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Posts Tagged ‘21st Century Cures Initiative

Mental Health Policy and its Impact on the American Population

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By: Fatima Chowdhry, MD

           In the last 50 years, the U.S. has seen a migration in which individuals diagnosed with a mental illness, defined by the Diagnostic and Statistical Manual of Mental Disorders as “a syndrome characterized by clinically significant disturbance in an individual’s cognition, emotion regulation, or behavior”, are treated not in a mental health institution but rather in prisons, nursing homes, and outpatient facilities. To understand the implications of this trend, it’s important to frame this issue as a cascade of events. For example, we can start with a member of law enforcement, not adequately trained to recognize someone in the throes of a manic phase or a schizophrenic not on their medication, arresting an individual with a mental illness. We then find that this individual, upon release, did not receive treatment and now has trouble reintegrating into their community and is unable to find gainful employment. The combination of a lack of treatment, stable community, and employment leads them to continuous run-ins with the law, restarting a vicious cycle that had led us to a prison population in which the majority has a mental illness.

The move to deinstitutionalize people with mental illness from mental institutions began in the 1960’s and accelerated with the passage of the Community Mental Health Act of 1963. This bill was an important step forward to improve the delivery of mental health care because it provided grants to states to set up community health centers. In 1981, President Ronald Reagan signed the Omnibus Budget Reconciliation Act, which sent block grants to states in order for them to provide mental health services. Aside from these two bills, and the Mental Health Parity Act of 1996, which ensured insurance coverage parity of mental health care with other types of health care, there has been little in the way of significant mental health legislation. Mental health was put on the backburner and the result is a mental health infrastructure in tatters.

During the Great Recession, states cut billions in funding dedicated to mental health. A vivid example of how decreased state funding affects mental health services can be seen in the state of Iowa. The current Governor has been put in the difficult position of balancing fiscal responsibility with maintaining access to mental health care. At one point, there were four state mental health hospitals that provided care to each corner of the state. The Governor closed down two of the facilities to save the state money. While they were old facilities built in the 19th century and cost millions to maintain, many people in Iowa felt that he moved too quickly before alternative services were in place. In addition to closing these mental health facilities, the governor obtained a waiver from the federal government to modernize the state’s Medicaid program and move from fee-for-service to managed care. Under fee-for-service, health care providers are paid for each service provided to a Medicaid enrollee. Under managed care, Medicaid enrollees get their services through a vendor under contract with the state. Since the 1990s, the share of Medicaid enrollees covered by managed care has increased, with about 72% of Medicaid enrollees covered by managed care as of July 1, 2013. The move can be difficult because hospital networks and providers have to contract with a vendor and Medicaid beneficiaries may have to switch providers. Needless to say, it can be an administrative nightmare. The transition in Iowa, to say the least, has been rocky with the vendors threatening to pull out because of tens of millions of dollars in losses. The vendors and the providers might not get paid as much as they want but the people getting the short end of the stick are people on Medicaid, which includes individuals with mental health illnesses.

Given the patchwork of mental health care across the country and the lack of funding, what can be done? According to NAMI, 43.8 million Americans experience a mental illness in a year. Many don’t receive the treatment they need. It’s a multi-faceted problem facing families, employers, health care providers and community leaders. At the federal level, lawmakers have introduced several bills to address mental health. In the United States Senate, a bipartisan group of four Senators introduced S. 2680, the Mental Health Reform Act of 2016. This bill encouraged evidence-based programs for the treatment of mental illness, provided federal dollars to states to deliver mental health services for adults and children, and created programs to develop a mental health workforce.

It was encouraging to see that many components of S.2680 were included in H.R 34, the 21st Century Cures Act, which was signed into law on December 13th, 2016.  H.R 34 faces some headwinds because some of the funding portions are subject to Congressional appropriations, and if Congress is feeling austere, they can tighten the purse strings. Moving forward, a major issue of concern for mental health is the future of the Affordable Care Act. Under the Affordable Care Act, states were initially mandated to expand their Medicaid rolls. A Supreme Court decision, however, made the decision to expand optional. So far 32 states, including Washington D.C., have expanded. Some red states, like Iowa, Arkansas and Indiana have utilized the waiver process of the ACA to expand their program. If the ACA is repealed, policymakers will have to contend with the effects on the private insurance market as well as Medicaid.

