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

Robotics, Technology, and the Patient’s Clipboard

As a little girl, The Jetsons used to be one of my favorite shows to watch. An animated sitcom produced by Hanna-Barbera, The Jetsons featured a quaint family of 4 living in a space-age, futuristic era where daily life was rendered simpler by the glories of robotics, whimsical advancements, and high-tech innovations. I can still close my eyes and picture myself as a 6 year old, daydreaming about what the future would be like. 

Fast forward to August 2017. I’m watching a client struggle to wrestle their insurance card out of their wallet while juggling a clipboard with patient intake papers, her phone, and a water bottle.  Peripheral neuropathy stemming from many cancer treatments has made grasping things difficult and excruciatingly painful.  “But nothing has changed since last week, do you still need another copy of my insurance card?” asks the exasperated patient, still trying to free the pesky card from the tiny pocket in her wallet. “Office policy”, replies the receptionist curtly without looking up, “And fill out the forms because we are updating the records in our system”. There are 3 pages of forms. The patient has been coming here for over 2 years. All of the information is EXACTLY the same. She slowly fills out everything as diligently and neatly as she can, refusing my offer to help do it for her.

As a private patient advocate, I often accompany patients and their care partners to a broad spectrum of appointments, spanning follow-ups with their primary care physicians to specialists such as oncologists, cardiologists, or rheumatologists, to imaging centers for MRI’s, CAT scans, PET scans, and mammograms, to hospitals for pre-admission testing, surgical consultations, or procedures, or to phlebotomy labs for routine blood draws. I’ve observed hundreds, if not thousands, of patient encounters with front desk receptionists. No matter what the setting, there’s a common denominator routine that unites most of these experiences:

(front desk receptionist):  “Please fill out this paperwork (handing a clipboard to the patient) and I’ll need a copy of your insurance card.”

The moments that follow can be filed under “Complete Waste of Time”.

• Patients can’t get their insurance card(s) out of their wallet.
• Patients can’t find their insurance card in their wallet because the last time they were asked for it they were so flustered juggling the clipboard they shoved in a random spot.
• Patients begin filling out the intake forms they’ve filled out many times before.
• Patients realize they didn’t bring their glasses so they can’t actually read the form that’s also  often poorly photocopied.
• The pen starts running out of ink a third of the way through.
• Patients start looking for a new pen.
• Some patients continue writing, pressing harder on the paper in hopes of reactivating the pen. Many curse, press harder, and rip the form.
• Patients wait for the receptionist to return their insurance card so they may proceed to fill out the insurance information on the intake form.
• Patients try to write their insurance information (ID number, group number, address, member phone number, etc.) on the miniscule lines on the form.
• Patients can’t find the group number on the insurance card. (Insurance companies, take note!)

We are in a fascinating era of robotics, artificial intelligence, virtual/augmented reality, and digital technologies that fulfill people’s wildest imaginations.  The cancer surgical landscape is being transformed by advancements such as the da Vinci System (Intuitive Surgical), a robotically assisted surgical device bringing surgeons enhanced precision, dimension, and control while extending the possibility of minimally invasive surgery to patients https://www.intuitivesurgical.com/products/davinci_surgical_system/.  Robear (Riken), a bear-like nursing robot, can perform care-giving tasks such as lifting patients from their bed, transferring them to a wheelchair, and helping patients stand from a sitting position http://www.riken.jp/en/pr/press/2015/20150223_2/.  Hospital acquired infections may have met their match with Xenex, the germ-zapping robot, which uses high intensity pulsed ultraviolet light from xenon lamps to significantly decrease microbial loads in hospital patient rooms https://xenex.com/video-demos.  And who can resist the cuteness of the PARO seal (AIST), a therapeutic robot that fulfills unmet needs within the animal-assisted therapeutic space http://www.parorobots.com.  This is just a sampling of the wonders out there. There are even wide spread reports anticipating a robotic takeover and fears of automation replacing humans in a variety of sectors. With all of this pure genious rocket science and technology, can someone please explain to me how the hell patients are dealing with clipboards, insurance cards, fax machines, and paper chart folders with 3-hole punched paper records? Dear technology, you’ve missed the mark. You need to go back to the basics and fill in the (MAJOR) gaps before aspiring to have robots take over the world.

Many aspects of healthcare are not the futuristic Jetsons-like encounter I daydreamed of as a little girl. Dear Apple, for the love of all that is holy, please tell me there is an solution in the making that will allow patients to wave their phone Apple Pay-style to confirm basic demographic patient and insurance information so we can simplify the patient in-take process at every healthcare facility touch point. And while you are at it, lets have the capability to pay any co-pays with a second wave of the phone.  I mean enough with this cash, checks, or digging for a credit card nonsense.

Alternatively, at this juncture, it may be easier to perhaps bring in Pepper (SoftBank Robotics), the humanoid robot, into waiting rooms across the nation as an acting patient assistant to fill out the damn paperwork and get the insurance cards out for patients https://www.ald.softbankrobotics.com/en/robots/pepper. I’ll bet you a dollar it may easier than solving the current shortcomings and interoperability issues surrounding electronic health records (EHR) and patient portals.  It may also be more pleasant of an experience than many of the interactions I’ve witnessed with some front desk receptionists. Dear tech companies, looking forward to what you come up with. In the meantime, excuse me while I go find a pen that works for my client.