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Laura Helms Reece of Rho

24/7/2020

Rho CEO, Laura Helms Reece, gained extensive experience in the world of business and pharmaceuticals, before returning home to join the family business. Laura talks to RARE Revolution about how the biggest motivator for her, and her team, is making a difference to people lives, and about the challenges and opportunities that lay ahead for clinical trials

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically Rho and what did that journey look like?

My parents founded Rho in our basement in 1984. My father was a professor of biostatistics and my mother ran a data management group at UNC-Chapel Hill. They started Rho because, as state employees, they were looking to earn extra money to send their kids to college. When they started, they hired graduate students and moonlighted faculty and staff from UNC to help run projects in our basement. I checked data for allowance money as did my brother.

I did not actually intend to stay in the family business. When I went to UNC as an undergraduate, I was a business major. I graduated with my master’s degree and had to decide which direction I wanted to take—stay on the path to become a finance professor or become a biostatistician. On a good day in finance, you make someone rich. On a good day in biostatistics, you make someone healthy. I decided I felt better about making someone healthy.

I still did not want to work in the family business, so in time between earning my master’s degree and getting my doctorate I worked for Glaxo. After my doctorate, I worked for Astra Zeneca in Wilmington, Delaware, and learned a lot about how to develop a drug. Despite having a wonderful experience, I wanted to come home to North Carolina. That is when I talked to my parents about working for the business.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Certainly, over the next several months, and possibly through much of next year, the novel coronavirus (COVID-19) will continue to have a major impact on how clinical trials are run. Patient safety is always a top priority, so it is important we make sure we have the right protocols in place to ensure patients feel safe about participating in clinical trials, especially during this time.

Our teams have helped many sponsors navigate and implement decentralised solutions for their clinical trials and programs. This includes providing recommendations for the level of decentralisation required (e.g. a fully virtual trial or a hybrid approach), as well as the development of strategic plans for remote and central monitoring, technology integration, IP management and site and patient engagement.

Based on our experience, we believe that the best decentralised solutions are patient-centric, customized and rooted in a robust Risk-Based Quality Management (RBQM) framework, as specified by the E6R2 guidance.

Additionally, Rho is coordinating a new study called Human Epidemiology and Response to SARS-CoV-2 (HEROS) to help determine the rate of COVID-19 in children and their family members in the U.S. The study also aims to determine what percentage of children infected with SARS-CoV-2, the virus that causes COVID-19, develop symptoms of the disease, as well as whether rates of SARS-CoV-2 infection differ between children who have asthma or other allergic conditions and children who do not. HEROS is sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH).

3.	What is your proudest moment in your career thus far?

I am proud of the work we do at Rho every single day. It is one of the blessings of working in our industry. Certainly there are huge highlights like the days that a therapy we’ve supported comes to market or a major publication we’ve supported comes out that will change lives for people.

The days when we discover a treatment doesn’t work are heart-breaking. And yet, I take pride in our work to test failed therapies vigorously. When we find a therapy doesn’t work we can help clients and patients move on to more hopeful options. I am also proud of the work we do at Rho to build a business that treats employees and clients honourably.

4.	What and who are your personal and professional inspirations and why?

Personally, I am inspired by my grandmother. She was widowed at the age of 38, with a high school education and three small children, during the height of the polio epidemic. She eventually became a very successful businesswoman but had some hard years. My parents, who both came out of childhood poverty and built a successful business, are also an inspiration to me.

My professional inspirations are many and varied. Dean Barbara Rimer has inspired me as she has overseen the Gillings Global School of Public Health at UNC. She has dealt with years of decreasing state funding while increasing the value that the Gillings School brings to public health. In contrast with Dean Rimer, whom I get to talk with regularly, I am also truly inspired by Dolly Parton, whom I’ve never met. She has built an impressive business empire, brought jobs to the depressed area where she grew up, and funded a number of valuable social support charities. And she did that with big blonde hair and a country twang.

