Government Reform and Oversight Committee
February 4, 1998
Mr. Charles B. Simone, M.MS., M.D.
Charles B. Simone, M.MS., M.D. graduated from Rutgers Medical
College (1971-1975). He is an Internist (trained at the Cleveland
Clinic 1975-1977), Medical Oncologist (trained at the National
Cancer Institute 1977-1982), Tumor Immunologist (trained at
the National Cancer Institute 1977-1982), and a Radiation
Oncologist (trained at the University of Pennsylvania 1982-1985).
Working with Senator Harkin, he helped to shape the Office
of Alternative Medicine, National Institutes of Health. In
addition, he also helped to organize a Department of Alternative
Medicine for Columbia and Mt. Sinai in NYC.
While at the NCI, his basic science research uncovered the
fundamental mechanism of how human white blood cells kill,
helped show how "complement" proteins aid in killing, demonstrated
how adriamycin works, and developed directed effector cells.
One of the first patients he consulted with at the NCI was
a senior statesman who was dying of malnutrition secondary
to his cancer. Later, a man his own age with a newly pregnant
wife came to him at the NCI and asked to be kept alive for
the birth of his child. An intensive course of chemotherapy
cleaned out the cancer cells, but the patient failed to improve.
"I decided at last resort to put him on high doses of vitamins
and minerals that quickly produced a temporary recovery.."
The man lived to see the birth of his son.
He began devoting some of his time investigating the effects
of nutrition on cancer and the possibilities of cancer and
disease prevention. The result was Cancer and Nutrition, A
Ten Point Plan to Reduce Your Risk of Getting Cancer (1981
McGraw-Hill, revised 1994 Avery). He also wrote Breast Health,
Shark Cartilage and Cancer, Prostate Health, KidStart, and
Sports and Nutridon.
He has testified as an expert in the fields of cancer, nutrition,
nutritional supplementation, disease prevention, and medical
care cost reduction before the United States Senate and the
United States House of Representatives of 1993, 1994, and
1995.
THE NEED FOR CHANGE
Billions of dollars have been invested in cancer research
and treatment since 1971 when President Nixon declared War
on Cancer. Each month, it seems, new therapies are trumpeted.
Some show promise, most fizzle quickly.
Cancer will emerge as the number one cause of death in the
United States by the year 2000. Despite the enormous effort
to combat cancer, the number of new cases of nearly every
form of cancer has increased annually over the last century.
Still worse, from 1930 to the present, despite the introduction
of radiation therapy, chemotherapy, and immunotherapy with
biological response modifiers, despite CT scans, MR scans,
and all the other new medical technology - life spans for
almost every form of adult cancer except cervical cancer and
lung cancer have remained constant, which means that there
has been no significant progress in cancer treatment (Figure
1 attached, data from the National Cancer Institute SEER Program,
published by the American Cancer Society each January).
"Cure" is largely elusive or statistically disguised. "Cure"
means surviving 5 years after treatment - if death occurs
at 5 years and one day, the "cure" is unaltered in the statistical
record.
The chilling prospect remains - by the year 2000, two of
every five Americans will develop cancer. And most will die.
Because of these dismal survival statistics with existing
conventional treatments, we need to redirect our attention
to two important areas. Prevention of cancer and other diseases;
and pursue totally New Substances or New Modalities that show
scientific merit for treatment even though they may not yet
be approved by the Food and Drug Administration for widespread
use.
FDA REFORM ??
Because FDA funding must be used for more food safety work,
it has been estimated that there will be a 60% to 70% reduction
in funds that would otherwise be assigned to tenure-track
scientists in the Division of Viral Products, Center for Biologics
Evaluation and Research (Feinstone SM, Lewis AM, Markoff LJ,
Carbone K, Golding H.[all lab chiefs in that Division] Science.
January 9, 1998; 279:157-159.
We certainly do not want to lose excellent scientists, however,
the dollars earmarked for research should be used only for
evaluation of Other governmental agencies are organized for
research.
Henry I. Miller, from the Hoover Institution, Stanford University,
writes about FDA Reform in the same cited Science article:
First, it calls for "promptly and efficiently reviewing clinical
research" "in a timely manner." But these words will not have
any impact on the agency's 30 year tradition of risk aversion
and foot-dragging.
Second, it calls on FDA to develop a plan by the year 2000
to clear the legendary backlog of products awaiting approval.
Congress here makes itself a hostage to an endless series
of demands for additional resources the FDA will say it needs
to do this.
Third, it codifies many policies that are already in place,
giving the impression of a lengthy list of improvements.
The most important provision offers drug companies greater
latitude in supplying scientifically sound information to
doctors about drugs' "off label" uses (those not yet approved
by FDA). Companies are currently prohibited from distributing
such critical information. But even this improvement comes
at a high price: substantial additional paperwork to convince
FDA that formal applications for approval of the new uses
are forthcoming. [This provision of "off label" uses is very
valuable for the patient. CB Simone, M.D.]
