I have received press credentials to attend the BIO 2025 International Convention, June 16-19 in Boston. Please let me know if you or your organization are interested in live coverage, with minimal expenses charged.
https://lnkd.in/ed92YWrM
Wednesday, May 28, 2025
I am Available to Provide Coverage from the 2025 BIO International Convention
https://lnkd.in/ed92YWrM
Presenting an Updated Partial List of My Select Publications Over the Past 25 Years
John J.
Otrompke, JD
347-370-6847
Skype ID: john.otrompke
https://www.linkedin.com/in/johnotrompke
www.otrompkescommentaries.blogspot.com
The changing US regulatory environment: Amid Chaos, Signs of
Opportunity
Nordic Life Science News, March 21, 2025
Recommendations Linked to Reduced Opioid Prescribing in
Michigan, but Challenges Remain
Anesthesiology News, Jan. 18, 2025
Treating high blood pressure with just two shots a year
Drug Discovery News, Apr 19, 2024
https://www.drugdiscoverynews.com/treating-high-blood-pressure-with-just-two-shots-a-year-15917
Small But Mighty: Three Breast Cancer Nonprofits Alleviating
Financial Toxicity Experienced by Cancer Patients
Dec. 3, 2023 (Enterprise journalism)
https://otrompke.blogspot.com/
A Tale of Two Courts: Eleven Distinctions in Search of a
Difference
Social Sciences Research Network, April 20, 2023
https://papers.ssrn.com/sol3/papers.cfm?abstract_id=4412146
One (Potentially) Giant Leap for Mankind
Cancer Wellness, April 10, 2023
https://cancerwellness.com/innovations-and-research/one-potentially-giant-leap-for-mankind/
FDA Approves Modulight Laser to Treat Age-Related Macular
Degeneration
FDA News, Feb. 8, 2023
Review of Arthur Peltomaa’s “Understanding
Unconstitutionality: How a Country Lost Its Way”
Int’l J. Const. L. Blog, Oct. 13, 2019
“It Starts With One”: Reflections on BIO 2019
The P&T Journal, August 2019
Can the insurance industry be a force for prevention in the
pandemic of child sex abuse?
Leader’s Edge Magazine, September 2019
US FDA Must Hire New Staff to Fulfill New Obligations and
Review New Technologies
Pharma DJ, June 12, 2019
Meeting
Highlights: Digestive Disease Week 2019
The P&T
Journal, July 2019
iCAN – Finland;s
Precision Oncology Toolbox.
HIMSS Insights
eBook, issue 7.4. April 1, 2019
CMS Utilizes
Dartboard Approach to Modernizing the Medicare Drug Benefit
Nashville
Medical News, Feb 12, 2019
AAN Issues New
Guidelines on Disease-Modifying Therapies in MS, Including Switching, Stopping
and Pregnancy
The Lancet
United States of Health Blog, April 30, 2018, p. 15
Why the Common Rule Should Permit Universal Psychological
Autopsy Studies of Child Suicides and School Shooters
Social Sciences Research Network, Dec. 3, 2019
http://ssrn.com/abstract=3488693
Review of Arthur Peltomaa’s “Understanding
Unconstitutionality: How a Country Lost Its Way”
Int’l J. Const. L. Blog, Oct. 13, 2019
Can the insurance industry be a force for prevention in the
pandemic of child sex abuse?
Leader’s Edge Magazine, September 2019
“It Starts With One”: Reflections on BIO 2019
The P&T Journal, August 2019
US FDA Must Hire New Staff to Fulfill New Obligations and
Review New Technologies
Pharma DJ, June 12, 2019
http://www.pharmadj.com/cms/detail.htm?item.id=6672f4078cb711e98efdfa163e227c38
Meeting
Highlights: Digestive Disease Week 2019
The P&T
Journal, July 2019
iCAN – Finland;s
Precision Oncology Toolbox.
HIMSS Insights
eBook, issue 7.4. April 1, 2019
https://pages.healthcareitnews.com/HIMSSInsights4.html
CMS Utilizes
Dartboard Approach to Modernizing the Medicare Drug Benefit
Nashville
Medical News, Feb 12, 2019
Study
presented at AACR and published in The Lancet Respiratory Medicine may set new
standard for rare sarcoma
The Lancet
United States of Health Blog, April 18, 2018, p. 25.
Some Children
May Now Receive Sex Change Hormone Therapy Before 16: ENDO Guideline
The Lancet
United States of Health Blog, March 26, 2018, p. 31.
https://els-jbs-prod-cdn.jbs.elsevierhealth.com/pb-assets/Lancet/lancet/TL_USA_Posts-1552663776617.pdf
Onco-cardiology-
targeted the double heat break
The Lancet United
States of Health Blog, March 16, 2018, p. 37.