5.	What advice would you give someone considering working in the rare disease space?

This is a space that requires innovation and often presents unique challenges. The rare disease community is one that faces a number of hurdles—if given the opportunity, interact with the patients and caregivers and listen to their voices. Patient and caregiver input in the rare disease space is valuable to researchers throughout the product development process. It is also critical to work closely with regulatory agencies, as the challenges of product development in the rare disease space may require creative study designs and agency feedback and support in uncharted territory.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

The rare disease community is a strong and resilient one, and they have worked hard to give themselves a stronger voice in the local government and afford themselves the opportunity to provide feedback. A state’s government has a critical role to play as to how a given state’s programs and policies can benefit the rare disease community. On a more central level, the Orphan Drug Act provides for status of designations such as orphan drug designation and regenerative medicine advanced therapy designation in order to incentivise companies to develop products for rare disease. The biggest gaps I see are accessibility and affordability. Rare disease therapeutics are extremely expensive to develop and in particular we have seen recent gene therapy products approved for rare diseases that have a high price tag. Congress will need to work with pharmaceutical companies and insurers to develop a strategy for financing these treatments. There are innovative, life-changing therapies being developed (and approved!) and the government must now give thought on how to determine the price of these treatments and how to finance them--the benefits of an innovative treatment should not be denied due to affordability.

7.	What would you say are some of the biggest motivators for your employees?

First and foremost, I would say the nature of our work is the biggest motivator for our employees. As I previously mentioned, on a good day in biostatistics, you make someone healthy. The fact that we get to work with sponsors to bring lifesaving or life-altering therapies to market to help improve health, extend life and enhance quality of life.

I think the culture Rho provides is also a key motivator. We are fortunate to have a collaborative work environment where we value a strong team spirit and are loyal to each other and to our clients—and there is a mutual trust and respect across the entire organisation. Rho also encourages a work-life balance that allows employees to bring their best selves to work while also being passionate about their lives outside of work.

Rho's new headquarters

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The toughest part is that the buck stops here. I am responsible for the decisions that are made at Rho and I take that very seriously. For my first eight years as CEO, I had a co-CEO who was also my brother. Sharing that burden with someone you trust completely makes the burden much easier to carry. I’m glad I had those years. I still have his support, as well as the support of my parents, who founded Rho. But I’m the one who has final responsibility.

The most rewarding part of being the CEO of Rho is getting to work with truly wonderful people. Because almost all of Rho works out of our Durham office (when we aren’t in a pandemic), I get to talk to people throughout the organisation regularly. I love working with the executives and project teams that I interact with frequently. And I also love the conversations I have over lunch in our hub and in the hallways. Rho is full of talented, dedicated, and delightful people.

9.	What would be your one wish for Rho for the year ahead?

I would love for us to increase the number of lives we improve by extending life, enhancing health, and improving the quality of life for the patients. We do that by expanding the number of clients and projects we serve. Our talented teams of experts make a difference in the projects they support. I’d love to help more projects to successfully execute their trials and submit to regulatory authorities world-wide.

10.

If you weren’t CEO of Rho, what was Plan B? What did your 10-year-old self want to do as a job?

My 10-year-old self wanted to be a Broadway star. Sadly, as my 9-year-old recently put it, “Mama just can’t catch a tune.” So that isn’t really a plan B for me!

The truth is, I love business. If I weren’t the CEO of Rho, I’d start another business with a worthy mission. I’m just a business geek.

To find out more about the work of Rho, please visit;
www.rhoworld.com

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Kristine Hoestermann and Theresa Thomas of RARE.

24/7/2020

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Kristine Hoestermann and Theresa Thomas' partnership was formed from a shared passion and drive to raise awareness for rare diseases. RARE. is not just a brand but a movement. They speak to RARE Revolution about what led them to creating RARE. and about their mission to make the invisible - visible

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically RARE and what did that journey look like?

Neither of us had a choice when it came to the world of rare disease, although we wouldn’t change it.

Kristine: At the age of 27 I was an avid runner and noticed a pain in my knee. What I didn't realise then was that pain was the beginning of my rare disease journey. I created RARE. initially as a way to express myself and the change that was happening in my life. After some time I realised that RARE. was a tool that not only could help me but could help others as well. The safe place I created was needed by more people than just myself. I began creating clothes and household items that helped raise awareness for rare diseases. Which eventually led me to connecting with Theresa.

Theresa: Two and a half years ago my son Owen was born weighing in at almost 12 pounds. I immediately knew that something was different about my son. He was very large and his tongue protruded from his mouth. Two weeks later we got his official rare disease diagnosis of Beckwith-Wiedemann Syndrome (BWS). A year after my son was born my husband was pushing me to start a blog. He knew I was struggling with our son's rare disease and he felt that writing would help me express myself. Little did I know six months later I would connect with Kristine through Instagram, and ultimately become her business partner and COO of RARE.

