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does any one know anything about Iliad Research and Trading, L.P
EXACTLY
ICH GCP
FDA
MHRA
JSQA
CRO List
Clinical Trials
Clinical Research Jobs
2020 Coronavirus Outbreak
ICH GCP
FDA
MHRA
JSQA
CRO List
Clinical Trials
Clinical Research Jobs
2020 Coronavirus Outbreak
ICH GCP | Clinical Trials Registry
A Phase III Clinical Trial Evaluating DCVax®-L, Autologous Dendritic Cells Pulsed With Tumor Lysate Antigen For The Treatment Of Glioblastoma Multiforme (GBM)
Study of a Drug [DCVax®-L] to Treat Newly Diagnosed GBM Brain Cancer
Sponsors
Lead Sponsor
Northwest Biotherapeutics
Source
Northwest Biotherapeutics
Oversight Info
Has Dmc
Yes
Brief Summary
The primary purpose of the study is to determine the efficacy of an investigational therapy called DCVax(R)-L in patients with newly diagnosed GBM for whom surgery is indicated. Patients must enter screening at a participating site prior to surgical resection of the tumor. Patients will receive the standard of care, including radiation and Temodar therapy and two out of three will additionally receive DCVax-L, with the remaining one third receiving a placebo. Patients randomized to the placebo arm will have the option to receive DCVax-L in a crossover arm upon documented disease progression. (note: DCVax-L when used for patients with brain cancer is sometimes also referred to as DCVax-Brain)
Detailed Description
This Phase III trial is designed to evaluate the impact on disease progression and survival time, as well as safety, in patients following treatment with DCVax(R)-L, an immunotherapy treatment for GBM. The experimental therapy uses a patient's own tumor lysate and white blood cells from which precursors of the dendritic cells are isolated. The dendritic cell is the starter engine of the immune system. The white cells are then made into dendritic cells and they are educated to "teach" the immune system how to recognize brain cancer cells. Eligible patients will receive a series of injections of DCVax-L, to activate and then boost the immune response to the tumor cells. The primary study endpoint is PFS (progression free survival), and the first secondary endpoint is overall survival (OS). Other endpoints include performance status, immune response, and also safety. Interim analyses to assess efficacy are incorporated in the trial design. Side effects reported from early trials are mostly mild, and may include skin reactions of redness, pain & swelling at the injection site.
Overall Status
Unknown status
Start Date
2006-12-01
Completion Date
N/A
Primary Completion Date
2016-11-01
Phase
Phase 3
Study Type
Interventional
Primary Outcome
Measure
Time Frame
The primary objective of this study is to compare progression free survival from time of randomization between patients treated with DCVax-L and control patients.
Time to tumor progression or death
Secondary Outcome
Measure
Time Frame
The secondary objective is to compare overall survival and time to disease progression between DCVax-L treated and control patients.
Until Death
Enrollment
348
Conditions
Glioblastoma Multiforme , Glioblastoma , GBM , Grade IV Astrocytoma , Glioma , Brain Cancer , Brain Tumor
Intervention
Intervention Type
Drug
Intervention Name
Dendritic cell immunotherapy
Description
Two intradermal (i.d.) injections of DCVax-L(treatment cohort) or autologous PBMC (placebo cohort) per treatment. Treatments will be given at days 0, 10, 20, and at weeks 8, 16, 32, 48, 72, 96 and 120.
