1. Fernando Ferrer RAPS 2016 Sept 20 FINAL PDF

La#n America, when and how to enter the orphan drugs markets

Fernando Ferrer, MBA

Mul/na/onal Partnerships LLC

Disclaimer

The views and opinions expressed in this session are those of the individual presenters and should not be aAributed to their employers or RAPS.

Fernando Ferrer, MBA Mul/na/onal Partnerships LLC 2

Abstract

•  Orphan Drugs are medicaments for Rare Diseases and low incidence Oncological Diseases. They are developed by the life science industry in order to treat, improve the health condi/on and quality of life of individuals and families affected by those diseases.

•  Star/ng with the US Orphan Drug Act of 1983 various na/ons have, created incen/ves and regula/ons to encourage companies to develop and offer orphan drugs.

•  The UN and Interna/onal Organiza/ons have launched universal declara/ons to grant people the right to health, and to guide ethics in healthcare. Countries across the globe include pa/ents’ right in their cons/tu/ons, as well as other norms to grant universal access to beAer health.

•  La/n America is aligned with this interna/onal trend and, liAle by liAle, people gaining access to new diagnoses, devices and medicines to treat rare diseases and cancer.

•  Pa/ent & associa/ons, health care prac//oners and providers, governments, industries and interna/onal organiza/ons must work together in a number of ini/a/ves across La/n America to accelerate the broad and fast adop/on of orphan drugs within the region


Content

Orphan Drugs and Rare Diseases in La1n America

①  the 1me is now ②  market access is a right ③  the value is much higher than the cost ④  ini1a1ves Conclusion


“First they came for the Socialists, and I did not speak out— Because I was not a Socialist.

Then they came for the Trade Unionists, and I did not speak out— Because I was not a Trade Unionist.

Then they came for the Jews, and I did not speak out— Because I was not a Jew.

Then they came for me— and there was no one leY to speak for me.”

-‐ Friedrich Gustav Emil Mar/n Niemöller, Lutheran pastor 14 January 1892, Lippstadt, German Empire – 6 March 1984, Wiesbaden, West Germany

①  the 1me is now


Rare Diseases are

•  OYen chronic and life-‐threatening •  ~ 7,000 diseases. Nearly 95% have no cures or treatments yet.

–  80% of rare diseases have iden/fied gene/c origins whilst others are the result of infec/ons (bacterial or viral), allergies and environmental causes, or are degenera/ve and prolifera/ve.

–  50% of rare diseases affect children. –  30% of children with rare disease will not live to see their 5th birthday –  Rare diseases are responsible for 35% of deaths in the first year of life

•  No single country or geographical/economic region is providing diagnosis and treatment for all these diseases.


FAQs About Rare Diseases

Rare diseases became known as orphan diseases because drug companies were not interested in adop#ng them to develop treatments. The Orphan Drug Act (ODA) created financial incen/ves to encourage companies to develop new drugs for rare diseases.


0

250

500

Requested Designa/on Approved

…and the industry responded!

516

FDA Orphan Drugs 1983 – 2015e Vs. Requested in average: 70% received Orphan Designa/on status,

and 12% were Approved

References are available upon request


Rare Diseases should not be mistaken for neglected diseases •  Neglected tropical diseases (NTDs)

–  are a group of parasi/c and bacterial infec/ous diseases(*) –  are a diverse group of communicable diseases that prevail in tropical and subtropical

condi/ons in 149 countries and affect more than one billion people, cos/ng developing economies billions of dollars every year. They mainly affect popula/ons living in poverty, without adequate sanita#on and in close contact with infec/ous vectors and domes/c animals and livestock. (**)

18 neglected tropical diseases

Sources: (*)The END Fund. (**) World Health Organiza/on (WHO)

Buruli ulcer Lympha/c filariasis Chagas Mazza disease Mycetoma Dengue and Chikungunya Onchocerciasis (river blindness) Dracunculiasis (guinea-‐worm disease) Rabies Echinococcosis Schistosomiasis Foodborne trematodiases Soil-‐transmiAed helminthiases Human African trypanosomiasis (sleeping sickness) Taeniasis/Cys/cercosis Leishmaniasis Trachoma Leprosy (Hansen's disease) Yaws (Endemic treponematoses)