Right now, the crystal ball is murky. Only time will tell.

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December 22, 2016 at 10:45 am

Science Policy Around the Web – December 13, 2016

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By: Allison Dennis, BS

Source: pixabay

Whistleblowers in Science

Keep your reviewers close and your online, anonymous, post-publication reviewers closer

A recent ruling by the Michigan Court of Appeals has ruled that anonymous online scientific reviews are a protected form of speech. Fazlul Sarkar, a former researcher at Wayne State University, had sued the site PubPeer in 2014 in an attempt to reveal the identity of several anonymous online reviewers to mixed success. Sarkar claimed that the defamatory and public nature of several online reviews posted anonymously to PubPeer had cost him a forthcoming tenure position at the University of Mississippi, one that came with a $350,000 a year salary. These reviews brought into question the validity of several images found in his published works.

While the initial ruling in March of 2015 largely sided with PubPeer to protect the anonymity of their online posters, a follow-up just two weeks later compelled PubPeer to reveal the IP address of a user who had gone as far as to repost quotes from an email response from the Senior Executive Assistant to the President of Wayne State University confirming their knowledge of the online allegations.

PubPeer filed an appeal of the decision by the end March, which garnished the collective support of science and internet moguls, Bruce Alberts, and Harold Varmus, Google, and Twittter in addition to the ACLU who filed amicus briefs in support of online anonymity. The summer brought more trouble for Sarkar as thirteen of his papers were retracted.

On December 9, 2016, the Michigan Court of Appeals found upon further review that Sarkar was “not entitled to unmask the identities of any speakers on pubpeer.com” citing “anonymity protections afforded by the First Ammendment.” Although this ruling does not dismiss Fazlul Sarkar’s case against John and Jane Doe, the protection of anonymity makes the suit moot. (Adam Marcus and Ivan Oranksy, STAT)

Federal Funding

Bipartisan cure found for stalled 21st Century Cures Initiative

In an end of the year push, the House and Senate passed the 21st Century Cures Initiative, a bill aimed at bringing legislation and regulation up to speed with biomedical research. At the end of November, a draft of the bill emerged from negotiations that were largely palatable to both Republican and Democrats across the House and Senate. A previous draft of the bill had successfully passed the House in July. However agreement over the source of funding could not be reached, arresting any further progress of the bill. The passing months brought Fred Upton, the Republican Representative who had originally spearheaded the bill close to the term limit afforded, as the chair of the Energy and Commerce Committee. The results of the recent elections seemed to be enough to incentivize compromise for Democrats in the final months of the Obama administration. Both parties returned to negotiations settling on a combination of funds derived from the selling of petroleum reserves and the Affordable Care Act.

In the end, the bill won 392-26 in the House and 94-5 in the Senate. Highlights of the bill under the title of Development include the accepted substitution of “data summaries” for full clinical trials when a new indication is to be added for a previously approved drug and expansion of off label-uses. The FDA has been tasked with evaluating evidence from the real world in an effort to speed-up and improve patient access. Highlights under the title of Discovery include a $4.8 billion boost to the NIH budget and $1.8 billion power pack for Joe Biden’s Cancer Moonshoot. A complete play-by-play of the winners and losers of the final version of the bill can be found in Sheila Kaplan’s article on STATnews. (Sheila Kaplan, STAT)

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December 13, 2016 at 10:38 am

Science Policy Around the Web – July 28, 2015

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By: Sara Cassidy, M.S., Ph. D.