Arm Group Label
Placebo Chohort
treatment cohort
Other Name
DCVax-L
DCVax
DCVax-Brain
Eligibility
Criteria
Inclusion Criteria: All patients must meet the following inclusion criteria. All tests and eligibility criteria must be completed within four weeks of completion of radiation and chemotherapy, following surgery. - Patients must have sufficient tumor lysate protein that was generated from the surgically obtained tumor material. Patients must also have sufficient DCVax-L product available after manufacturing. These determinations will be made by Cognate BioServices, Inc. (Cognate) and communicated to the clinical site through the Sponsor, or its designee. - Patients with newly diagnosed, unilateral GBM (Grade IV) are eligible for this protocol. An independent neuropathologist will review this diagnosis during the enrollment process. - Subjects ≥18 and ≤70 years of age at surgery who are capable of informed consent. Patients must be able to understand and sign the informed consent documents indicating that they are aware of the investigational nature of this study. - Patients must have a life expectancy of >8 weeks. - Patients must have a KPS rating of ≥70 at the baseline visit (Visit 3). - Primary therapy must consist of surgical resection with the intent for a gross or near total resection of the contrast-enhancing tumor mass, followed by conventional external beam radiation therapy and concurrent Temodar chemotherapy. Patients having a biopsy only will be excluded. These primary treatments must be completed at least two weeks prior to first immunization. - Patients may have received steroid therapy as part of their primary treatment. Steroid treatment must be stopped at least 10 days prior to leukapheresis. - Patients must not have progressive disease at completion of radiation therapy. Patients with suspected pseudoprogression will be enrolled and analyzed separately. - Patients must be willing to forego cytotoxic anti-tumor therapies except temozolomide essentially according to the schedule of the Stupp Protocol (Stupp et al. N Engl J Med 352: 987-96, 2005) while being treated with DCVax-L. DCVax-L treatment must be given as described and temozolomide/Temodar treatment schedules must be given essentially according to the Stupp Protocol. - Patients must have adequate bone marrow function (e.g., hemoglobin >10 g/dl, white blood count 3600-11,000mm3, absolute granulocyte count ≥1,500/mm3, absolute lymphocyte count ≥1,000/mm3, and platelet count ≥100K/mm3. Eligibility level of hemoglobin can be reached by transfusion. - Adequate liver function (SGPT, SGOT, and alkaline phosphatase ≤1.5 times upper limits of normals (ULN) and total bilirubin ≤1.5mg/dl), and adequate renal function (BUN or creatinine ≤1.5 times ULN) prior to starting therapy.
Gender
All
Minimum Age
18 Years
Maximum Age
70 Years
Healthy Volunteers
No
Overall Official
Last Name
Role
Affiliation
Linda Liau, M.D.
Principal Investigator
University of California, Los Angeles
Marnix L. Bosch, MBA, PhD
Study Director
Northwest Biotherapeutics
Location
Facility
University of Alabama at Birmingham
Birmingham Alabama 35294 United States
University of Arkansas for Medical Sciences
Little Rock Arkansas 72205 United States
Sutter East Bay Neuroscience Institute-Eden Medical Center
Castro Valley California 94546 United States
City of Hope
Duarte California 91010 United States
UCSD Moores Cancer Center
La Jolla California 93093 United States
Kaiser Permanente - Los Angeles
Los Angeles California 90027 United States
UCLA Medical Center
Los Angeles California 90095 United States
Hoag Memorial Hospital Presbyterian
Newport Beach California 92663 United States
St. Joseph Hospital of Orange
Orange California 92868 United States
University of California, Irvine Medical Center
Orange California 92868 United States
Kaiser Permanente - Redwood City
Redwood City California 94063 United States
Sutter Institute for Medical Research
Sacramento California 95816 United States
University of Colorado Cancer Center
Aurora Colorado 80045 United States
