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

International Alliance of

Patients´ Organizations

A global voice for patients

Esperantra

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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

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…

Importance of the Patient Voice

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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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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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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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Mature Economies

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

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Value proposition of biosimilars is to control spiraling healthcare

costs due mostly to aging populations, but without compromising quality care

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

Emerging Markets

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

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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

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

Lower-resource Settings

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

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Value proposition of biosimilars is to increase affordability and

public sector access to biotherapeutics, in the context of still-existing dual burden of disease (NCDs + transmissibles)

Patients should be outright cautious…interchangeability is still

far from possible

Potential Framework for Patient Advocacy

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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This geo-political analysis can lead to ideas for what patient-

based organizations might want to ask for in order to get to interchangeability

Potential Framework for Patient Advocacy

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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This geo-political analysis can lead to ideas for what patient-

based organizations might want to ask for in order to get to interchangeability

Potential Framework for Patient Advocacy

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 post-marketing pharmacovigilance to prove similarity after-the-fact

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This geo-political analysis can lead to ideas for what patient-

based organizations might want to ask for in order to get to interchangeability

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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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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Maurice (Mo) Mayrides Teléfono: +51 (1) 2215518

Cel. +51 994681819 mmayrides@esperantra.org

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