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Archive for the ‘Patient-centered Medicine’ Category


Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2017:

This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee

Novartis defends the eye-popping price of its pioneering gene therapy with arguments about its $1 billion expenditure—and novel “value-based” pricing.

https://www.fastcompany.com/40461214/how-novartis-is-defending-the-record-475000-price-of-its-pioneering-gene-therapy-cancer-drug-car-t-kymriah

 

On 8/30/2017 we wrote:

FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of KitePharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/08/30/fda-has-approved-the-worlds-first-car-t-therapy-novartis-for-kymriah-tisagenlecleucel-and-gileads-12-billion-buy-of-kite-pharma-no-approved-drug-and-canakinumab-for-lung-cancer-may-be/

 

The Price for the Treatment was published on 8/31/2017, a Value-based Pricing Payment Model of a $475,000 per patient charge for the responding patients after ONE month of treatment. Novartis says it takes an average of 22 days to create the therapy, from the time a patient’s cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy. 

CAR-T gained national attention three years ago when Carl June, a researcher at the University of Pennsylvania, used to put a young girl’s acute lymphoblastic leukemia. Genetically altering the girl’s immune cells had made her deathly ill, but June had used a Roche drug, Actemra, to treat the side effects. She lived, and the results were published in The New England Journal of Medicine. Novartis bought the rights to the Penn treatment for just $20 million up front.

Pharma Buying the right to use from an Academic Institution is a known route to leap frog the R&D lengthy process of Drug discovery.

“I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis’ Chief Executive Joseph Jimenez, told Forbes in a cover story about the work then.

The FDA calls this CAR-T therapy treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S., according to the FDA.

Two gene therapies for rare, inherited diseases have already been approved in Europe.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA’s Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

FDA commissioner Scott Gottlieb in a statement.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” 

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

The Protocol

A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

https://www.technologyreview.com/s/608771/the-fda-has-approved-the-first-gene-therapy-for-cancer/?utm_campaign=add_this&utm_source=email&utm_medium=post

CAR-T Therapies: Product/Molecules/MOA under Development:

  • Similar CAR-T treatments were being developed at other institutions including
  • Memorial Sloan-Kettering Cancer Center,
  • Seattle Children’s Hospital, and
  • The National Cancer Institute.
  • The Memorial and Seattle work was spun off into a startup called Juno Therapeutics, which has fallen behind. Juno Therapeutics ended a CAR-T study earlier this year after patients died from cerebral edema, or swelling in the brain.
  • The NCI work became the basis for the product being developed by Kite Pharma. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

On Cambridge Healthtech Institute’s 4th Annual Adoptive T Cell Therapy, Delivering CAR, TCR, and TIL from Research to Reality, August 29 – 30, 2017 | Sheraton Boston | Boston, MA

TUESDAY, AUGUST 29 – I covered in Real Time the talk on Juno Therapeutics: Building Better T Cell Therapies: The Power of Molecular Profiling by Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

Precision Medicine is Costly and not a Rapid manufacturing process

All of the CAR-T products are expensive to make, and must be manufactured on an individual basis for each new patient from the patient’s own T-cells, a type of white blood cells, a process that takes weeks.

  • How quickly companies can speed up manufacturing.
  • Kymriah will be manufactured at a facility in Morris Plains, N.J.
  • CAR-T technology, which has so far been used only in patients with blood cancers that have not been cured by other treatments, can be used earlier in the disease or for solid tumors: Breast, Prostate, Melanomas.

https://www.forbes.com/sites/matthewherper/2017/08/30/fda-approves-novartis-treatment-that-alters-patients-cells-to-fight-cancer/#2aecb25b4400

Prediction How Patients will Far Well – Researchers use a big-data approach to find links between different genes and patient survival.

https://www.technologyreview.com/s/608666/a-cancer-atlas-to-predict-how-patients-will-fare/?set=

A pathology atlas of the human cancer transcriptome

+ See all authors and affiliations

Science  18 Aug 2017:
Vol. 357, Issue 6352, eaan2507
DOI: 10.1126/science.aan2507

Modeling the cancer transcriptome

Recent initiatives such as The Cancer Genome Atlas have mapped the genome-wide effect of individual genes on tumor growth. By unraveling genomic alterations in tumors, molecular subtypes of cancers have been identified, which is improving patient diagnostics and treatment. Uhlen et al. developed a computer-based modeling approach to examine different cancer types in nearly 8000 patients. They provide an open-access resource for exploring how the expression of specific genes influences patient survival in 17 different types of cancer. More than 900,000 patient survival profiles are available, including for tumors of colon, prostate, lung, and breast origin. This interactive data set can also be used to generate personalized patient models to predict how metabolic changes can influence tumor growth.

