Evolution of Biopharma R&D Through COVID-19 - Crisis as an opportunity - Pharma ...
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Evolution of Biopharma R&D Through COVID-19 Crisis as an opportunity
Introductions
Presenter Contributor
Daniel Chancellor Duncan Emerton, PhD
Director, Thought Director, Custom
Leadership Intelligence & Analytics
Informa Pharma Informa Pharma
Intelligence Consulting
LinkedIn LinkedIn
informa | Pharma Intelligence 2
Agenda
Clinical research
• Biopharma’s R&D effort against COVID-19
• COVID-19 disruption to clinical operations Research
• Diversified, patient-centric research
Regulatory environment Regulatory
• Short-term trial guidance
• Access to COVID-19 therapeutics Drug
• Lifting barriers permanently lifecycle
Commercial
Commercial implications
• Top- and bottom-line effects in Q1
• Adjusting commercial models for telehealth Supply
• Individual and collective industry profit
Conclusions and Q&A
informa | Pharma Intelligence 3
Clinical research
COVID-19 research effort
Surge in clinical activity matches global spread of coronavirus
Pharma Intelligence’s latest daily counts:
COVID-19 R&D tracker
• 456 pipeline drugs
3,000
• 2,451 clinical trials 2,451
2,500
Number of trials/drugs
2,000
Disparity between trials and industry progress:
1,500
• Trials largely driven by repurposed drugs
with academic and government sponsors 1,000
456
• Few clinical-stage industry programs 500 271
50
0
Industry pipeline 9 16 23 30 6 13 20 27 4 11 18 25 1 8 15 22 29 6 13
Phase III Phase II
Preregistration 10% Mar Apr May Jun Jul
2%
0% Clinical trials Industry pipeline drugs
Phase I
4% “More haste, less speed”
Preclinical
• Rush to establish clinical infrastructure without adequate central oversight
84% • Still lacking robust, conclusive clinical trial data, months into the outbreak
• Focus on quality – master protocols, adaptive designs, multiple arms
Source: Trialtrove; Pharmaprojects informa | Pharma Intelligence 5
Treatment paradigm
Biopharma is progressing multiple therapeutic approaches at varying rates
Initial wave Second wave Second wave
Repurposed drugs Specific antivirals Prophylactics
Rapid screening of existing Drug discovery using Therapeutic interventions
anti-microbials for effects known coronavirus to lower transmission in
against SARS-CoV-2 sequence to produce the community and/or
Also includes therapies for novel, specific treatments produce herd immunity
COVID-19 complications
Includes • Antivirals • Monoclonal antibodies • Vaccines
• Antimalarials • RNA interference • Antivirals
• Convalescent plasma • Cell therapies
• Immunological drugs
• Anticoagulants
Timeline Immediate Efficacy trials started in summer Efficacy trials started in summer
Emergency approvals granted Emergency approvals from Q3–4 Emergency approvals from Q4
informa | Pharma Intelligence 6
Innovative R&D
In time, biopharma will deliver more effective therapies, suitable for stockpiling and prophylaxis
The first novel antivirals have entered clinical testing
this summer
• Competing with huge numbers of existing trials
• Validation requires community-based transmission
continuing through 2020
• Can also be positioned as prophylactics and
providing bridge to broad vaccine availability
Vaccines are the most desirable and scalable
treatment option, attracting concerted industry effort
• Aggressive timelines condensing R&D work
• Calculated risks offset by sheer weight of
development effort
• ~170 programs across 10 discrete vaccine technologies
• Manufacturing scale-up in tandem to meet
demand
Each box denotes manufacturing capacity each quarter, beginning Q4 2020
Source: Pharmaprojects; RA Capital informa | Pharma Intelligence 7Collaborations
Industry is combining forces to tackle COVID-19 in unison
1 16
Biopharma companies are partnering with government bodies,
NGOs, academics and each other to share resources and expertise
Non-profit Industry
Consortia:
• NIH: Accelerating COVID-19 Therapeutic Interventions and Vaccines
• Bill and Melinda Gates: COVID-19 Therapeutics Accelerator ACTIV
• World Health Organization: Access to COVID-19 Tools Accelerator
5
COVID-19 drug discovery partnerships
60
50
40 Government
30
20
10 Partnerships:
0
Jan Feb Mar Apr May Jun Jul • Increasing numbers of R&D alliances and licensing deals
2020 2020 2020 2020 2020 2020 2020 • Bodies such as BARDA, CEPI also funding early research
Vaccine Biologic NME Device Non-NME
Source: Biomedtracker informa | Pharma Intelligence 8Disruption to clinical operations
Social distancing and point-of-care disruption can affect clinical operations
Almost all biopharma and CRO companies have stated disruption to ongoing and planned clinical trials
• New sites and patient recruitment is generally on hold, except for key focus areas
