Rare Voices Podcast

After years of tests, after countless of late-night WebMD searches, and a sea of referrals … after hours on the phone with insurance companies and payment plans. After all of that. That is when you FINALLY receive the words: We think your son has a rare condition.

And just when you thought you would get answers. That — for patients with rare and orphan conditions — is only where your journey begins.

At least that is where the journey began for Monica Weldon, our guest for this episode of Rare Voices.

Monica was able to absorb those words and the emotional roller coaster attached to them and create a network of support and a vision for what advocacy can mean for patients with rare and orphan conditions.

In 2014, Monica founded Bridge the Gap: SYNGAP Education and Research Foundation. While most patient advocacy groups focus on an illness, Monica focused her foundation on the research involving a rare gene mutation. As a result, she has been able to build a coalition with other advocacy groups, research institutions, and, most impressively, with manufacturers.

Today, Monica is a force for change in patient advocacy, an author, and keynote speaker at countless conferences even beyond health care. I’m excited to bring you her story during National Caregivers Month, because, most importantly, Monica has redefined what it means to be a caregiver with what she has accomplished on behalf of her son, Beckett.

Anyone who has invested their time in creating improved outcomes for patients becomes familiar with a kind of balancing act. You must be committed to the science and precision involved in diagnosis and therapy — the research, the development, the trials, the care plan. You must also be devoted to the art of the therapy — the patient’s well-being, the expectations, the lives impacted.

After all, the patients we care for are … people. And so, anyone who has invested time in creating actual, improved outcomes knows: it’s a balance.

Our guest on this episode of Rare Voices is an example of what happens when science meets art. He is a master at understanding how to balance what seem like two competing ideas. Dave Penake, CEO of Saol Therapeutics, combines his training as an engineer and systems designer with his passion for serving patients with rare and orphan diseases.

Previous to Saol, Dave served as Vice President, Commercial Operations at Corcept Therapeutics and held roles of increasing responsibility at Genentech. In addition to his experience in biotechnology and pharmaceuticals, Dave has designed and launched devices and has experience in a number of different technologies.

As you listen to our conversation, be prepared to be provoked by big questions, the kind that will check your balance and set you on a new course — one where you can really put the patient first.

In healthcare, it is easy to become cynical. We are surrounded by the shortcomings of our own industry — it’s easy to criticize and point out where we fall short.

Everyone… frontline workers, patients, doctors, pharmacists, researchers, administrators… we all have, at some time or another, felt powerless to make things better. This powerlessness is made worse by our pandemic and the complications and uncertainties that surround us.

But, once in a while, we meet people who inspire us to pick up our burden and move forward. Rather than preaching from some lofty hilltop, they do this by their example.

This episode of Rare Voices features one of those examples. At least, he has been one for me. Our guest today is Bill Jollie, an account executive for a global drug manufacturing company. But, more importantly, Bill is a husband, a foster parent, a cancer survivor, a nonprofit founder, a Marine, and an inspiring human.

Some people are the unique combination of visionary genius and thoughtful character. They connect the head and the heart. Our guest for Rare Voices Episode 3 is one such person, Ken Greathouse. Ken is a veteran commercialization guru and a great friend. Ken has a decades-long point-of-view on developing innovative therapies with a proven track record in commercialization, sales and marketing for pharmaceuticals. When Ken sold Manchester Pharmaceuticals, he created the Greathouse Family Foundation with his wife, three sons, and daughter-in-law, in an effort to fund research for patients with orphan and rare disorders. The phrase, “That’s the way we have always done it,” does not apply to Ken. If you are ready to be provoked into a new way of thinking about how we could bring new therapies to market for people who need them the most, you won’t be disappointed with this conversation in Episode 3.

One of the least understood components of commercialization is the role of the payer. For manufacturers, it is hard to know how to set the right strategy. For specialty pharmacies, lack of clarity in payer strategy can create challenges with consistent patient experience. For patients, insurance is a black box that they often feel like they have to navigate alone. Our guest for the second episode of Rare Voices, Jim Clement, a partner at Coeus Consulting, demystifies the assumptions that many of us make about payers. Jim has been there. He has seen the good, the bad, and the ugly of commercialization from every vantage point in the system. Simply put: If you want to be successful in bringing new therapies to patients with rare and orphan disorders, then you need to know Jim.

You never get a second chance to introduce your first episode. So, we wanted to start Rare Voices with someone who has granted opportunities for being heard to so many patients, Candace Lerman. Candace, a self-identified disease warrior, is an unconventional advocate for creating a more fulfilling life experience for patients with chronic and rare illnesses. Her work is sprawling — from courtrooms, to policy-maker offices, to living rooms and the virtual world, she has empowered so many, exposed the shortcomings of our healthcare systems, and created new pathways to improved care.

Introducing Rare Voices. The show that reveals the wisest path to a fulfilled life for patients with rare and orphan disorders. Brought to you by the people at Optime Care, a pioneering specialty pharmacy. Hosted by Donovan Quill. Subscribe at www.optimecare.com/rare-voices or anywhere you listen to podcasts.