The Other Side of Pinktober

It’s October, or Pinktober, as we witness the transformation of basically everything, from tortilla chips, to dogs, clothes, billboards, feather boas, and all of social media for breast cancer awareness. It’s time to refresh the message. We are aware of breast cancer. What many aren’t aware of is that a specific type of breast cancer, metastatic breast cancer (MBC), has no cure. It will kill every single person diagnosed with it. That’s a far cry from all the hopeful pink washing that happens every October. It’s not pink, cute, fluffy, or fashionable. The only thing that will save those with MBC is research that will drive cures and improve the lives of those living with the disease.

So what’s it like living life with MBC?

During our #bcsm tweet chat 2 weeks ago, a question was posed about what we each could do to bring awareness to MBC during Pinktober. I struggled for 2 weeks to try to find a good answer. One I could act upon. I needed to DO something.

As an oncology patient advocate, I’m passionate about elevating the patient & carepartner’s voice. I am a firm believer in sharing patient stories. That’s where the authenticity is. That’s where we can all learn and best support each other. I have been blessed to be a part of the #bcsm community and have learned so much over the last 2 years. One night it dawned on me. I wanted to bring all of the lived MBC patient and carepartner experiences to reside in one location. While anyone can look up the meaning of MBC, it’s hard to find what it’s like really living with MBC.

My colleague and good friend, Jen Horonjeff, is the founder of Savvy Cooperative, a patient co-op that emphasizes the importance of including patient insights in designing healthcare. Savvy’s patient community recently launched a new initiative: Animate Health. Animate Health does just that, it goes beyond the clinical and textbook definitions of a condition and helps animate what people actually go through in light of a diagnosis and how it impacts them physically, socially, emotionally, financially, and beyond.

Animate Metastatic Breast Cancer

I asked Jen if we could create one for MBC and she was fully supportive and spear-headed the initiative. We would like this to be our way as advocates to bring awareness to the patient experience of living with MBC during the month of October (and beyond!). We would like to understand what areas are of most interest to the MBC community. We want to hear your stories of living with MBC, the stories of loved ones, and the stories of those who have been stolen too early from us by MBC.

We would be honored if you would join us in this quest to bring reality to Pinktober and breast cancer awareness as we know it. Let’s animate MBC for those who don’t understand its dramatic impact. Our hope is that everyone who wants to can contribute their thoughts, experiences, and stories throughout the month of October and continue the conversation throughout the year. Those with MBC know that awareness does not end when the month of October is over.

Fulfilling a promise

In one of my last messages to Beth Caldwell, a fierce advocate for MBC who was killed by this horrible disease, I told her how grateful I was to have met her through the #bcsm tweet chats and how much I have learned from her. I also promised her I would find every way I could to drive more awareness about MBC and to elevate the patient voice. I hope I can work towards fulfilling that promise with this project and with your support. Please let me know if you have any questions.

If you would like to help Animate Metastatic Breast Cancer, please:

• Visit Savvy’s Animate Metastatic Breast Cancer

• Register to create a Savvy login (all information on Savvy is kept private, never sold)

• Once logged in, select Animate Health from the menu, click on “Metastatic Breast Cancer”

• Review the current animation categories that span a wide variety of topics (currently over 20 cards)

• Add comments or stories in any or all categories with your lived experiences and perspectives

• Add a new category if a topic of interest is not represented

• Rate how much these areas impact your life, on a 0–5 star scale (no impact to huge impact)

Questions?

Reach out to jen@savvy.coop or enlighteningresults@gmail.com (Grace)

I look forward to continuing to learn from all of you. Looking forward to refreshing breast cancer awareness together.

The Secret to Authentic, Human Centered Point-of-Care

Traditionally, point-of-care (POC) has been the space where diagnoses or treatment decisions are made or where disease state education takes place, typically within the confines of doctor’s office or hospital. Patients and their carepartners no longer live in this arbitrary space. Why do we continue to incentivize this model? Why do we continue to prioritize and dedicate billions of dollars annually to innovating and marketing in this outdated space? Why do we continue to design innovations and digital tools that can only assist patients within the confines of this traditional space, as opposed to connecting the dots and streamlining continuity of care where it happens the most, in real-life?