Pa/ents with Rare Diseases in La/n America

Popula/on: 625 Million •  Rare Disease 6% – 8% = 37 -‐ 50 million SA, 415,

66%

CA&C, 87, 14%

NA, 123, 20%

446 -‐ 600 million people with a Rare Disease in the world

EU 30 – 40 Million USA 25 – 30 Million 19 La/n American 587 m popula/on : Argen/na, Brazil, Chile, Colombia, Ecuador, Mexico, Peru, Puerto Rico, Venezuela

Cuba, PR, DR, T&T, Guatemala, El Salvador, Honduras, Nicaragua, CR, Panama

19 top La#n American countries:

47 million Fernando Ferrer, MBA Mul/na/onal Partnerships LLC 10

Access to orphan drugs is granted in La/n America by diverse norms

interna/onal trea/es and conven/ons

na/onal cons/tu/on

federal law

statutes / codes

decrees

②  market access is a right


The Universal Declara/on of Human Rights (1) Everyone has the right to a standard of living adequate for the health and well-‐being of himself and of his family, including food, clothing, housing and medical care and necessary social services, and the right to security in the widowhood event of unemployment, sickness, disability,, old age or other lack of livelihood in circumstances beyond his control.

(2) Motherhood and childhood are en/tled to special care and assistance. All children, whether born in or out of wedlock, shall enjoy the same social protec/on.


Interna/onal Covenant on Economic, Social and Cultural Rights (ICESCR)

Par/es and signatories to the ICESCR: signed and ra/fied (164) signed but not ra/fied (6) neither signed nor ra/fied (27)

Ar#cle 12 1. The States Par/es to the present Covenant recognize the right of everyone to the enjoyment of the highest aTainable standard of physical and mental health.

It commits its par/es to work toward the gran/ng of economic, social, and cultural rights (ESCR)


Physician-‐Pa/ent rela/onship is the cornerstone of medical prac/ce

“The health of my pa/ent will be my first considera/on” -‐  Declara/on of Geneva, World Medical Associa/on

“A physician shall owe his/her pa/ents complete loyalty and all the scien/fic resources available to him/her” -‐  Interna/onal Code of Medical Ethics


American College of Physicians (ACP) Ethics Manual Physicians have an obliga/on to promote their pa/ents' welfare in an increasingly complex health care system. This entails

forthrightly helping pa/ents to understand clinical recommenda/ons and

make informed choices among all appropriate care op/ons. It includes management of the conflicts of interest and mul/ple commitments that arise in any prac/ce environment, especially.

It also includes stewardship of finite health care resources so that as many health care needs as possible can be met, whether in the physician's office, in the hospital or long-‐term care facility, or at home.


Rare Disease regula/ons in La/n America

Argen/na Brazil Chile Colombia Mexico

ANMAT ANVISA ISP INVIMA COFEPRIS

Law 26689, EPF/2011 Na/onal law for rare diseases. Disposi/on 4622 August 2012

Decree 2577/2006 – 768/2006, RDC 28/2007 RDC 16/2008 allows for priority review-‐

Ricate Soto Law January, 2015; medica/ons for rare diseases and cancer. Law 19966 AUGES-‐GES

Law 1751 of 2015. Law 1392 of 2010 and Law 1438 of 2011; orphan designa/on Decree 481 of 2004

Ar/cle 224 Bis Mexican Health Law, Official GazeAe, January 2012; orphan drug designa/on


Prevalence and Incidence of rare diseases: Bibliographic data

These low incidence diseases are being treated in La/n America.

Why?