Colorado Neurological Institute
Englewood Colorado 80113 United States
Georgetown University Medical Center
Washington District of Columbia 20057 United States
University of Florida
Gainesville Florida 32611 United States
Memorial Cancer Institute
Hollywood Florida 33021 United States
Mount Sinai Community Clinical Oncology Program
Miami Beach Florida 33140 United States
Moffitt Cancer Center
Tampa Florida 33612 United States
Piedmont Atlanta Hospital
Atlanta Georgia 30309 United States
Rush University Medical Center
Chicago Illinois 60612 United States
Illinois Cancer Care
Peoria Illinois 61615 United States
Cadence Cancer Center
Warrenville Illinois 60555 United States
IU Simon Cancer Center
Indianapolis Indiana 46202 United States
University of Kansas Cancer Center
Westwood Kansas 66205 United States
Markey Cancer Center/University of Kentucky
Lexington Kentucky 40536 United States
Norton Cancer Institute
Louisville Kentucky 40202 United States
Tufts Medical Center
Boston Massachusetts 02111 United States
Beth Israel Deaconess Medical Center
Boston Massachusetts 02215 United States
University of Michigan Cancer Center
Ann Arbor Michigan 48109 United States
Henry Ford Hospital
Detroit Michigan 48202 United States
Spectrum Health
Grand Rapids Michigan 49503 United States
John Nasseff Neuroscience Institute at Abbott Northwestern Hospital
Minneapolis Minnesota 55407 United States
St. Luke's Hospital
Kansas City Missouri 64111 United States
Washington University School of Medicine
St. Louis Missouri 63110 United States
The Brain Tumor Center at JFK Medical Center
Edison New Jersey 08818 United States
John Theurer Cancer Center at Hackensack University Medical Center
Hackensack New Jersey 07601 United States
The Valley Hospital
Ridgewood New Jersey 07450 United States
Overlook Hospital
Summit New Jersey 07902 United States
Capital Health Regional Medical Center
Trenton New Jersey 08638 United States
Long Island Brain Tumor Center at Neurological Surgery, P.C.
Lake Success New York 11042 United States
North Shore University Hospital
Manhasset New York 11030 United States
New York University Clinical Cancer Center
New York New York 10016 United States
Mount Sinai Medical Center
New York New York 10029-6574 United States
Columbia University Medical Center
New York New York 10032 United States
University of Rochester Medical Center
Rochester New York 14642 United States
Stony Brook Medicine
Stony Brook New York 11794 United States
Brain and Spine Surgeons of New York and Northern Westchester Hospital
White Plains New York 10604 United States
University of North Carolina, Chapel Hill
Chapel Hill North Carolina 27514 United States
Wake Forest Baptist Medical Center
Winston-Salem North Carolina 27157 United States
University of Cincinnati Cancer Institute
Cincinnati Ohio 45267 United States
University Hospitals Seidman Cancer Center
Cleveland Ohio 44106 United States
Cleveland Clinic Foundation
Cleveland Ohio 44195 United States
Ohio State University
Columbus Ohio 43210 United States
OhioHealth
Columbus Ohio 43214 United States
Oklahoma University Health Science Center
Oklahoma City Oklahoma 73104 United States
Geisinger Medical Center
Danville Pennsylvania 17822 United States
Hospital of the University of Pennsylvania
Philadelphia Pennsylvania 19104 United States
Jefferson Hospital for Neuroscience
Philadelphia Pennsylvania 19107 United States
Temple University School of Medicine
Philadelphia Pennsylvania 19140 United States
Rhode Island Hospital
Providence Rhode Island 02903 United States
Medical University of South Carolina Hospitals and Clinics
Charleston South Carolina 29425 United States
Saint Thomas Research Institute
Nashville Tennessee 37205 United States
Vanderbilt Ingram Cancer Center
Nashville Tennessee 37232 United States
Baylor Research Institute
Dallas Texas 75246 United States
The Methodist Hospital
Houston Texas 77030 United States
University of Texas Health Science Center at Houston
Houston Texas 77030 United States
Cancer Therapy & Research at University of Texas Health Science Center San Antonio