Legislative policy

House and Senate pass legislation to assist ALS sufferers

Both chambers passed the Steve Gleason Act of 2015 in a verbal vote on July 15th, sending the bill to the President’s desk for signing. The bill modifies Medicare provisions for people with amyotropic lateral sclerosis (ALS, A.K.A. Lou Gehrig’s Disease) to allow for the purchase of medical equipment that aids in speech generation (speech-generating devices, SGDs). ALS is a neurodegenerative disease that affects motor neurons and sufferers progressively lose the ability to move limbs and facial muscles necessary for speaking. However, cognitive abilities remain intact. SGDs are electronic systems that allow for verbal communication for individuals with severe speech impairments. The famous physicist Steven Hawking relies on an SGD for communication, for example. These devices often use gaze or eye-tracking to relay information to a computer to generate sound or written messages. The bill is named after a former New Orleans Saints football player diagnosed with ALS and was championed by legislators from Louisiana, Sen. David Vitter (R) and House Majority Whip Steve Scalise (R). Steve Gleason played with the Saints for 8 years (from 2000-2008) and famously blocked a punt in 2006 in the first game played in the Superdome after the devastation caused by Hurricane Katrina. Gleason wrote an op-ed for the Washington Post about the value his SGD gives to the remaining years of his life, and the importance of Medicare coverage of these devices for other ALS sufferers in 2014. (Cristina Marcos, The Hill)

Legislative policy

Congressman Upton urges momentum for medical innovation legislation

Fred Upton (R-MI), chairman of the House Energy and Commerce Committee, is asking the Senate to move forward with a bill that they have been crafting in the Health, Education, Labor, and Pensions (HELP) committee that aligns with the recently passed 21st Century Cures Bill in the House. The goal is to potentially combine the bills in conference negotiations so the final product can be enacted by the end of the year. “We all know what happens in presidential years, right? Things really just sort of get gummed up,” Upton explained to an audience at the Alzheimer’s Association International Conference in Washington, D.C. “We don’t want it to be put into next year.”

The 21st Century Cures Act (H.R. 6), co-authored by Diana DeGette (D-CO), passed the House in a 344-77 vote July 10th. Upton plans to meet the Senate HELP committee chairman Lamar Alexander (R-TN) and ranking Democrat Patty Murray (D-WA) to explain the House legislation by the end of this week. “We want to just walk them through what we went through so that they don’t feel like we jammed them,” Upton said. “Do interoperability [of electronic medical records], do some FDA reform, do something that’s relevant to what we’ve done and we’ll go to conference and we’ll accept it,” he said.

The HELP committee is expected to have a medical innovations bill in September, but recent efforts have been focused on education. On July 16th, Alexander and Murray advanced their committee’s bipartisan elementary and secondary education bill (S. 1177) through the Senate by an 81-17 vote.

Upton said the goal of the Cures Bill was to get more than 300 votes on the House floor to get the attention of the Senate and prod the chamber to act. “Our goal was always 300 votes,” he mentioned in a previous interview, “344 was icing on the cake. We’ll continue to work with all of our colleagues.” However, while the bill did garner bipartisan support, 70 of the 77 unsupportive votes came from Upton’s own party. One of the dissenters, Budget Chairman Tom Price (R-GA) took issue with the $8.75 billion slated for the NIH, because it is categorized as mandatory funding and therefore not subject to budget caps. Price believes this issue could hold up the Senate’s acceptance of the bill. Notably, Sen. Richard Burr (R-NC), who authored a white paper on medical innovation with Sen. Alexander, said he supports keeping the NIH budget discretionary. “This is a discretionary program today; it should stay there,” he noted. And importantly, an amendment to the 21st Century Cures Bill, presented to the House by Dave Brat (R-VA), that would have made the NIH funds discretionary, failed in a 281-141 vote.

Funding authorized by the bill could begin as early as Oct 1st, Upton noted, if the Senate can enact legislation by then.

(Melissa Attias, CQ Roll Call; Caitlin Owens, The National Journal)

Publishing policy

Scientists fed up with sexist overtones at Science Magazine

Science is one of the oldest and most prestigious journals in the academy. In recent years, it has branched out beyond primary scientific research to cover topics like science policy (Science Insider) and career advice for scientists (Science Careers), among others. However, some recent Science publications have proponents of diversity up in arms; so much so that Aradhna Tripati, Jennifer Glass, and Lenny Teytelman authored an open letter to Science (signed by more than 300 scientists) urging its editors to use their influence in the scientific community for the promotion of diversity in science, instead of elevating outdated gender stereotypes typified by the offending posts.