Source: Orphanet report series , March 2016 Fernando Ferrer, MBA Mul/na/onal Partnerships LLC 17

The stakeholders needs must be addressed independently of market size 1.  Pa/ents, caregivers and pa/ent advocacy organiza/ons 2.  Non-‐governmental (NGO) and Inter-‐governmental (IGO) Organiza/ons 3.  Clinicians, nurses, pharmacists, biochemists, other health-‐related professionals and their

professional associa/ons (HCPs) 4.  Life science-‐related industries and services, and their associa/ons 5.  Employers and unions 6.  Na/onal legisla/ve, execu/ve, and judiciary powers 7.  Government agencies 8.  Civil society 9.  Ins/tu/onal health care providers, such as hospital systems and medical clinics 10.  Purchasers and payers, such as employers and public and private insurers 11.  Health care industry representa/ves 12.  Healthcare policy makers at the federal, state and local levels 13.  Healthcare researchers and research ins/tu/ons 14.  Interna/onal and suprana/onal organiza/ons 15.  Media and communica/on channels.

③  the value is much higher than the cost


Diagnosis and treatment for few pa/ents are more costly

Who is gonna pay the bill !?

Source: The Telegraph, UK, CREDIT: REUTERS


The ques/on should be: Who is gonna pay the bill if we do nothing? More people… more needs Humankind took millions of years to grow to 1 billion people … and only 113 years to reach 2 billion

… and 95 years to become 8 billion !

•  Fernando Ferrer, MBA Mul/na/onal Partnerships LLC 20

Who should be diagnosed and treated?

Individual Society . .

•  Fernando Ferrer, MBA Mul#na#onal Partnerships LLC •  21

The common good must include the individual

The need of the individual must be properly addressed

It is not about the person the society. It is about the person AND the society


La/n America is the place for Orphan Drugs

ü  We have 50 millions pa/ents

ü  The global industry has developed / is developing diagnos/cs, medicines, devices and treatments that are ready for LA

ü  The HCPs are well prepared and trained

ü  Health Care centers have the ability / resources to treat pa/ents

ü  There are interna/onal and na/onal regula/ons and codes

ü  Pa/ents are being treated using local budgets

Nothing is missing but you ! Fernando Ferrer, MBA Mul/na/onal Partnerships LLC 23

Challenge

To bring beAer health and quality of life for individuals and families affected by rare diseases and cancer

④  ini1a1ves


Ini/a/ves (1) Centers for Rare Disorders

Areas in health care facili/es for the diagnosis, treatment and supply of Orphan Drugs – Mul/disciplinary team of Health Care Prac//oners (HCPs), in situ and remote

–  Collabora/on with local and interna/onal health care systems, public and private organiza/ons including academic ins/tu/ons, and KOLs

–  Treatments with Approved Orphan Drugs and with Orphan Drugs’ Designa/on status granted by US/PR and EU


Ini/a/ves (2) Newborn Screening & Pa/ent Registry

Homogenize/Harmonize and expand the implementa/on of – newborn screening (NBS) programs – pa/ent registry

to allow beAer monitoring of the disease incidence and prevalence, which is essen/al to make reliable predic/ons for disease management across La/n American


Ini/a/ves (3) The year of Rare Diseases in La/n America

Following the European Year for Rare Diseases 2019 •  Pa/ents/rela/ves/associa/ons, healthcare prac//oners and providers, governments, industries, na/onal and suprana/onal organiza/ons, any and all stakeholders are invited to support the Year of Rare Disease in La/n America.


Conclusions

①  50 million of pa/ents to receive Orphan Drugs in La/n America

②  There are regula/ons in place. The right of pa/ents to have access has being granted and the medical ethic supports it

③  The way is open; pa/ents are receiving high cost treatments already

④  New ini/a/ves will help accelerate the adop/on of Orphan Drugs in La/n America. There are strategies and knowledge suitable for each market

What are you wai/ng for to offer your products in La/n America?


Thank you Muchas Gracias Muito Obrigado

Fernando Ferrer Mul/na/onal Partnerships LLC

+1 908 219 9291 fferrer@mul/na/onalpartnerships.com

Orphan Drugs and Rare Diseases La/n America

1. Fernando Ferrer RAPS 2016 Sept 20 FINAL PDF

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