1. Speeding access to therapies
Suzanne M. Sensabaugh, MS, MBA

2. Panacea Pharmaceuticals, Inc.
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. 2

3. Panacea Pharmaceuticals, Inc.
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 3

4. Panacea Pharmaceuticals, Inc.
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 4

5. Panacea Pharmaceuticals, Inc.
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 5

6. Panacea Pharmaceuticals, Inc.
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 6

7. Panacea Pharmaceuticals, Inc.
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 7

8. Panacea Pharmaceuticals, Inc.
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 8

9. Panacea Pharmaceuticals, Inc.
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 9

10. Panacea Pharmaceuticals, Inc.
Any questions? 10