San Antonio Texas 78229 United States
Benaroya Research Institute at Virginia Mason
Seattle Washington 98101 United States
Swedish Hospital Neuroscience Research
Seattle Washington 98122 United States
Aurora Saint Luke's Medical Center
Milwaukee Wisconsin 23215 United States
Montreal Neurological Institute, McGill University
Montreal Quebec H3A 2B4 Canada
CHUS - Hôpital Fleurimont
Sherbrooke Quebec J1H 5N4 Canada
Universitätsklinikum Heidelberg Neurochirurgische Klinik
Heidelberg Baden-Württemberg 69120 Germany
Katharinenhospital
Stuttgart Baden-Württemberg 70174 Germany
Universitätsklinikum FrankfurtKlinik und Poliklinik für Neurochirurgie
Frankfurt Hesse 60528 Germany
Universitätsklinikum Bonn Nervenklinik (Zentrum), Klinik und Poliklinik für Neurochirurgie
Bonn North Rhine-Westphalia 53105 Germany
Universitätsklinikum Klinik für allgemeine Neurochirurgie
Köln North Rhine-Westphalia 50924 Germany
BG-Kliniken Bergmannstrost, Klinik für Neurochirurgie
Halle Saxony-Anhalt 06112 Germany
Klinik für Neurochirurgie, Klinikum Chemnitz gGmbH
Chemnitz Saxony 09116 Germany
Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden
Dresden Saxony 01307 Germany
Neurochirurgische Klinik
Hamburg 20246 Germany
Addenbrookes NHS Trust
Cambridge Cambridgeshire, East Anglia CB2 2QQ United Kingdom
Kings College Hosital NHS Foundation Trust
London Greater London SE5 9RS United Kingdom
University College Hospital London
London Greater London WC1E 6BT United Kingdom
University Hospital of Birmingham NHS Foundation Trust
Birmingham West Midlands N15 2WB United Kingdom
Location Countries
Country
Canada
Germany
United Kingdom
United States
Verification Date
2016-10-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
oncology , neurology , glioma , brain tumor , brain cancer , glioblastoma multiforme , glioblastoma , newly diagnosed glioblastoma , immunotherapy , dendritic cells , immune therapy , GBM , Brain cancer, primary , tumor vaccine , grade IV astrocytoma , DCVax , Grade IV brain cancer
Has Expanded Access
No
Condition Browse
Glioblastoma , Glioma , Brain Neoplasms , Astrocytoma
Number Of Arms
2
Arm Group
Arm Group Label
treatment cohort
Arm Group Type
Active Comparator
Arm Group Label
Placebo Chohort
Arm Group Type
Placebo Comparator
Description
Autologous PBMC
Firstreceived Results Date
N/A
Acronym
GBM
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Study First Submitted
September 17, 2002
Study First Submitted Qc
September 18, 2002
Study First Posted
September 19, 2002
Last Update Submitted
October 12, 2016
Last Update Submitted Qc
October 12, 2016
Last Update Posted
October 14, 2016
Last Known Status
Active, not recruiting
ClinicalTrials.gov processed this data on May 22, 2020
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Medical Technologist II (1st shift)
Indianapolis, Indiana, United States
CRA I/ II - sponsor dedicated
Budapest, Hungary
Proposal Associate
Madison, Wisconsin, United States
Principle Investigator/Lead Scientist II-Immunochemistry
Somerset, New Jersey, United States
Clinical Research Manager (6 months FTC)
Lisbon, Portugal
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Is that us ?
it was 450,000 some was filled
The snowball has now been let go from the top of the mountain by the time it stops rolling it will be huge
NWBO has started to arrive
Thank You.
Does anyone know anything about this company
Novan Inc. on Monday said it hired H.C. Wainwright & Co., LLC "to assist the company in exploring and evaluating a range of strategic and financial alternatives, intended to maximize shareholder value."
Novan said it advanced several late-stage development programs in the field of dermatology, including its lead product candidate, SB206, currently in Phase 3 clinical trials for molluscum contagiosum, "a primarily pediatric contagious skin infection."
The company said it "believes that its clinical and preclinical data, technology platform and market potential, will provide for a range of opportunities in order to maximize shareholder value."
