Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:


MGH cancer specialists propose new approval track for targeted drugs


’Selective approval’ would require follow up studies to identify most appropriate patients

One of the most promising new strategies for cancer treatment is the development of drugs that directly target molecular abnormalities that lead to the growth of tumors. Several such drugs have received Food and Drug Administration (FDA) approval in recent years, but some have been controversial because the data on which they were approved did not clearly define which patients will most benefit from the new medications.

Writing in the July 29 New England Journal of Medicine, two specialists from the Massachusetts General Hospital (MGH) Cancer Center propose an expansion of the FDA’s "fast-track" programs for drug approval. In their "Sounding Board" piece, Thomas Roberts Jr., MD, and Bruce Chabner, MD, call on the FDA to require pharmaceutical companies to carry out studies identifying the patients most likely to respond to a targeted-therapy drug, as a condition of the drug’s approval.

"Throughout the 1980s members of the AIDS community were incredibly effective in influencing the FDA to allow easier access to antiretroviral drugs," says Roberts. "The resulting fast-track programs allow more rapid review and approval of drugs to treat many serious conditions that have no effective treatment."

While fast-track approval has opened up important therapeutic options for many patients, Roberts adds, without the detailed data provided by fully controlled clinical trials, physicians may not have information that clarifies who should or should not receive the medications. "With a new drug that costs $15,000, you don’t want to shoot in the dark and just hope this patient will be the one in ten who will benefit. And since choosing one of these drugs means you may have to exclude a different one, you don’t want to waste time with an agent that won’t help that patient."

He and Chabner propose institution of a mechanism they call "selective approval," which could be piloted for cancer drugs and eventually used for medications treating other conditions. Under selective approval, targeted drugs could receive fast-track approval based on consistent antitumor responses in early clinical trials, provided that the sponsoring pharmaceutical company has begun studies designed to identify the patients most likely to respond to the therapy. The approval would be reviewed annually and could be withdrawn if the clarifying research were not completed. On the other hand, completion of controlled clinical trials would convert the approval to full status.

"The advent of targeted cancer reatments has brought great enthusiasm for what some refer to as personalized medicine," says Roberts, "but there has also been disappointment that so few of these individualized approaches have become available to patients. One of the FDA’s fast-track programs, called accelerated approval, which looks at surrogates for clinical benefit [biomarkers] instead of waiting for definitive evidence of clinical benefit [e.g. improvement in survival time], is an important first step that we think should go further. The next step will require investing in the studies that will help us, as clinicians, better target therapies to the patients that will benefit the most."

Roberts is an instructor in Medicine at Harvard Medical School, and Chabner, clinical director of the MGH Cancer Center, is a professor of Medicine. Their report is supported by grants from the National Cancer Institute and the PhRMA Foundation.

Sue McGreevey | EurekAlert!
Further information:

More articles from Health and Medicine:

nachricht Inflammation Triggers Unsustainable Immune Response to Chronic Viral Infection
24.10.2016 | Universität Basel

nachricht Resolving the mystery of preeclampsia
21.10.2016 | Universitätsklinikum Magdeburg

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: New 3-D wiring technique brings scalable quantum computers closer to reality

Researchers from the Institute for Quantum Computing (IQC) at the University of Waterloo led the development of a new extensible wiring technique capable of controlling superconducting quantum bits, representing a significant step towards to the realization of a scalable quantum computer.

"The quantum socket is a wiring method that uses three-dimensional wires based on spring-loaded pins to address individual qubits," said Jeremy Béjanin, a PhD...

Im Focus: Scientists develop a semiconductor nanocomposite material that moves in response to light

In a paper in Scientific Reports, a research team at Worcester Polytechnic Institute describes a novel light-activated phenomenon that could become the basis for applications as diverse as microscopic robotic grippers and more efficient solar cells.

A research team at Worcester Polytechnic Institute (WPI) has developed a revolutionary, light-activated semiconductor nanocomposite material that can be used...

Im Focus: Diamonds aren't forever: Sandia, Harvard team create first quantum computer bridge

By forcefully embedding two silicon atoms in a diamond matrix, Sandia researchers have demonstrated for the first time on a single chip all the components needed to create a quantum bridge to link quantum computers together.

"People have already built small quantum computers," says Sandia researcher Ryan Camacho. "Maybe the first useful one won't be a single giant quantum computer...

Im Focus: New Products - Highlights of COMPAMED 2016

COMPAMED has become the leading international marketplace for suppliers of medical manufacturing. The trade fair, which takes place every November and is co-located to MEDICA in Dusseldorf, has been steadily growing over the past years and shows that medical technology remains a rapidly growing market.

In 2016, the joint pavilion by the IVAM Microtechnology Network, the Product Market “High-tech for Medical Devices”, will be located in Hall 8a again and will...

Im Focus: Ultra-thin ferroelectric material for next-generation electronics

'Ferroelectric' materials can switch between different states of electrical polarization in response to an external electric field. This flexibility means they show promise for many applications, for example in electronic devices and computer memory. Current ferroelectric materials are highly valued for their thermal and chemical stability and rapid electro-mechanical responses, but creating a material that is scalable down to the tiny sizes needed for technologies like silicon-based semiconductors (Si-based CMOS) has proven challenging.

Now, Hiroshi Funakubo and co-workers at the Tokyo Institute of Technology, in collaboration with researchers across Japan, have conducted experiments to...

All Focus news of the innovation-report >>>



Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

Agricultural Trade Developments and Potentials in Central Asia and the South Caucasus

14.10.2016 | Event News

World Health Summit – Day Three: A Call to Action

12.10.2016 | Event News

Latest News

New method increases energy density in lithium batteries

24.10.2016 | Power and Electrical Engineering

International team discovers novel Alzheimer's disease risk gene among Icelanders

24.10.2016 | Life Sciences

New bacteria groups, and stunning diversity, discovered underground

24.10.2016 | Life Sciences

More VideoLinks >>>