Millions around world to observe Rare Disease Day on Sunday

• Author: Health Informer
• Filed under: Health News
• Date: Feb 24,2010

A coalition of more than 300 U.S. patient organizations, medical societies, government agencies and companies will join the National Organization for Rare Disorders (NORD) in observing World Rare Disease Day on Sunday, Feb. 28. At the same time, corresponding groups around the globe will be doing the same thing.

“People with rare diseases remain a medically underserved population,” said NORD President and CEO Peter L. Saltonstall. “Studies have shown that it often takes several years to get a diagnosis. Little or no research is being done on many rare diseases. And, even when there is a treatment, patients may have difficulty accessing it because of reimbursement or other issues.”

For those reasons, he said, patient organizations around the world are uniting to support an annual day to focus attention on rare diseases as a public health issue and on the need for more research on these diseases.

Events planned in the U.S. for Rare Disease Day 2010 include:

– An Advocacy Day on Capitol Hill hosted by an organization of college football players known as Uplifting Athletes

– Exhibits and displays such as a 60-foot paper link chain in the lobby of Children’s Hospital Boston bearing the names of more than 350 rare diseases that do not have clinical trials

– Open houses and gatherings hosted by patient organizations

– An awareness day organized by pre-medical students at the University of Connecticut

– A rare disease symposium at the Sanford-Burnham Medical Research Institute in San Diego

– A “Wear Your Jeans on Rare Disease Day” campaign

– Online postings of patient stories and videos, newspaper and TV reports, and a “radio media tour”

– Creation of a Research “Hall of Fame”

Rare Disease Day was launched in 2008 by the European Rare Disease Organization, EURORDIS. Last year, EURORDIS asked NORD to sponsor Rare Disease Day in the U.S. Now the day is also being observed in Australia, South America, China and–for the first time this year–Japan.

In the U.S., a disease is considered rare if it affects fewer than 200,000 Americans. There are thought to be nearly 7,000 such diseases affecting nearly 30 million Americans, or about 1 in 10 people.

“More than half of the patients are children,” Saltonstall said. “And many of these diseases are extremely disabling or even life-threatening. Patients and their families often have to deal with very challenging medical, financial and emotional issues.”

Rare Disease Day 2010 will highlight the need for more medical research. While progress has been made in the development of treatments in recent years, about 15 million Americans still have rare diseases for which there is no FDA-approved treatment. In many cases, no research is being done on those diseases.

For information about Rare Disease Day activities by NORD and its partners in the U.S., go to www.rarediseaseday.us. For information about global Rare Disease Day activities, go to www.rarediseaseday.org.

FDA announces possible safety concern for HIV drug combination

• Author: Health Informer
• Filed under: Health News
• Date: Feb 24,2010

Review of data indicating life-threatening heart abnormality underway

The U.S. Food and Drug Administration announced preliminary data suggesting that Invirase (saquinavir) in combination with Norvir (ritonavir) may have potentially important adverse effects on the heart.

When used together, the drugs may cause prolongation of the QT and PR intervals on an electrocardiogram. Prolongation of the QT interval may lead to a condition known as torsades de pointes, an abnormal heart rhythm. Prolongation of the PR interval may also lead to an abnormal heart rhythm known as heart block. With torsades de pointes or with heart block, patients may experience lightheadedness, fainting, or abnormal heart beats. In some cases, torsades de pointes may progress to a life-threatening irregular heart beat known as ventricular fibrillation.

Review of the data is ongoing. Preliminary findings suggest that some patients using Invirase and Norvir may be at an increased risk for heart abnormalities leading to irregular heart rhythms. For example, the risk for torsades de pointes may be increased in patients who are also using medications known to cause a heart disturbance called QT interval prolongation. The risk may also be increased in patients who have a history of QT interval prolongation.

Patients using Invirase should talk to their health care professional about any questions or concerns they have about Invirase. Patients and health care professionals should report any side effects from the use of Invirase to the FDA’s MedWatch program: http://www.fda.gov/safety/MedWatch/default.htm

Invirase is an antiretroviral medication that was first approved in 1995. Invirase is used in combination with Norvir and other antiretroviral medicines to treat HIV in adults. Invirase does not cure HIV infection, may not prevent you from developing HIV-related illnesses, and may not prevent you from spreading HIV to other people.