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The Future of Hospitals – How Medical Care and Technology Work Together to Advance Patient Care 

Curator: Gail S. Thornton, M.A.

Co-Editor: The VOICES of Patients, Hospital CEOs, HealthCare Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures

 

Gap Medics (https://www.gapmedics.com/blog/), the world’s leading provider of hospital work experience placements for high school and university students, recently released their “Futuristic Hospitals” infographic. The infographic reviews a collection of top hospitals in the world based on several key factors:

  • overall patient care,
  • innovative medical and technological excellence,
  • efforts toward sustainability,
  • environmental stewardship, and
  • social responsibility, as well as
  • other innovative health care features

to help advance the field of medicine and, ultimately, patient care.

Futuristic Hospitals Infographic

Image SOURCE: Infographic of Futuristic Hospitals courtesy of Evolved Digital and Gap Medics. Reprinted here with Permission from the Source.

 

“Many leading hospital facilities are now rolling out significant improvements and changes that couldn’t have been envisioned 10 years ago,” said Ian McIntosh, Director, Evolved Digital (http://evolveddigital.co.uk/), a U.K.-based digital marketing company specializing in search engine optimization and content marketing, whose team created the infographic for Gap Medics.

Science and innovation are working together to help convey higher expectations for quality medical and health care and advancements in the hospital experience for health care providers, patients and their families.

Particularly, the infographic analyzed prominent hospitals around the world so patients and their families can learn about the latest advances and efforts in patient care and hospital and medical technology.

In this infographic, we investigated the most cutting-edge hospital facilities in the world, where best-in-class technology and innovative medical care are making a difference in providing a quality experience all over the world.

“Gap Medics creates programs offered to thousands of students from Europe, Asia and the United States so they have the opportunity to gain insights into the work of doctors, nurses, physician assistants, midwives and dentists before the students begin their clinical training,” said Dave Brown, Director, Gap Medics, a U.K.-based company that provides hospital work experience between 1-8 weeks to students 16 years of age and older.

This one-in-a-lifetime opportunity helps students better understand their chosen career path, develop as people, and strengthen their university application process.

 

REFERENCE/SOURCE

http://evolveddigital.co.uk/

https://www.gapmedics.com/blog/2017/03/27/futuristic-hospitals/

Other related articles published in this Open Access Online Scientific Journal include the following:

 

“Sudden Cardiac Death,” SudD is in Ferrer inCode’s Suite of Cardiovascular Genetic Tests to be Commercialized in the US

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2014/02/10/sudden-cardiac-death-sudd-is-in-ferrer-incodes-suite-of-cardiovascular-genetic-tests-to-be-commercialized-in-the-us/

 

Hybrid Cath Lab/OR Suite’s da Vinci Surgical Robot of Intuitive Surgical gets FDA Warning Letter on Robot Track Record

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/07/19/hybrid-cath-labor-suites-da-vinci-surgical-robot-of-intuitive-surgical-gets-fda-warning-letter-on-robot-track-record/

 

3D Cardiovascular Theater – Hybrid Cath Lab/OR Suite, Hybrid Surgery, Complications Post PCI and Repeat Sternotomy

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/07/19/3d-cardiovascular-theater-hybrid-cath-labor-suite-hybrid-surgery-complications-post-pci-and-repeat-sternotomy/

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Pharmacotyping Pancreatic Cancer Patients in the Future: Two Approaches – ORGANOIDS by David Tuveson and Hans Clevers and/or MICRODOSING Devices by Robert Langer

Curator: Aviva Lev-Ari, PhD, RN

 

This curation provides the resources for edification on Pharmacotyping Pancreatic Cancer Patients in the Future

