Wednesday, 20 November, 2013 Latin America Biotherapeutic Conference Day 2

1. A Patient-based Perspective on the
Regulation of Biosimilars
Maurice (Mo) Mayrides
Director, Policy and Programs
Esperantra
IFPMA Congreso Latinoamericano sobre Medicamentos
Bioterapéuticos
19-20 November 2013
Lima, Peru

2. Esperantra
We are a patient-based advocacy organization working on behalf of
cancer patients in Peru since 2005. Much of our work is dedicated to
coalition- and alliance-building in Peru, in Latin America, and globally
International Alliance of
Patients´ Organizations
A global voice for patients
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3. Importance of the Patient Voice
 Patient groups have not had many opportunities to participate in
these kinds of regional and international consensus meetings
 We have been passive listeners mostly because it is difficult to
convey and difficult for a lay audience to understand many of the
technical concepts that are at the heart of this issue
 But patient advocates are generally good at one thing, which is
helping to tell the story of why a policy issue matters, whether
this be to legislators, health authorities, or to the general public
through the media
 On biologics/biosimilars, the “story” we would like to be able to
tell, or to help you the expert stakeholders better tell, is the
following…
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4. Full Potential of Biosimilars
 Clinical enthusiasm not only for more options, but more good options,
to aid in the practice of medicine
 Industry enthusiasm
 For new business models working in collaborative (but still contractual)
scenarios where more and more companies across the globe are involved in
interesting arrangements to bring biosimilars to market
 Where because of competition, established pharmaceutical companies have
accelerated discovery and development programs for new biologics
 Government enthusiasm not only for cost savings, but an opportunity
for NRAs and their corresponding MoHs to build capacity and have
greater influence over health system improvements
 Patient enthusiasm because all of this translates into INNOVATION
 Pushing the frontiers of science and medicine
 Accelerating technological advances
 Moving us all closer to delivering on the promise of personalized medicine
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5. Full Potential of Biosimilars
However, we cannot tell this great story of global INNOVATION fully
without:
INTERCHANGEABILITY
“Interchangeability and substitution are likely to be game-changers”
– IMS Health
“introduction and proliferation of biosimilars in the global market strongly
depends on supporting legal and regulatory frameworks settled in each
country, which must ensure safety, efficacy, quality and hence
interchangeability”
– R. Ibarra-Cabrera etal., Biotechnol Adv. 2013 May 25
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6. How do we get to Interchangeability?
 Sophisticated regulations, harmonized among countries
 But why has this been so difficult?
 Especially when mechanisms (both official and unofficial) for harmonization
and international consensus exist and have been active
 Because the value proposition of biosimilars differs by type of country
 Geo-politics and the world we live in
 Geo-politics doesn’t in itself have the answers, but using it as a lens for
analysis can help identify the current status and look for solutions to
bridge gaps
 Mature economies
 Emerging markets
 Lower-resource settings
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7. Mature Economies
Value proposition of biosimilars is to control spiraling healthcare
costs due mostly to aging populations, but without
compromising quality care
 Substitution not allowed and interchangeability generally not
recommended as policy
 National (or regional in the case of the EU) decision-making on this is
delegated to provinces/states (countries for the EU), and then on to
treating MDs
 US FDA is an exception, but even then the decision is at the state level, and
many states are considering amendments to make it an MD issue too
Patients should be relatively comfortable with this…local
authorities have the capacity to regulate, we trust physicians
highly, and over time we can reasonably expect
interchangeability will exist in the practice of medicine
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8. Emerging Markets
Value proposition of biosimilars is to broaden access to
biotherapeutics, but also as an important economic growth
driver because of industrial policy favoring local national
production
 Dual pathway and/or openness by NRAs to case-by-case consideration
does not inspire confidence among patients that interpretations will not
be based on things other than safety, efficacy, and quality
 For example, how well-characterized are reference molecules elsewhere
 Having regulations in place is in many respects the beginning, not the
end, of the issue
Patients should be cautiously optimistic…having regulations in
place has taken considerable time and should be
congratulated, but we will need real-time data to show these
systems are working
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9. Lower-resource Settings
Value proposition of biosimilars is to increase affordability and
public sector access to biotherapeutics, in the context of stillexisting dual burden of disease (NCDs + transmissibles)
 Where draft rules exist, they are characterized by reaching minimum
sufficiency to claim alignment with the WHO guidelines
 Continued risk of entry of bio-copies through older rules,
compromising the entire modernization exercise
 Surveillance systems are not robust, so success of risk management
and pharmacovigilance plans remains suspect
Patients should be outright cautious…interchangeability is still
far from possible
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10. Potential Framework for Patient Advocacy
This geo-political analysis can lead to ideas for what patientbased organizations might want to ask for in order to get to
interchangeability
Mature Economies
 Clinicians can be encouraged to consider interchangeability, per local
and national rules, of course, and always in the best interest of the
patient
 Patients generally prefer to stay on the same medicine, but we need to
start gathering real-world data and experience at the clinical level
 We need to continue to try to work through the technical and ethical
issues raised by possible cross-over clinical trials
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11. Potential Framework for Patient Advocacy
This geo-political analysis can lead to ideas for what patientbased organizations might want to ask for in order to get to
interchangeability
Emerging Markets
 Insist that NRAs and corresponding MoHs keep the doors open on
established rules to consider—in a proactive manner—refinements to
the regulations as necessary
 Transparency in how the rules are being implemented
 Surveillance data-sharing to show public health is protected
 Continued openness to international consensus on the development of
product-specific guidance
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12. Potential Framework for Patient Advocacy
This geo-political analysis can lead to ideas for what patientbased organizations might want to ask for in order to get to
interchangeability
Lower-resource Settings
 Insist there is a very important timing issue here and draft rules need
to make it to the implementation stage
 Insist draft rules incorporate suggested changes based on
international consensus, and that NRAs take a step back and read the
final draft not with a checklist mentality, but with the overall “spirit”
of the WHO guidelines in mind
 Continue to build surveillance capacity, but do not over-rely on postmarketing pharmacovigilance to prove similarity after-the-fact
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13. Conclusions
 Patient advocates are important in that we can help “paint the
picture” for health decision-makers of the world we want to live in
 This world, already half-built, is characterized by ENTHUSIASM among
all sectors and stakeholders around biosimilars
 Interchangeability is the “game-changer” will allow for the full
potential of biosimilars to be realized
 Using the geo-political lens allows us to identify opportunities and
gaps, and to build a framework for patient advocacy
 Patient advocacy organizations in Latin America look forward to
working more closely with you the technical experts to further build
this framework
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14. Conclusions
All of this needs to keep moving forward, always
with the foremost consideration that patients now
and in the future deserve the best chance to access
quality products and quality care, which for them
means continued survival, freedom from
significant disease morbidity, and potential cures.
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15. Maurice (Mo) Mayrides
Teléfono: +51 (1) 2215518
Cel. +51 994681819
mmayrides@esperantra.org
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