This early communication is in keeping with FDA’s commitment to inform the public about ongoing safety reviews of drugs. FDA will communicate its findings to the public as soon as the review is complete.

Invirase is marketed by San Francisco-based Genentech, a subsidiary of the Roche Group. Norvir is marketed by Abbott Park, Ill.-based Abbott Laboratories.

FDA approves first new inhaled antibiotic for Cystic Fibrosis in more than a decade

• Author: Health Informer
• Filed under: Health News
• Date: Feb 23,2010

CF Foundation’s $1 Million Investment Helped Drug Known as Cayston(R) Become a Reality

The U.S. Food and Drug Administration approved an important new inhaled antibiotic called Cayston® (aztreonam for inhalation solution) today for the treatment of cystic fibrosis. The drug was made possible by significant support from the Cystic Fibrosis Foundation, including a $1 million investment by a Foundation subsidiary to help develop the therapy.

Developed by Gilead Sciences, Inc., Cayston offers a much-needed antibiotic alternative for CF patients who battle recurrent lung infections and develop resistance to existing antibiotics. Lung infections are the leading cause of death in CF.

Cayston is the first CF drug to advance — from beginning to end — through the Cystic Fibrosis Foundation’s Therapeutics Development Program, which is designed to speed the creation of new CF therapies. In addition, Cystic Fibrosis Services, Inc, a wholly owned subsidiary of the Foundation, will be one of only several pharmacies in the country distributing the drug.

Cayston will be available by the end of next week.

“The approval of Cayston demonstrates that our drug development model is working and making a real difference in the lives of people with cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We are delighted to have a new antibiotic in the arsenal to help fight the life-threatening infections associated with this disease, as we continue to push forward with other therapies that address the underlying cause of CF.”

The Foundation supported Cayston at each stage of development, from providing an initial therapeutics development award, to making the Foundation’s clinical trials network available to test the drug in patients, to advocating with the FDA for the drug’s approval.

“The Cystic Fibrosis Foundation’s early funding and ongoing support for Cayston played an important role in bringing this new therapy to patients,” said Bruce Montgomery, M.D., senior vice president, head of Respiratory Therapeutics, Gilead Sciences. “We are thrilled to help meet an urgent medical need for CF patients, and to support the Foundation’s mission to improve the lives of people with this terrible disease.”

Cayston is administered with a new device called the Altera(R) Nebulizer System that allows patients to take the medicine in less than five minutes, a fraction of the time required for other inhaled antibiotics. This shortened delivery time reduces the burden on patients, who – on average – have a treatment regimen of three-to-four hours per day.

Patients interested in learning more about Cayston should consult their physicians.

To support patients, Gilead is launching a Cayston Access Program, a call center developed with a nonprofit subsidiary of the Cystic Fibrosis Foundation.

The program will assist people with CF and members of their care team with insurance verification, co-pay assistance and claims support. For information, call 1-877-7CAYSTON (877-722-9786).

In 2004 Cystic Fibrosis Foundation Therapeutics, Inc., a nonprofit affiliate of the Cystic Fibrosis Foundation, gave a therapeutics development award to Corus Pharma to support an experimental drug now known as Cayston. In 2006, Gilead Sciences acquired Corus and continued the program, filing a New Drug Application with the FDA in 2007.

Source: Cystic Fibrosis Foundation

Americans remain split on stalled health care legislation

• Author: Health Informer
• Filed under: Health News
• Date: Feb 23,2010

But Some Provisions Popular Among Majorities of Democrats, Independents and Republicans

Most See Delays As Driven By Politics Rather Than Policy

The latest Kaiser Tracking Poll finds the public still split on health care reform legislation, with 43 percent in favor and 43 percent opposed. However, the poll also finds that majorities of Americans of all political leanings support several provisions in the health reform proposals in Congress and most attribute delays in passing the legislation to political gamesmanship rather than policy disagreements.

