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Contributing Author: Jeanette
Bowers Weaver
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The information contained and presented
herein is intended to provide general information only. It
should not be construed or relied upon as individual legal
advice and does not constitute an offer to represent you.
The legal considerations discussed herein may differ widely
based upon the specific situation. In addition, some information
may be incomplete, out-of-date, or inapplicable to your jurisdiction
(State).
JEANETTE BOWERS WEAVER AND JEANETTE
BOWERS & ASSOCIATES, PLLC MAKE NO WARRANTY, EITHER EXPRESS
OR IMPLIED, REGARDING THE COMPLETENESS, ACCURACY OR CURRENCY
OF THIS INFORMATION OR ITS SUITABILITY FOR ANY PARTICULAR
PURPOSE.
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PROKARIN™ is a compounded drug exempt from FDA approval
The Federal Food, Drug and Cosmetic Act (FFDCA) prohibits
the marketing of any “new drug” without prior approval
of the Food and Drug Administration (FDA).[1]A “drug”
is defined as “articles recognized in the official United
States Pharmacopoeia” and “articles (other than
food) intended to affect the structure or any function of
the body of man . . .”[2] The term refers not only to
the active ingredient in a drug product, but refers to the
entire product.[3] The term “new drug” is defined
as “[a]ny drug . . . [which is] not generally recognized,
among experts qualified by scientific training and experience
to evaluate the safety and effectiveness of drugs, as safe
and effective for use under the conditions prescribed, recommended,
or suggested in the labeling thereof. . .” or any drug
that “has become so recognized, but which has not . .
. been used to a material extent or for a material time under
such conditions.”[4]The newness of a drug may arise from
a new drug use of a combination of two or more substances,
none of which is a new drug, or the newness of the proportion
of a substance in a combination, or the newness of the method
of administration or application of such drug, even though
the drug when used in another method of administration or
application is not a new drug.[5]
When a drug product is compounded by a licensed pharmacist
or licensed physician using drug substances that are components
of FDA approved drugs, then FDA approval is not required to
prescribe the product.[6]The FDA has long recognized that
“once a product has been approved for marketing, a physician
may prescribe it for [off-label] uses or in treatment regimens
of patient populations that are not included in approved labeling.”[7]
Prokarin™ is an off-label drug prescribed by physicians
and compounded by pharmacists using drugs that are FDA approved.[8]The
FDA entrusts physicians and pharmacists with the knowledge
and skill to provide safe and effective off-label prescriptions
to their patients, and consequently does not require off-label
drugs such as Prokarin™ to be approved by the FDA for
the use in treatment of MS.
PROKARIN™ does not require an NDC number
NDC numbers are 10-digit numbers used to identify manufactured
drug products.[9]They are assigned when establishments engaged
in the “manufacture, preparation, compounding, or processing”
of drugs register their business and list their products with
the Secretary of Health and Human Services (Secretary). [10]Generic
drugs that have the same active ingredients and excipients
together are covered by the NDA of the pioneer drug copied
and do not require an NDA to be marketed.[11]EDMS is not engaged
in any activity that requires registration of its business
or product. Pharmacies which prepare Prokarin™ for sale
in the regular course of their business are exempt from the
requirement to register and list the drug because Prokarin™
consists of drug articles already registered and listed by
other manufacturers.[12]Subsequently, Prokarin™ will
never receive an NDC number even if EDMS elects to submit
listing information because the most it can obtain is a 5-digit
Labeler Code.13]
PROKARIN™ is not an “investigational new drug”
in practice.
The Secretary has been granted authority to promulgate regulations
for the efficient enforcement of the Federal Food, Drug, and
Cosmetic Act (FFDCA) (§§ 201-902, 52 Stat. 1040
et seq., as amended, codified at 21 USC §§ 301-392).[14]
These regulations define the term “investigational new
drug” as a “new drug . . . that is used in a clinical
investigation. 15]A clinical investigation is “any experiment
in which a drug is administered or dispensed to, or used involving,
one or more human subjects.” [16]An experiment is “any
use of a drug except for the use . . . in the course of medical
practice.”[17] Because Prokarin™ is not a new drug
currently undergoing clinical trials, it is not an "investigational
new drug" as that term is defined by the FFDCA.
However, patients have two methods of obtaining access to
investigational new drugs. One method is to obtain the drug
from a licensed pharmacist who compounds the drug based upon
an unsolicated prescription. [19]The other method involves
obtaining the drug directly from the manufacturer or distributor
of the drug, which is currently not the method used for obtaining
Prokarin™. [20]Patients now have the right to obtain
access to investigational drugs intended for serious diseases
such as MS directly from a manufacturer through their physician
[21]This method of access can be achieved by several methods,
one of which is when a protocol for widespread use is submitted
by the drug sponsor or a physician to the Secretary. [22]The
Secretary must permit this method of expanded direct access
to the new drug if the following conditions are met:
- the new drug is intended for use in the treatment of
a serious disease or condition;
- there is no comparable or satisfactory alternative therapy
available to treat the disease or condition in the patients
for which the drug is intended;
- the drug is under investigation in a controlled clinical
trial for the use intended under an investigational drug
application in effect under 21 USC § 355(i);
- the sponsor is actively pursuing marketing approval for
the use intended with due diligence; and
- there is sufficient evidence of safety and effectiveness
to support the intended use. [23]
However, when a so-called investigational new drug is obtained
from and compounded by a licensed pharmacist, it is exempt
from FDA application and approval procedures normally required
of all investigational new drugs. [24]
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References
[1]21 USC § 355.
[2]21 USC § 321(g)(1).
[3]See United States v. Generix Drug Corp., 460 U.S. 453, 454 (1983).
