The Rise (Rare Disease) Selling Framework: Rising Above Traditional Skill Sets. Transformational Rare and Ultra-Rare Disease Therapeutics Demand Transformational Customer Interfacing

By Peter W. Carbone

The RISE (Rare Disease) Selling Framework is meant to be disruptive! It is designed to help commercial teams identify how to inspirationally RISE and become a rare voice for rare disease patients. It is challenging oneself and not the healthcare professional. In keeping aligned with biopharmaceutical organizations' respective mission statements, industry's customer interfacing teams must always keep patients top of mind. It is an important journey, a journey of putting patients first by challenging oneself to reach beyond and become a cogent rare voice for rare disease patients. The RISE Selling Framework is a transformational approach that focuses on Inspiration. It is about mustering the daily will and skill to reach beyond, because it is the patients who are fighting every day to reach beyond. They are creating Inspirational steps that lead to Inspirational stories. We must RISE every-single-day because our healthcare professionals RISE every-single-day and our patients RISE every-single-day to fight, touch others, and share their gifts. RISE, it's a new day. Are you ready to reach beyond your current skill set for rare disease patients?

• Language: English
• Publisher: AuthorHouse
• Release date: Dec 16, 2019
• ISBN: 9781728328775

Peter W. Carbone

Peter W. Carbone has worked in the biopharma industry since 1992 with Fortune 500 companies. His responsibilities are comprised of leadership, sales and learning & development within the biopharma sector, which include therapeutics for an array of diseases and conditions. Throughout his tenure within the industry he learned that customer interfacing within the rare disease market is different and require a shift in approach and mindset. In terms of other interests, Peter is an avid martial artist where he has earned belts in multiple disciplines. He also enjoys walking to his own beats (so to speak) - he is a drummer.

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© 2019 Peter W. Carbone. All rights reserved.

No part of this book may be reproduced, stored in a retrieval system, or transmitted by any means without the written permission of the author.

Published by AuthorHouse  01/23/2020

ISBN: 978-1-7283-2876-8 (sc)

ISBN: 978-1-7283-2877-5 (e)

Any people depicted in stock imagery provided by Getty Images are models,

and such images are being used for illustrative purposes only.

Certain stock imagery © Getty Images.

Illustration © Andrea Montano (https://www.montanogallery.com)

Because of the dynamic nature of the Internet, any web addresses or links contained in this book may have changed since publication and may no longer be valid. The views expressed in this work are solely those of the author and do not necessarily reflect the views of the publisher, and the publisher hereby disclaims any responsibility for them.

Dedicated to my beloved departed sister, TARA

Who has inspired me

To Always Reach Above!

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A fleeting, unfinished life – a lifelong inspiration!

Extraordinary Rare Mountain of Inspiration

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I pledge a journey for my rare mountain view

With a snow topped peak and a blazing red hue.

I dare to climb that battered, cold peak

And risk my life as I stride and seek.

From here, I see the ceaseless time

The sweat, the tears, the never-ending climb.

I see a river of risk coursing down that mountaintop –

Others may recoil but I’ll never stop. (No, I’ll never stop!)

Not even after I’m atop that blazing red hue –

That rare summit of inspiration – marveling its view!

It is a cataclysmic wonder – a perplexing thought

That one would evade such a journey, a journey yet sought.

As for me, I pledge to embrace my

extraordinary rare mountain view,

And become awestruck atop its blazing red hue.

Why firmly risk fate with no hesitation?

It is my extraordinary rare mountain of inspiration.

Peter Carbone

The RISE (Rare Disease) Selling Framework©

Readiness – Inspiration – Signals – Energizer

Rising Above Traditional Skill Sets. Transformational Therapeutics

Demand Transformational Healthcare Professional Interfacing

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CONTENTS

Chapter 1   What is Rare About the RISE Selling Framework?