• Existing trials are continuing, with modifications where necessary to ensure continuity
Difficult to quantify scale of disruption, although different metrics are now pointing towards stabilization and recovery
All known COVID-19-affected trials Effect of COVID-19 on industry trial status
900 16,000 4.0%
Ongoing industry-sponsored trials
Of which temporarily closed
800 14,000 3.5%
700 12,000 3.0%
10,000 2.5%
Number of trials
600
500 8,000 2.0%
400 6,000 1.5%
300 4,000 1.0%
2,000 0.5%
200
0 0.0%
100 30 6 13 20 27 4 11 18 25 1 8 15 22 29 6 13
0 Mar Apr May Jun Jul
30 6 13 20 27 4 11 18 25 1 8 15 22 29 6 13
Mar Apr May Jun Jul Temporarily closed Ongoing industry trials Closed %
Source: Trialtrove informa | Pharma Intelligence 9Spotlight on COVID-19-affected trials
Trial disruption is occurring across the spectrum of drug development
Status Number of trials
Trialtrove notes degrees of disruption to studies, with many able to continue
Temporarily closed 345
However, the effect is universal across many segments of biopharma
Open 322
• Clinical phases – early, mid and pivotal stages all affected Planned 66
• Therapy areas – relative rankings consistent with all ongoing trials Closed 57
• Trial locations – bias towards US and EU5; China, Japan absent from top 10 Completed 28
Terminated 25
Cross-section of trials disrupted by COVID-19
I 23% Oncology 33% United States 71%
I/II CNS 22% United Kingdom 22%
11%
Autoimmune/Inflammation 19% France 19%
II 30% Spain
Metabolic/Endocrinology 11% 18%
II/III 2% Germany 17%
Cardiovascular 8%
III 20% Canada 15%
Infectious Disease 6% Italy
III/IV 0% 14%
Ophthalmology 4% Belgium 11%
IV 12% Vaccines (Infectious Disease) 3% Poland 11%
Other 0% Genitourinary 2% Netherlands 11%
0 100 200 300 0 50 100 150 200 250 300 0 200 400 600 800
Number of trials Number of trials Number of trials
Source: Trialtrove informa | Pharma Intelligence 10Industry response to mitigate clinical disruption
How can biopharma maintain R&D momentum through the COVID-19 pandemic?
Biopharma is compelled to maintain as much continuity as possible to improve patient outcomes
Disruption to trials delays access to new treatments, which introduces indirect harm, counterbalancing
potential increased exposure to coronavirus
• Risk assessments • Delays and • Willingness to Willingness of patients to engage in
• Protocol postponements participate trials may take longer to recover
adjustments • Strategic • Diversity and
prioritization equality Opportunities to focus on patient-
centricity, increasing diversity and
Ongoing
New starts
Patient equality of R&D
trials engagement
Elastic timelines, stable demand should show minimal long-term effect
Patient bolus should build and return, provided pandemic continues to ease
Opportunities to modernize clinical trial design and conduct
informa | Pharma Intelligence 11Shift towards decentralized, virtual trials
COVID-19 is accelerating the uptake of clinical trial innovations
Decentralized, virtual trials offer the potential to continue Decentralized, virtual trials by start year*
clinical research by moving the point of care 140
120
Number of trials
100
Uptake by industry remains low, but interest is growing and 80
sparked by COVID-19 60
40
20
Digital and virtual innovations within clinical trials include: 0
• Online consent forms, tied to electronic health records 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020
YTD
• Decentralized care, including mobile clinics
• Patient-reported outcomes and wearables *Explicitly stated in trial description/notes/protocol
• Telemedicine for remote patient monitoring
• Real-time tracking of clinical trials J&J’s 2020 virtual trials
Heartline: Mobile app and Apple
Wide-ranging benefits include cost, time, convenience, Watch for stroke reduction
diversity, and resilience to COVID-19 disruption CHIEF-HF: Invokana real-world study
Source: Trialtrove; In Vivo informa | Pharma Intelligence 12Diversification of clinical research
Extending the scope and inclusivity of trials to increase speed
Trial sponsors should also seek to cast the net wider Dimensions in scope and access to clinical trials
to increase the pace of clinical research
• Broader geographic spread: less COVID-19
exposed, recovering/resilient countries
Inclusion/exclusion criteria
Non-interventional
• Looser inclusion criteria: co-morbidities, ages Patient registries
studies
• Patient demographics: targeting minority groups
This will speed up the pace of clinical research, and
also improve quality/generalizability to real world
Early phase Late phase
Trade-off with homogeneity of sample introducing
variables that might require larger samples for
equivalent statistical power
Geographic spread
Rationalization of exclusion criteria needed; taken to