Kristine

Theresa and her family

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

We believe our biggest challenges are exposure and reach. Although we have been connecting with so many amazing families there are still so many people suffering in silence with no community or voice. We want to reach them as quickly as we can so they know that they are not alone.

Some of our biggest opportunities have been, and continue to be, working with philanthropists in the rare disease network. We are working with authors such as Deborah G. Edwards, David Fajgenbaum, and Sarah Ramey. We are currently working on a few projects with non-profit organisations as well as other rare disease fighters, in addition to working on next year's Rare disease day event.

3.	What is your proudest moment in your career thus far?

One thing we value the most is the ability we have created to connect with people across the world. To date RARE. is in 31 states and six countries. We also worked incredibly hard on our shop with a purpose initiative. When you check out on RARE. you now have the ability to choose between several non-profits where 15% of your purchase total will be donated to.

4.	What and who are your personal and professional inspirations and why?

Kristine: Taylor Swift has always inspired me because she built her brand from the bottom up using her own story to touch the masses. She is always authentic and real and connects with her fans despite the criticism and negativity she receives. She continues to stay true to who she is. She built her brand through personal connections with her fans and continues to cultivate kindness and fight for equality which correlates to the brand we are building—one that is authentic and made from genuine connections and stories.

Theresa: My inspiration has always been my older sister Michelle. She was born with one arm but has never let it hold her back. She deals with everything head on and is now a paediatric nurse at one of the top Children’s Hospital. She has faced adversity her whole life but you would never know it. She has always been a source of inspiration and strength for me.

5.	What advice would you give someone considering working in the rare disease space?

Listen first, be patient, and respond with kindness.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

We feel there are gaps from diagnosis all the way through to workplace accommodations. Until we put the patient at the centre of the solutions these gaps will continue to grow.

7.	What would you say are some of the biggest motivators for you?

Kristine: The biggest motivators for me are the kind messages and affirmations that RARE. receives. We are constantly reminded that the work we are doing is reaching the people who need it most.

Theresa: My biggest motivator is my son Owen. Watching him grow and go through life with a rare disease, one that most people have never heard of, continues to motivate me to create change and awareness for him and other BWS children.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

One of the hardest things about being a CEO, one with a rare disease, is allowing myself the time to rest and recover. When you have a dream and a business and tons of ideas it can be challenging when your rare disease gets in the way.

The most rewarding things are seeing the impact that our products and our brand have made. They have helped people find their voice.

9.	What would be your one wish for RARE for the year ahead?

Our wish is that we are able to continue our journey of cultivating change, impacting lives, and creating designs that people resonate with.

10.

If you weren’t CEO of RARE, what was Plan B? What did your 10-year-old self want to do as a job?

RARE. actually was our plan B.

Kristine: Prior to getting sick I was a clinical social worker.
Theresa: Prior to having Owen I was a registered cardiovascular invasive specialist.

Our 10 year old selves both wanted to do jobs that made us happy and had a tremendous positive impact on others. We both were in fields that accomplished that, however our lives took a different turn and we are now able to continue to live out our 10 year old dream through RARE.

To find out more about the work of RARE. please visit;
www.findyourrare.com

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Patrick Howie of MediFind

7/7/2020

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Patrick Howie, CEO of MediFind, knows all too well the challenges patients can face in trying to obtain the right care and learn about new treatment options. Motivated by personal experience, he founded MediFind, to try and ensure precious time isn't wasted; by helping patients find the expertise and treatments they need, faster

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically MediFind and what did that journey look like?

I was at brunch with my brother, Dennis, who had been fighting a rare cancer for four years at that point, when he said, “I’m running out of options, any ideas?” I literally couldn’t breathe for a minute. I also knew that I had to come up with some ideas. While I’m not a doctor, as the head of Global Analytics for one of the largest pharmaceutical companies in the world, I did have access to unparalleled data capabilities and an intricate understanding of the world of healthcare. I realised then that he had lost a lot of time trying to find experts and trying to learn about new treatment options, and that I could leverage analytics to help him, and patients like him, find better care, faster. That’s how MediFind was born.

