FDA News and Views: Cybersecurity Month, Expanded Access, Rare Disease Grants, Mammography Compliance, Priority Review Vouchers, Opioid Policy Steering Committee, Complex Generic Drug Development – Drug and Device Digest

FDA BRIEF: Week of October 2, 2017

National CyberSecurity Awareness Month

Medical devices increasingly interconnected through wired and wireless connections
  • more vulnerable to cybersecurity threats
  • could potentially impact patient safety
FDA works with industry to identify cybersecurity issues to consider in design and development of medical device
  • 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


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



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

Photos of Mammography devices

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

Laptop, gavel and stethoscope, representing medical product-related legislation

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


Image of a hand reaching for a pill bottle in a medicine cabinet

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

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