[4]21 USC § 321(p)(1), (p)(2).
[5]See 21 CFR § 310.3 (h).
[6]See 21 USC § 353a(a) (effective Nov. 21, 1998). NOTE: the
Secretary of Health and Human Services will eventually issue regulations
to implement § 353a, as required under § 353a(d)(1), and
has reserved 21 CFR Pt. 216, Subpt. A and 21 CFR § 216.23 presumably
for these regulations when made available.
[7]FDA Drug Bulletin 12:4, 1982, reprinted in 59 FR 59820, 59821,
1994.
[8]Histamine Phosphate (also known as Histamine di-phosphate) and
caffeine.
[9]See 21 CFR § 207.35.
[10]21 USC § 360(b); See also 21 CFR § 207.20 and §
207.35.
[12]See 21 USC § 360(a),(g).
[13]See 21 CFR § 207.20 (b) and § 207.35(2)(i) (owners
of an establishment not otherwise required to register that distribute
under their own label or trade name a drug manufactured or processed
by a registered establishment “may” elect to submit listing
information directly to the FDA and obtain a 5-digit Labeler Code).
[14]See 21 USC § 371(a) and § 321(d).
[15]21 CFR § 312.3(b).
[16]21 CFR § 312.3(b).
[17] Id.
[18] At the moment, Prokarin™ is listed on the Internet as
currently undergoing clinical studies. See http://www.cairx.com/page8.html
[19] See 21 USC § 353a(a).
[20] See 21 USC § 360bbb(b).
[21] See 21 USC § 360bbb(b). The term “serious disease”
is not defined by the Act, but a debilitating disease with no known
cure such as MS would likely fall within its meaning.
[22] See 21 USC § 360bbb(c).
[23] 21 USC § 360bbb(c).
[24] See 21 USC § 353a(a)(exempting pharmacy compounding from
the provisions of § 355 on new drug applications).
[25] See Hoffman, supra note 25.
[26] Id. citing to Catherine A. Voigt & Kevin J. Conlon, Insurance
Coverage for Experimental Treatment: New Hope for Patients, 83 Ill.
B.J. 396, 398 (1995).
[27]See E. Farnsworth, Contracts 479-80 (1982); R. Jerry, Understanding
Insurance Law 95-98 (1987); K. Abraham, Insurance Law and Regulation
47 (2d ed. 1995).
[28]See Johnson v. District Z Marine Eng’rs Beneficial Ass’n,
857 F.2d 514, 516 (9th Cir. 1988), cert. denied, 111 S.Ct. 581 (1990).
[29]See Zuckerberg v. Blue Cross & Blue Shield, 119 Misc.2d
834, 464 N.Y.S.2d 6787, 682 + n 83 (Sup. Ct. 1983), rev’d on
other grounds, 108 A.D.2d 56, 487 N.Y.S.2d 595 (1985) (disagreeing
in dicta with the trial court’s holding of ambiguity), aff’d,
67 N.Y.2d 688, 490 N.E.2d 839, 499 N.Y.S.2d 920 (1986) (declining
to address the ambiguity issue).
[30]See Dozsa v. Crum & Forster Insurance Co., 716 F.Supp. 131,
139 (D.N.J. 1989) (cancer drug HDCT-ABMT held not experimental).
[31]See Zuckerbergsupra note 30.
[32] I.V. Services of America, Inc. v. Trustees of the American
Consulting Engineers Council Ins. Trust Fund, 136 F.3d 114, 119
(2nd Cir. 1998).
[33] Id.
[34] Id.
[35] See Pirozzi v. Blue Cross-Blue Shield, 741 F. Supp. 586, 591
(E.D.Va. 1990).
[36] Id.
[37] Id.
[38] See Hoffman, supra note 25.
[39] Id., citing Harris v. Mutual of Omaha Cos., 992 F.2d 706 (7th
Cir. 1993); Holder v. Prudential Ins. Co., 951 F.2d 89, 90 n.3 (5th
Cir. 1992) (the policy excluded coverage of therapies that were
not “commonly and customarily recognized throughout the doctor’s
profession as appropriate in the treatment of the diagnosed sickness”
or that were “educational or experimental in nature”).
[40]Id., citing Henderson v. Bodine Aluminum, Inc., 70 F.3d 958,
961 (8th Cir. 1995) (The health insurance plan in question covered
high dose chemotherapy (HDCT) only for particular cancers, not including
breast cancer, since it considered the treatment experimental for
all but the specified cancers. The Eighth Circuit reversed and remanded
the lower court's denial of plaintiff's motion for a preliminary
injunction, finding that she showed a likelihood of success on the
merits in proving that HDCT is an "accepted" therapy for
breast cancer).
[41] Id., citing Schnitker v. Blue Cross/Blue Shield, 787 F. Supp.
903, 904 (D.C. Neb. 1991) (plan excluded from coverage any treatment
that had not received final approval from the appropriate governmental
entity or had been accepted by a consensus of opinion based on scientific
evidence).
[42] , citing Leonhardt v. Holden Bus. Forms Co., 828 F. Supp. 657,
663 (D. Minn. 1993) (plan defined as "experimental" any
treatment that is: (a) not proven in an objective manner to have
therapeutic value or benefit; (b) restricted to use at medical facilities
capable of carrying out scientific studies; or (c) of questionable
medical effectiveness); Dozsa v. Crum & Forster Ins. Co., 716
F. Supp. 131, 134 (D.N.J. 1989) (plan excluded coverage for treatments
that were not "commonly and customarily recognized throughout
the doctor's profession as appropriate in the treatment of the sickness
or injury" or that were "educational" or "experimental
in nature" or were "provided primarily for research purposes").
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