We must stop visualizing POC as a stationary moment and open the aperture to see the potential of a dynamic continuum of care. We must stop viewing POC as where the doctor is and explore the broad spectrum of places where patients are and live. Disease state education, treatment decisions, and the pursuit of health predominantly happens beyond the 4 walls of the exam room and hospital. POC is happening at retail clinics and via telemedicine. Social media has led to the development of powerful peer-to-peer networks that support patients and carepartners with round-the-clock dialogue with global perspectives and expertise. We must stop solely focusing on digital, sleek, buzzword-filled innovations for the future. We must not forget the power of the human touch, rolling up our sleeves and venturing out into local communities to connect with real people in their daily lives. A study recently published by the NEJM reports on the successful management of uncontrolled hypertension in non-Hispanic African American males at barbershops. You read that correctly. Barbershops. Specialty-trained pharmacists fostered trusting relationships with barbershops and their clientele and demonstrated significant reductions in blood-pressure with medication management. This may be the most powerful POC yet. We need to blow the doors off of this example of real-life POC STAT.

Think about your life right now. Where are places in your local community that you faithfully frequent? Perhaps it is a house of worship, where you exchange in conversations with others from your local community regularly. Conversations about health are happening here. Perhaps it is a barbershop or, for ladies, a beauty parlor or nail salon. Some people may skip their follow-up appointment with their primary care doctor, but not their monthly haircut, color and set, or gel manicure appointment. Conversations about health are happening here. Perhaps it’s the local laundry mat or dry cleaners. Information about health is being exchanged here. Perhaps it’s the local deli or bagel shop. Some may skip filling their prescriptions, but never their Sunday paper, coffee, and buttered rolls. Some may not manage their blood glucose or count calories regularly, but they’ll never skip playing their lucky numbers at the corner bodega. We have become so wrapped up in innovating to improve healthcare that we have become impervious to the sound of the pulse of our local communities. Where is all of the budgeting and innovation for these avenues? Where are the priorities? Where are these conversations?

Please tell me. I’m here waiting, listening.

Grace Cordovano, PhD, BCPA is a board-certified oncology patient advocate, patient experience enhancer, and Citizen Health 2018 Ambassador.

Follow her on Twitter: @GraceCordovano

Shattering the Mold: It’s Time for Reality Conferences

Thematically and structurally, the majority of healthcare conferences are all predominantly (sadly) the same. A celebration of successes, achievements, and pats-on-the-back. Shows of sheer brilliance. Accomplishments beyond the average person’s wildest dreams. Demonstrations and exhibits that are mesmerizing to the beholders’ eyes. Thought leaders in sharp suits with pocket squares or classic pumps are illuminated in spotlights, while the audience sits in silence with their eyes fixated on the stage. Reporters are scattered throughout the room, furiously taking down words of triumph for their next breaking press release.

Healthcare conferences are addicted to demonstrations of achievement and, in ways, rightfully so. Healthcare and medicine are not for the faint of heart. Grit, resilience, foresight, dedication, blood, sweat, and extreme sacrifice pave the way to success. But why do we continue to omit shortcomings, barriers, failures, and the herds of pink elephants that stand among us? Why do we continue to solely feed attendees with promises of potential, displays of futurism, and innovations that push the boundaries of tangibility, while avoiding discussions of real-time, foundational problems that need attention NOW? Poverty, mental health, addiction, substance abuse, drug prices, healthcare costs,barriers to access, fax machines, poor coordination of care, lack of universal access to information, food deserts, impact of tobacco and alcohol, and social isolation only scratch the surface of the daily realities of those struggling to achieve true wellness. Bionic contact lenses and sending a Tesla to Mars aren’t going to help any patient today or tomorrow.

Highly respected conferences have some of the most admirable, powerful, and influential people in attendance as well as gracing their microphones. How ironic is it to have the likes of these people spending the majority of their time at a conference sitting in silence, focused on listening to unidirectional informational flow? Phones are encouraged to be silenced. Live tweeting and sharing of information from presentations is some cases still considered controversial. Limited networking breaks offer small talk over wellness beverages or individually brewed espressos topped with the endless search for a free outlet to charge whatever electronic device is on the brink of dying. This is not innovation. This is not disruption. The is not advancing anything besides perhaps personal agendas.

