence
The Expectation: Accelerate Time to Market
KEY STAKEHOLDER PERSPECTIVES
Such expectations create a unique need for early cooperation to maximize orphan medicinal product potential.14,35
In response to the complexities of the R&O sector, Syneos One deploys its proprietary ADAPT (Accelerate, Drive, Anticipate, Partner, be Transparent) diagnostic tool to enable companies to:
• Accelerate time to market by applying and implementing efficiencies, opportunities and incentives that are available in the R&O space
• Drive data collection and generate the evidence required at each inflection point for the asset during the life cycle
• Anticipate risks, unknowns and evolving therapeutic landscape
• Partner with all key stakeholders early and throughout an asset’s life cycle
• Be transparent in processes and communication between all stakeholders involved
To create the ADAPT diagnostic tool, the Syneos One team evaluated a cohort of 218 assets with 436 orphan drug designations granted (74 percent of assets have more than 1 designation per asset) in 40 therapeutic classes that have been launched since 2016 (Fig. 1-2).
Effectively Demonstrating Value within the Challenging Rare & Orphan LandscapeAs mentioned above, orphan medicinal products pose unique challenges when it comes to demonstrating value, owing to an often-limited evidence base, small dispersed patient populations, lack of knowledge about the condition and its natural history,19,20 and affordability and variable availability.51,52
10AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
Many factors affect OMP value. Until now, there has been little consensus on the most appropriate assessment criteria.
—ORPH-VAL, 201782
11
Figure 1-2. ADAPT Sample
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
THERAPEUTIC CLASS FOR 218 ASSETS
LAUNCHED SINCE 2016 (ADAPT SAMPLE)
436 ORPHAN DRUG STATUS GRANTED TO
218 ASSETS LAUNCHED (ADAPT SAMPLE)
Antianemic Antiarthritic Antibody, non-monoclonal Anticancer Antidepressant Antidiarrheal Antiepileptic Antifungal Anti-inflammatory Antimalarial Antithrombotic
Antiulcer Antiviral Antiviral, anti-HIV Blood fraction Cardiovascular Cellular therapy Cystic fibrosis treatment Dermatological Diagnostic Fusion protein
Gene therapy Hepatoprotective Hormone Infection Metabolic and enzyme disorders
Monoclonal antibody Muscle relaxant Musculoskeletal Neurological Neuroprotective
Protozoacide Recombinant Reformulation Respiratory RNA interference Urological Antisense therapy, Other Antisense therapy, Musculoskeletal
12
The Syneos One team structured the findings from these assessments into a system of 51 levers by five categories (Fig. 3):
LEVER EXAMPLES
Consensus on
definitions and
diagnosis
Novel feasibility
Dynamic Assembly
LEVER EXAMPLES
Stakeholders regular
alignment during
the life cycle and
arrangement for the
benefit of a patient.
Avoid asymptomatic
information
Integrated evidence
generation plan (value
over time requirements,
each phase in right
order completion)
LEVER EXAMPLES
Early patient
engagement
Synergy between
orphan drug
designation, expedited
regulatory designations
and early market access
LEVER EXAMPLES
Completion and
superiority evidence
Disease / OMP budget
impact vs. preferences:
society / family / patient
LEVER EXAMPLES
Net outcomes
improvement evidence,
meaningful to patients
and KOLs
Early knowledge (e.g.,
surrogate or composite
intermediate endpoints,
that are qualified and
validated in advance)
data collection and
generate the evidence
required at each
inflection point for the
asset during the life
cycle to support
acceleration in time
risks, unknowns, new
condition information,
regular revisions of
experience, new market
realities and trends,
shifts in stakeholders’
need, new data
and technology
with all key stakeholders
early in the process
and through OMP
lifetime / life cycle
time to market with
single or a combination
of the R&O efficiencies,
opportunities and
incentives,
opportunities and
incentives tailored per
OMP, therapy area and
patient needs
We start with accelerating time as our patient is there and is waiting
Acceleration is always evidence driven. We need to show evidence to receive expedited
pathway designations
While working with evidence in the R&O space, we need to keep the door open to uncertainties
We expect unknowns and uncertainties. Yet, to accelerate our time to patient, we need clear
partnering with all stakeholders
To support partnering with all stakeholders, we need clear communication and processes
Drive Anticipate PartnerAccelerate
How we came to these
Categories
in processes and
communication
between all parties
involved to deliver the
best care to the patient
with unmet need
Be Transparent
ACCELERATION IS ALWAYS
EVIDENCE DRIVEN
WORKING WITH UNCERTAINTIES IS
ESSENTIAL
EARLY ENGAGEMENT STRUCTURE
REQUIREMENT
TRANSPARENT COMMUNICATION AND PROCESSES
1
2
3
4
5
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
Figure 3. ADAPT Levers Examples
Our analysis suggests that applying between 23 and 51 of these levers could significantly reduce the time to approval and strengthen the value proposition of the asset for all key stakeholders (Fig. 4).