Chapter 2   Defining Rare and Ultra-Rare Diseases (Orphan Diseases)

Chapter 3   The RISE (Rare Disease) Selling Framework & Passion

Chapter 4   Readiness

Chapter 5   Inspiration & The Rare Disease Pyramid

Chapter 6   Signals

Chapter 7   Energizer

Chapter 8   A Rare and Transformational Approach (Incorporating Adult Learning Principles)

Chapter 9   A Rare Closing

CHAPTER 1

Readiness – Inspiration – Signals – Energizer

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What is Rare About the

RISE Selling Framework?

What is Rare About the RISE Selling Framework?

Why do those affiliated with rare and ultra-

rare disease sectors (orphan diseases)

require a shift in mindset?

No treatment options. No diagnosis. No research. No cure. No diagnostics. No financial means. No solution. No support. No voice.

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Imagine the plight of rare and ultra-rare disease patients. Imagine the pain and suffering of people and their family members who live with a rare or ultra-rare disease. These are often life-threatening or severely debilitating diseases. Imagine the frustration of forging ahead down a path that upon every turn, you find a frozen, dead end. In fact, the more you pledge to forge ahead, the more you grow mystified. For rare disease patients, it is not only hearing the sounds of but also walking against a bitter cold, unforgiving, wind-driven rain, which relentlessly attacks their vulnerable bodies and frightened souls. Even caregivers and parents of these rare disease patients suffer. In fact, they catastrophize, which will often spread throughout and negatively impact all elements of their lives. They must feel as if they are adrift upon a fading star. How can this happen to me? Why isn’t there a solution? Why my child? What now? As daunting as it is for many a patient and many a family, there is reason for hope.

RISE, it’s a new day! Are you ready? We are now witnessing past medical limitation getting obliterated by advanced technologies. Through the dedication and imagination of many, are innovative solutions for the relatively few who have become afflicted with rare diseases. According to EvaluatePharma® worldwide prescription drug sales will continue to grow by approximately 5% per year, leading to a forecast of nearly $1 trillion in sales by the year 2020. [1] In terms of regional breakdown comparing 2012 to 2013 growth, prescription drug sales grew in the United States, Germany, France, Italy, England, Spain and Japan by 7.2%, 8.5%, 1.8%, 4.2% 6.3%, -2.9% and 0.7%, respectively. [¹] As biopharma companies increase their gross revenue, they also increase their investment in research and development (R&D). Much of this R&D is in pursuit of solutions for unmet medical needs. The US Orphan Drug Act of 1983 provided pharmaceutical companies incentives for rare disease drug development. In fact, new drugs serving unmet medical needs is one of the key value drivers for research-based pharmaceutical companies.

The biopharmaceutical industry is not acting on their own accord with this endeavor. It is reasonable to say there is a shared international commitment by patient advocacy groups, government research supported by funding, scientists, medical doctors, and a host of other critical stakeholders who have contributed to a surge of success in the rare and ultra-rare disease sectors. Organizations like the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) are strong advocates for the continued support and search for life altering and life-saving therapies. The European Organization for Rare Diseases (EURORDIS) is a unique, non-profit alliance of 864 rare disease patient organizations from 70 countries. They work in tandem to improve the lives of an estimated 30 million people living with a rare disease in Europe.

Another organization is the International Rare Diseases Research Consortium (IRDiRC), which support rare disease patients and has expressed their two all-encompassing objectives: to contribute to the development of 200 new therapies and the means to diagnose most rare diseases by the year 2020.[2] As a result of impressive concerted worldwide efforts, the Consortium’s 2020 goal for 200 new therapies was achieved three years ahead of schedule. Moreover, the goal for diagnostics is within reach. [²] The Genetic and Rare Diseases Information Center (GARD) is another organization for rare disease patients. They maintain a list of diseases to help healthcare professionals and patients find reliable information.

Rare disease advocacy organizations help rare disease patients in a multitude of ways. Most notably, they are aware of how a rare condition has been coded for other patients with the same diagnosis. They may be able to recommend one or more International Classification of Diseases (ICD) codes providers can utilize for patients’ medical records, health insurance reimbursement, administration, epidemiology and research.