The first offense listed by the letter’s authors was in June 2014. That month, one cover of the weekly Science Magazine was a photo of transgender sex workers in Jakarta whose heads were cropped out of the image. According to Retraction Watch, which was given permission to publish excerpts of the letter, “The cover photo of headless transgender sex workers of color with the caption ‘Staying a step ahead of HIV/AIDS’ fed into stereotypes associating prostitution and HIV/AIDS with three underrepresented communities – women, people of color and the transgender community – along with its general harmful representation of disembodied female bodies”, the authors note. In July of that month Science editor Marcia McNutt did add an addendum to the webpage displaying that cover apologizing for the offense. Congresswomen Jackie Speier (D-CA) also weighed in on the photo, “The use of headless, sexualized women of color on the cover of the most prestigious science publication in the United States sends the message that women and minorities still do not fully belong in the ‘boy’s club’ of science.”

On June 1st, 2015, a post-doc wrote to a Science Careers advice column penned by virologist Alice Huang (Senior Faculty Associate, CalTech) complaining that her new male advisor constantly stares down her blouse. Dr. Huang effectively told her to grin and bear it. To their credit, Science Careers retracted the article within hours, however her advice drew immediate attention from the Internet and was cited by the Washington Post, among others.

The final straw came earlier this month, when Science Careers published an article by Dr. Eleftherios Diamandis, wherein his advice on how to “make it” in academic science involved passing off all domestic responsibilities to your wife in favor of working excessively long hours. “I worked 16 to 17 hours a day, not just to make progress on the technology but also to publish our results in high-impact journals. How did I manage it? My wife—also a Ph.D. scientist—worked far less than I did; she took on the bulk of the domestic responsibilities. Our children spent many Saturdays and some Sundays playing in the company lobby. We made lunch in the break room microwave,” he wrote. And while some commenters at Inside Higher Ed were unfazed by this advice, others are fed up. Janet Stemwedel (associate professor at San Jose State University) told Retraction Watch, “My big issue with the Science Careers career advice/exemplars of people succeeding that are clearly meant to convey something advice-like is how mired they are in a status quo that many of us have been trying to dismantle for (what feels like) forever. Advisor who views you as a pair of boobs rather than a fully human future colleague? Grin and bear it! Need to make an impression to get noticed in your field? Work an unhealthy number of hours and foist the (unpaid/undervalued) domestic and emotional work on your wife! Tips on how to make it assume that nothing’s going to get better — and indeed, they give people following them no reason to work to change the system to make it any better.”

Science Careers published an apology penned by Marcia McNutt, July 16th, acknowledging some mistakes but also asserting their commitment to promoting diversity in science. (Retraction Watch, Rachel Feltmen, Washington Post)

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July 28, 2015 at 11:00 am

Science Policy Around the Web – July 14, 2015

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By: Agila Somasundaram, Ph.D.

Climate Change

Climate Change Is Shrinking Where Bumblebees Range, Research Finds

Climate change has caused the geographical range of bumblebees in North America and Europe to shrink, according to a recent study published in Science. Increasing temperatures have caused the southern limits of bumblebee travels to shrink by 170 miles since the 1970s, but there is no corresponding increase in the northern limits of their travels, likely because the northern areas may be less habitable to the bees. The researchers, led by Jeremy T. Kerr, conversation biologist at University of Ottawa, Canada, studied 67 species of bumblebee in North America and Europe. They compared populations from 1974-2010, when temperatures started increasing, to 1901-1974, when climate change was less of a concern. They found that the southernmost geographical limits of the bumblebee retreated north at about 3 miles per year, and that the trends were quite similar for Europe and North America. Dave Goulson, a bee biologist at the University of Sussex in England, says that he is not surprised by the findings given that bees thrive in cooler climes and would be affected by temperature changes. Many entomologists who disagree with the findings say that the study provides a correlation between climate change and decreasing bumblebee habitat, but not direct evidence that warming climates are the major cause. Entomologists Sydney Cameron and James Strange warn that there are other factors that could affect bumblebee habitat, such as pesticide use, parasites and habitat destruction, and it is too early to say that climate change is the main reason for bumblebees’ shrinking habitat. (Nicholas St. Fleur, The New York Times)

