Tag: drug approval

Bioethics News

Drugs that Go Through FDA’s Expedited Review Might Be Better Drugs

August 14, 2017

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A new paper in the latest issue of Health Affairs is worth a look. “Drugs Cleared Through The FDA’s Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process“:

We investigated whether drugs approved by the Food and Drug Administration (FDA) through expedited review have offered larger health gains, compared to drugs approved through conventional review processes. We identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) associated with drugs approved in the period 1999–2012 through expedited (seventy-six drugs) versus conventional (fifty-nine) review processes.

The FDA has a strict process for drug approval, requiring a number of phases of testing to establish safety and efficacy. It can, however, expedite this process when it thinks a drug may address an unmet clinical need. Of course, you want such a process to be fair and work. You want it to be focused on “better” and “more important” drugs. The 21st Century Cures Act moved even further. Some worry that loosening standards will lead to increased exposure to harm, especially if there isn’t proof that these drugs provide more benefits.

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The Incidental Economist

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The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Erika Lietzan on ‘The Week in Health Law’ Podcast

By Nicolas Terry and Frank Pasquale Subscribe to TWIHL here! This week’s TWIHL is a deep dive into pharmaceutical patent protection and its intersection with the FDA new drug approval process. We touch on molecular drugs, biosimilars, data exclusivity, market exclusivity, the runway to generics, and fascinating differentials … Continue reading

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Web Roundup: Water by Lily Shapiro

Before I began graduate school, I worked in water-related public health, and have continued to follow the news around water. This month, some stories (mostly) about water.

Trump signed an order last week to “expedite” the construction of the Dakota Access Pipeline, which jeopardizes the water source for the Standing Rock Sioux Reservation, and for many others who drink water from the Missouri River. Opponents of the pipeline are not surprised, and are gearing up for a fight. Although most of Silicon Valley has come out against Trump (though perhaps in somewhat tepid terms), particularly in light of his recent executive order on immigration, Peter Thiel has been tapped to help Trump pick someone to lead the FDA, with the goal of decreasing its regulations around drug approval process. Vox goes into detail about why that’s a bad idea. (And, before we move away from the topic of Trump’s horrifying first week in office, if you haven’t read CultAnth’s interview with prof of anthropology and lawyer Darryl Li on the travel ban, I recommend it).

California has recently gotten some much-needed rain (and snow) this month. Researchers and farmers there are experimenting with methods, which include flooding fields during the winter, to use this rain to recharge the depleted groundwater aquifers. Also, check out this time lapse of the California drought from 2011-present. The storms also toppled an iconic, if controversial, “drive-through” Sequoia tree, one of the last still standing in a public park.

Meanwhile, the state of Tamil Nadu, in South India, is experiencing its worst monsoon in nearly 150 years, a crisis worsened by the demonetization on November 9 (in which the Rupees 500 and 1000 notes were declared invalid), resulting in the deaths of numerous farmers and recent agitations by farmer’s groups.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

EpiPen Controversy Fuels Concerns Over Generic Drug Approval Backlog

September 6, 2016

(Kaiser Health News) – Consumers and Congress members pushing for cheaper alternatives to the EpiPen and other high-priced drugs are seeking answers about a stubborn backlog of generic drug applications at the Food and Drug Administration that still stretches almost four years. As of July 1, the FDA had 4,036 generic drug applications awaiting approval, and the median time it takes for the FDA to approve a generic is now 47 months, according to the Generic Pharmaceutical Association, or GPhA. The FDA has approved more generics the past few years, but a flood of new applications has steadily added to the demand.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Accelerated Drug Approval and Health Inequality

Since the 1960s, the U.S. FDA has served as a model for drug regulation around the world with its stringent standards for approval of new drugs. Increasingly, however, a coalition of libertarians, patient advocates, and certain commercial interests have been pressing for a relaxation of these stringent standards. Examples of legislative initiatives that would weaken regulatory standards of evidence for drug approval include the “Regrow Act,” “21st Century Cures Act,” as well as various “Right to Try” laws passed in U.S. states.

Much has been written in support- and against- relaxation of current regulatory standards. Typically, these debates are framed in terms of a conflict between public welfare (i.e. the public needs to be protected from unproven and potentially dangerous drugs) and individual choice (i.e. desperately ill patients are entitled to make their own personal decisions about risky new drugs).