Single payer
healthcare debate takes the stage in managed care
The Lancet
United States of Health Blog, March 13, 2018, p. 39.
More Aggressive Surgery Linked to Arm Problems in Young
Women With Breast Cancer
HEALTHCENTRAL, Jan. 29, 2018
Metformin linked with better survival in RCC patients with
diabetes
IMNG / Frontline’s MD Edge,
April 10, 2017
A Compelling Legal History of a Contemporary Civil Rights
Catastrophe (Book review: Rogue Justice: The Making of the Security State by
Karen J. Greenberg)
Illinois State Bar Association’s HUMAN RIGHTS Newsletter,
October 2016
Large Database Study Finds 11 Genes Associated with
Increased Ovarian Cancer Risk
TARGETED ONCOLOGY, June 9, 2016
Population Health Management- Worth the Effort?
HIMSS EUROPE INSIGHTS, Vol. 4, no. 3, p. 22
Moving toward personalized medicine for melanoma
DERMATOLOGY TIMES, Jan 7, 2016
Risk Factors for Melanoma and Genetic Testing
DERMATOLOGY TIMES, January 7, 2016
Tumor Treating Fields Device Shows Survival Advantage
TARGETED ONCOLOGY, December 18, 2015
Bills, Quills and Stills offers something for everyone, but
most of all the serious scholar
Illinois State Bar Association’s HUMAN RIGHTS Newsletter,
September 2015
A World of Difference: Biosimilars and Biobetters Offer
Unique Benefits- and Risks
BIOPROCESS INTERNATIONAL, June 2015
Recap ADA 2015
Elsevier’s PRACTICE UPDATE, June 5-7 2015
http://www.practiceupdate.com/c/26075/48/8
HIMSS Keynote Speakers Highlight the Speed of Innovation
BIO-IT WORLD, April 17, 2015
http://www.bio-itworld.com/2015/4/17/himss-keynote-speakers-highlight-speed-innovation.html
Radioactive Iodine Overused in Papillary Thyroid Cancer
CANCER NETWORK, March 9, 2015
http://www.cancernetwork.com/thyroid-cancer/radioactive-iodine-overused-papillary-thyroid-cancer
Investigating treatment strategies for the Middle East
respiratory syndrome coronavirus, ROYAL PHARMACEUTICAL SOCIETY’S PHARMACEUTICAL
JOURNAL, Oct. 20, 2014
US antibiotic stewardship reduces drug resistance and cost,
THE PHARMACEUTICAL JOURNAL, Sept. 15, 2014
Micafungin well tolerated by leukaemia patients
THE PHARMACEUTICAL JOURNAL
Eric Topol on the Medical Geographic Information System
HEALTH INFORMATICS NEWS, July 31, 2014
Movement toward exchanges faster than anticipated
NATIONAL UNDERWRITER’s LIFE & HEALTH, June 16, 2014
http://www.lifehealthpro.com/2014/06/16/movement-toward-exchanges-faster-than-anticipated
Top 10 Recent ICU Quality Initiatives
HCPLive, October 31, 2013
Experimental derivative of spectinomycin could help fill gap
for new TB treatment
THE PHARMA LETTER, Sept. 16, 2013
Focusing on the Long-Term Needs of Cancer Survivors
US NEWS & WORLD REPORT, September 9, 2013
Initial Data from CCS Registry Shows Low Complication Rate
in Intervention Cases, MD CONFERENCE EXPRESS, July 27, 2013
Top Ten Recent ICU Quality Initiatives
HCPLive, October 31, 2013
Study Indicates That Anti-Diabetic Therapies Do Not Increase
Cancer Risk
HCPLive, June 25, 2013
Participation in VA’s MOVE! Associated with Weight Loss
MD CONFERENCE EXPRESS, June 21-25, 2013
Skin Cancer Warning Signs
ASSOCIATED BODYWORK AND MASSAGE PROFESSIONALS, April 2013
Tightening of Ethical Standards in Bioengineering Brings
Teaching to the Fore,
The IEEE’s PULSE Magazine, January-February 2013,
Significant Changes to Institutional Review Boards Proposed
IEEE’s PULSE Magazine, November-December 2012,
Even HIV-Infected Men With Undetectable HIV in Blood May
Shed HIV in Semen, TheBODYPRO.com, October 11, 2012
Black MSM Caught in HIV's Net by Complex Web of Factors
TheBODYPRO.com, October 22, 2012
Chance Encounter: The Rise in the Number of Incidental
Findings Brings Ethical and Medical Dilemmas to the Forefront
The American College of Radiology’s BULLETIN, September 12,
2012,
Presentations at JAHC Discuss Relationship of Homeopathy to
Allopathic Medicine, THE TOWNSEND LETTER, August-September, 2012,
www.townsendletter.com/AugSept2012/AugSept2012.html
Referring Out: Spotting the
Spots that Need a Doctor
The Associated Skin Care
Professionals’ SKIN DEEP Magazine, July/August 2012,
Future Boom in Chronic Cancer
Patients Likely to Change the Face of Disease, suggest ASCO Findings
PHARMACEUTICAL EXECUTIVE, June
25, 2012
Drug Innovation: Alive and Well
at ASCO!