Biomedical research funding

21st Century Cures bill sails through House

The U. S. House of Representatives recently approved the 21st Century Cures Act, a bill to speed the discovery and development of new health cures. The bill supports new funding for the National Institutes of Health (NIH) – $8.75 billion over 5 years, $500 million per year to fund research within NIH’s 27 institutes, including research on precision medicine, while the remaining money would fund high-risk, high-reward research, intramural research and others. The bill also authorizes $550 million to the Food and Drug Administration (FDA) to handle new responsibilities including priority review process for certain antibiotics, speedy approval for certain medical devices, and creation of new guidance documents for drug developers. New provisions include ones that would allow the FDA to approve cures for rare, life-threatening diseases based on smaller clinical trials, given the difficulty in recruiting large number of patients for these studies. Though the bill has the support of many industries, research and patient organizations, it has raised concerns about whether speeding cures might compromise patient safety. There are concerns about “…the misperception that you might be able to speed innovation by lowering standards for safety and efficacy, and we think that would be a terrible mistake,” said former FDA commissioner Margaret Hamburg. Safety concerns include risks of using fast track antibiotics on patients who may not fit the profile of people the drugs were originally tested on, and the risk of increased antibiotic resistance that might result from increased use of newer antibiotics by hospitals, encouraged by higher Medicare reimbursements. An amendment passed with the bill directs the Center for Disease Control and Prevention to study whether increased incentives for the use of new antibiotics would lead to increased antibiotic resistance. The bill is now headed to the Senate. (Kelly Servick, ScienceInsider)

Drug safety

Should you take painkillers? FDA bolsters safety labels, warns of heart attack, stroke risk.

The FDA recently strengthened its safety warnings on over-the-counter and prescription painkillers. Painkillers can increase the risk of heart attack and stroke, and these new labels will be added on non-steroidal anti-inflammatory drugs (NSAIDs), a commonly used class of painkillers for temporary and chronic pain, including ibuprofen (Advil and Motrin) and naproxen (Aleve). A safety label has already been on NSAIDs since 2005, but now the wording would be changed from “may cause” problems, such as heart attack and strokes, to “cause.” A number of research studies have shown that long-term use of high doses of NSAIDs can increase the risk of cardiovascular diseases and death. But recent studies show that the risk of heart attack and stroke are associated with even short-term use of these drugs. Though the risk is higher for people with a history of cardiovascular diseases, everyone may be at risk, says Judy Racoosin, deputy director of FDA’s Division of Anesthesia, Analgesia and Addiction Products. The FDA says that consumers can still take NSAIDs, given that they are effective in treating pain, but people should avoid taking more than one NSAID containing drug at the same time. Also, people should watch for symptoms of a heart attack and stroke, and seek medical help. (Ariana Eunjung Cha, The Washington Post)

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July 14, 2015 at 9:00 am

Science Policy Around the Web – May 26, 2015

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By: Sara Cassidy M.S., Ph.D

photo credit: Matti Mattila via photopin cc

Biomedical Research Funding

21st century cures chugs along

A $13 billion package (HR 6) to spur new medical cures sailed through the House Energy and Commerce Committee Thursday (5/21). The non-partisan so-called 21st Century Cures bill, authored by Fred Upton (R-MI), Frank Pallone (D-NJ), Joe Pitts (R-PA), Gene Green (D-TX) and Diana DeGette (D-CO), aims to modernize and personalize health care, encourage greater innovation, support research, and streamline the system to deliver better faster cures to more patients. The unanimous 51-0 vote sets up floor consideration for the legislation, which would overhaul how the Food and Drug Administration (FDA) evaluates new medical products and provide $10 billion in mandatory funding for the National Institutes of Health (NIH). Rep. Pitts remarked, “Today’s vote is an important next step for this committee as we work to get 21st Century Cures enacted into the law by the end of the year. I want to thank my colleagues on both sides of the aisle for their hard work to modernize our discovery, development and delivery system, which will give hope to millions of Americans for an accelerated path to cures.” Additional markups to the applauded legislation include, $550 million in mandatory funding to the FDA over five years, a mandate to the National Institute on Minority Health and Health Disparities to increase the representation of underrepresented minorities in clinical trials, and specific language on research surrounding Lyme and other tick-borne diseases. Parallel efforts in the Senate, led by the Health, Education, Labor and Pensions Chairman Lamar Alexander (R-TN) are on a much slower timeline, but Rep. Upton had said he thinks the bill could land on the president’s desk before the end of the year. (Melanie Zanona, CQ Roll Call)

