Access to Investigational Therapies: Regulations and Criteria
This content discusses the access to investigational drugs outside of clinical trials, highlighting Treatment Use Criteria and Emergency Use situations. It covers Subpart E regulations, including the definition of life-threatening and severely-debilitating illnesses. The importance of safeguards like informed consent, IRB, and IND safety reports are also emphasized.
- Investigational Therapies
- Treatment Use Criteria
- Emergency Use
- FDA Regulations
- Life-Threatening Illnesses
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Presentation Transcript
Speeding access to therapies Suzanne M. Sensabaugh, MS, MBA smsensabaugh@hw-fda.com www.hw-fda.com
Suzanne Sensabaugh, MS, MBA Former FDA: Reviewer of hundreds of industry submissions. Committee member who drafted FDA Guidelines: Received numerous awards. Former industry: Drafted submissions for FDA review. Since 2009, assisted in the development of over 120 drugs. www.hw-fda.com 2
Introduction Access to an investigational drug outside of clinical trials Treatment Use Emergency Use Speeding review and approval Subpart E Fast Track Accelerated Approval Priority Review Not mutually exclusive www.hw-fda.com 3
Treatment Use Criteria The drug is intended to treat a serious or immediately life- threatening disease There is no comparable or satisfactory alternative The drug is under investigation in a controlled clinical trial under an IND or all clinical trials have been completed The sponsor is actively pursuing marketing approval of the investigational drug with due diligence A drug is made available during Phase III In some circumstances, a drug may be made available for treatment use during Phase II Safeguards Informed consent, IRB, and IND safety reports www.hw-fda.com 4
Emergency Use Use of an investigational drug in a life-threatening situation where no standard acceptable treatment is available and in which there is not sufficient time to obtain IRB approval Allows FDA to authorize use of an investigational drug in an emergency situation that does not allow time for submission of an IND Also used for patients who do not meet the criteria of an existing study protocol or if an approved study protocol does not exist www.hw-fda.com 5
Subpart E regulations 21 CFR 312, Subpart E (1988) Life-threatening and severely-debilitating illnesses, especially where no satisfactory therapy exists Life-threatening: diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted and diseases or conditions with potentially fatal outcomes, where the endpoint of clinical trial analysis is survival Severely-debilitating: diseases or conditions that cause major irreversible morbidity Procedures reflect the recognition that physicians and patients are generally willing to accept greater risks or side effects from products that treat life- threatening and severely-debilitating illnesses than they would accept from products that treat less serious illnesses In addition, benefits of the drug need to be evaluated in light of the severity of the disease being treated Meet early on with the Agency to review and reach agreement on the design of necessary preclinical and clinical studies www.hw-fda.com 6
Accelerated Approval 21 CFR 314, Subpart H, and 21 CFR 601, Subpart E (1992) Drugs to treat serious or life-threatening illnesses and provide meaningful therapeutic benefit over existing treatments Approval based on a surrogate endpoint or an effect on a clinical endpoint other than survival or irreversible morbidity Surrogate endpoint: an endpoint intended to relate to a clinically meaningful outcome, but does not itself measure a clinical benefit an indirect or substitute measurement that represents a clinically meaningful outcome Can considerably shorten the time for FDA approval Tumor shrinkage in cancer patients reasonably likely to predict clinical benefit Studies must be adequate and well-controlled Post-marketing confirmatory studies must be conducted to verify the anticipated clinical benefit Must be adequate and well-controlled Usually underway when approval is granted If studies do not demonstrate clinical benefit, product removed from market www.hw-fda.com 7
Priority Review Prescription Drug User Fee Act, 1992 Performance goals for review of BLAs/NDAs Standard: drugs which offer only minor improvement over existing marketed products 10 month time frame Priority: major advances in treatment or provide a treatment where no adequate therapy exists 6 months time frame Receive an action letter at the end of this review time frame Major advances may include Increased effectiveness in treatment, prevention, or diagnosis of disease Elimination or substantial reduction of a treatment-limiting drug reaction Enhancement of patient willingness or ability to take the drug according to the required schedule and dose Safety and effectiveness in a new subpopulation, such as children www.hw-fda.com 8
Fast Track FDA Modernization Act, 1997 Process designed to facilitate the development and expedite the review of drugs to treat serious diseases and fill an unmet medical need Serious: matter of judgement that includes such factors as survival, day- to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious disease AIDs, Alzheimer s disease, cancer Unmet medical need: providing a therapy where non exists or providing a therapy which may be potentially superior to existing therapy Advantages More frequent meetings and written correspondence with/from the Agency Rolling review: company can submit completed sections of the NDA or BLA rather than wait until the entire application is complete www.hw-fda.com 9
Any questions? smsensabaugh@hw-fda.com www.hw-fda.com 10