the extreme, requires regulatory receptivity towards
real-world evidence
informa | Pharma Intelligence 13Regulatory environment
Guidance for clinical trials during pandemic
FDA and EMA have offered advice to sponsors about trial conduct
All decisions to be made on a trial-by-trial basis. Regulatory advice
can be loosely grouped into three themes:
Prioritize patient safety Patient
• Sponsors to determine risk to patients and trial conduct safety
• Halt recruitment or pause trials as necessary, but avoid scenarios
where suspension compromises patient wellbeing
Ensure continuity for trial integrity
• Safety monitoring remains paramount, even if trial is disrupted
Trial Protocol
• Distribution and administration of treatments at home if required
integrity flexibility
Manage protocol flexibility
• Adapt patient monitoring in light of COVID-19 disruption
• Handle deviations in protocols without compromising trial
informa | Pharma Intelligence 15Facilitating access to COVID-19 drugs
FDA is expediting and prioritizing coronavirus research and evaluation
Regulators have multiple avenues in which to expedite COVID-19 research and facilitate access to treatment
Coronavirus Treatment Compassionate use Emergency use Formal regulatory
Acceleration Program and expanded access authorization review
Triage and 1-day turnaround Access to investigational Controversially awarded to Unknown timescale, but likely
for development plans for medicines outside of clinical HCQ, sets precedent for other conducted in record time,
new COVID-19 treatments trial setting COVID-19 drugs while retaining scientific rigor
“When we do [receive data], we intend to use a flexible and “The FDA is also a partner in the ACTIV partnership,
innovative approach. Once we observe favorable results, we which aims to develop a collaborative framework to
intend to be very proactive about patient access.” rapidly respond to COVID-19.“
Source: Pink Sheet; US FDA informa | Pharma Intelligence 16Expediting clinical development
Applying lessons learned from COVID-19 to general R&D
Industry R&D benchmarks, 2010–19
• Despite advances over past decade, average time in
clinical development stubbornly remains at 10 years Phase I 9% 2.2 years
• However, companies in COVID-19 are promising Phase II 16% 3.5 years
development timelines better measured in months
Phase III 53% 3.2 years 10.2 years
• What learnings can industry and regulators apply to
enable time/cost savings in general R&D? Registration 90% 1.3 years
0% 20% 40% 60% 80% 100%
Likelihood of approval
↑ biomarker or ↑ regulatory prioritization
Preclinical Clinical Regulatory genetic validation and flexibility
20% LOA, 5 year timeline
• Pre-competitive • Skipping or • Real-time review
collaboration overlapping phases • Conditional ↓ R&D expense
• In silico R&D and • Early indicator, approvals
characterization surrogate endpoints ↑ patient outcomes, access
• IND prioritization
Source: Pharmapremia informa | Pharma Intelligence 17Blurring the lines between clinical phases
Recognizing opportunities for pivotal Phase II trials, real-world/confirmatory Phase III
Tufts CSDD: $2.6bn per drug approval Breakthrough therapy benchmarks
• Much of the cost of R&D belongs to Phase III in
terms of time/spend Phase I 9%
90%
• There is still a high fail rate (53% LOA)
• Are there pockets of R&D where success is much 16%
Phase II
higher? 90%
• Can Phase III be justifiably replaced by RWD in
these instances? Phase III 53%
92%
The FDA is already good at picking winners Registration 90%
96%
• Breakthrough therapy drugs have 90% LOA
even at early-stage development 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100%
• Routine accelerated approval could shave 2.5 Likelihood of approval
years off development
All drugs Breakthrough therapy
Other potential targets could include de-risked opportunities:
• Biomarker-driven R&D, line extensions, biosimilars, high unmet need
Source: Pharmapremia; Tufts CSDD informa | Pharma Intelligence 18Commercial implications
Global economic outlook
COVID-19 is plunging the world into recession and eradicating years of market gains
Expected 2020 recession set to eclipse 2007–08 crisis:
• 4.6% global GDP decline, pronounced in developed YTD market returns, S&P 500 vs Nasdaq Biotechnology Index
economies
• Rebound to pre-coronavirus levels by end of 2021,
although US and Eurozone may lag further
Fitch Ratings Global Economic Outlook
10
8
6
4
GDP growth
2
0
-2 2019 2020F 2021F 2022F
-4
-6
-8
-10
US Eurozone China Japan UK Developed Emerging World
Source: Fitch Ratings; Google Finance informa | Pharma IntelligenceBiopharma Q1 earnings calls
Business and underlying demand resilient in Q1, although longer-term outlook less clear
Big Pharma companies are generally reporting strong
