Department of Health and Human Services Food and Drug AdministrationFDA ORPHAN DRUG DESIGNATION REQUEST FORMForm Approved OMB Control Number: 09100167 Expiration Date: January 31, 2021 See OMB Statement.
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How to fill out the FDA Form 4035 online
Filling out the FDA Form 4035 online can seem complex, but with clear guidance, you can navigate each section confidently. This form is essential for requesting orphan drug designation, which recognizes drugs and biologics intended to treat rare diseases.
Follow the steps to complete the FDA Form 4035 online.
- Click ‘Get Form’ button to obtain the form and open it in the editor.
- Enter the date of the request in the format mm/dd/yyyy. If this is an amended request, include the designation request number.
- Fill in the sponsor contact information. Required fields include business country, primary contact country, business name, primary contact business name, and primary contact email address.
- If applicable, provide details of the sponsor U.S. resident agent, including name, address, contact numbers, and email.
- Complete the product information section by indicating if the product is a biologic, combination product, or drug. Fill in the UNII, chemical name, generic/descriptive name, code name, and trade name.
- Specify whether the sponsor is the manufacturer and provide the manufacturer’s name and address when applicable.
- Identify the orphan disease or condition requesting designation and define whether the request is for treatment, prevention, or diagnosis.
- Describe the disease or condition and the proposed use of the drug, including the need for therapy. Use the attachment feature for relevant tables or charts.
- Provide the scientific rationale supporting the use of the drug for the specified disease. Include details about clinical and animal data, if available.
- Indicate if the same drug has been previously approved for the same use. If applicable, provide a hypothesis for clinical superiority.
- Determine if the request is for an orphan subset of a common disease and provide the necessary explanation.
- Document the regulatory status and marketing history of the drug, including any orphan drug designations held previously.
- Complete the population estimate section, ensuring it is less than 200,000 persons for orphan drug designation eligibility. Attach any relevant documentation.
- Once all fields are filled out and necessary attachments are included, save your changes. You may download, print, or share the completed form.
Begin your online submission of the FDA Form 4035 today.
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May 19, 2021 — The content and format of a request for orphan drug designation is...
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What are the benefits of orphan drug status?
7-year marketing exclusivity to sponsors of approved orphan products. 25% federal tax credit for expenses incurred in conducting clinical research within the United States. ... Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs.
What does Orphan Drug mean?
Listen to pronunciation. (OR-fun ) A drug used to treat, prevent, or diagnose an orphan disease. An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States.
When do you submit orphan drug designation?
The orphan designation is part of the approval process In fact, the orphan application can be filed anytime in the drug development process before NDA/BLA submission, even prior to IND filing.
What is the criteria for a drug to receive orphan drug designation?
Criteria for Orphan Drug Designation The product must be intended for use in a rare disease or condition. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.
What is an example of an orphan drug?
An orphan drug can be defined as one that is used to treat an orphan disease. For example, haem arginate, used to treat acute intermittent porphyria, variegate porphyria, and hereditary coproporphyria [12], is an orphan drug.
How do you get an orphan drug exclusivity?
Orphan Drugs receive a 7-year period of exclusivity from product approval - effective on the date of FDA approval of a marketing application. For seven years, FDA will not approve a subsequent sponsor of the same drug for the same use or indication (except as otherwise provided).
What is an FDA orphan drug?
According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US" (which equates to approximately 6 cases per 10,000 population) "or meets cost ...
How do I submit an orphan drug designation request?
Mail the CD along with a signed cover letter as described at Corresponding with the OOPD Drug Designation Programs. Alternatively, during this public health emergency associated with the COVID-19 pandemic, orphan designation requests may be submitted by email to orphan@fda.hhs.gov.
When should I request orphan drug designation?
The orphan designation is part of the approval process In fact, the orphan application can be filed anytime in the drug development process before NDA/BLA submission, even prior to IND filing.
What is required for orphan drug designation?
Criteria for Orphan Drug Designation The product must be intended for use in a rare disease or condition. ... There must be a scientific rationale establishing a medically plausible basis for the use of the product for the rare condition.
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