In short, the new millennium has brought with it transformational medical technologies and medical breakthroughs, which now requires a shift in mindset from healthcare professionals and the commercial function of biopharmaceutical companies who are marketing within the complex rare and ultra-rare disease sectors. It is a mindset that must match these transformational medical breakthroughs. Exciting new technologies are shaping the rare and ultra-rare disease landscapes. To illustrate, cytologists have the ability to view an individual’s karyotype, which is an organized profile of a person’s chromosomes. The advancements of companion and complimentary diagnostics, genetic marker testing as well as next generation sequencing (NGS), to name a few, have annihilated the restricted walls that limited comprehension of some rare and ultra-rare diseases and opened a new frontier of disease state management, enabling new hope and new solutions. Scientists have even mapped the entire human genome. Undeniably, more diseases are treatable by enzyme replacement therapy or substrate deprivation. Science leads to greater advancements, more awareness, increased disease diagnoses, research and publications.

But, what matters most? For years, rare disease patients have been resigned to the fact that they have to battle their afflictions on their own or were limited to the support of loved ones. They draw strength they find from deep within each other. They often must fight, not with an arsenal of medications that cure their diseases, but with grace and hope. A patient may be subject to several diagnoses by multiple diagnosticians before the right conclusion is reached. Even worse, imagine a patient being repeatedly informed that there is no diagnosis from which he or she is suffering. The healthcare professional is simply unable to diagnose the condition. In fact, Your problem may simply be in your head – they call it psychosomatic. Perhaps you should go to a psychiatrist.

As explained by Susan Ann Vitale in her online October 2017 publication titled: Individual and Family Issues Surrounding a Rare Disease, Individuals with a rare disease are geographically separated and may encounter care by providers unfamiliar with their disease. They often face diagnostic delays, inadequate treatment options or a lack of hope for a cure. Economic and societal implications include high costs associated with morbidity and mortality. While there is some research on individual rare disease experiences, a review of experiences common to a wide variety of rare diseases for potential commonalities has not been established.

For some rare or ultra-rare disease patients, there are solutions, but now they require a strong voice for others to notice and that is the nature of the RISE Selling Framework. In reality, there are unmet needs for millions of patients around the world, many people afflicted with diseases that are currently being studied. Still other unmet needs will soon be reached while today, more and more are currently approved for use. Today, there is hope for some patients who find themselves searching for answers; hoping someone is speaking on their behalf.

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What is rare about the RISE Selling Framework? It brings to healthcare professionals – possibilities! It brings to patients – hope! Think about it. With the right fortitude, skill set and desire, one person can inspire another to help a patient who otherwise may not be helped. It takes a team of scientists, hundreds of millions of dollars of investments, state-of-the-art technology and thousands of others to create a product, with the goal of filling an unmet medical need. Considering all those resources and effort, it may take only one person to create the necessary awareness that leads to a rare disease patient gaining access to therapy via a healthcare professional who is willing to listen.

A representative’s responsibility is to encourage an appropriate yet impassioned dialogue. It must be a dialogue that is designed to get a healthcare professional to stop looking in the past when referencing a disease or condition and become forward thinking; to consider the possibilities and realities of today’s advancements. It is harboring one’s desire to reach beyond the obstacles and impossibilities and becoming a vessel of transformative results that lead to shifts in mindsets and aspirational achievements. Together, we have the wherewithal to make a significant difference for those who have unmet needs; to become a song of hope and healing. Indeed, that is the essence of the RISE Selling Framework – to inspire others to help others. To reach beyond.

Today the biopharma industry finds itself in a new day, which begets its own internal unmet need. This is the need to shift the minds of their respective commercial teams. The rare disease and ultra-rare disease businesses of the biopharmaceutical sector require a significant mindset shift with not only the healthcare professionals but also the biopharmaceutical industry who has recently entered this rare but exciting space. But, there is so much more room for advancements in both finding solutions to unmet needs and the way in which the industry may inspire others to help others. In short, these treatments are transformational, therefore, the commercial teams’ approach must also be transformational.