What should the purpose of conferences be? Is there anything more valuable than making connections, bridging silos, and collaborating to dismantle barriers in real-time? Have you ever attended a conference and witnessed something actionable happen on stage? What if participating speakers were encouraged to address pressing problems as part of their speaking commitment? Taking a play from the show, Who Wants to be a Millionaire, what if speakers were offered lifelines, such as phone-a-friend, to begin connecting the right people and influencers to address major problems patients are struggling with right now? I guarantee the majority of invited keynoters have the necessary connections within the confines of their contacts to truly move the needle. What if leaders with solutions could dial in virtually and be given the opportunity to join problem solving sessions? Imagine if phones at conferences were ringing off the hook like on a Wall Street trading floor, with solutions and ideas pouring in, truly connecting the dots. What if the criteria for giving a keynote address was driven by which attendee connected the most dots in real-time by deadlines set throughout the duration of a conference? Talk about flipping the script! Keynote speeches would then become real-time draft plans as to how a problem affecting patients was going to begin to be solved. Uncharted and rough dirty? Yes, but all of the world’s most brilliant diamonds start that way.

Is this going to be well scripted and seamless? Far from it. Could this get messy? Absolutely! Are there going to be disagreements and heated debates?I hope so. Tough times call for tough discussions. Reality TV has produced some of the most memorable and highly acclaimed TV shows. Is it time to consider reality conferences in healthcare?

There are only 2 camps of people in healthcare: those who want to transform it and those who just want to talk about transformation. How can we create and execute a never-been-seen-before conference that gives those that reallywant to roll up their sleeves, connect the dots, and truly get stuff done an opportunity to showcase what they are made of and capable of doing?

Grace Cordovano, PhD is a professional oncology patient advocate, patient experience enhancer, and Citizen Health 2018 Ambassador.

Follow her on Twitter: @GraceCordovano

Direct-To-Consumer Genetic Tests: Who Are They REALLY Benefitting?

The FDA approved the Personal Genome Service Genetic Health Risk (GHR) Report for BRCA1/BRCA2 (Selected Variants), the first direct-to-consumer BRCA test by 23andMe that may be performed in the comfort of one’s home without the need for a prescription. Press releases and even the FDA announcement do not thoroughly detail the many limitations and conditions associated with the test. Individuals many incorrectly interpret what the test’s results may show or may over-extrapolate how the test is meant to be used. In healthcare, it is of essence to be considerate of people’s health literacy levels so as to encourage informed decision-making based on factual, credible information. It’s also important to be fully transparent as to how the test’s results are benefitting the actual company, in this case 23andMe, as well as other third party companies. This needs to be spelled out front and center. Not just buried in the legalese of the terms and conditions and privacy policy.

Here are the limitations, caveats, and fine print that everyone needs to know about regarding the latest FDA approved 23andMe offering:

1. The test only detects 3 out of more than 1000 BRCA mutations. BRCA1/BRCA2 breast cancer gene mutations are most common in Ashkenazi Jewish populations. This test does not benefit the general population.
2. A negative result does NOT rule out the possibility of other BRCA mutations which may pose an increased risk in cancer.
3. Not all cancers are BRCA mutation driven or hereditary. Some cancers develop sporadically. A negative result does NOT mean one has no risk for developing cancer.
4. People who test positive should seek the guidance of a medical professional and understand that they will most likely be retested in a supervised, clinical setting.
5. A positive test result may lead to fear, anxiety, and distress.
6. Patients and clinicians should NOT make medical decisions, such as opting for prophylactic surgery or anti-hormone therapy, based on 23andMe test results.
7. This test is NOT a substitute for consulting with your doctor for cancer screenings, genetic counseling, or managing lifestyle factors that may impact one’s cancer risk.
8. Anyone with a strong family history of cancer should seek professional medical guidance for appropriate management.
9. Your web behavioral, genetic, or self-reported information may be used by the company internally to improve their services or sold to third party companies for research, marketing and advertising, or commercial purposes. Read the privacy policy and the terms and conditions.

The test may only benefit a very small population of people, specifically those of Ashkenazi Jewish descent, however it is being marketed and offered to the general public. People will purchase the test for a variety of reasons. Regardless of what their reasons are, who the test benefits (or doesn’t), all of the people who submit their saliva samples for testing will have their data and results utilized in one way or another or sold to third party vendors for commercial purposes. Any improvements in company services, commercial, or research advancements that happen as a consequence of using customer data offers zero recognition or compensation for their “participation”. In the end, who really gets the most benefit?