Policy Development

OSTP seeks experts to weigh in on microbiome research

The Office of Science and Technology Policy (OSTP) released a Request For Information (RFI) on Wednesday (5/20) seeking advice from industry, academia, research laboratories, and other stakeholder groups involved in microbiome research. The goal of the RFI is to identify the unifying questions in microbiome research, as well as the tools, technologies, and training needed to answer these questions. OSTP is specifically looking for information matching the mission statements of multiple Federal agencies, private sector interests, and current White House Policy Initiatives. Due in large part to improved sequencing technologies over the past 10 years, human microbiome studies have exploded onto the research scene, with potential implications in human health in diverse settings such as obesity and cancer. There is currently one FDA approved microbiome based treatment, fecal microbial transplant, for the treatment of relapsing Clostridium difficile infections. However, most of the field is currently stalled in correlative associations with disease and would like to progress to causative or predictive of disease. The OSTP hopes the RFI will result in a focusing of the microbiome research field, potentially harnessing its therapeutic capacity. Responses will be accepted until 6/15/15. (American Society for Microbiology, Public Affairs Office)

Legislative policy

Senators create a new caucus specifically for the NIH

Sens. Linsdey Graham (R-SC) and Dick Durbin (D-IL) will co-chair a nascent caucus aimed at boasting funds for the National Institutes of Health (NIH). The NIH has lost 25% of its purchasing power since 2003, which the senators attribute to sequestration and flat budgets. The senators believe inadequate funding has stalled the ability of the agency to find cures for some the countries most devastating diseases, like heart disease, Alzheimer’s, and cancer. Despite the fact that many Congress members have recently publicly voiced their support for increasing the NIH budget (including Newt Gingrich and Eric Cantor), paying for it will continue to be an enormous challenge in a political climate where budget cuts and sequestration are de rigeur. And although many are in favor, none have yet proposed a way to pay for it. The other members of the caucus include Sens. Tammy Baldwin (D-WI), Richard Blumenthal (D-CN), Ben Cardin (D-MD), Robert Casey (D-PA), Joe Donnelly (D-ID), Al Franken (D-MN), Angus King (I-ME), Amy Klobuchar (D-MN), Edward Markey (D-MA), Claire McCaskill (D-MO), Jim Moran (R-KS), Gary Peters (D-MI), Brian Schatz (D-HI), and Roger Wicker (R-MS). (Sarah Ferris, The Hill)

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May 26, 2015 at 9:00 am

21st Century Cures Initiative

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By: Danielle Friend, PhD

One year in the making, with several hearings, roundtable discussions, and white papers released, the 21st Century Cures Initiative is nearing completion. The bill has been spearheaded by the conservative Representative, Fred Upton (R-Mich.) and the more liberal Representative Diana DeGette (D-Colo.), both of whom are members of the Energy and Commerce Committee. While biomedical research has advanced at what seems like lightning speed, unfortunately the translation of research into treatments and cures has been slow and costly. This revolutionary new bill aims to update the regulatory processes and to keep legislation readily informed of new developments in the biomedical sciences so that patients see treatments faster.

During the early phases of the 21st Century Cures initiative, the Health Subcommittee Chairman Joe Pitts (R-P.) organized hearings, roundtable discussions, and white papers to collect feedback on the bill from health care stakeholders, patients, manufacturers, medical associations and research institutions. “While increasing accountability, this legislation would invest in the basic research so critical to equipping our nation’s best and brightest with the tools they need to discover the underpinnings of disease”, he stated. The most updated version of the bill was released on April 28, 2015 and following the release, the Heath Subcommittee held a legislative hearing to receive testimony regarding the new bill. Dr. Kathy Hudson, Deputy Director for Science, Outreach, and Policy at the National Institutes of Health (NIH), Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA), and Dr. Jeff Shuren, Director of the FDA Center for Devices and Radiological Health all testified in support of the bill. On May 6, 2015 the committee also hosted a meeting of biomedical leaders, including NIH Director Francis Collins, in the first public discussion regarding the state of biomedical innovation in the United States.

