November 1, 2018
On Monday, October 12th, FDA issued a draft guidance regarding the role of Pre-IND Meetings in the development of drugs to treat rare diseases. The document, entitled "Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings," touches on four main issues to consider in the early phases of development and Pre-IND Meetings. These include:
During the Pre-IND Meeting for a rare disease drug development program, it is important that sponsors clearly summarize the type and amount of CMC information that will be included in their IND. Sponsors must also justify the appropriateness of the information in supporting the clinical trials being proposed. In addition to standard meeting background material, FDA recommends that sponsors "include additional detailed information to enable meaningful discussion of the specific questions posed to FDA." The draft guidance provides a list of various topics that may be appropriate based on the questions of interest. Some of these include a description of the:
Nonclinical studies are used to determine whether it would be reasonably safe for the investigational drug to be tested in humans. As such, they must be completed before clinical trials can be started. The type(s) of studies required varies based on the investigational drug’s intended use, the proposed clinical trial population, and the proposed treatment regimen.
"In addition to providing the standard meeting package elements, sponsors should include the following information to support specific nonclinical questions posed to the FDA review division/office:
The draft guidance notes that "FDA may exercise flexibility in the types and amount of nonclinical data to accept to support drug development for serious and life-threatening diseases." Furthermore, during the Pre-IND Meeting, sponsors can ask the FDA about any additional nonclinical studies that may be necessary to support clinical trials as well as the timing of those studies during (as applicable).
Clinical pharmacology studies serve as the link between nonclinical studies and the drug’s targeted population. These studies provide sponsors with critical information regarding a drug’s mechanism of action, pharmacokinetic and pharmacodynamic properties, potential for clinical benefit, safety profile, and dose- or exposure-response relationship.
Information from these studies can inform clinical trial design and provide supportive evidence of effectiveness. Sponsors should carefully plan and prepare for the clinical pharmacology aspects when developing a drug to treat a rare disease.
FDA recommends that sponsors "include the following information to address specific clinical pharmacology questions:"
Developing drugs for rare diseases comes with many potential challenges, one of which is the small number of disease-affected individuals. Because of this limitation, it is critical for sponsors the maximize each patient’s contribution during clinical development.
"Although FDA has no specified minimum number of patients needed to establish drug safety and efficacy, the number of patients should be adequate to assess benefit and risk." While the Agency’s approval standards are the same for drugs treating rare and nonrare diseases, the broadest possible scientific judgement is used when applying the evidentiary standard in the case of rare diseases. As such, the Agency will consider the following:
During the Pre-IND Meeting, sponsors should be prepared to discuss the following topics with the FDA:
On top of the four main areas discussed above, FDA’s draft guidance provides sponsors with a number of additional considerations. These considerations touch on a variety of topics, including:
Details on the Agency’s additional considerations are available in FDA’s full draft guidance. This draft guidance for comment purposes only and the Agency is asking interested individuals to submit written or electronic comments by December 17, 2018.
Are you developing a drug or biological product to treat a rare disease? Whether your product is indicated for a rare or nonrare disease, we can help you achieve successful interactions with the FDA, starting with the Pre-IND Meeting. To learn more about our services and how we can help get your product approved by the FDA, contact us today.
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