13
Figure 4. Best Practice, Sector Standard, Below Average Cases
218
2 31
23 5112
Below Average
Number of Levers Used
Num
ber o
f OM
Ps
Sector Standard Best Practice
Significant acceleration times to approval and strengthen the asset value proposition to all key industry stakeholders
ADAPT enables us to benchmark an asset’s clinical and regulatory strategy against best practice. We perform this by determining the applicability of each of the 51 levers for the asset in question using an R-W-W (Real-Win-Worth it) screening approach.
From the findings of the ADAPT tool, we typically build an integrated asset roadmap that includes activities to address the areas highlighted by the diagnostic. The roadmap is “integrated” in the sense it incorporates:
• Patient-focused fast adaptive R&D
• Accelerated regulatory pathways and scientific advice
• Patient engagement measures
• Value proposition and market access optimization (Fig. 5)
Importantly, it determines if there is a potential valuation lift of the asset by considering alternative clinical and regulatory approaches.
“Real” analysis
Determines if each lever’s activity has been completed, is ongoing, is planned
by company or is feasible and timely
“Win” analysis
Identifies the additional value of each lever to help reduce the time-to-market, find ways to de-risk development and
realize efficiencies
“Worth it” analysis
Tests each lever’s viability according to effort/result ratio
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
R-W-W (Real-Win-Worth it) screening approach
14AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
Figure 5. Asset road map: high-level illustration
PRE- CLINICAL PHASE I PHASE II PHASE III PHASE IV COMMERCIALIZATION
& POST-APPROVAL
OMP Case for UseOMP Case for ReimbursementOMP Case for ApprovalCurrent Drug Development
ADAPT Diagnostics
Outcome
R&D Program and Strategy
OMP Case for Approval
Adaptive Trial(s) Design and R&D strategy
Accelerated regulatory pathways
Expedited HTA/P&R
EAP/CUP/Hospital Exemptions Early Market Access
Additional therapeutic benefits
Clinical and Real-World Research and Evidence Generation
Post-Authorization Evidence Generation
Commercialization, Market Access and Pricing
OMP Case for Reimbursement OMP Case for Use
MAA Review P&R
15AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
INTEGRATED ASSET PLANNING AND DELIVERY IS ESSENTIAL TO DE-RISK PATH FORWARDOnce the asset road map has been developed, a detailed integrated planning process can be initiated. This requires multidisciplinary collaboration to capture all the required functional area insights, enabling companies to:
• Promote a sense of urgency by selecting an innovative research and development design that includes timely and relevant reported outcomes (meaningful to patient, observer and physician)
• De-risk R&D with early protocol reviews, scientific consultation and frequent interactions with regulators and payers
• Take advantage of the efficiencies and incentives such as orphan drug designation, regulatory early access tools and expanded market access programs
• Implement priority insights to help strengthen the case for investment
• Ensure key stakeholders’ needs are addressed early on
For stakeholders, using the ADAPT tool results in:
ADAPT-BASED INTEGRATED SOLUTION IMPROVES ASSET VALUE
Patient, Family, SocietyEarly access with evidence that
the treatment is effective and
results in better quality of life
Patient OrganizationsPatient-focused adaptive
and efficient evidence
generation for OMP coverage
without restrictions
InvestorsImprovements in asset value
through optimized planning,
accelerated delivery and
de-risked operations
IndustryNiche and sustainable
advantages to expand and
maximize OMP value
BoardIntegrated evidence to
optimize OMP value via clinical
/ commercial competitive
advantage in cost, time,
resources and quality
Small / Mid-size companies (SME)Biotechnology integrated
business model with single
contact managing
multifunctional teams
onward for maximum NPV
RegulatorsStrong OMP evidence for
approval based upon dynamic
results that evolve with new
data and RWE
KOLsRobust evidence to support
rare disease understanding
and clear differentiation of
OMP in the treatment pathway
16
A DEFINED PARTNERSHIP MODEL TO CREATE, CAPTURE AND MAXIMIZE OMP VALUESyneos One uses a specific partnership model with companies looking to develop rare and orphan medicines. Building from the diagnostic outputs of the ADAPT tool, we work with our customers to design and execute an integrated product development approach for their assets spanning clinical and commercial activities. Leveraging this approach, our customers immediately gain global operational scale, allowing them to help de-risk the use of funds for their clinical and commercial activities.