Pharma's 50 Shades of Gray: Including Patients in the Clinical Trial Life Cycle

“Is there a form I could fill out to provide my feedback?”

The silence in the room was deafening.

I exhaled with a furrowed brow, feeling extremely disappointed, wanting to stand up and apologize on behalf of the entire room that was seated so quietly. Apologize for the practices, procedures, and ideologies that had failed this woman panelist on stage, and so many others. Apologize on behalf of an industry that is advancing on so many levels, yet dropping the ball in too many priceless ways.

“Is there a form I could fill out to provide my feedback?”.

This simple question came from a vibrant young woman who just delivered a powerful narrative that brought a quiet room to a roar with the applause of a standing ovation.  This young woman detailed her harrowing patient experience of dying of advanced cancer to a tremendous success story reveling in the miraculous power of personalized medicine. She is years post the clinical trial that saved her life.

“Is there a form I could fill out to provide my feedback?”.

If she hadn’t stood at the podium and told us her story, no one would’ve known the horrors she witnessed, felt, experienced, or thought. If she hadn’t stood on the stage and articulated her story, no one would have known about her difficulties of finding the trial that saved her life. Without her story, no one would’ve heard her suggestions for improvement. If she just sat in her seat like every other member of the audience, no one would’ve known how scary, how exhausting, and how painful her journey was. Thank goodness she was invited to share her story! Otherwise, she’d just be another anonymous clinical trial endpoint successfully met.

It’s gravely concerning that after participating in a successful trial, no one invited her to help improve the process. If an educated, well-spoken, personable individual with a miraculous response to a cutting-edge therapy isn’t included in the enhancement, refinement, and co-design of clinical trials, who will be?

It’s common to hear from patients that they are not asked for their insights during or after participation in clinical trials. There is an aura of reverence surrounding people who have participated in trials. These individuals have made a profound sacrifice and leap of faith. Trial participants have agreed to introduce a foreign substance, a hypothesis, into their body, realizing that this unknown may save their life, end it, or run the gamut of all of the possibilities in between.

Imagine being diagnosed with terminal cancer, being sent home to die. Shattered. In physical, mental, and spiritual pain. Would you risk trusting an unknown therapy and introducing it into your frail, already dying body? Could you picture yourself taking an experimental agent and hoping for the best? How many of us are truly brave enough to put our trust and fate into the hands of science? Wouldn’t you agree that these experiences are profound, rare, and worth their weight in gold to thoroughly comprehend?

There are over 21,000 clinical trials listed in clinicaltrials.gov as recruiting in the US.  Were these trials designed to be tailored to the lives of the people living with a diagnosis and inclusive of all lives with a diagnosis? It depends on whether the expertise of patients and carepartners were included throughout the continuum of the clinical trial life cycle.

Over the last year, it’s been my personal curiosity to ask members of pharma if their company includes patients in the clinical trial life cycle. When I broach the subject, there is a broad spectrum of reactions, the majority of which are accompanied by clear discomfort, agitation, defensiveness, dismay, and confusion. These responses all fall into an ambiguous gray area. Here are 50 answers I have received in response to the question: Does your company include patients’ expertise and insights in the clinical trial life cycle, from study design planning, during trial implementation, after trial closeout, and post-launch of product?

1.     There are compliance issues.

2.     There are regulatory hurdles.

3.     What business incentive is there to do that?

4.     Patients don’t know anything about clinical trial design, management, or operations.

5.     We don’t have a budget for that!

6.     The FDA doesn’t require patient feedback or insights in the clinical trial process for drug approval.

7.     (Look of confusion)

8.     Our company is patient-centered. We don’t need patient insights.

9.     We don’t have the staffing to do that.

10. Pharma doesn’t traditionally speak with patients.

11. Our stringent timelines cannot accommodate this.

12. Where would we find patients to participate?

13. (eyes narrowing) Are you one of those patient activists?