The company's stock was moving higher after hours Monday following its announcement. At 4:58 p.m. EDT, shares had risen 2.47%, to trade at 41 cents. Over 3.1 million shares had traded.
--Write to Stephen Nakrosis at
He reminds me a lot like Turtle.
scott Tell us why you feel the need to give us your advice? why do you care what happens with the company if your not invested ? Do you have some sort of job that pays you to write what you write?
Im having trouble understanding why anyone would waste so much of their time doing what you do for no reason. Not trying to insult you at all Im really trying to see the point of what your doing.
YW. GL to all
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The Wall Street Journal
PRESS RELEASE
NW Bio To Discuss Projected Schedule For Data Lock, Unblinding and Top Line Data From Its Phase 3 Clinical Trial At Annual Shareholder Meeting
April 18, 2020 1:10 pm ET
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TEXT
BETHESDA, Md., April 18, 2020 /PRNewswire/ -- Northwest Biotherapeutics (OTCQB: NWBO) ("NW Bio"), a biotechnology company developing DCVax(R) personalized immune therapies for solid tumor cancers, today announced that its CEO, Linda Powers, will discuss the projected schedule for reaching data lock, unblinding and reporting of top line data from its 331-patient Phase 3 trial of DCVax(R)-L for Glioblastoma brain cancer at the Company's 2019 Annual Meeting. The Meeting is being held virtually due to the current public health crisis.
The Company has been working since last year with the contract research organization (CRO) that managed the trial and numerous independent service companies to make the final in-person monitoring visits to all the clinical trial sites (hospitals) across the US and Europe, and to finish collecting and confirming the Phase 3 trial data and resolving queries.
Despite nearly two months (during March and April to date) in which hospital trial sites stopped allowing in-person data monitoring visits and became too overwhelmed to continue helping with data confirmation, the Company's data collection and confirmation process has continued moving forward in part through workarounds.
The data collection process is including certain epigenetic and genetic information that is recognized as important in Glioblastoma, such as MGMT methylation status. As part of this process, the Company has also identified a method that can potentially enable an additional important genetic factor -- IDH mutation status -- to be analyzed using bio samples collected years ago during the trial, and to be analyzed in the same timeframe as the data lock. This IDH mutation factor was unknown when the Company's trial began and through much of the trial period, but has become recognized as very important in recent years.
After factoring in the March and April shutdowns, and the additional genetic analysis, the Company believes it can reach data lock by approximately the end of May.
Upon reaching data lock, the data will be unblinded to the independent statisticians (i.e., the statisticians will be given access to the trial database containing all of the raw data points). The Company will not yet become unblinded at this time.
The independent statisticians will then use the raw data to calculate the relevant measures, such as median survival times and survival percentages at various time points. The statisticians will also calculate various statistical measures and prepare graphs and tables. This work is anticipated to take several weeks. The Company will become unblinded when it receives these results from the statisticians.
The Company will then discuss the information from the statisticians with its expert advisors, including its Scientific Advisory Board and the Steering Committee of the Phase 3 trial. Any questions or comments raised by the experts will be addressed and the results will be prepared for public announcement.
Based on these expectations, and taking account of the two months lost in March and April, the current estimate of public disclosure of top line data would range from the end of June to early July.
About Northwest Biotherapeutics
Northwest Biotherapeutics is a biotechnology company focused on developing personalized immunotherapy products designed to treat cancers more effectively than current treatments, without toxicities of the kind associated with chemotherapies, and on a cost-effective basis, in both the North America and Europe. The Company has a broad platform technology for DCVax(R) dendritic cell-based vaccines. The Company's lead program is a 331-patient Phase III trial of DCVax(R)-L for newly diagnosed Glioblastoma multiforme (GBM). GBM is the most aggressive and lethal form of brain cancer, and is an "orphan disease." The Company is also pursuing development of DCVax(R)-Direct for inoperable solid tumor cancers. It has completed a 40-patient Phase I trial, and is preparing for Phase II trials. The Company previously conducted a Phase I/II trial with DCVax-L for advanced ovarian cancer together with the University of Pennsylvania.