PHARMACEUTICAL EXECUTIVE, June
6, 2012
Billions of dollars for research at stake in health-reform
case
NATURE MEDICINE, May 4, 2012
http://www.nature.com/nm/journal/v18/n5/full/nm0512-637.html
During Red Tides, Blue Light Triggers Microalgae To
Release Brevetoxin
The American
Society of Microbiology’s MICROBE Magazine, May 2012
Joint
Accreditation of Continuing Medical Education
MIDWEST
MEETINGS March 2012
Plans begin
ACO development in commercial markets
MANAGED
HEALTHCARE EXECUTIVE, October 1, 2011
Fever-Causing Bacteria Replacing Malaria in West African
Countries
MICROBE, September
2011
RESET: Largest Study to Date of Xience, Cypher Shows
Noninferiority
CARDIOVASCULAR RESEARCH FOUNDATION, August 2011
GRAVITAS Analysis: Lower Cutpoint May Enhance Functional
Platelet Count, CARDIOVASCULAR RESEARCH FOUNDATION, August 2011
Number of Preterm Births on the Rise
MEDSCAPE
January 2011
Atomoxetine Does Not Increase Cardiac QT Interval in Healthy
CYP2D6 Poor Metabolisers
DOCTOR’S GUIDE, November 2010
Making New Medical Innovations Available in Developing
Countries First, Where They’re Needed Most
CASE STUDIES FOR GLOBAL HEALTH, published by the Bill and
Melinda Gates Foundation and the Association for University Technology
Managers, November 2009
http://issuu.com/ipha/docs/case_studies_for_global_health
Goodbye to the bubble: Medical researcher John Cunningham’s
stem-cell–transplant technique gives offers a chance at a normal life to a
child with a rare immune disorder, UNIVERSITY OF CHICAGO MAGAZINE, May-June
2009,
http://magazine.uchicago.edu/0906/investigations/bubble_boy.shtml
New Federal Approval for Some Stem Cell Research May Benefit
People with SCI/D: First Human Clinical Trial Enrolling Now
SCILife, published by the National Spinal Cord Injury
Association, July 2009
Social networking, offline and on (co-author)
MOMENTUM, a publication of the National Multiple Sclerosis
Society, Summer 2009
Drug Discovery: Back to the Future
DRUG DISCOVERY AND DEVELOPMENT, February 2007
Improving Melanoma Immunotherapy Outcomes
SKIN AND AGING, February 2007
Federal Court Firmly
Rejects FDA Prohibitions, Affirms Constitutional Right to Access Experimental
Lifesaving Drugs
LIFE EXTENSION
FOUNDATION, November 2006
Intravenous Form and Labeling Approved for Ibandronate:
Presented at AACE, DOCTORS GUIDE, May
2006
Intravenous Ibandronate May Be Associated With Higher
Incidence of Flu-like Symptoms, Urinary Incontinence, and Other Side Effects
DOCTORS GUIDE, May
2006
Melanoma Immunology Trials Surveys
DERMATOLOGY TIMES, March 2005
New rules restrict promotion and education
PHARMACEUTICAL BUSINESS STRATEGIES, February
2005
Recruiting in the Workplace: Sponsors are turning to large
employers for help with large trials
BIO-IT WORLD, January 2005
Health care needs and America's college students: public
health dilemma or opportunity? UNIVERSITY BUSINESS, December 2004
Hospital pharmacy worries over workforce supply
DRUG TOPICS, November 2004
Hospitals headed the
way of managed care?
RISK AND INSURANCE, May 2004
Navigating US Conflict-of-interest
Rules When Commercializing Research
NATURE
BIOENTREPRENEUR, 2003
http://www.nature.com/bioent/2003/030801/full/bioent760.html
ASCO Takes On Conflict Of Interest
Medical Meetings, Sep 1, 2003
Patently Conflicted Medical schools have gotten into the
health technology business in a big way
UNIVERSITY BUSINESS, 2003
An industry under suspicion: pharmacy benefit managers are
under increased scrutiny- and are increasingly the defendants in lawsuits
RISK AND INSURANCE, January 2003
Risk News Exclusive Special Report: Managed Care Medicine
Under Scrutiny
RISK & INSURANCE, JUNE 2002.
HIPAA Will Bring Major Change to Industry Routines but Labs
Have Certain Advantages in Compliance
CLINICAL LAB PRODUCTS, April 2002, at 34.