14. Patients aren’t well spoken.

15. Patients aren’t professional enough to participate in these conversations

16. Our legal team would go into cardiac arrest.

17. We do not have the technology in place to do this.

18. It sounds like a great idea but upper management would never approve.

19. Our marketing team is working on a campaign.

20. We only work with non-profits.

21. It’s on the backburner for this fiscal year.

22. This is a conflict of interest.

23. We’re one of the top biopharmaceutical companies in the industry. We know what we are doing.

24. Do you know how much it costs to execute a clinical trial?

25. (eye roll)

26. Our clinical operations team runs like a well-oiled machine.

27. Do you have any references or data to support the benefit of including patients in clinical trial design?

28. Patients are unreliable and volatile.

29. That’s a waste of time.

30. Working with patients is a pain in the @ss.

31. How could pharma work with patients? They don’t trust us!

32. Sounds great in an ideal world, but we have pressing deadlines to meet.

33. Patients have their own agenda and won’t collaborate.

34. We can’t base our clinical trials on patient complaints.

35. Our company runs successful trials and gets approvals without patients involved in the process.

36. That’s another trend that will phase out soon.

37. Whose responsibility would that even fall under at our company?

38. We are watching to see how other companies are doing it.

39. Those tactics are for companies that can afford the “bells and whistles”.

40. We tried. It didn’t work.

41. We are too small of a company to implement this.

42. We don’t need more paperwork.

43. What is the return on investment?

44. We’ll talk to patients but we can’t pay them.

45. We are too big of a company, with locations all over the world. How could we coordinate this?

46. We are so overwhelmed, we can’t take on another initiative.

47. Is there a standard operating procedure for implementing this?

48. What happens if they all start talking about the adverse events of their treatments?

49. How can we trust patients?

50. Our investors don’t care about patient-centered practices and co-design. They want drugs going to market.

Pharma’s 50 shades of gray does not run industry wide. There are pioneers who are committed to changing the status quo, actively working with patients and including them on many fronts. It is evident that these trailblazers are excited by their partnerships with patients. They speak loudly and proudly about their patient-centered initiatives, with an enthusiasm that is invigorating and resounding. They acknowledge that this isn’t a just a nice gesture or outreach program. Partnering with patients is solid business sense.  Collaborating with patients bridges silos, brings meaning to daily work, and creates unique opportunities for experiential learning.

It is important to note that those within pharma who collaborate with patients are more open to leveraging technology and machine learning, discussing mobile health, digital platforms, and telehealth, exploring benefits of utilizing blockchain, as well as recognizing the importance of patient-reported outcomes. Coincidental? Absolutely not.  There is unprecedented value and power in including patients in optimizing the drug lifecycle. Augmenting trials to patients’ values, preferences, and needs is a solid win on all fronts. Do not mistake this as another passing trend!

Is your company somewhere in the 50 shades of gray? What is holding you back? Is your company a pioneer in being authentically patients first and collaborating with patients? How can early adopters share best practices to eliminate every single one of these 50 gray areas so as to collectively move the industry forward to being truly patients first?

The Need for Healthcare Experience Literacy

As an advocate and carepartner for over 20 years, I have seen things I can not unsee. I have experienced things I can not forget. I have heard things I can not unhear. I have come to understand all of my experiential learnings as reflection of a single, major omission in US healthcare. We don't teach anyone how to be a proactive member of the US healthcare system. I'm not just talking about patients or their carepartners. What about healthcare policy decision makers? How about those within pharma designing new medications? What about innovators creating new solutions to advance healthcare?  Shouldn't we all have the same foundation of knowledge to be members of our healthcare system? How are we preparing the next generation for healthcare?

Our pre-K through grade 12 public education is lacking severely in healthcare experience literacy. Literacy alone is not enough. Health literacy not enough. This is a call-to-action for healthcare experience literacy. Imagine the possibilities of ingraining the essentials of the healthcare system incrementally from early childhood through young adulthood! See my latest publication for more details: 

https://medium.com/savvy-cooperative/conquering-the-next-frontier-healthcare-experience-literacy-97eb5f0ffd8d

Book Review: In Shock by Rana Awdish, MD

This May at the Cleveland Clinic Patient Experience Summit , I had the opportunity to hear a mind-blowing talk by a physician named Dr Rana Awdish. Her story was a personal account of how she literally died at her own hospital, losing her entire blood volume into her abdomen due to a ruptured liver tumor. Pregnant at the time, she lost the baby. She continued with her journey of pain, suffering, recovery, and healing while the audience sat pin drop silent, tears and sobbing audible. Her experiences as a patient transformed her understanding of medicine and how it should in actuality be practiced; with compassion, with empathy, and through making space for suffering and vulnerability. Needless to say, I couldn't wait to dive in to the pages of her book. Here's my review. 