What should you know about the bill?

The new draft of the bill comes in at approximately 200 pages (which is, surprisingly, about half the size of the previous draft!). The current draft is divided into three sections: Discovery, Development, and Delivery. Discovery generally focuses on the budget and organization of the NIH. Development focuses on the FDA’s regulation of clinical trials, drug development and precision medicine. Currently, Delivery focuses on changes to the Social Security Act and Medicare, however much of this section is still being drafted. Below are some of the most important highlights and take-home points from the current draft of the bill.

Title 1: Discovery

Title I of the new bill primarily concerns the organization and budget of the NIH. In fact, through the “NIH Innovation Fund”, the new draft increases the NIH budget by $2 billion per year for five years beginning in fiscal year 2016. The current NIH budget is approximately $30 billion a year, and an additional $2 billion each year is a small but significant increase. Although the programs that the “NIH Innovation Fund” will support are not yet defined, they may include precision medicine and programs for young emerging scientists, like those included in the Capstone Award (Section 1061). More details and definitions of the “NIH innovation fund” are surely to come.

The remainder of Title 1 focuses on the organization and administration of the NIH and the ways in which biomedical innovation can be better translated to the clinic. For example:

  • Subtitle E, “Promoting Pediatric Research Through the National Institutes of Health” states that the “National Institutes of Health should encourage a global pediatric clinical trial network through the allocation of grants, contracts, or cooperative agreements to supplement the salaries of new and early investigators who participate in the global pediatric clinical trial network,” with the goal of combining resources to combat pediatric diseases and birth defects.
  • Subtitle F, “Advancement of National Institutes of Health Research and Data Access” calls for data “generated through NIH-funded research” to be standardized and made available to other researchers.
  • Subtitle G, “Facilitating Collaborative Research” calls for the establishment and release of de-identified clinical trial data from “qualified clinical trials,” including trials on drugs and medical devices so that this data can be used for future discovery. Additionally, this section establishes a national neurological diseases surveillance system.
  • Subtitle H, “Council for 21st Century Cures” establishes a “nonprofit corporation to be known as Council that consists of a public-private partnership” with “the purpose … to accelerate the discovery, development, and delivery in the United States of innovative cures, treatments, and preventive measures for. ”

Title II: Development

As mentioned above, the overall purpose of the bill is to accelerate the speed with which discovered treatments reach the patient in the clinic. Title II of the new bill focuses on ways in which this may occur through the help of the FDA.

  • Subtitle B, “Qualification and Use of Drug Development Tools” aims to establish new biomarkers and other endpoints to accelerate the approval of new treatments. The bill states “The development of biomarkers and other drug development tools can benefit the availability of new medical therapies by helping translate scientific discoveries into clinical applications.” The bill also establishes that the FDA will develop guidelines regarding the use of these biomarkers. The legislation would require FDA and a sponsor to enter into an “accelerated approval development plan” to support the approval of a drug using surrogate endpoints.
  • Subtitle C, “Advancement of Precision Medicine” establishes that the FDA will produce guidelines regarding the definition of a “precision drug.” This part of the bill also establishes that the FDA will release guidelines on investigations can be designed to answer specific questions about narrow sub-populations of patients.
  • Subtitle D, “Modern Trial Design and Evidence Development” aims to accelerate clinical trials. One way this will occur is through the development of FDA guidelines for clinical data collected outside clinical trials, which may include observational trials, product registries and therapeutic use, according to the bill.
  • Section E, “Expediting Patient Access” overhauls “compassionate use”, allowing patients’ easier access to experimental drugs. This policy designates that drug companies company have a point of contact who will process patients’ requests, details of the procedures for making an expanded access request, the criteria for enrolling in a trial and the amount of time the company expects to take to process a request.
  • Section G, “Antibiotic Drug Development” creates a “limited population pathway” for antibacterial and antifungal drugs. This will allow sponsors to seek approval for a product intended to treat “a serious or life-threatening disease, condition or indication” that is currently not adequately served by existing therapies. FDA is also required to set up a website to provide recommendations on which bacteria/fungi are susceptible to specific drugs.
  • Subtitle K, “Priority Review for Breakthrough Devices”, establishes that medical devices are eligible to receive “breakthrough” designation by FDA and provides priority review for these devices. “Breakthrough devices” are those which represent “breakthrough technologies,” are intended to treat conditions “for which no approved alternative exist,” offer “significant advantages over existing approved or cleared alternative,” and are “otherwise in the best interest of patients.”
  • Subtitle L, “Medical Device Regulatory Process Improvements” aims to make the approval process for medical devices faster and easier. For example, in this section, companies would be allowed to make recommendations for the types of expertise FDA should include on an advisory committee panel.