As Democratic and GOP lawmakers prepare for the upcoming health reform summit, the February Kaiser Health Tracking Poll finds that at least six of every ten Republicans, Democrats and independents back at least some of the key provisions in the reform bills that have passed the House and Senate. They include measures that would: reform the way health insurance works, such as preventing insurers from excluding people because of pre-existing conditions; offer tax credits to small businesses to help their workers get coverage; create a new health insurance marketplace; help close the Medicare “doughnut hole” so that seniors would no longer face a period of having to pay the full cost of their medicines; and expand high-risk insurance pools for individuals who cannot get coverage elsewhere. Providing subsidies to lower and middle income people also received strong support from Democrats and independents and near majority support from Republicans.

“While the intense debate over health care reform has divided the public, it looks like there is bipartisan support on at least some elements of health reform legislation, and more bipartisan support outside the beltway than there is inside,” said Kaiser President and CEO Drew Altman.

Along with these areas of agreement were differences of opinion that broke along party lines. Expanding Medicaid received majority support from Democrats and independents, but was deemed important to pass into law by a much smaller share of Republicans, 34 percent. On the other hand, limiting malpractice lawsuit payments ranked second among priorities for Republicans, but barely cracked the top 10 for Democrats and independents.

More and Less Popular Provisions

Given a list of various elements of health care reform, more than two-thirds of the public overall said it was either “extremely” or “very important” that these changes be passed into law: reforming the way health insurance works ( 76%), providing tax credits to small businesses (72%), creating a health insurance exchange (71%), helping close the Medicare “doughnut hole” (71%), expanding high-risk insurance pools (70%), and providing financial help for lower- and middle-income people (68%). At the bottom of the list, though still receiving significant levels of support, were measures to allow insurers to sell policies across state lines (50%) or to limit future increases in Medicare provider payments (46%).

The February Tracking Poll did not examine public opinion on the full range of measures to finance health reform or the public option; subjects which have been addressed comprehensively in previous tracking polls.

All Sides Point to Politics as the Reason for the Delay in Passing Health Reform

Most Americans attribute the delays in passing health care reform to political gamesmanship, with 59 percent saying the delays are “more about both sides playing politics” and 25 percent saying they are “more about Republicans and Democrats having disagreements.” This sentiment runs the political gamut, with 61 percent of Democrats, 62 percent of independents and 53 percent of Republicans fingering political considerations as the primary reason for the delays.

The large majority of Democrats reported feeling frustration and disappointment about the legislation being stalled, as did a narrow majority of independents. Most Republicans reported feeling “relieved.”

Moving Forward from Here

The poll shows that after a long and intense debate, the public has mixed views about how quickly and how comprehensively to move on reform, but relatively few hope the matter is dropped altogether. Overall, 32 percent hope Congress will send some version of the House and Senate’s comprehensive overhaul legislation to the president, and another 20 percent support pulling out some key provisions on which there is broad agreement and just passing those. Another 22 percent would rather Congress put health care on hold and deal with other priorities. And a similar percentage (19%) would like to pull the plug on health care reform for 2010.

When all is said and done, 58 percent of Americans say they will be either disappointed or angry if Congress decides to stop working on health care reform. Thirty-eight percent say they expect to feel happy or relieved. The numbers are roughly reversed when the public is asked how they will feel if Congress passes reform, with 54 percent naming a positive emotion and 38 percent a negative one.

The survey was designed and analyzed by public opinion researchers at the Kaiser Family Foundation and was conducted February 11 through February 16, 2010, among a nationally representative random sample of 1,201 adults ages 18 and older. Telephone interviews conducted by landline (800) and cell phone (401, including 192 who had no landline telephone) were carried out in English and Spanish. The margin of sampling error for the total sample is plus or minus 3 percentage points. For results based on subgroups, the margin of sampling error is higher.

The full question wording, results, charts and a brief on the poll can be viewed online at http://www.kff.org/kaiserpolls/8051.cfm.

Source: Henry J. Kaiser Foundation