Disclaimer
Statements made in this news release that are not historical facts, including statements concerning future treatment of patients using DCVax and future clinical trials, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "expect," "believe," "intend," "design," "plan," "continue," "may," "will," "anticipate," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We cannot guarantee that we actually will achieve the plans, intentions or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. Actual results may differ materially from those projected in any forward-looking statement. Specifically, there are a number of important factors that could cause actual results to differ materially from those anticipated, such as risks related to the Company's ability to enroll patients in its clinical trials and complete the trials on a timely basis, uncertainties about the clinical trials process, uncertainties about the timely performance of third parties, risks related to whether the Company's products will demonstrate safety and efficacy, risks related to the Company's ongoing ability to raise additional capital, and other risks included in the Company's Securities and Exchange Commission ("SEC") filings. Additional information on the foregoing risk factors and other factors, including Risk Factors, which could affect the Company's results, is included in its SEC filings. Finally, there may be other factors not mentioned above or included in the Company's SEC filings that may cause actual results to differ materially from those projected in any forward-looking statement. The Company assumes no obligation to update any forward-looking statements as a result of new information, future events or developments, except as required by securities laws.
CONTACTS
Dave Innes Les Goldman
804-513-4758 dinnes@nwbio.com 240-234-0059 lgoldman@nwbio.com
--------------------------------
View original content to download multimedia:http://www.prnewswire.com/news-releases/nw-bio-to-discuss-projected-schedule-for-data-lock-unblinding-and-top-line-data-from-its-phase-3-clinical-trial-at-annual-shareholder-meeting-301043144.html
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Looks like the news yesterday made the WSJ
Fuggedaboutit Its on now!
Absolutely.. Enjoy the weekend.
Turtle?
Agree. We will continue to add to our position.
I have to agree with your view on tomorrow. I also know a lot of investors in the company who have been loyal to them for a long time. We have spoken and all have the same feelings. If tomorrow is the same old story we will have no reason to trust anymore.
Thank you I thought we were testing the site to see if it was working. GL to all of us
Hi Senti i tried logging on with my control number and used NWBO2020 for the password. The site said incorrect password
So they use him like a rolodex ?
cybersecurity
Agreed. 7 years worth of BS now. Sometimes I think black was hired to protect them instead of us.
Exactly.
Dr Duffy left for a better offer? Thats what they are telling us. This wont change anything with the company so they are saying. Ive been around for a long time with this company they have lead us down a river of hope. I wish I could say I trust what they tell us however its been the same story over, and over,year, after year we are left disappointed with the never ending dreams of success. The years are gone and the money is as well. I hope April 18th will bring the answers we all have been waiting for. If not maybe we should all leave for a better offer as well.
Maybe margin calls on other stocks.
csh long island ?
I know how you feel. A lot of us are in that same position. From 3$ up to 12$ then down to .17 and never selling. Trusting the science and believing that the company was being upfront with the little amounts of information they would release. We have all heard it so many times that great news is just around the corner. I have to say after all the years and a lot of money lost I would agree that the company seems like they are losing the faith of so many long term holders. Im sure along the way this has effected so many investors and their families as well. We all know that there are no guarantees when we invest however honesty from the company is something we should expect and demand. That being said i will remain holding my position and watching everyday. GLTA
.
Neon Therapeutics (NTGN) said Monday that a phase 1b trial evaluating its NEO-PV-01 vaccine combined with Bristol-Myers Squibb's Opdivo showed prolonged median progression-free survival, or PFS, in multiple types of cancer.
The company evaluated the drug combination in patients with advanced or metastatic melanoma, smoking-associated non-small cell lung cancer and bladder cancer.