The On-Line Pharmacy and More
PLAN SPONSOR, March 2, 2002, at 78.
A New Format for Making More Cost-Effective Drug Coverage
Decisions Takes Off
BUSINESS & HEALTH, January 31, 2002.
Plans poised to embark on new policies of broader experimental
coverage
MANAGED HEALTHCARE EXECUTIVE, March 2005
New Ideas in Pharmacy Cost Control
RISK & INSURANCE, January 2002, at 26.
Changing the Formulary Puts the Focus Back on Value,
MANAGED HEALTHCARE EXECUTIVE, November 2001, at 33.
Cracking Down on Fraud in Managed Care can Prove
Challenging
MANAGED HEALTHCARE EXECUTIVE, November 2001, at 45.
Pharmacy, the Internet & Expatriates
RISK & INSURANCE,
September 16, 2001, at 26.
Stark II and the O & P Industry
ORTHOPEDIC TECHNOLOGY REVIEW, September-October 2001, at 1.
A Stark Exception, the American Hospital Association’s
HOSPITALS & HEALTH NETWORKS, September 2001, at 17.
Stark Allows Federal Regulators to Review Claims Records
Fully
HEALTH PLAN NETWORKER, August 17, 2001, at 4.
Stark II regulations debut with new guidelines that
warrant close study by clinical laboratorians
CLINICAL LAB PRODUCTS, August 2001, at 30.
Pharmacy Benefits Managers Use the Internet for Savings,
BUSINESS AND HEALTH, July/August 2001, at 43.
Stark Allows Federal Regulators to Review Claims Records
Fully
MANAGED CARE, July 2001, at 69.
HCFA Carves Out Requests by Pathologists for Clinical Lab
Services, Pathology Exam
CLINICAL LAB LETTER, March 15, 2001, at 1.
Waiving Co-payments and Deductibles for Indigent
Patients
FAMILY PRACTICE DIGEST, February 2001, at 13.
Navigating Your Way Through the Fraud and Abuse,
Legislation
MEDICAL LABORATORY OBSERVER, November 2000, at 46.
Internet Likely to Open a Brave New World in Global
Pharmaceuticals Trade
Faulkner & Grey’s MEDICINE & HEALTH PERSPECTIVES,
October 8, 2000, at 1.
FTC Uses Unusual Tool Against Rogue Internet Pharmacies
INTERNET HEALTHCARE, October 2000, at 30.
Class Action Against Managed Care May Be a Trend
The American Dental Hygienists’ Association’s ACCESS, March
2000, at 30.
Advocates Gear Up as Privacy Deadline Draws Near,
TELEHEALTH, December 1999, at 27.
See You in Court…Oral Health Lawsuits Heat Up,
The American Dental Hygienists’ Association’s ACCESS,
September-October 1999, at 26.
Visiting the Front Lines of Day-to-Day Medicine
ILLINOIS MEDICINE, June 26, 1998, at 1.
Illinois Court Decision Further Clouds State Tort Reform
Issue
PROACTIVE RISK MANAGEMENT, June 1998, at 73.
Advocate Extraordinaire
PHYSICIAN’S PRACTICE DIGEST, March/April 1998, at A1.
Thursday, December 26, 2024
I have received press credentials for the 2025 BIO CEO and Investors Conference
I have received media credentials to attend and cover the 2025 BIO CEO and Investors Conference, February 10-11 in New York City. I will be on hand, and if you are interested in live, independent coverage, I need charge no expenses.
Thanks for your consideration, and Happy Holidays.
One article I am proposing as coverage from this conference has to do with bone marrow transplants for breast cancer. This therapy (along with high dose chemotherapy) was considered experimental when I was in law school, and was the subject of bitter litigation.
So fast-forward to the present, and what happened? Survival rates for breast cancer have increased markedly, especially with those women who have genetically-driven forms of the disease. Is the gain in survival associated with more use of bone marrow transplants for breast cancer?
No. High dose chemotherapy with bone marrow transplant is not routinely used for patients with breast cancer; instead, the benefit has come about partially because the biopharma industry has developed several targeted therapies addressing genetically-associated forms of the disease.
Is it possible that the managed care companies were right?
Sunday, December 3, 2023
Small But Mighty: Three Breast Cancer Nonprofits Alleviating Financial Toxicity Experienced by Cancer Patients
By John Otrompke
The
eleventh annual Dancing For The Survivors event was held tomorrow, Friday,
October 13, at The Mint in Lathrup Village, Michigan.
“One of our dancers is Lisa McCall,
a breast cancer survivor who was the dance choreographer for Aretha Franklin,”
said Molly MacDonald, founder and CEO of The Pink Fund, based in Southfield,
Michigan. The goal was to raise $300,000 from the event, she said.
