1. What is a Humanitarian Use Device (HUD)? a. Definition. The FDA defines a HUD as a device that is intended to benefit patients in the treatment and diagnosis of diseases or conditions that affect or is manifested in fewer than 4,000 individuals in the United States per year. An investigator, or a sponsor, may submit a request for HUD designation to the FDA, and must include the following information:
b. Request for HUD designation. An investigator, or a sponsor, may submit a request for HUD designation to the FDA, and must include the following information: i. A statement that the applicant is requesting HUD designation for a rare disease or condition,
ii. The name and address of the applicant,
iii. A description of the rare disease or condition for which the device is to be used,
iv. A description of the device, and
v. Documentation, with authoritative references appended, to demonstrate that the device is designed to treat or diagnose a disease or condition that affects or is manifested in fewer than 4,000 people in the U.S. per year.
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2. What is a Humanitarian Device Exemption (HDE)? After a request for a HUD designation is submitted to the FDA, the FDA will determine if it should grant an HDE for use of the device. As part of its review, FDA will take into consideration the following issues: a. Whether there is a comparable device on the market. There must be no comparable device on the market before an HDE may be granted. b. FDA will determine from the information submitted that the Quality Systems Regulation (formerly Current Good Manufacturing Regulation) will be followed, or FDA may waive this requirement. If the FDA grants an HDE, a sponsor/investigator is authorized to market the HUD, but only under the limited indications approved by FDA and only if the use has been reviewed and approved by an IRB. The holder of the HDE is responsible for ensuring that the HUD is used only in facilities with a constituted JHM IRB. The JHM IRB may approve use of the HUD under a protocol or on a case-by-case basis.
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3. Is JHM IRB’s review required for use of HUDs? If at all possible, an investigator should apply for and receive JHM IRB review and approval before a HUD may be used at Johns Hopkins. In an emergency situation, if a physician determines that JHM IRB approval cannot be obtained in time to prevent serious harm or death to a patient, the physician should try to obtain the concurrence of one of the JHM IRB’s Chairs. In emergency situations, a physician may act and administer the HUD without the prior approval of the JHM IRB or the concurrence of an IRB Chair. However, in such situations, the physician must provide, within five (5) days after the use of the device, written notification to the Chairman of the JHM IRB of the use. This written notification must include:
- The identification of the patient involved;
- The date on which the device was used; and
- The reason for the use of the HUD.
The JHM IRB will review this notification to determine if the HUD use met with FDA regulations. The physician will be informed of the outcome of this review.
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4.Is consent required for use of a HUD at Johns Hopkins? It is the opinion of counsel for JHHS and JHU that consent must be obtained from patients who are to receive a HUD at Johns Hopkins. A special HUD consent form template has been provided by the JHM IRB, and is on the JHM IRB website at: http://irb.jhmi.edu/Forms/HUDTemplate.rtf. Each protocol’s consent form must be approved by JHM IRB. The consent form should specifically explain the HUD, discuss the risks and benefits of the device, and state that effectiveness of the device for the labeled indication has not been demonstrated. For assistance with the consent form you may contact a Consent Form Specialist at the JHM IRB office at 410-955-3008.
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5. What about "Compassionate Use" of a HUD? a. The JHM IRB may approve compassionate use of the HUD, off label and in an emergency situation for one patient. Before the device is used, the following criteria must be satisfied: i. authorization from the holder of the HDE (either a sponsor or a physician);
ii.the JHM IRB chair’s concurrence, if possible
iii. informed consent from the patient or the patient’s Legally Authorized Representative (LAR) (Note: the consent form must be approved by the JHM IRB; a standard Johns Hopkins Hospital consent form is not acceptable); and
iv. an independent assessment of the use of the HUD by an uninvolved physician.
b. The JHM IRB may also approve compassionate use of a HUD if the physician determines that there is no emergency, but there is no alternative device for the patient’s condition. The physician who wishes to use the HUD should provide the HDE holder with the following: i. a description of the patient’s condition and the circumstances necessitating treatment with the device
ii. a discussion of why alternative therapies are unsatisfactory, and iii. information to address patient protection measures.
The HDE holder then should submit an HDE amendment for FDA approval prior to the use. The FDA will review the information expeditiously and issue its decision to the HDE holder. Once approval is granted, the physician using the HUD should monitor the patient, taking into consideration the limited information available regarding the potential risks and benefits of using the HUD for the specific patient.
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6. What must the treating physician do after compassionate use occurs? After the compassionate use occurs, the treating physician is responsible for notifying the JHM IRB as set forth in Section 3, above, and for ensuring one of the two follow-up procedures noted below occurs: a. The treating physician should contact the sponsor/manufacturer of the device to find out if an Investigational Device Exemption (IDE) exists for the device. If an IDE does exist, the treating physician should provide the holder of the IDE with sufficient patient follow-up information to allow the sponsor to comply with the FDA reporting requirements of the IDE regulations. The treating physician should provide to the JHM IRB a copy of the information sent to the sponsor. b. If an IDE does not exist, the treating physician should submit a follow-up report on the use of the device to the JHM IRB and to the FDA IDE staff. This report should contain a summary of the conditions constituting the emergency or other rationale for use, patient protection measures that were followed, and patient outcome information.
Post-approval procedures for compassionate use cases are set out in www.fda.gov/cdrh/ode/idepolcy.html. For more information on emergency use of investigational devices, see 50 FR 42866 and 21 CFR 812.35(a).
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7. What is the Cost of the HUD? The manufacturer cannot charge more than the cost of research, development, fabrication, and distribution of the device. If the amount charged is $250 or more, the HDE applicant must submit a report by an independent certified public accountant, or in lieu of such a report, an attestation by a responsible individual of the organization, verifying that the amount does not exceed the cost of research, development, fabrication, and distribution. If the amount charged is under $250, the FDA waives this requirement.
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8. What must be done to bill for use of a HUD? Please check with the sponsor who holds the HDE from the FDA for procedures to be followed to gain authorization to bill on an insurance provider for therapy involving a HUD. Prior authorization from a patient’s insurer must be sought and the consent form must explain to the patient that authorization was sought.
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9. How might a device lose HUD Status? The FDA may withdraw the HDE if:
- The FDA determines that the disease or condition affects more than 4,000 people in the U.S. per year.
- The device gets approved through the PMA process or cleared through the 510(k) process.
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10. How can I learn more about HUDs and HDEs? The federal government has issued guidance in this area. You can find this information at the following site: www.fda.gov/cdrh/ode/guidance/1381.pdf.
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