Determining expertise

One of a kind—example data sources

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Our biggest challenge will be to make sure everything we do is easily understood by patients and their caregivers. Healthcare can be extremely complex, especially the terminology. We are working very hard to translate this complex medical terminology so patients can more easily understand and act on it. Many patients want to be more actively involved in their treatment decisions, and once the medical jargon is removed, they will better be able to use their voice to get the care they need.

3.	What is your proudest moment in your career thus far?

As we started to build out MediFind, an American colleague of mine heard about what I was doing and asked for help. His son had some significant health issues from birth and was being treated at one of the medical centres considered to be a world leader in his condition. They were recommending his son start a treatment, but the father wanted a second opinion. I pointed him to some experts in Europe, who willingly reviewed his son’s history and records, and told the father that the treatment being recommended had recently been shown to not work. The European doctors consulted with the local doctors to come up with a different treatment plan that is dramatically better than the original plan.

4.	What and who are your personal and professional inspirations and why?

Obviously my original inspiration was my brother, but I am now inspired by the millions of people like him around the world who tackle their medical situations head on and are looking for a better way. Everyone deserves the best chances at the best health.

5.	What advice would you give someone considering working in the rare disease space?

In my opinion, passion and persistence are requirements to working in the rare disease space. Tackling a rare disease is particularly challenging because it is hard to get enough critical mass to really drive progress for any single rare disease. The good news is that the rare disease communities are extremely passionate and frequently have an impact much bigger than their numbers would indicate.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

Given that I am based in the United States of America, our approach is obviously quite different from much of the world. While every government faces a situation where there are more demands on resources than can be fully met, I do believe governments are missing out on a big opportunity to help rare disease patients (and, quite frankly, all patients) by not investing in maximising the impact of generic medications. There are thousands of generic medications that alone, or in combination, might be able to help rare disease patients but there is no economic incentive for private companies to explore those options. A government program to maximise the impact of these low-cost treatments could have a huge impact to patients and could reduce costs to the system.

7.	What would you say are some of the biggest motivators for your employees?

Just about every one of our employees has a friend or family member who has struggled with a rare disease. For them, the motivation is easy, as they understand the impact we can make. For those that don’t have that direct connection, I believe they all understand the greater human impact we can have, even if it isn’t as personal for them. We are also solving some pretty tough challenges, so many get an added motivational boost from working on difficult problems.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The most difficult part for me is having to spend more time than I would like on operational tasks. I know they are needed, which is why I do them, but I want to spend as much time as I can trying to solve those problems that will help the most patients.

9.	What would be your one wish for MediFind for the year ahead?

I want to release the first complete and constantly updated list of treatments for every rare disease. I believe that one of the greatest opportunities we have in the near term is to learn from the experience of others and which treatments, or combinations of treatments, have helped patients with a specific disease or condition related to a disease. This information is hard to get, and difficult to synthesize, and we have been working for the past three years to develop a system to capture this information in a comprehensive and ongoing way. This is related to the notion of medication repurposing—many doctors are having success using combinations of readily available treatments to treat rare disease but it is hard to find out what has been tried and what is working (or not).

10.

If you weren’t CEO of MediFind, what was Plan B? What did your 10-year-old self want to do as a job?

The truth is that I didn’t seek out this job, but I knew that there was an important need that we could uniquely solve. My 10-year-old self was blissfully ignorant of rare diseases and I didn’t have a personal connection to them until my 40s, but helping patients with rare diseases has become my life’s mission.

Patrick's brother Dennis - his inspiration

To find out more about the work of MediFind please visit;
www.medifind.com

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Jeff D'Angelo of The CHAMP1 Research Foundation

6/7/2020

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Motivated by a desire to make a difference to his son's life, and the lives of others with the same condition, Jeff D'Angelo co-founded The CHAMP1 Research Foundation. He talks to RARE Revolution about his drive to accelerate research and about his hopes of achieving the ultimate goal of a treatment

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically The CHAMP 1 Reasearch Foundation and what did that journey look like?

My wife Katis and I had our world rocked, completely blindsided. We were convinced that my son's challenges were the result of a birth injury. A phone call on April 6th, 2017, shortly after his 4th birthday, confirmed my son was 1 of 38 kids in the world with a rare genetic mutation to a gene called CHAMP1. It was only discovered a few years before in 2015 and there were only two scientists studying the gene. It became evident that we would not see things progress fast enough to impact this generation of kids unless we took action. We decided if we wanted to see change we would have to drive it ourselves. We immediately launched the CHAMP1 Research Foundation to accelerate research and treatment options for our son JJ and all of the other children affected with CHAMP1.