The Pink Fund’s work alleviating
the financial toxicities suffered by breast cancer patients was also
highlighted this month by the Detroit People Mover, in partnership with Priority
Waste Management. It was the light rail train’s first public service
announcement.
“The Pink Fund is totally unique;
they provide financial assistance for 90 days for people going through breast
cancer, not for your co-pay or your insurance deductible, but for life bills
like your car note, mortgage and electric bill,” said Heidi Floyd, patient
advocate, influencer, and breast cancer patient sand survivor, who recently
joined the Board of Directors. The Pink Fund is based in Detroit, but operates
in all 50 states, she said.
“In the US, health care is not just
provided just because you exist here; you have to get your own, so people have
to make decisions about what to pay that month. Can I afford radiation
treatment or do I have to keep the water on for my family? Three months’ worth
of someone paying your mortgage could be a life changer,” she explained.
Floyd, who was first diagnosed with
ER-positive, stage 3B breast cancer 18 years ago, knows the realities of the financial costs of
breast cancer from first-hand experience.
“I was a young mom. There’s a list
of things not supposed to do. I wish I had known, because it would have changed
everything for my family generationally. We did all the wrong things. We had a
college fund for the kids; we didn’t have credit cards. When We started getting
bills to the tune of hundreds of thousands of dollars after insurance, we got
every credit card we could and maxed them out, without knowing we could go to
the cancer center and negotiate, or call the mortgage company.
“Many people say, I didn’t think I
needed financial assistance. I thought I had decent insurance, but I didn’t
think about how I’d have to pay for the car to get to the cancer center, and $50
for parking, as well as child care. This month, I won’t pay the gas bill; next
month, I won’t pay the electric,” she added.
“To me, the most amazing thing if that when you’re in the middle of a serious medical issue, you immediately realize where the gaps are in the system. There are some people who it’s inherent in their nature to say, I’m just going to do what I need to survive, and then they immediately try to do what they can to help other people. Molly is not going to repair the financial toxicity in the entire system; that would be a Sisyphean challenge. But she’s going to do what she can with what she has. That’s what weaves this tapestry together,” Floyd explained.
Avoiding
the Financial Toxicity of Cancer
“When you’ve been through cancer
treatment, a long trail of side effects follow you quite a while,” said The
Pink Fund’s MacDonald, who also sits on the board of directors of the National Breast
Cancer Coalition.
“In 2013, two doctors, Yusuf Zafar and Amy Abernathy, did a study in which they coined the term financial toxicity, which is less known than the physical toxicities of cancer treatment, but it’s becoming quite a hot topic as it effects patients regardless of the time in making their decisions. The most egregious example is that, according to our work, 41% of breast cancer patients will skip or stop treatment to return to work.”
Like Floyd, MacDonald knows
first-hand about the financial toxicity of cancer treatment. Diagnosed in 2005
with ductal carcinoma in situ (stage 0), she had 23 surgeries and 6 weeks of
radiation, followed by five years of aromatase inhibitors. In 2006, she founded
The Pink Fund with her husband.
“Being diagnosed at a time of job
transition left me unemployed and unemployable while in treatment. I was
already negotiating with my health care provider to cover deductible, which it
took me 3 years to pay off. My credit report really tanked. My home did go into
foreclosure. My mother was had dementia at the time was not sure who I was, but
she knew this person Molly had cancer, and she asked how she could help. I told
her about the house, and she gave me $20,000 to pull it out of foreclosure. I
ended up in line at the food bank. Fortunately, people brought foods while I
was in treatment,” she explained.
So MacDonald started The Pink Fund,
to address the issue of financial toxicity by paying non-medical bills in women
and men in active treatment who have experienced a loss of household income. “You
have to be at or below 50% of the federal poverty level, and we pay patient’s
creditors, we don’t pay the patient,” noted MacDonald. “We pay between $3,000
to $6,000 per qualified, fully supported application, which amounts to about
57% of our income for housing every year,” she said.
The Pink Fund also covers health
insurance premiums, COBRA, car payments and insurance and transportation, as
well as utilities for 3 months. “If think of life like a Jenga tower, when your
life starts to sway, and eventually it’s going to collapse, we’re not going to
fix it, but we’re coming around providing financial scaffolding around you to
stabilize you, so you can take a breath and figure out what your long-time financial
strategy is,” MacDonald said.
Supporting Moms and Children
Like
MacDonald, Audrey Guth was a successful businesswoman when she was diagnosed
with breast cancer. “When I was diagnosed 15 years ago, I was the owner of a
nanny agency. My first cancer was thyroid. I thought I would get over it without
sharing my information with very many people. A few years later I was diagnosed
with estrogen receptor-positive breast cancer. I didn’t know anyone who had breast
cancer, but it was a totally different ball game, because it was life
threatening. After the first surgery, I was told there was nothing left to do,
but the margins weren’t clear, so it took 2 more surgeries to be able to remove
all the physical cancer. Then I went through radiation,” said Guth, who today
is the Founder and Volunteer Executive Director of Nankind, a charitable
organization that transforms the cancer experience for families with free
childcare and vital support programs, based in in Toronto.