In a recent commentary, my co-author Alex London and I take a different tack on this debate. Rather than framing this as “public welfare” vs. “individual choice,” we examine the subtle ways that relaxed standards for drug approval would redistribute the burdens of uncertainty in ways that raise questions of fairness. We suggest weakened standards would shift greater burdens of uncertainty a) from advantaged populations to ones that are already suffer greater burdens from medical uncertainty; b) from research systems toward healthcare systems; c) from private and commercial payers toward public payers; and d) from comprehending and voluntary patients towards less comprehending and less voluntary patients. We hope our analysis stimulates a more probing discussion of the way regulatory standards determine how medical uncertainty is distributed.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Research Ethics Roundup: Diversifying Clinical Trials, Women Researchers Don’t Receive Top Billing, and More

From the role of women in research to a poll that found the majority of Americans don’t approve of an expedited drug approval process in exchange for lower standards, this week’s Research Ethics Roundup examines some of the latest controversial issues occurring in research.

The post Research Ethics Roundup: Diversifying Clinical Trials, Women Researchers Don’t Receive Top Billing, and More appeared first on Ampersand.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

Zika Highlights Need for Research on Pregnant Women

By Anne Drapkin Lyerly, Carleigh Krubiner, Ruth Faden

 

This op-ed was originally published by the Baltimore Sun.

 

The rapid spread of the Zika virus — and its now clear association with microcephaly in babies exposed prenatally — has put extraordinary pressure on the research community to develop a vaccine as rapidly as possible. But accelerating the development of this vaccine is not only scientifically and logistically complicated, it is ethically complicated.

 

Pregnant women are at the crux of Zika’s most devastating consequences. Their needs must be uppermost in Zika prevention plans. While this will not be easy, the knee-jerk response that research with pregnant women is too complex to contemplate is not acceptable.

 

Current recommendations for women to delay or avoid pregnancy are unfair and unrealistic. In many areas hit hardest by Zika, women have limited access to contraception; there are, moreover, high rates of unplanned pregnancy worldwide. Preventing pregnancy may be the right course for some women, and preventing Zika in women before they get pregnant is critical. But these responses cannot be the whole answer. If we are serious about addressing the devastating impacts of Zika on normal brain development (Zika congenital syndrome), we must consider all approaches to preventing infection during pregnancy. This will require, at the very least, conscientious consideration of the role of pregnant women in the vaccine development agenda.

 

No doubt research with pregnant women is complicated. But ethical research with pregnant women is not impossible. Some might propose avoiding these complexities by testing vaccines in non-pregnant individuals, with a promise to collect safety data among pregnant women later.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

Gene Therapy Drug Approval Granted to GSK

May 31, 2016

(BBC) – Regulators have given one of the world’s largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. Commentators say the development marks the beginning of many more genetic medicines from so-called “Big Pharma”.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Methodological Miasma not mental dystrophy plagues drug trials

by Arthur Caplan, Ph.D. and Bruce Levin, Ph.D.

The Wall Street Journal and many other media outlets chose to beat on the FDA for its recent decision to deny approval of eteplirsen, a treatment for Duchenne muscular dystrophy being developed by Sarepta Therapeutics.

Actually it was the FDA’s scientific advisors not the FDA who concluded that there wasn’t sufficient evidence the drug was effective.

Still the WSJ sneered that “Here’s the gist of FDA’s objection: 12 patients are too few, and thus we don’t know if the drug helps boys walk longer or if the results are skewed. FDA prefers large trials that inject half of patients with a placebo. The agency has all but told patients to blame the lack of approval on Sarepta for its trial design.”

It is not just the FDA but most of the scientific community that prefers large placebo trials that produce stronger evidence. But both the WSJ, other FDA bashers and the FDA’s scientific advisors need to move the ball forward on trials. It is not placebo-controls but the importance of randomization in small trials that ought to be the issue for demonstrating safety and efficacy in any trial using small numbers.

In the comparison of eteplirsen to the matched control group there was no randomization.

Consider this fourfold table:

Still walking    Not still walking     Total

Treated                  10                      2                  12

Comparison             1                    10                  11

The P-value for testing the null hypothesis of no difference is 0.0006.  The likelihood ratio weight of evidence in favor of an efficacy hypothesis and against the null hypothesis is 71.6—that

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

What do doctors know about FDA drug approval standards and the breakthrough therapy designation? Less than we’d hope.

By Dalia Deak A study published this week in JAMA examined how much physicians know about FDA approval standards for new drugs and the breakthrough therapy designation. The investigators found major gaps in understanding with regard to both issues, despite … Continue reading

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.