multinational partnerships orphan drugs rare diseases jan 21 2016 final pdf

Orphan Drugs Rare Diseases or Disorders

Reaching out to pa3ents and markets in La3n America

Basking Ridge, NJ. January 21, 2016

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© Mul3na3onal Partnerships® LLC 2

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Fernando Ferrer

Orphan Drugs for Regulators, HCPs and Industry

Orphan Drug Regulatory Status

Rare Disease

Cancer

Medical and Marke3ng Approach


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Fernando Ferrer

Rare diseases are beyond our current scope of control

–  ~ 7,000 diseases. Nearly 95% have no cures or treatments yet. –  80% of rare diseases have iden3fied gene3c origins whilst others are the

result of infec3ons (bacterial or viral), allergies and environmental causes, or are degenera3ve and prolifera3ve.

–  50% of rare diseases touch 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 to all these diseases.

References are available upon request


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Fernando Ferrer

FDA Orphan Drugs 1983 – 2015

0

250

500

Requested Designa3on Approved

516

Vs. Requested: in average 70% received Orphan Designa3on status, and 12% were Approved



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Fernando Ferrer

Designa3ng an Orphan Product: Drug and Biological Products

•  FDA, Q&A. “What if the sponsor has difficulty finding data on prevalence? What if data is not available? What are the best prevalence es4mate resources? What should a sponsor do if the best resource they can find is 10-‐20 years old (or from other countries only)?”

–  “If data is old, the sponsor should explain why the data is s4ll per4nent and, if from a foreign source, why data with that country’s populaBon could also be representaBve of US populaBon.”



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Is La3n America a foreign source representa/ve of US popula/on?

420 to 560 million people with a Rare Disease in the world

EU 30 – 40 Million USA 25 – 30 Million 10 La3n American popula3on considered: Argen3na, Brazil, Chile, Colombia, Cuba, Ecuador, Mexico, Peru, Puerto Rico, Venezuela

10 La3n American countries: 40 million

USA 10 La/n America 2016 -‐ Popula/on in Million 321 524 White/Mes/zo/Hispanic -‐ Million 257 363 WMH in % 80% 69% Black/African/Mulato/Moreno -‐ Million 41 127 BAMM in % 13% 24% Indigenous/Other -‐ Million 23 34 IO in % 7% 7%


INTRODUCING ORPHAN DRUGS FOR RARE DISEASES IN LATIN AMERICA

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Growing Strategies


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Fernando Ferrer

Orphan drugs to treat rare diseases calls for different strategies vs. the tradi3onal

marke3ng of the pharma industry

An entrepreneurial, mul3disciplinary, and systema3c approach to unveil areas of opportunity for the product and the rare disease, and with each one of the many stakeholders

The expansion to La3n America demands a rela3vely low investment but working with an experienced business team with cultural awareness in both, the interna3onal, and the local environments


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Fernando Ferrer

Some “few” in Rare Diseases •  few products in the pormolio of a rare disease company •  few local data about the RD •  few specific diagnos3c •  few treatment op3ons for few pa3ents diagnosed and

treated by few MD/specialists •  few sales reps and MSL in the field, but also with the

mindset/sensibility to work in RDs •  few units delivered, in few (or unique) dose and

pharmaceu3cal form


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Some “many” in Rare Diseases

•  many unrelated therapeu3c areas

•  many misdiagnosis, and diagnosis by elimina3on, ”it is not this or this ... perhaps is that”

•  many stakeholders, including social media

•  many family / rela3ves / geographies involved

•  many thousands of dollar per treatment

•  many opera3onal procedures to get reimbursement


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Strategic Management principles to capture the market

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Strategy Formula3on

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Strategy Implementa3on

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Control

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Environmental scanning


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A resource-‐based model to determine core and dis3nc3ve competencies

Resources

Capabili3es

Competences

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Effec3ve assessment of the poten3al markets by research and interviews

Gain knowledge on KOLs / HCPs’ percep3ons

and prac3ces for a specific disease en3ty

Get the KOLs / HCPs’ perspec3ve on an

innova3ve product using the target product profile

Suitable sample size u3lizing properly defined screener that we define in a case by case basis with the companies

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The Stakeholders needs must be addressed independently of the market size

1.  Pa3ents, caregivers and pa3ent advocacy organiza3ons 2.  Non-‐governmental (NGO) and Inter-‐governmental (IGO) Organiza3ons 3.  Clinicians, nurses, pharmacists, biochemists, other health-‐related professionals

and their professional associa3ons (HCPs) 4.  Life science-‐related industries and services, and their associa3ons 5.  Employers and unions 6.  Na3onal legisla3ve, execu3ve, and judiciary powers 7.  Government agencies 8.  Civil society 9.  Ins3tu3onal 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 representa3ves 12.  Healthcare policy makers at the federal, state and local levels 13.  Healthcare researchers and research ins3tu3ons 14.  Interna3onal and suprana3onal organiza3ons 15.  Media and communica3on channels.

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Other key considera3ons when developing the business opportunity

•  The 5Ps marke3ng mix in rare diseases: Product-‐Promo3on-‐Place-‐Price-‐Pa/ent

•  Con3nuous monitoring and par3cipa3on in local regula3ons

•  Innova3on in communica3on & market access

•  The profile of the local organiza3on in an “Organic Marke/ng Partner” business model

•  Support local ac3vi3es of Advocacy Groups

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Conclusion •  For the purposes of prevalence data La3n America might be

considered representa/ve of US popula/on

•  40 million people suffering from some rare disease in the 10 selected popula3ons of La3n America

•  The marke/ng approval and market access regula/ons are constantly evolving, demanding a con3nuous monitoring and ac3ons/investments from the companies penetra3ng the growing and highly-‐demanding La/n American market

•  The internal capabili/es of the mul3na3onal companies can be supplemented with experienced external teams, so as to grow successfully in La3n America while using and adap3ng the core competences of the entrant organiza3on

If you see something

do something !

Pa3ents, Families, Governments, Industry, Pa3ent Associa3ons, Health and Insurance Providers need the help and support of each other

Thank you

Fernando Ferrer Phone + 1 908 326 6417 -‐ [email protected]

®

Mul3na3onal Partnerships® is a Registered Trade Mark of Mul3na3onal Partnerships LLC

multinational partnerships orphan drugs rare diseases jan 21 2016 final pdf

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