A welcome provision permits manufacturers to submit "health
care economics information," such as data on a drug's cost-effectiveness,
to hospitals and HMOs.
The bill contains other minor improvements, such as loosened
restrictions on health claims for food products and expanded
use of third party, including academic institutions, to review
medical devices.
However, one provision actually increases the scope of FDA's
regulation by expanding its jurisdiction to activities that
occur completely within a single state - small-scale research
by an academic or a practicing physician testing an innovative
therapy.
Many critical reforms recommended by blue-ribbon panels are
absent. These include reducing the redundancy of regulation
of early-stage clinical trials and a binding reciprocity provision
that, for example, would limit the duration of FDA review
of a new drug to a maximum of, say 60 days after its approval
in the United Kingdom or by the European Medicines Evaluation
Agency (thereafter, the FDA would have to show cause why the
drug should not be marketed in the United States, or it would
automatically be approved).
Following Congress's failure to accomplish significant FDA
reform, the costs of drug development (already averaging more
than $500 million to bring a single product to market) will
continue to rise, fewer drugs will be developed, and market
competition will erode. Patients will suffer higher prices
and benefit from fewer breakthrough drugs.'
FDA Should Not Be In The Drug Discovery Business
"FDA finds potential cancer treatment" (USA Today 12-12-97)
The Associated Press reported in USA Today on December 12,
1997 that the FDA "found a promising new treatment for cancer
and licensed it" to Neopharm Inc. an Illinois biotechnology
company. The private firm will need FDA approval to sell it.
Obviously, the FDA has a conflict of interest for this approval.
The chief executive of Neopharm, William Govier, said "his
company may have saved $100 million in dug-development work
by merely licensing the FDA's discovery." The FDA Reform Bill
passed by Congress includes requirements to speed the review
of new vaccines and drugs and to reauthorize the Prescription
Drug User Fee Act. To accomplish this, the FDA will tap "user
fees" that are charges to companies that submit products for
FDA review and approval.
However, the FDA uses about $10 million a year (USA Today
article) in industry fees to fund their research labs. The
biotechnology industry protested the new drug discovered by
the FDA. "It should stick to regulation and leave discovery
to industry."
The taxpayer has essentially funded a private company's Research
and Development. That company will have saved over $100 million.
The taxpayer should be compensated and the FDA should deal
only with regulation and not discovery. There are many other
Federal research labs that are in the business of discovery.
The Dietary Supplement Health and Education Act of 1994
Having helped to write some of the key language for the Dietary
Supplement Health and Education Act of 1994 with Senators
Harkin and Hatch, I was very disappointed in the proposed
statements of the President's Commission on Dietary Supplement
Labels. The Commission issued a draft report for public comment
before it made its final recommendations to the President,
Congress, and the FDA.
The report was a disaster. It completely ignored the subject
Congress created the Commission to address: namely, "how best
to provide truthful and non-misleading information to consumers
so that such consumers may make informed health care choices."
Instead, the Commission simply placed its stamp of approval
on the FDA's current prior restraint on all health claims,
except those pre-approved and recommended the adoption of
safety, reporting, and OTC botanical regulations that are
beyond the scope of its delegated authority. The tragedy is
compounded by the fact that the Commission's recommendations
are required, by law, to be published by FDA as proposed rules,
making it possible that the agency will adopt one or more
of the suggestions.
Government Interference Pith Choice of Treatment by Informed
Patients
I have been called upon many times by patient advocates to
determine whether the treatment outcome desired by the patient's
guardians or patient will equal or be better than the outcome
of existing conventional treatment. Often times, these patients
or their guardians find themselves entangled with legal issues
because the physician wants to impose the conventional treatment
indicating "it will save the patient's life," or "it will
cure you." Or, the patient has received all conventional treatment
without success and then wants to try an Investigation New
Drug approved by the FDA for research purposes. The patient
attempts to obtain this drug but finds himself or herself
ineligible according to the strict research criteria. In these
instances, when a patient has an unwanted treatment unposed
upon him or her by a governmental agency, or when he or she
desires an Investigational New Drug, I review the medical
records to make a determination of the various effective treatment
options, and whether any one option is superior to another,
and importantly, whether a particular treatment option will
increase life span. Remember that a critical measurement in
cancer care is whether a specific treatment will increase
the life span of the patient. Examples:
You have already heard the eloquent and heart wrenching story
of Zachary McConnell by Jonathan Emord, Esq. Zachary, age
5, had a rare brain tumor and was enrolled in a FDA approved
Investigational New Drug protocol. While under this treatment
the FDA decided to stop the protocol. His parents fought this
decision legally.