Dr. Awdish tears down the walls housing her personal experiences as a doctor turned patient. She masterfully guides the reader through multiple vantage points: as a doctor expecting a child, to the loss of a child and her own “death”, to miraculous survival, painful determined recovery, multiple set backs, surgeries, and medical emergencies, with victories and profound revelations throughout.  You will need to put the book down to dry the tears that fall out of pure sympathy. The reader will experience complete disbelief that a single person could possibly endure such devastation, pain, and grief.  The story captivates with a raw authenticity, transporting the reader through a broad spectrum of emotions the reader couldn’t possibly be prepared to experience: disbelief, grief, devastation, sadness, victory, joy, and true hope.  Dr. Awdish’s deep inner reflections are juxtaposed with her seemingly endless battle for life, providing a thought-provoking framework for redefining the terms patient experience, patient engagement, doctor satisfaction, and the meaning of true healthcare. There is no doubt that medical education must be refreshed after experiencing this harrowing account.

Readers will undoubtedly leave this book permanently changed and rocked to the core. In Shock professes a convincing perspective on the need for compassion and human connection in medicine.  Dr. Awdish makes a clear case for the need for more empathy for doctors, patients, and their carepartners. She is a visionary enlightened by her profound experiences as a patient. This should be required reading by every medical and nursing student, every hospital administrator, technician, and staff member. There isn’t a single person who wouldn’t benefit from reading In Shock and experiencing the consequent opening of the soul first-hand. 

Gratitude

Pausing to reflect can be a powerful, grounding moment. I’m grateful for all that I am blessed with. A loving, supportive family. Happy, healthy children. A comfortable home. More than enough food to eat. A circle of incredible friends. A career path that is challenging and rewarding. Health and a mind filled with burning curiosity.

If I had to narrow my focus and more clearly define what I’m most grateful for, it would be the people in my life. They are priceless. My children, my husband, my family and friends. Additionally, there are the people I’ve met through my advocacy work. The patients. The carepartners. The advocates. The physicians. The nurses.  The students. The academics. The innovators. The disruptors. The challengers of the status quo.  The endless numbers of people who inspire me and leave me speechless with their works. For the people who disagree with me and challenge me to continuously consider other points-of-view.  For the many who continue to push and motivate me, and show me there is good happening in the world.

On this Thanksgiving, I’m eternally grateful for all of the people in my life.  For the people who make time to be a part of my life. For the people who continue to welcome me into theirs.

Wishing all of you and yours a very Happy, Happy Thanksgiving!

Photo by Pro Church Media on Unsplash

My (REJECTED) Letter to the Editor

          As a researcher, I can fully appreciate the review process and recognize that there is simply only so much space allotted in any given journal. My recent submission was a Letter to the Editor of the New England Journal of Medicine (NEJM) regarding a perspective “Saying Goodbye to Lectures in Medical School- Paradigm Shift or Passing Fad?” by Schwartzstein et al, on alternatives to traditional teaching formats in medical school.  Given the elite status of the NEJM, I wasn’t entirely surprised to receive a rejection. Perhaps my simple little opinion wasn’t written in cream-of-the-crop style. The more concerning alternative is that the submission was rejected because it was not representative of the masses of submissions received or the popular vote. Perhaps no one else feels strongly about incorporating patients and care partners in the design and delivery of medical education. I find this gravely alarming.

            Revising the manner in which knowledge is delivered, i.e., virtually, in Q&A sessions, flipped classrooms, augmented reality, etc., addresses only part of the issue of sustained learning in medical trainees. It is important to incorporate the power and influence of storytelling to enhance the medical education experience. It is important for curated curricula to foster life-long learning not only in students, but also in the medical experts responsible for shaping young minds.  Current medical curricula offer learning as a 1 way street with information being disseminated from professors to students. Medical education is missing patient and care partner stories and experiences. More on that here: http://www.kevinmd.com/blog/2017/07/every-medical-student-needs-hear-patient-caregiver-stories.html

            Here’s my formal rejection from NEJM in response to my submission below. Does anyone else believe in the power of incorporating patient and care partner stories to enhance medical education?