Like
Floyd and MacDonald, Guth’s experience with cancer taught her about financial
toxicity. “When I was diagnosed, I recognized that there is a gap in the health
care system, where mothers had to make a choice between their own career and
the care of their children. No mother is going to tell you they’d choose
themselves over their children, so a lot of these moms were going without
treatment,” said Guth, who has turned over her nanny for-profit agency to her
daughter.
Today, Guth provides not only her
network of highly screened and trained Volunteer Angels to parents with cancer
and their children, but grief counselling as well. “Moms no longer here, so
what do we do, abandon these children? So I went to hire a specialist in grief
and bereavement, called a thanatologist, and asked her to create a treatment.
During that time I learned grief is not just death, but a loss of normalcy. If
children don’t grieve in an age-appropriate way, it can have long-term
psycho-social impacts. For example, 80% of people in prisons have unresolved
grief issues, as do drug abusers, those who engage in self-harming or have
eating disorders,” explained Guth. “They also have a 50% higher incidence of
mental health disorders. So we built the Nankind Program for Children. Every
week for four hours, our Volunteer Angels engage with children and teach them
coping strategies and mechanisms,” she said.
“One of the things we do that no
one else offers is intense bereavement support, such as what to say to kids at
a funeral. When you say, ‘They’re in a better place,’ that’s like saying to the
kid they’d rather be there than with you,” Guth said.
Nankind provides support to over
150 families each year, to mothers, fathers,
and primary caregivers with all types of cancer, not just breast cancer Guth
noted. “As of today, we have provided over 40,000 prepared meals to 2,000
families and empowered 3,500 children with the tools to build lifelong
emotional resilience,” she added.
Providing
Translation and Navigation Services to the Uninsured
Dora
Arias narrowly avoided the effects of financial toxicity when she was diagnosed
with stage 0 estrogen receptor-positive breast cancer in 2003. “I always had
very lumpy breasts, and always went to my annual gynecological appointments.
The doctor said, ‘Why don’t you start screening earlier, because it’s going to
be very hard for you to determine what’s good and bad.’ At age 37, my mammogram
was clear, and the following year I almost cancelled my appointment. Because I
was under 40, my insurance would not cover it, and I was not sick. My husband
was like, ‘No, don’t be crazy. If we have to pay out of pocket, we’ll pay out
of pocket,’” explained Arias, founder and executive director of Curémonos
(which means ‘healing together’ in Spanish’ in Mountainside, New Jersey.
“It
came back, and the doctor recommended a mastectomy because I had calcifications
spread throughout my breast. There was a 99% chance of a cure and no radiation,
whereas even if I had a lumpectomy, my breast was going to be disfigured. I was
scared, because I was very young, I was active, and I was working in the city
for JP Morgan. I learned that it’s not vanity, it’s the fact that you’re having
an amputation, and losing part of your body. He recommended plastic surgery
right away,” she added.
In
2009, Arias founded Curémonos, which has provided translation services as well
as patient navigation, education, and support, connecting more than 5,000 women
(most of whom are uninsured) to resources within and outside the hospital.
Today Arias provides the service on a purely voluntary basis.
“All of
these people have been through a horrible situation. Instead of dwelling on
that, saying, ‘Why me?’, they contemplate why anyone else should have to go
through it,” said Heidi Floyd. “The world is awash with people who want to
influence without actually making an impact. These women are the opposite of
that. The world has enough people telling you what kind of makeup to buy, how
to put it on, and what clothes you should wear. These women aren’t seeking
adulation or attention, but they should have all of it,” she added.
© 2023 John Otrompke
Monday, December 12, 2022
FDA’s Approval of IND Application Allows Kyverna Therapeutics to Explore Role of CARs in Lupus, and Possibly Other Immune-Moderated Diseases
By John Otrompke
Immunology
company Kyverna Therapeutics, Inc., will be exploring a new role for CAR
T-cells in treating lupus nephritis, after the company received approval on
Nov. 11 from the FDA for its investigational new drug application to begin
trying its agent, KYV-101, in humans.
“CAR Ts are very effective
depleters of B-cells, which in lupus nephritis are abnormally located in
nephrotic tissues,” explained chief medical officer James Chung, MD PhD. The
Emeryville, California, company plans to begin a phase 1 / 2 trial next year, and
should have clinical data within 6 months, he added.