An eleven year old girl, EU, diagnosed with non-metastatic
high-grade osteosarcoma of the left distal femur was treated
with three cycles of appropriate chemotherapy with adequate
doses for osteosarcoma. Her mother, with whom I spoke, wanted
no further chemotherapy and wanted her daughter to proceed
to surgery. Her physicians wanted her to have several more
months of chemotherapy before the surgery. After reviewing
all the data, including the patient's records and imaging
scans, I was convinced from the published medical journal
articles that the patient should proceed with surgery because
no benefit in lifespan or local control is achieved when more
than three cycles of chemotherapy is administered before surgery.
Any delay in surgery brought a higher risk for distant metastasis.
The judge in the case overturned the court order requiring
the patient to have more chemotherapy.
TRUE FDA REFORM SHOULD INCLUDE
Easier access to Drugs for terminally ill patients and once
a patient is enrolled in an FDA approved protocol, never stop
that treatment. Allow physicians to have access to "off label"
information. Assign New Time Limit for FDA Review of Applications,
and limit FDA review of a new drug to 60 days if that drug
has prior approval in United Kingdom or European Medicines
Evaluation Agency. Discontinue FDA 's Discovery Research,
and Recover for the American taxpayer the $100 million saved
by Neopharm, Inc., and other monies possibly gleaned in a
similar fashion by other companies. In the short term, hire
additional staff to clear the backlog of products that await
approval. Rescind FDA 's regulatory authority within a single
state.
Answers to the Following:
1) How many applications per year are submitted for protocol
approval (Investigational New Drug, New Drug Approval, etc)?
2) How many of these are approved and in what period of time?
3)How many potential applicants stop the process after attempting
to complete the paperwork for the application.
4)How many applications are submitted by "professional" application
writers - attorneys, past FDA people, etc.
5) To how many potential applicants does the hiring of these
"professionals" present an impediment?
6) What is the average cost of getting an application ready
for submission to FDA?
7) What is the average length of time needed to complete an
application by the applicant?
8)How effective is the FDA at helping potential applicants
to complete the application if an applicant requests help
from the FDA?
9) How many patients request from the FDA an "off-label use"
treatment or one that has an IND for which they may not be
eligible?
10) How many of those patients receive such treatment?
Informed Consent could be a very powerful tool for the patient.
If the concept of Informed Consent was truly enforced and
fully explained, patients would then understand the limitations
of many treatments. Misinformation is sometimes given to patients.
A glaring example is found in a Sunday front page New York
Times article on October 26, 1997 entitled 'Vitamin Mania,
Millions Take a Gamble on Health.' Larry Norton, M.D., a staff
person from Memorial Sloan Kettering, a highly regarded institution,
was interviewed and stated:
"Research at his institution showed that large doses of vitamin
C could blunt the beneficial effects of chemotherapy for breast
cancer. The research showed that breast cancer cells had large
numbers of receptors, or docking places, for vitamin C, suggesting
that the vitamin acted like a tonic for cancer cells.
And a recent experiment showed that free radicals, chemicals
that damage cells in ways that may lead to cancer, are also
necessary for some of the mechanism that stop cancer once
it gets going. So a substance like vitamin C, in large doses,
could have unpredictable effects. It is also known that folio
acid can negate the effects of methotrexate, a drug used to
treat cancer."
This "information" is absolutely incorrect. Over 200 peer-reviewed
scientific articles have been published in medical journals
in the 1970s, 1980s, and 1990s. Summarized in books and medical
journals, the correct information shows that nutritional modification,
including the use of certain nutrients, and proper lifestyle
can dramatically decrease the morbidity and side effects of
chemotherapy and radiation therapy as well as increase response
rates. There have even been some reports that nutritional
and lifestyle modification actually increase survival.
Simone, CB. Cancer and Nutrition (1992 revised Avery Publisher).
Simone, CB. Breast Health (1994 Avery Publisher)
Simone CB, Simone NL, Simone CB II. Oncology care augmented
with nutritional and lifestyle modification. J Ortho Mol.
Med 1997; 12(4): 197-206.
Simone CB, Simone NL, Simone CB II. Folic acid does not interfere
with methotrexate. Lancet. 1997; 350: 1556.
Simone CB, Simone NL, Simone CB II. Lifestyle modification
in oncology care. In: Prasad, KN ed. Cancer and Nutrition.
1997. Amsterdam, Netherlands, IOS Press.
Simone CB. Nutritional and Lifestyle Modification in Oncology
Care. In: Torosian M. Integrated Cancer Management: Surgery,
Medical Oncology, and Radiation Oncology. 1998. Marcel Dekkar,
New York. (University of Pennsylvania)
Summary
The patient's well being must come first in all instances.
Patients need to have access to treatments and information
that potentially may benefit them. The FDA should serve the
public and not be an obstruction.
|