 

  • Professor Hans Clevers at Clevers Group, Hubrecht University

https://www.hubrecht.eu/onderzoekers/clevers-group/

  • Prof. Robert Langer, MIT

http://web.mit.edu/langerlab/langer.html

Langer’s articles on Drug Delivery

https://scholar.google.com/scholar?q=Langer+on+Drug+Delivery&hl=en&as_sdt=0&as_vis=1&oi=scholart&sa=X&ved=0ahUKEwixsd2w88TTAhVG4iYKHRaIAvEQgQMIJDAA

organoids, which I know you’re pretty involved in with Hans Clevers. What are your plans for organoids of pancreatic cancer?

Organoids are a really terrific model of a patient’s tumour that you generate from tissue that is either removed at the time of surgery or when they get a small needle biopsy. Culturing the tissue and observing an outgrowth of it is usually successful and when you have the cells, you can perform molecular diagnostics of any type. With a patient-derived organoid, you can sequence the exome and the RNA, and you can perform drug testing, which I call ‘pharmacotyping’, where you’re evaluating compounds that by themselves or in combination show potency against the cells. A major goal of our lab is to work towards being able to use organoids to choose therapies that will work for an individual patient – personalized medicine.

Organoids could be made moot by implantable microdevices for drug delivery into tumors, developed by Bob Langer. These devices are the size of a pencil lead and contain reservoirs that release microdoses of different drugs; the device can be injected into the tumor to deliver drugs, and can then be carefully dissected out and analyzed to gain insight into the sensitivity of cancer cells to different anticancer agents. Bob and I are kind of engaged in a friendly contest to see whether organoids or microdosing devices are going to come out on top. I suspect that both approaches will be important for pharmacotyping cancer patients in the future.

From the science side, we use organoids to discover things about pancreatic cancer. They’re great models, probably the best that I know of to rapidly discover new things about cancer because you can grow normal tissue as well as malignant tissue. So, from the same patient you can do a comparison easily to find out what’s different in the tumor. Organoids are crazy interesting, and when I see other people in the pancreatic cancer field I tell them, you should stop what you’re doing and work on these because it’s the faster way of studying this disease.

SOURCE

Other related articles on Pancreatic Cancer and Drug Delivery published in this Open Access Online Scientific Journal include the following:

 

Pancreatic Cancer: Articles of Note @PharmaceuticalIntelligence.com

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/05/26/pancreatic-cancer-articles-of-note-pharmaceuticalintelligence-com/

Keyword Search: “Pancreatic Cancer” – 275 Article Titles

https://pharmaceuticalintelligence.wordpress.com/wp-admin/edit.php?s=Pancreatic+Cancer&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

Keyword Search: Drug Delivery: 542 Articles Titles

https://pharmaceuticalintelligence.wordpress.com/wp-admin/edit.php?s=Drug+Delivery&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

Keyword Search: Personalized Medicine: 597 Article Titles

https://pharmaceuticalintelligence.wordpress.com/wp-admin/edit.php?s=Personalized+Medicine&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

  • Cancer Biology & Genomics for Disease Diagnosis, on Amazon since 8/11/2015

http://www.amazon.com/dp/B013RVYR2K

 

 

VOLUME TWO WILL BE AVAILABLE ON AMAZON.COM ON MAY 1, 2017

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Forthcoming e-Book on Amazon.com

The VOICES of Patients, HealthCare Providers, Care Givers and Families: Personal Experience with Critical Care and Invasive Medical Procedures

2017 

 

Author, Curator and Editor

Larry H Bernstein, MD, FCAP

Chief Scientific Officer

Leaders in Pharmaceutical Business Intelligence, Northampton, MA

Larry.bernstein@gmail.com

and

Contributing Editor and Author

Gail S. Thornton, PhD(c)

Leaders in Pharmaceutical Business Intelligence, New Jersey

gailsthornton@yahoo.com

Aviva Lev-Ari, PhD, RN

Editor-in-Chief BioMed e-Series of e-Books

Leaders in Pharmaceutical Business Intelligence, Boston

avivalev-ari@alum.berkeley.edu

BioMedical e-Books e-Series:

Cardiovascular, Genomics, Cancer, BioMed, Patient-centered Medicine

https://pharmaceuticalintelligence.com/biomed-e-books/

Abbreviated electronic Table of Contents (eTOCs)

Part One: Perceptions of Care

Chapter 1

1.1    Personal Tale of JL’s Whole Genome Sequencing
1.2    Live Notes from @AACR’s #cbi16 Meeting on Precision 
1.3    Live Notes from @AACR’s #cbi16 Meeting on Precision 
1.4    Supportive Treatments: Hold the Mind Strong During Cancer
1.5    Finding My Voice: A Laryngectomee’s Story
 

Chapter 2

2.1     Delivery of Care – See Live links in the body of the e-Book, below

2.2  Hospital CEO:  A New Standard in Health Care – Farrer Park 
2.3  Drug Discovery for Cancer Cure:  Value for Patients – Turning 
2.4  Hospital CEO: A Rich Tradition of Patient-Focused Care 
2.5  Hospital CEO:  University Children’s Hospital Zurich 

2.6 Hospital CEO: Swiss Paraplegic Centre, Nottwil, Switzerland – A World-Class Clinic for Spinal Cord Injuries

Part Two: The Voice of Cancer Survivors

Chapter 3

3.1    Cancer Companion Diagnostics

3.2    lncRNAs in Human Cancers
3.3    Liquid Biopsy Assay May Predict Drug Resistance
3.4    Pharmacogenomic Biomarkers for Personalized Cancer 
 

Chapter 4

4.1 Personalized Medicine: Cancer Cell Biology and Minimally Invasive Surgery (MIS)

4.2    Cardiotoxicity and Cardiomyopathy Related to Drugs Adverse 
 

Chapter 5

5.1       Thyroid Cancer

5.1.1    Experience with Thyroid Cancer
5.1.2    Cancer Signaling Pathways and Tumor Progression
5.2      Brain Surgery
5.2.1   A Cousin’s Experience with a Pituitary Acromegaly
5.2.2    Loss of Normal Growth Regulation
5.2.3    Glioma, Glioblastoma and Neurooncology
5.3       Breast Cancer
5.3.1    Faces of Breast Cancer – Find Your Story, Join the Conversation
5.3.2    An Emotional and Thoughtful Decision Over BRAC1 and Surgery
5.4       Ovarian Cancer
5.4.1    A Curated History of the Science Behind the Ovarian Cancer
5.4.2    Good and Bad News Reported for Ovarian Cancer Therapy
5.4.3    Almudena’s Story:  A Life of Hope, Rejuvenation and Strength
5.5       Hematological Malignancies
5.5.1    Hematological Malignancy Diagnostics
5.5.2    Hematological Cancer Classification
5.5.3    Chemotherapy in AML
5.5.4    Update on Chronic Myeloid Leukemia
5.5.5    Rituximab for a variety of B-cell malignancies
5.5.6    T cell-mediated immune responses & signaling pathways 
5.5.7    Gene expression and adaptive immune resistance mechanism
5.5.8    Sunitinib brings Adult Acute Lymphoblastic Leukemia (ALL) to R
5.5.9    Management of Follicular Lymphoma
5.5.10   Gene Expression and Adaptive Immune Resistance Mechanisms 
5.6       Other Types of Cancer
5.6.1    Lung Cancer Therapy
5.6.2    Non-small Cell Lung Cancer drugs
5.6.3    Colon Cancer
5.6.4    GERD and esophageal adenocarcinoma
5.6.5    Melanoma
5.6.6   Adenocarcinoma of the Duodenum – Nathalie’s Story: A Health 
5.7     Organ Transplantation
5.7.1     Marcela’s Story: A Liver Transplant Gives the Gift of Life 
 

Chapter 6

6.1   Nutrition: Articles of Note @PharmaceuticalIntelligence.com
6.2   Epigenetics, Environment and Cancer: Articles of Note 
6.3   The relationship of stress hypermetabolism to essential 
6.4   Cancer Drug-Resistance Mechanism
6.5   Biochemistry and Dysmetabolism of Aging and Serious Illness
6.6   Experience of and Alleviation of Pain
 