“This is sort of a ‘Goldilocks’ moment,
because the agent has efficacy on B-cells, and on the other hand, it also has a
great safety profile. These lupus nephritis patients can live 20 or 30 years,
so we can’t give them the level of side effects you see in oncology patients,”
noted CEO Peter Maag, PhD, who was hired by the company in October. (Maag had
previously served as the CEO of Brisbane, California-based CareDx, Inc., a
personalized medicine company in the transplant medicine space).
The announcement comes on the heels
of an article published
in October in Nature Medicine, which described the success of the therapy in treating
five patients. (“Anti-CD19 CAR T cell therapy for refractory systemic lupus
erythematosus,” Mackensen A, Müller F, Mougiakakos D, et al. Nat Med. 2022
Oct;28(10):2124-2132. doi: 10.1038/s41591-022-02017-5. Epub 2022 Sep 15.
Erratum in: Nat Med. 2022 Nov 3;: PMID: 36109639).
“Seventeen months out, they still
have no need for additional immune suppression. If that’s true in five
patients, the discovery might amount to a paradigm shift, achieved by pushing
the immune system’s reset button through the deep depletion of B-cells,” added
Maag.
Lupus occurs mostly in young women
with about 65% of cases occurring in those between the ages of 16 and 55.
Approximately 40% of adults will develop lupus nephritis, 60% of whom will fail
standard of care and approved treatments, said Chung, and up to 40% of those
with diffuse disease will ultimately develop kidney failure, requiring dialysis
or a kidney transplant to stay alive.
The treatment, an anti-CD19
chimeric antigen receptor T-cell (CAR T) construct for which Kyverna has obtained
exclusive, global licensing from the NIH to use in both autologous and
allogeneic CAR T-cell therapies, will be tested first in open label clinical
trials, Maag said.
“The FDA and other health agencies
have accepted objective endpoints for lupus nephritis, such as serum
creatinine, which is a measure of renal function,” Chung noted.
While multiple gene signatures have
been identified in lupus patients, KYV-101 does not target them; instead, the
company hopes to target a broader population of lupus patients through
depletion of B cells.
“There are a number of diseases in
which B-cell depletion might be relevant; there is some evidence in multiple
sclerosis, for example,” said Maag, adding that the company is in talks with
the FDA regarding such an application.
Friday, November 4, 2022
The Clinical Imperative of Personalized Medicine Makes Broader Reimbursement for Genetic Diagnostics Assured
A Discussion of Several Panels at the 16th Annual Meeting of the Personalized Medicine Coalition
by John Otrompke
Although reimbursement coverage is now available for select use cases for molecular diagnostics, these use cases have been demonstrated to be too narrow. Almost 10% of colorectal cancer patients treated at Mayo Clinic Cancer Centers for the two years before the pandemic had actionable germline genetic variants that would not have been identified by contemporaneous guidelines from medical societies such as the National Comprehensive Cancer Network (NCCN), or the 20-gene sequencing panel based on those guidelines, according to a recent article. (Uson P, Riegert-Johnson D, Boardman L, et al. Germline Cancer Susceptibility Gene Testing in Unselected Patients With Colorectal Adenocarcinoma: A Multicenter Prospective Study, Clinical Gastroenterology and Hepatology 2022;20:e508–e528).
Next generation sequencing was performed on the 361 patients using the 84-gene Multi-Cancer Panel from Invitae Corp. (NVTA, San Francisco). Researchers found incrementally actionable variants in 9.4% patients that would not have been identified by the 20-gfene panel from the same company.
“We collaborated with the Mayo Clinic, and the study showed that family history and age are imperfect predictors of whether you have Lynch syndrome, so we ought to test every patient,” explained Robert Nussbaum, MD, CMO at Invitae, a co-author on the article.
The company also recently launched its personal care monitoring service, which measures minimal residual disease in the form of cell-free DNA. “Does the patient need adjuvant treatment? Will they relapse, and if so, when?” added Nussbaum, who also spoke on a panel about diagnostics at this year’s annual Personalized Medicine Conference at Dana Point, California, in May. Some payors are reimbursing for the diagnostic test, Nussbaum said.
Reimbursement for genetic diagnostics is likely to grow in the future as researchers develop more evidence about the clinical and economic benefits that such testing can provide. If genetic diagnostics are not reimbursed consistently, drugmakers may have a harder time marketing genetically guided therapeutics.
“Even in the primary care setting, virtually every patient should be assessed for hereditary risk factors,” noted Lisa Alderson, CEO and founder of Genome Medical, Inc., in South San Francisco, CA. “While 7% of the population has a single-gene variant that impacts their health, a much larger percentage, approximately 17%, have moderate risk variants that may influence treatment or preventive options.” Fully 85% of people have genetic factors that impact their response to prescribed drugs and could change dosing or therapeutic selection, particularly in the areas of pain management, oncology, and mental and behavioral health, added Alderson, who also spoke at a session at the PMC conference on precision medicine in differently-structured health systems.
