Subject:
Orphan Drugs
Description:
_______________________________________________________________________________________
IMPORTANT NOTE:
The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.
Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.
__________________________________________________________________________________________________________________________
Orphan drugs are medications or biologics intended for use in rare diseases. The Food and Drug Administration (FDA) defines a rare disease as a disease that affects:
- < 200,000 people in the United States; OR
- > 200,000 people in United States for which there is no reasonable expectation that the cost of developing and providing availability in the United States will be recovered from sales in United States.
Drug sponsors may submit a request for orphan drug designation at any time during the filing of a New Drug Application (NDA) and/or an Investigational New Drug (IND) application. The orphan drug designation process will allow the sponsors to qualify for incentives such as tax credits and marketing exclusivity. The following are some of the requirements for the “designation” process:
- Description of the rare disease for which the drug is being or will be investigated, the proposed indications for use of the drug, and the reasons as to why such therapy is needed;
- Data from non-clinical laboratory studies, clinical investigations, and other relevant information that are available (whether positive, negative, or inconclusive) to support the scientific rationale for the use of the drug for the rare disease and description of the medication;
- Statement that acknowledges that the manufacturer submitting the request is the real party in interest for the development and the intended or actual production and sales of the product.
Orphan grant programs are available for the development and funding of clinical trials for drugs to be studied in rare diseases, which could lead to FDA approval for marketing in the United States. The requirements for NDA/IND include the following phases of clinical trials:
- Phase 1 studies include limited number of healthy volunteer subjects. These studies are designed to determine the metabolic and pharmacological actions of the drug in humans, the adverse events associated with increasing doses, and, if possible, to gain early evidence on effectiveness.
- Phase 2 studies are controlled clinical trials to obtain some preliminary data on the effectiveness of the drug for a particular indication or indications in several hundreds of patients with the disease. This phase of testing helps to determine the common short-term adverse events and risks associated with the drug.
- Phase 3 studies are intended to gather the additional information about effectiveness and safety that is needed to evaluate the overall benefit-risk relationship of the drug in several hundred to several thousand of people. Phase 3 studies also provide an adequate basis for extrapolating the results to the general population and transmitting that information in the package insert.
The FDA requires one to two phase 3 clinical trials for an orphan drug to be reviewed and approved for marketing. Usually, orphan drugs receive expedited FDA review and approval secondary to the life-threatening diseases that they are typically indicated to treat. Once the FDA approves an orphan drug for marketing to a sponsor, the FDA will also grant the manufacturer seven years of market exclusivity of the orphan drug from the date of approval.
Hence, orphan drug designation is NOT the same as orphan drug approval for marketing.
Policy:
(NOTE: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance)
1. Drugs that are designated as orphan drugs by the FDA but have not received FDA approval for marketing in the United States are considered investigational and thus, ineligible for coverage.
2. Drugs that are designated as orphan drugs and have received FDA approval for marketing in the United States for a specified disease entity are eligible for coverage based on the FDA approved package insert.
3. Drugs that are designated as orphan drugs and FDA approved for specific indications but are being used for unapproved indications will be subject to the “Off-Label Use of Prescription Drugs” policy (DRUGS# 008).
Medicare Coverage
There is no National Coverage Determination (NCD). In the absence of an NCD, coverage decisions are left to the discretion of Local Medicare Carriers. Novitas Solutions, Inc, the Local Medicare Carrier for jurisdiction JL, has not issued a determination for this service. Therefore, Medicare Advantage Products will follow the Horizon Policy.
Medicaid Coverage
For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf
________________________________________________________________________________________
Horizon BCBSNJ Medical Policy Development Process:
This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.
___________________________________________________________________________________________________________________________
Index:
Orphan Drugs
Orphan Drug Regulations
References:
1. FDA: U.S. Food and Drug Administration: Orphan Drug Regulations. Available at: https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-regulations-regulatory-history. [accessed 12/23/2019].
2. FDA: U.S. Food and Drug Administration: Orphan Drug Regulations. Available at: https://www.govinfo.gov/content/pkg/FR-2013-06-12/pdf/2013-13930.pdf. [accessed 12/23/2019].
3. CDER (Centers for Drug Evaluation and Research): Frequently Asked Questions on Drug Development and Investigational New Drug Applications Available at: www.fda.gov/cder/about/smallbiz/faq.htm. [accessed 12/23/2019].
4. FDA: U.S. Food and Drug Administration: The Orphan Drug Act (as amended): Congressional Findings for the Orphan Drug Act. Available at: www.fda.gov/orphan/oda.htm. [accessed 12/23/2019].
5. FDA: U.S. Food and Drug Administration: The Orphan Drug Designation. Available at: www.fda.gov/orphan/designat/. [accessed 12/23/2019].
6. FDA: U.S. Food and Drug Administration: Frequently Asked Questions Concerning the Orphan Product Designation. Available at: www.fda.gov/orphan/designat/faq.htm. [accessed 12/23/2019].
7. FDA: U.S. Food and Drug Administration: Orphan Product Designation. Available at: www.fda.gov/orphan/designat/index.htm. [accessed 12/23/2019].
8. FDA: U.S. Food and Drug Administration: List of Orphan Designations and Approvals. Available at: www.fda.gov/orphan/designat/list.htm. [accessed 12/23/2019].
9. FDA: U.S. Food and Drug Administration: Developing Products for Rare Diseases & Conditions. Available at: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm. [accessed 12/23/2019]
Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)
CPT*
HCPCS
* CPT only copyright 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.
_________________________________________________________________________________________
Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.
The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy
____________________________________________________________________________________________________________________________ |