Our model is based on a close strategic partnership between our client and the Syneos One team. This partnership is driven by our “Asset Lead,” who works closely with the customer to support the development of the optimal clinical, regulatory and commercial strategy and the enablement of successful integrated operational delivery. This streamlined product development approach ensures that our clients receive a tailored service in an “easy to do business with” model but with the benefit of the global scale of Syneos Health. We supplement our customers’ existing expertise with broad clinical, market
access, regulatory and commercial capability, and integrated thinking with no stone unturned. This tightly integrated approach across the entire product development program is essential in the rare and orphan space.
The effort typically involves a series of distinct phases where the end of each phase is linked to a value inflection point for the asset. This provides our customers with clear opportunities to review achievements and change course if desired.
The best strategic idea means nothing in isolation.
—Nilofer Merchant
Harvard Business Review, 201150
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
ConclusionThe scale of the “rare disease problem”—thousands of rare diseases, the vast preponderance of them with no approved treatment and decades‐long diagnostic odysseys for many patients—has led to the realization that the time has arrived for global cooperation and collaboration among the many stakeholders active in rare disease research, to capitalize on proofs of principle and maximize the output of rare disease research efforts around the world.51
Government research funders, companies, scientists and patient advocacy groups have all demonstrated their commitment and effectiveness in contributing to progress in rare diseases research. We are all in this together—enabling earlier access to new, effective treatments.
17AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
ABOUT THE AUTHOR
Maryna Kolochavina
Executive Director, Syneos One, Rare Diseases
18
Maryna has over 15 years in life cycle management of a number of complex rare and orphan
conditions, including pediatric rare and ultra-rare diseases. Maryna has strong experience in
planning and execution of multiple studies/project phases (pre-clinical, I, IIA/IIB, IIIA/IIIB, IV) and
designs, such as disease and product observational registries with prospective and retrospective
data collection, pregnancy registries, observational FUP and retro chart review studies;
compassionate use programs (including Named Patient Program, Expanded Access Programs,
ATU, Special Access, IND); safety surveillance studies (including active and passive surveillance
studies, descriptive drug utilization studies, PASS, PAES); non-interventional studies (NIS) with
ePRO, HEOR and EPI modules; rare ATMP and stem cells studies, device studies, computer adaptive
testing and Omics studies (tissue research). Her educational background includes PharmD in
clinical pharmacology from National Medical Academy, Gdansk, Poland, and translational work in
rare skin diseases. Maryna is certified ACRP, BARQA, ASQ and PMI. She is an active patient advocate
for rare and orphan patients in the Patient Voice program at Charité, Berlin, Germany.
For more information about Syneos One and the ADAPT Diagnostic Tool, please go to syneoshealth.com.
AN INTEGRATED DEVELOPMENT PLAN IN RARE & ORPHAN MEDICINAL PRODUCTS (OMPs)
19
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3. Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat. Rev. Drug Discov. 9, 921–929 (2010)
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