Dear Dr. Cordovano,

I am sorry that we will not be able to publish your recent letter to the editor regarding the Schwartzstein article of 17-Aug-2017.  The space available for correspondence is very limited, and we must use our judgment to present a representative selection of the material received.  Many worthwhile communications must be declined for lack of space.

Thank you for your interest in the Journal.

Sincerely,
XXXXXXXX Editor

(Original Submission) To the Editor,

It was disappointing Schwartzstein et al. (Aug 17 issue)1 did not include patients and care partners in the design, development, and delivery of medical education. Students are only learning part of the story from cadavers and medical experts at respective institutions. We must foster the creation of curricula that are not only engaging, thought provoking, and evidence-based, but also unite patients and physicians, promote shared-decision making2, and delivery of compassionate care. Patients and care partners bring an authentic breathe of experience and perspective3 that textbooks and medical experts cannot impart or convey. It’s time to invest in participatory medicine and welcome patients and care partners to share their experiences living with disease significantly earlier into the careers of medical trainees. This model offers a unique opportunity to foster interactive, collaborative learning for all involved: medical trainees, seasoned medical experts, and patients and care partners. A change in the culture of medical education is more profound than revising curricula alone.

References:

1.     Schwartzstein R, Roberts D. Saying Goodbye to Lectures in Medical School- Paradigm Shift or Passing Fad? N Engl J Med 2017; 377: 605-607

2.     Barry M, Edgman-Levitan S. Shared Decision Making- The Pinnacle of Patient-Centered Care. N Engl J Med 2012; 366: 780-781

3.     Awdish R. A View from the Edge. N Eng J Med 2017; 376: 7-9

The Glaring Omission in Kymriah Drug Approval Press Releases

Novartis received F.D.A. approval for Kymriah (tisagenlecleucel), as the first CAR-T gene therapy approved in the US for pediatric and young adult patients with relapsed B-cell acute lymphoblastic leukemia (ALL).  Kymriah is being hailed as a paradigm and life changing therapy. Clinical studies have shown that within 3 months of treatment, patients achieved overall remission rates of 83%, survival of at least six months by 89%, with a 79% chance of survival of at least a year. These are tremendous clinical feats for patients with limited to no treatment options. The price for the single treatment therapy is $475,000. Novartis is working with the Centers for Medicaid and Medicare Services where patients will only be responsible for payment if they respond after 1 month of treatment. The company has also reported it would provide assistance to those without insurance and the underinsured.

The CEO of Novartis Oncology, Bruno Strigini, said, “We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program(1). The majority of press releases (2-14) reporting the approval all had one glaring omission. Not a single press release formally recognized the importance of the patients that volunteered to participate in the trials. Not a single one expressed gratitude for the dedication of the care partners that committed their time to the success of these trials. Many trials fail to recruit and retain enough patients (15), leading to early termination, delays, or inadequate data for statistical reporting. Considering Kymriah is a treatment geared to a very specific subset population of ALL, it is incredible that these trials were able to accrue and retain the necessary numbers of patients to generate statistically significant findings.  Clinical trial participation requires dedication, sacrifice, and an uncanny belief in the unknown.  Imagine the degree of mental, physical, emotional, and spiritual duress a parent or guardian experiences in deciding whether their child, who is in the throes of a terminal cancer diagnosis, should participate in a clinical trial.  In the cases of pediatric and young adults patients, one parent, if not both, often need to significantly decrease the number of hours they may work, take an unpaid leave of absence, or leave their job permanently to assume the role of care partner for the patient so as to follow trial protocol. One can only imagine the impact on a single parent household. While there were clearly never seen before successes, with many precious lives saved, it wasn’t a miraculous ending for ever patient that was treated.  There are also life-threatening sides that come with treatment. Even the success stories may have had many dark moments along the way.

At a time when the healthcare industry is exploring and investing in initiatives to promote patient-centricity, it is important to acknowledge all stakeholders involved in successes such as these.  While Kymriah is undoubtedly a groundbreaking approval representative of the epitome of exceptional science, dedication, and innovation, let’s not forget to credit the patients and care partners who made this innovation and approval a reality as well. While researchers may have dedicated years of their careers developing the basic science and pharma may have invested close to a billion dollars to bring this living drug to market, the most invaluable part of Kymriah’s product lifecycle was patients’ volunteering their lives to the development of what’s being hailed as historic breakthrough science.