What is the RISE Selling Framework? It is disruptive. It is designed to help commercial teams identify how to inspirationally RISE to the occasion of becoming a rare voice for rare disease patients. Commercial teams must elevate their skill set, transform their approach and enhance their knowledge base enabling them to become cogent voices for patients. As we work through the anatomy of the RISE Selling Framework, you will learn the importance of challenging yourself to expand your talents, creativity and understanding of the impact you may have for the rare disease community.

To some degree, you will better understand the difficult journey to which the rare disease patients are subject. The success of this framework however is dependent upon one’s experience, expertise, integrity and finesse. If you don’t believe in the product for which you are responsible, the data or disease, you won’t engage healthcare professionals with passion and you may appear forceful. If you are only concerned about your business, RISE is not for you.

This is a framework that requires a team to sincerely believe that they can make a difference for patients and families; to be a rare voice for rare disease patients. If that is the fuel that will motivate you in the field, then the RISE Selling Framework will absolutely help you engage critical healthcare professionals, which will help them identify appropriate patients. This is a tool that has the potential to enrich rare lives. Which lives? Turn the page.

Imagine the possibilities for rare disease patients who often suffer with life-threatening and/or severely debilitating symptoms…

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Anatomy of a Successful Rare and Ultra-

Rare Disease Representative

(Evolving Needs, Transformational Approach)

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CHAPTER 2

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Defining Rare and Ultra-Rare Diseases

(Orphan Diseases)

Defining Rare and Ultra-Rare Diseases

(also known as Orphan Disease)

According to the National Organization for Rare Disorders (NORD) and the Food and Drug Administration:

In the United States,

is the condition Too

Rare to Care?

• There are approximately 7,000 known rare diseases.

• Approximately 80% of rare diseases are genetic.

• Approximately 50% of all rare diseases affect children.

• Rare diseases are defined differently around the globe. In the United States, a disease is rare if it afflicts fewer than 200,000 people, annually.

• Approximately 30 million Americans are afflicted with a rare disease.

• Approximately 250 million (~4%) people worldwide are afflicted with a rare disease.[3]

http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm293213.htm

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Globally, is the Condition

Too Rare to Care?

According to the Global Genes:

• 8 in 10 Rare Diseases are caused by a faulty gene.

• 95% of Rare Diseases lack a FDA approved treatment.

• The average time it takes for rare disease patients to receive an accurate diagnosis is 8 years.

https://globalgenes.org/rare-facts/

According to Michael Beck in his editorial Rare and Ultra-Rare Disease:

• In Japan, orphan disease is a condition with a prevalence of 2.5 cases per 10,000.

• In Europe, an orphan disease is defined as a disorder that affects 5 or less per 10,000.

• The definition for Ultra-Rare disease is universally lacking. In the United Kingdom it is described as a prevalence of less than 1 case per 50,000.

• In the United States an ultra-rare orphan disease is defined as a disorder that affects less than 2,000 people.

According to the International Federation of

Pharmaceutical Manufacturers & Associations (IFPMA):

• The rare disease prevalence (cases per population) and definition of rare disease across various counties:

o United States of America: 7.5 per 10,000 (affecting fewer than 200,000).

o European Union: 5 per 10,000.

o Japan: 1 per 2,500.

o Singapore: ~37 per 100,000 (affecting fewer than 20,000 patients).

o Russia: 1 per 10,000.

o Australia: 1 per 10,000 (affecting fewer than 2,000 people per year).

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The delineation between prevalence and incidence is important.

• Prevalence: The proportion of persons who have a condition at or during a particular time period.

• Incidence: The proportion or rate of persons who develop a condition during a particular time period.

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Title 21, section 316 defines rare disease drugs (aka, Orphan Drugs) and provides procedures to encourage and facilitate the development of drugs for rare disease or conditions, including biological products and antibiotics. The FDA