underlying demand and limited COVID-19 effect Select biopharma Q1 results and COVID-19 impact
Company Q1 COVID-19 impact 2020 guidance
Chronic Rx and OTC drug sales are resilient, although
one-time treatments often deferred AstraZeneca Low-to-mid single digit boost Unchanged
Eli Lilly +$250m sales increase Unchanged
• Many companies experienced short-term COVID-
19 revenue boosts on account of wholesaler GSK “Positive impact on growth” Unchanged
stockpiling and increased Rx renewals in Q1 J&J “Net favorable” to pharma 7–9% decline
• J&J medical devices business exposed because of Merck Negligible 3–4% decline
fewer elective procedures Novartis +$400m sales increase Unchanged
• Other businesses such as ophthalmology, Pfizer Limited impact Unchanged
veterinary medicine adversely affected Roche Limited impact Unchanged
Sanofi +€300m sales increase Unchanged
Longer-term picture is less clear, but most companies
have restated their initial 2020 guidance
Source: Scrip; various quarterly results presentations informa | Pharma Intelligence 21Commercial models during and post-COVID-19
Telemedicine offers a different playing field for biopharma
COVID-19 pandemic has necessitated near universal adoption Routine patient triage during and post COVID-19
of telemedicine, despite logistical challenges
• Given practical benefits, this trend is unlikely to be reversed
• Biopharma must adapt to new physician-patient interaction Digital Virtual Physical
with digital sales strategies
Desirable drug characteristics for telehealth practice
Telemedicine will also cause realignment in desirable
target product profiles
• Sanofi raising expectations for Dupixent
Clean safety profile Companion diagnostic
• Is inclisiran still worth $10bn?
Clinical familiarity Differentiated MOA
• Interesting case study to follow with risdiplam vs
Infrequent or self Extensive patient- Spinraza and Zolgensma in SMA market
administration follow up
Aligned with other Reimbursement tied
• Developing a drug with telemedicine in mind in clinical
digital solutions to outcomes trials could confer competitive labelling advantage
informa | Pharma Intelligence 22Transitioning from clinical to commercial
COVID-19 poses an essential opportunity to rebuild public reputation and trust
Public opinion polls routinely and consistently portray Biopharma scorecard: so far, so good
biopharma in an extremely negative light
• Harris Poll from 2016 stated more than 9 out of 10
Americans think pharma values profits over patients
• 2019 Gallup poll ranked pharma last of 25 Clinical trials
industries, below federal government Repurposed and
novel drugs
Collaborations
However, since the COVID-19 outbreak, Harris is noting
Charitable
steady improvements in biopharma perceptions donations
Not-for-profit
“As much as the pandemic has devastated many
industries, it has offered Big Pharma a chance to
shine as never before, winning back the trust of a Reputational pitfalls that pharma needs to manage include:
public infuriated with years of soaring drug prices.
Will they seize the moment?” • Overpromising and underdelivering on timelines
• Managing the transition from clinical to commercial access
• Resisting nationalist behaviors for drug/vaccine tenders
“A rising tide lifts all boats”
Source: The Harris Poll; Gallup; Fortune informa | Pharma Intelligence 23Conclusions and Q&A
Concluding thoughts
COVID-19 poses numerous challenges and opportunities for biopharma to modernize
Clinical research
• Unprecedented R&D is only just bearing fruit, although scale of collaborations will certainly yield further successes
• COVID-19 poses unique challenges to the industry, most pressingly to the conduct of clinical trials
• Companies can innovate both to continue operations through the pandemic and modernize for longer-term benefit
Regulatory environment
• Regulators are acting in an advisory role, empowering biopharma to apply judgement to clinical trial conduct
• They also have a range of tools to facilitate and expedite research into COVID-19 therapies
• Speed of COVID-19 development leaves questions as to whether the current R&D model is outdated or even harmful
Commercial implications
• Biopharma valuations and earnings are relatively well insulated against the specific COVID-19 threat, although the
wider macroeconomic situation poses longer-term uncertainties
• The emergence and solidification of telehealth will require adjustments in commercial practice and reprioritization
• Industry must restore credibility and prioritize long-term collective value over short-term individual pandemic gains
informa | Pharma Intelligence 25Thank you for your attention Questions? daniel.chancellor@informa.com duncan.emerton@informa.com
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