At 13.4-month median follow-up in 34 patients with metastatic melanoma, median PFS had not yet been reached. In 27 patients with metastatic NSCLC, median PFS was 5.6 months, and in 21 patients with metastatic bladder cancer, median PFS was 5.6 months.
Neon said the data support further development of the vaccine, including randomized phase 2 trials.
How Have We Got It So Wrong?
By John F. Smyth, MD
June 10, 2019
Get Permission
John F. Smyth, MD
John F. Smyth, MD
The past 20 years have seen an unprecedented increase in the development of effective drugs for the management of cancer. The advent of immunotherapy offers even the promise of cure for some previously highly resistant diseases. The science is brilliant, the need is ever increasing—but the cost is unaffordable! How have we got this so wrong?
It is easy to criticize and blame those involved in the separate components of drug development, but I suggest that ultimately, this is a responsibility for the whole of society to address. Innovation comes from academia and the pharmaceutical industry; we have developed excellent partnerships, and the time from bench to bedside has been considerably reduced. The latter has been helped by genuine cooperation between clinical researchers and regulators, especially the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), in designing trials that are much more likely to result in regulatory approval than was the case in the past. However, the biggest hurdle is post licensing, when payers and health technology assessors have to decide whether the price is acceptable to achieve value for money—and the crunch is to assign that concept of “value” to a new intervention, in comparison to alternatives and the opportunity cost/loss when budgets are limited, which they always will be.
Defining Value on Both Sides of the Atlantic
In 1892, Oscar Wilde famously defined a cynic as “a man who knows the price of everything and the value of nothing.” I do not think that any of us needs to be cynical about the costs of new medicines, but we do need to better define “value.”
In an excellent article by Hagop M. Kantarjian, MD, in The ASCO Post,1 he addresses the U.S. position as to whether the Trump administration’s plan to reduce cancer drug prices will work. Dr. Kantarjian quotes that the average price of a cancer drug ranges from $10,000 per year to $170,000 between the years 2000 and 2017.2 With so many new medicines being approved, it was hoped that market forces would push prices down, but this has not happened. Dr. Kantarjian quoted the U.S. Department of Health and Human Services Secretary Alex Azar as saying the drug industry’s repeated mantra that it must make large profits to pay for research and innovation was a tired point, and he maintained that the biggest problem was simple: drug prices are too high.
One of Dr. Kantarjian’s proposed solutions is to follow the European Union (EU) practices based on objective benefit (value-based pricing). However, in Europe, we still have far too much variation in what we call “value.”3
In most, but not all, countries in the EU, following an approval from the EMA, a new project is subject to health technology assessors. In the UK, the National Institute for Clinical Excellence (NICE) decides for all (except Scotland, where we have our own Scottish Medicines Consortium). Recently, the latter rejected chimeric antigen receptor T-cell therapy with tisagenlecleucel for adults with diffuse large B-cell lymphoma, despite a positive approval for the rest of the UK by NICE—extraordinary!
The challenge facing health technology assessors is enormous, and the very success of research across many areas of medicine compounds this problem when faced with fixed budgets. Our National Health Service in the UK is more than creaking at the seams for many reasons, and the shameful arguments over Brexit will only compound this. However, we have to find more acceptable ways to agree on “value for money” to allow the right patients access to potentially curative treatment (in this example).
Difficulty in Assessing New Cancer Treatments
Great progress has been achieved in the past decade by regulators, especially at the FDA and EMA. In a thoughtful “perspective” in The New England Journal of Medicine, published in February 2019,4 Tatiana Prowell, MD, and colleagues used the example of neoadjuvant treatment of breast cancer to highlight the difficulties for everyone involved in assessing new agents for a cancer where there are so many other possible influences on overall survival. They highlighted the
The actual cost of developing an individual drug is difficult to determine, but, surely, we need to rebalance the ambition of needing to reward shareholders with the cost of new medicines.