Chapter 7

7.1   Metabolomics: its applications in food and nutrition research
7.2   Neutraceuticals

Part Three: The Voice of Open Heart Surgery Survivors

Chapter 8
8.1   A Patient’s Perspective: On Open Heart Surgery
8.2   Triple-bypass operation at age 69 – Ralph’s Story
8.3   A Fantastic Vessel-Clearing Innovation on The vessel-clearing 

 

VIEWS – All time for HEALTH CARE PROVIDER INSTITUTIONS –

LIVE Interviews by Gail Thornton

 

TITLE

URL

# Views

on 4/12/2017

Swiss Paraplegic Centre, Nottwil, Switzerland – A World-Class Clinic for Spinal Cord Injuries

# Views

on 4/12/2017

60

 

https://pharmaceuticalintelligence.com/2017/03/23/swiss-paraplegic-centre-nottwil-switzerland-a-world-class-clinic-for-spinal-cord-injuries/

 

60
University Children’s Hospital Zurich (Universitäts-Kinderspital Zürich), Switzerland – A Prominent Center of Pediatric Research and Medicine

# Views

on 4/12/2017

84

 

https://pharmaceuticalintelligence.com/2016/12/21/university-childrens-hospital-zurich-universitats-kinderspital-zurich-switzerland-a-prominent-center-of-pediatric-research-and-medicine/

 

84
A Rich Tradition of Patient-Focused Care — Richmond University Medical Center, New York’s Leader in Health Care and Medical Education

# Views

on 4/12/2017

 139

 

https://pharmaceuticalintelligence.com/2016/10/17/a-rich-tradition-of-patient-focused-care-richmond-university-medical-center-new-yorks-leader-in-health-care-and-medical-education/

 

139
Value for Patients – Turning Advances in Science: A Case Study of a Leading Global Pharmaceutical Company – Astellas Pharma Inc.

# Views

on 4/12/2017

329

 

https://pharmaceuticalintelligence.com/2016/08/24/value-for-patients-turning-advances-in-science-a-case-study-of-a-leading-global-pharmaceutical-company-astellas-pharma-inc/

 

329
A New Standard in Health Care – Farrer Park Hospital, Singapore’s First Fully Integrated Healthcare/Hospitality Complex

# Views

on 4/12/2017

670

https://pharmaceuticalintelligence.com/2016/06/22/a-new-standard-in-health-care-farrer-park-hospital-singapores-first-fully-integrated-healthcarehospitality-complex/

 

670

VIEWS – All time for Patient Experience

LIVE Interviews by Gail Thornton

 

TITLE

URL # Views
WEGO Health Awards Competition Focuses on Patients

 

# Views

on 4/12/2017

17

https://pharmaceuticalintelligence.com/2016/09/13/wego-health-awards-competition-focuses-on-patients/

 

17
Almudena’s Story: A Life of Hope, Rejuvenation and Strength

 

# Views

on 4/12/2017

109

https://pharmaceuticalintelligence.com/2016/08/10/almudenas-story-a-life-of-hope-rejuvenation-and-strength/

 

109
Nathalie’s Story: A Health Journey With A Happy Ending

 

# Views

on 4/12/2017

300

https://pharmaceuticalintelligence.com/2016/07/30/nathalies-story-a-health-journey-with-a-happy-ending/

 

300
Ralph’s Story: An Entertainer at Heart

# Views

on 4/12/2017

143

 

https://pharmaceuticalintelligence.com/2016/07/30/ralphs-story-an-entertainer-at-heart/

 

143
Marcela’s Story: A Liver Transplant Gives the Gift of Life

 

# Views

on 4/12/2017

48

https://pharmaceuticalintelligence.com/2016/07/23/marcelas-story-a-liver-transplant-gives-the-gift-of-life/

 

48
The Role of Big Data in Medicine

 

# Views

on 4/12/2017

55

https://pharmaceuticalintelligence.com/2016/05/16/the-role-of-big-data-in-medicine/

 

55
A Revolutionary, Personalized Approach in Brain Tumor Research

 

# Views

on 4/12/2017

46

https://pharmaceuticalintelligence.com/2016/04/11/a-revolutionary-personalized-approach-in-brain-tumor-research/

 

46

 

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