Lastly, much of Title III, Delivery, is currently still being drafted and the Energy and Commerce Committee will be adding sections covering interoperability and telemedicine soon. We’ll look forward to reading the completion of these sections in the near future.

Developing effective treatments and cures for disease is a bipartisan, national priority. However, in order for biomedical discoveries to be translated to the clinic in an efficient and cost effective manner, regulatory processes to facilitate that must keep up to speed. The new 21st Century Cures Initiative aims to do just that. In the developing phases of the bill, Representatives Upton and DeGette explored issues that occur during the discovery of clues to treatments in basic science, during the development of these new treatments, and during the delivery of the identified cures. The current draft of the bill has developed policy to accelerate the regulatory processes involved and to facilitate each of these steps on the road to clinical treatments and cures.

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May 13, 2015 at 9:00 am

Posted in Essays

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Science Policy Around the Web – November 14, 2014

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By: Nicholas Jury, Ph.D

Photo Credit: Lester V. Bergman/Corbis

 

Mental Health

Recent evidence linking gut microbes and mental disorders grab neuroscientists’ attention

Dietary supplement companies selling “probiotics” have often claimed that adding them to a diet can enhance the overall mental health of an individual. Even though these gut-derived microbes are readily available over-the-counter, neuroscientists are not completely sold on their utility. However, recent pre-clinical studies have provided some evidence linking mental conditions such as autism and depression to the gut microbiome. These studies have spurred new interest in the potential connection between the “gut-brain axis.”

More than $1 million has been spent this year by the National Institute of Mental Health on research projects investigating the connection between the gut microbiome and the brain. Furthermore, John Cryan at the University of College Cork in Ireland will present evidence linking gut microbes to depression-like behavior in mice at the Society for Neuroscience Annual Meeting in Washington, D.C. His study demonstrated that mice that were born via caesarean section acted in a more depressed-like manner when compared to mice that were born vaginally. “The microbiome is likely to have its greatest impact on early life,” said Cryan.

Another study conducted in mice demonstrated a potential link between autism-like symptoms and the gut microbe Bacteroides fragilis. Mice with autism-like symptoms were fed these gut bacteria and symptoms ceased. Furthermore, this same group found that the mice with autism-like symptoms had high levels of a bacterial metabolite, and that injecting this metabolite into normal mice caused them to exhibit autism-like symptoms.

These pre-clinical studies provide some evidence of the interaction between our gut microbiome and the brain, and the potential therapeutic utility of probiotics in mental health disorders.

(Sara Reardon, Nature)

 

Federal Science Policy – Regulatory

The 21st century cures initiative drums up bipartisan support on Capitol Hill

Amidst the partisan rancor and gridlock on Capitol Hill, there appears to be some resemblance of bipartisanship with a new biomedical legislative priority. The 21st Century Cures Initiative aims to reduce unnecessary regulation and decrease the time to bring new biological and pharmacological treatments to market. Specifically, the initiative could address regulation regarding clinical trials and institutional review boards. The initiative was conceived by two unlikely bedfellows on the House Energy and Commerce Committee: a conservative, Representative Fred Upton (R-MI), chairman, and a liberal Representative Diana DeGette (D-CO). The first draft of the legislation is expected to be announced at the beginning of the 114th Congress.

(Kelly Servick,  ScienceInsider)

 

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Written by sciencepolicyforall

November 14, 2014 at 11:12 pm