“We have a full-time team of leading molecular geneticists, genetics counsellors and a pharmacist, as well as a contract network of specialists and primary care doctors whom we bring into specific patient’s cases as needed. We see patients directly and also support providers in appropriately utilizing genomics. We are simplifying the process of finding patients with need through our patient assessment and clinical support tools. Physicians just point patients to our platform, which identifies those who meet NCCN guidelines for oncology testing and hereditary cancer risk assessment,” she added.
“Genome Medical is a covered benefit for approximately 170 million people, including all beneficiaries of United Healthcare, CIGNA, and most of the Blues,” said Alderson. “Many payors are bringing Genome Medical in network to better support this complex area of medicine. Today, we have both underutilization and overutilization of genetic testing in the market. The majority of patients meeting guidelines are not identified and when testing is ordered, up to 25 percent of the time, it is the wrong test. We also support clinical decision making to ensure action is taken on the results.
“Take colon cancer as an example. If somebody is at an elevated risk for cancer because of their genetic make-up, that changes the standard of care for cancer screening. The standard of care is for the average patient to have their first colonoscopy at age 45, but if they are at higher risk, the first colonoscopy may take place when they are in their 30s. If they have a colonoscopy earlier, the cost is quite low compared to that of treatment, because removing polyps means you can prevent colon cancer from forming,” she explained.
“There is a pretty steep lag between when you demonstrate clinical utility and when you have broad-based adoption, but we’re trying to build a future model in which providers order genomics earlier and particularly for complex cases. There is a gap in knowledge which is creating a gap in clinical care, but by reducing underutilization and overutilization, we show a very immediate return-on-investment, a 5-times ROI,” Alderson said.
Speeding the Time to Clinical Acceptance
Colon cancer is far from the only example of a disease state in which genetic science is changing medicine and saving lives. “Sepsis accounts for half of all hospital deaths in the US. As it is a time-critical disease, earlier detection or prediction can help save lives. Currently, there is no single biomarker that can accurately predict sepsis. However, research has shown that the combination of a few in vitro diagnostic tests can significantly improve the prediction of sepsis,” said Okan Ekinci, MD, global head of marketing and innovation for Roche Diagnostics Information Solution in Basel, Switzerland. A recent study suggested that a combination of five biomarkers improved sepsis prediction in children compared with C-reactive protein alone, added Ekinci, who also spoke at the PMC conference panel on diagnostic tools. (Rautiainen L, Cirko A, Pavare J, et al. Biomarker combinations in predicting sepsis in hospitalized children with fever. BMC Pediatrics [2022] 22:272).
Success stories such as those of Genome Medical and Invitae come as some payors express frustration with the lack of transparency regarding some diagnostic tools, such as smaller, locally-designed tests.
“It can take 14 to 17 years for a new test to get to widespread adoption, but hopefully we can get it under 10 years, and maybe as low as 7,” agreed Jill Hagenkord, MD, CMO at Optum Genomics in Eden Prairie, Minnesota. (Optum Genomics is currently part of United Healthcare).
To help reduce the time lag, Hagenkord is leading the roll-out of the Optum Evidence Engine, a service which connects developers of diagnostic tests with market access experts who can help design studies and communicate with investors.
“There is a lack of consistent regulatory oversight due the FDA’s exercise of its enforcement discretion, so that anything can go on the market, and does, without any clear criteria. It’s impossible to determine whether the tests do or don’t work at all. Nobody even knows how many tests are on the market, because we don’t have unique identifiers for them, which could make it easier for payors to distinguish between those that do and those that don’t work. So some payors decide that they’re either just going to pay for all laboratory-designed tests, or pay for none of them.” Those at the Optum Evidence Engine have finally begun to assign unique identifiers to the locally-designed tests within the past year, added Hagenkord, who spoke on a panel about reimbursement at this year’s annual PMC conference.
While there may be a significant time lag in personalized medicine between the demonstration of clinical utility and widespread adoption, it is undeniable that broader reimbursement for genetic testing is a fait accompli. Breast cancer is one important example of a clinical area where the clinical importance of personalized medicine has perhaps been longest-established.
“Anybody who has hormone receptor-positive breast cancer, and that’s 80% of breast cancers, is getting targeted therapy,” said Kevin Hughes, MD, director of cancer genetics at the University of South Carolina in Charleston.
Genetic testing for the approximately 13 gene signatures associated with breast cancer is almost always reimbursed, added Hughes, who is also a board member for the American Society of Breast Surgeons. “We’re trying to get the NCCN to change to guidelines to match ours, because they have a very complex set of criteria that ends up with about 50% of breast cancer patients testable,” he noted.
The author is a member of the Personalized Medicine Coalition
© 2022 John J. Otrompke, JD
Wednesday, January 19, 2022
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