FDA BRIEF: Week of October 2, 2017
National CyberSecurity Awareness Month
- more vulnerable to cybersecurity threats
- could potentially impact patient safety
- Shared responsibility including: medical device manufacturers, government agencies, health care organizations, health care professionals, cybersecurity researchers, and medical device users
- FDA works with several public and private organizations to raise awareness of medical device safety and cybersecurity
Expanded Access: FDA Describes Efforts to Ease Application Process
Expanded access (Compassionate Use) programs that provide investigational drugs and devices to patients with serious conditions
- FDA authorizes 99% of applications; simplified via Form FDA 3926
- Time is critical for seriously ill patients who do not have alternative therapies
Lifting another potential burden for applications
- Simplification of IRB process
- Just one IRB member can now approve the treatment
Addressing uncertainty about assessment of adverse event data from expanded access
- Recognize patients outside of controlled clinical trial setting; with more advanced disease, be receiving other drugs and have other diseases
- Guidance clarifies that suspected adverse reactions must be reported “only if there is causal relationship”
Other activities
- Addressing issues raised by Government Accountability Office (GAO) on adverse event data
- New online tool called the Expanded Access Navigator
- Working with the Reagan-Udall Foundation to include FDA’s Rare Disease Program
FDA Rare Disease Grants: For clinical trials, For Natural History Studies
CLINICAL TRIAL GRANTS
Awarded 15 new clinical trial research grants totaling more than $22 million
- Through the Orphan Products Clinical Trials Grants Program ( creation in 1983) funded by Congressional appropriations
- To boost development of products for patients with rare diseases
- Awarded to principal investigators from academia and industry
- A total of 76 grant applications received; funding rate of 20%
Grants awarded for
- Rare forms of cancer: glioblastoma, anaplastic astrocytoma, pediatric neuroblastoma
- Hyperphagia in Prader-Willi syndrome, idiopathic osteoporosis in premenopausal women, sickle cell disease, pulmonary tuberculosis (TB), including multidrug-resistant TB
NATURAL HISTORY STUDIES
Awarded 6 grants for natural history studies in rare diseases totaling $6.3 million
- First time to fund through Orphan Products Grants Program for natural history studies
- Inform medical product development by better understanding diseases progression
- More than 80 grant applications reviewed by more than 60 rare disease and natural history experts
Grants awarded for
- Friedreich’s ataxia, pregnancy and lactation-associated osteoporosis, sarcoidosis, chronic kidney disease, Angelman syndrome, Myotonic Dystrophy Type 1
Good News for Public Health: Most Mammography Facilities are in Full Compliance with MQSA Regulations
Mammography Quality Standards Act (MQSA) established in 1992
- Facilities must meet specific standards and abide by FDA regulations
- Services must be accredited by FDA- approved accrediting body
- Facility must hold an active MQSA certificate
> 87% of mammography facilities have no MQSA violations
- 8,700 MQSA certified mammography facilities, perform > 39 million mammograms/year, using ~ 18,000 mammography units
- MQSA inspection includes performance, quality assurance records, quality control testing, personnel qualifications, medical records, and medical audit and outcome analysis records
- Certified MQSA inspectors have performed > 175,000 MQSA inspections
Priority Review Vouchers
Priority Review Vouchers (PRVs) can be used to obtain priority review designation for a subsequent application that does not itself qualify for priority review
- May be transferred (including by sale) the entitlement; no limit on the number of transfers
- Application for drug/biological product must contain no previously approved active ingredient (including salt/ester)
Tropical Disease PRV: Application for the prevention or treatment of a “tropical disease”
Rare Pediatric Disease PRV: Application for “rare pediatric disease” that is serious or life-threatening, affecting individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents
Medical Countermeasure PRV: Application intended to prevent or treat harm from a biological, chemical, radiological, or nuclear agent
Opioid Policy Steering Committee
Opioid Policy Steering Committee (OPSC) proposed
- To address opioid addiction and the resulting overdoses and deaths
- FDA seeking public input on how use of its authorities to address crisis
Seeking public input on 3 key areas
- What more can FDA do to ensure that the full range of available
information, including about possible public health effects, is considered
when making opioid-related regulatory decisions - What steps can FDA take with respect to dispensing and packaging
to facilitate consistency of and promote appropriate prescribing practice - Should FDA require some form of mandatory education for health care professionals who prescribe opioid drug products, and if so, how should such a system be implemented
Reducing the Hurdles for Complex Generic Drug Development
Drug Competition Action Plan to advance new policies aimed at bringing more competition to the drug market
- Improve the efficiency of the generic drug approval process
- Closing loopholes that allow branded drug companies to game FDA rules to forestall generic competition
- Facilitate generic competition to complex drugs
Complex drugs
- Such as metered dose inhalers used to treat asthma, injectable drugs
- Have feature(s) difficult to “genericize”
- Have lost exclusivity, but have no generic competition
- Can be a high-value opportunity for a generic drug maker
Provide scientific and regulatory clarity with respect to complex generic drugs
- Adopt more rigorous and sophisticated science, including sophisticated quantitative methods and computational modeling, in drug development, evaluation, and review
- Draft guidance on pre-ANDA meeting requests to discuss regulatory strategy
- Draft guidance on ANDAs for peptides manufactured using recombinant DNA technology such as namely, glucagon, liraglutide, nesiritide, teriparatide, teduglutide
Other Initiatives
- Address challenges with bioequivalence testing
- Identify gaps in the science and develop more tools, methods, and efficient alternatives to clinical endpoint testing
- Workshops on quantitative modeling approaches, new analytical tools for demonstrating active ingredient sameness in complex products
Image credits: FDA