Resources:

1.     Novartis. (2017, Aug 30). Novartis received first ever FDA approval for a CAR-T cell therapy, Kymriah (tisagenlecleucel, CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice [Press Release]. Retrieved from https://novartis.gcs-web.com/novartis-receives-fda-approval-for-KymriahTM

2.     Berkrot, B. (2017, Aug 30). Novartis gene therapy approval signals new cancer treatment era [Press Release]. Retrieved from http://in.reuters.com/article/us-novartis-fda-idINKCN1BA1ZY

3.     Cortez, M., Edney, A., &Paton, J. (2017, Aug 30). Breakthrough cancer therapy for dire cases gets FDA approval [Press Release]. Retrieved from https://www.bloomberg.com/news/articles/2017-08-30/breakthrough-genetic-treatment-for-dire-cancers-approved-by-fda

4.     The Food and Drug Administration. (2017, Aug 30). FDA approval brings first gene therapy to the United States [Press Release]. Retrieved from https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

5.     Garde, D. (2017, Aug 30). Pioneering cancer drug, just approved, to cost $475,000- and analysts say it’s a bargain. [Press Release]. Retrieved from https://www.statnews.com/2017/08/30/novartis-car-t-cancer-approved/

6.     Glenza, J. (2017, Aug 30). US approves first cancer drug to use patient’s own cells- with a $475,000 price tag [Press Release]. Retrieved from https://www.theguardian.com/us-news/2017/aug/30/cancer-drug-kymriah-leukemia-novartis

7.     Grady, D. (2017, Aug 30). FDA approves first gene-altering leukemia treatment, costing $475,000 [Press Release]. Retrieved from https://www.nytimes.com/2017/08/30/health/gene-therapy-cancer.html?mcubz=3

8.     Herper, M. (2017, Aug 30). Novartis CEO’s dilemma: Is $475,000 too much for a leukemia breakthrough? Or is it not enough? [Press Release]. Retrieved from https://www.forbes.com/sites/matthewherper/2017/08/30/novartis-ceos-dilemma-is-475000-too-much-for-a-leukemia-breakthrough-or-is-it-not-enough/#772e4a77556e

9.     Nedleman, M. (2017, Aug 30). FDA announces first US gene therapy approval for cancer treatment [Press Release]. Retrieved from http://www.cnn.com/2017/08/30/health/fda-first-gene-therapy-leukemia/index.html

10. NPR. (2017, Aug 30). FDA approves first gene therapy for leukemia [Press Release]. Retrieved from http://www.npr.org/sections/health-shots/2017/08/30/547293551/fda-approves-first-gene-therapy-treatment-for-cancer

11. Ramsey, L. (2017, Aug 30). A medical breakthrough that hacks genes to fight cancer just got approved, and it’s the beginning ‘of a big new field of medicine’ [Press Release]. Retrieved from http://www.businessinsider.com/why-the-fda-approved-kymriah-a-car-t-cell-therapy-to-treat-cancer-2017-8

12. Scotti, A. (2017, Aug 31). New cancer drug’s ‘astronomical price’ only affordable for the 1% [Press Release]. Retrieved from http://www.nydailynews.com/life-style/health/new-cancer-drug-astronomical-price-affordable-1-article-1.3458071

13. Smith, M., Goodman, B. (2017, Aug 30). FDA approves first-of-its kind cancer treatment [Press Release]. Retrieved from http://www.webmd.com/cancer/news/20170830/fda-approves-breakthrough-cancer-treatment

14. Univeristy of Pennsylvania. (2017, Aug 30). FDA approves personalized cellular therapy for advanced leukemia developed by University of Pennsylvania and Children’s Hospital of Philadelphia Retrieved from https://www.pennmedicine.org/news/news-releases/2017/august/fda-approves-personalized-cellular-therapy-for-advanced-leukemia

15. Kolata, G. (2017, Aug 30).  A cancer conundrum: Too many drug trials, too few patients [Press Release]. Retrieved from https://www.nytimes.com/2017/08/12/health/cancer-drug-trials-encounter-a-problem-too-few-patients.html?mcubz=3