— John F. Smyth, MD
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development of protocols that use increasingly complex, expensive, multidrug regimens, which expose patients to overtreatment with unnecessary toxicities, both in the short and long term—the very antithesis of what precision medicine seeks to avoid.
The development of biomarkers and genetic profiling has led to the concept of smaller, well-characterized groups of patients being selected for pivotal trials to facilitate smaller and faster regulatory submissions. This challenges the statisticians5 but “should” result in both better patient outcomes and reduced financial burden for patients and society. But is this happening? There is little evidence to date that it is.
‘One of the Greatest Challenges of Our Time’
Ultimately, it is for the highly profitable pharmaceutical industry to reduce the costs of medicines, and there “are” ways to achieve this without losing support from shareholders. This is where society as a whole needs to play its part in refining what we mean by “value” and perhaps accepting a lower return on investment across the health-care spectrum.
Late in 2018, ASCO and the European Society for Medical Oncology published a joint assessment of their value frameworks, highlighting concordance but also revealing significant discordance.6 The latter is attributed to using different approaches to the evaluation of relative and absolute gains for overall survival, and progression-free survival, crediting the tail of the curve gains and assessing toxicity.6 It is hoped that the development of robust tools to assess “value” will help balance the argument for pricing vs the opportunity cost in allocating budgets for health care.
I believe that this issue of how we value health care poses one of the greatest challenges of our time. Continued successful research will only compound our choices, but tough decisions must be made. The actual cost of developing an individual drug is difficult to determine, but, surely, we need to rebalance the ambition of needing to reward shareholders with the cost of new medicines. This past year, in an interview with the Financial Times, the chief executive of a major pharmaceutical company was quoted as saying quite openly that, “their primary responsibility was to their shareholders”; there was no mention of patients or the needs of health-care deliverers at all! Surely, we can do better than that.
Reducing Cost of New Agents: A Benefit for All?
Of course, we need investment in the pharmaceutical industry—its products are essential, and despite the costs of failure, the rewards make it one of the most successful area for investors. Businesses increasingly wish to be credited with an ethical approach. I am therefore suggesting that for sustainability and above all to make good medicines available to those most in need, there may be a case globally for being less ambitious about returns that impact directly on cost. Since everyone eventually needs health care, is it beyond the realm of possibility that investors and shareholders, who represent an important sector of society, could accept smaller gains than they currently expect and thereby help the “market forces” argument to reduce the cost of new medicines? Would it not be to everyone’s benefit to do so? ¦
Dr. Smyth is Professor Emeritus of Medical Oncology at the University of Edinburgh, Scotland.
DISCLOSURE: Dr. Smyth has served as an advisor/consultant for Bristol-Myers Squibb, Merck, and Northwest Biotherapeutics (NWB); and has stock ownership in NWB and AstraZeneca.
REFERENCES
1. Kantarjian HM: Will the Trump administration’s plan to reduce cancer drug prices work? The ASCO Post. December 25, 2018.
2. Kantarjian H, Patel Y: High cancer drug prices four years later: Progress and prospects. Cancer 123:1292-1297, 2017.
3. National Institute for Health and Care Excellence: Cost/Benefit of Cancer Drugs. November 5, 2018. Available at www.nice.org.uk/search. Accessed May 22, 2019.
4. Prowell TM, Beaver JA, Pazdur R: Residual disease after neoadjuvant therapy: Developing drugs for high-risk early breast cancer. N Engl J Med 380:612-615, 2019.
5. Vogl SE: The risks of drug approval based on shaky evidence. The ASCO Post. March 25, 2019.
6. Cherny NI, de Vries EGE, Dafni U, et al: Comparative assessment of clinical benefit using the ESMO-Magnitude of Clinical Benefit Scale version 1.1 and the ASCO Value Framework Net Health Benefit Score. J Clin Oncol 37:336-349, 2019.
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