Jeff and his beautiful son JJ

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

God is teaching me patience! It has felt like an eternity for our community to position ourselves where we now have research tools available. In just under two years we have cell lines and mouse models available to scientists. The opportunities are really just beginning. The biggest challenges ahead are funding research and accelerating the process from basic science to translational research. We have had a tremendous amount of urgency since the beginning and fighting time in the hopes that something will impact this generation of CHAMP1 kids.

3.	What is your proudest moment in your career thus far?

It's hard to see progress when you are so focused on the end goal of a potential treatment, I have to force myself to appreciate what our community has done to advance the research process. The most fulfilling moment was hosting and getting to spend time with 20 other CHAMP families from around the world at our first family conference in Orlando, Florida.

The CHAMP1 Foundation's first family conference in June 2018

4.	What and who are your personal and professional inspirations and why?

Giving my son the best chance at the fullest life possible is what inspires me. My life was changed forever on the day my son was born and he is the reason I’m on this mission, I fight for him every day. I want things to be different for future generations so they don’t experience the isolation that comes with a diagnosis and the challenges of so many unknowns for the future.

5.	What advice would you give someone considering working in the rare disease space?

Anyone looking to get involved in the rare disease community, you are entering an environment which consists of some of the most selfless and inspiring people you will ever meet. I’m not sure you will find a career with a more rewarding atmosphere. Coming from self-experience, your eyes are open to a world of compassion and understanding. Be as relentless as the parents of the children you're fighting for.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

I believe they are moving in the right direction but unfortunately it's slow. It's crucial that patient advocates continue to voice their needs and work closely with policy makers. It’s going to take the rare disease community to drive and accelerate the pace of change. I don’t believe anyone sitting in my position would be satisfied. There is a tremendous amount of difficulty to gain recognition, access funding or get services for rare and often neglected diseases.

7.	What would you say are some of the biggest motivators for your employees?

Currently the community and our partners involved are motivated by the belief in the mission to ultimately help CHAMP1 kids. I think besides the obvious motivation for parents to fight for their children and choosing to live in hope, others are excited to be a part of driving this forward and fostering change.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The toughest part is managing the emotional rollercoaster of raising a child with special needs, working my day job and trying to complete an endless amount of tasks on a weekly basis. It's difficult to put your heart and soul into something you're so passionate about, especially knowing there is still so much to overcome in order to reach your goal. What I find most rewarding is knowing I have the honor to play a part of reaching new milestones. It's a privilege to work with these families, engaging with researchers and traveling the country to learn and collaborate with like-minded people on similar missions.

9.	What would be your one wish for The CHAMP 1 Research Foundation for the year ahead?

My biggest wish is to be in a position where we have gained enough knowledge and have the financial ability to significantly impact the children with CHAMP1. Ultimately to have a treatment in place.

10.

If you weren’t CEO of The CHAMP 1 Research Foundation, what was Plan B? What did your 10-year-old self want to do as a job?

This gave me a good laugh. My 10-year-old self wanted to be a professional athlete, and although I did play sports competitively, that clearly didn’t work out. Even though I wouldn’t have chosen the difficulty of this journey, I feel blessed and my life has been fulfilled with a far greater purpose.

To find out more about the work of The CHAMP1 Foundation please visit;

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CEO
Series

Laura Helms Reece of Rho

Kristine Hoestermann and Theresa Thomas of RARE.

Patrick Howie of MediFind

Jeff D'Angelo of The CHAMP1 Research Foundation

Author

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Categories

Rare Revolution is published by NRG Collective, a not-for-profit media company specialising in rare disease content
Contact Us
rstewart@rarerevolutionmagazine.com

CEO​Series

Laura Helms Reece of Rho

Kristine Hoestermann and Theresa Thomas of RARE.

Patrick Howie of MediFind

Jeff D'Angelo of The CHAMP1 Research Foundation

Author

Archives

Categories

Rare Revolution is published by NRG Collective, a not-for-profit media company specialising in rare disease contentContact Us​rstewart@rarerevolutionmagazine.com

CEO
Series

Rare Revolution is published by NRG Collective, a not-for-profit media company specialising in rare disease content
Contact Us
rstewart@rarerevolutionmagazine.com