The Boomer Esiason Foundation

Category: CF News

Every day, researchers around the world come one step closer to discovering a cure for cystic fibrosis. In the meantime, the CF drug “pipeline” ensures therapies are moving from the laboratory to the marketplace. We keep an eye on medical news sources from around the world and report on these developments as they occur.

  • Legislation to Expedite Approval of Rare-Disease Drugs Moving Through Congress

    Source: Cystic Fibrosis Foundation

    The U.S. House of Representatives has passed the Expanding and Promoting Expertise in the Review of Rare Treatments Act, better known as the “EXPERRT” Act.

    Introduced by Reps. Edward Markey (D-MA), Tom Marino (R-PA) and Cliff Stearns (R-FL), the legislation enables expanded consultation between the Food and Drug Administration (FDA) and external rare disease experts and patient advocates during the FDA drug approval process. EXPERRT passed as part of the Food and Drug Administration Safety and Innovation Act, which reauthorizes the FDA’s user-fee program that funds its drug and device evaluation.

    “The EXPERRT Act will help expedite the approval of safe and effective new rare-disease drugs and treatments for patients by ensuring that the FDA has the most complete information during its evaluation,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This legislation is critically important to the 30,000 Americans with cystic fibrosis and millions more with rare diseases who desperately need access to sophisticated therapies for their complex conditions. We extend our special thanks to Representatives Markey, Marino and Stearns for the critical work they have done to advance this legislation.”

    Passage of this legislation follows the recent approval of Kalydeco™, a cystic fibrosis drug developed by Vertex Pharmaceuticals with major financial, scientific and clinical support from the Cystic Fibrosis Foundation. Kalydeco’s swift approval, announced by the FDA just three months after it was submitted for review, was a result of the overwhelming, conclusive clinical evidence of its safety and efficacy. The review process benefitted greatly from expertise provided by the Foundation and its partners, as well as patients and other experts. Kalydeco is the first CF drug to address the underlying cause of the disease in select CF patient groups.

    “As new promising cystic fibrosis treatments come through the pipeline, we hope they will be reviewed with the same speed and agility as Kalydeco,” said Beall. “Our aim is to establish the best practice we saw with Kalydeco’s review as the standard, not only for cystic fibrosis treatments, but for all rare disease drugs.”

    The legislation, which previously passed the Senate, passed the House with changes. It now goes back to the Senate for final approval. If it is passed again by the Senate, the legislation will then go to the White House for President Obama’s signature. 

  • CF Foundation Kicks Off ‘Make Every Breath Count’ Campaign

    Volunteers from all over the country will meet with their elected officials in their hometowns this summer as part of Make Every Breath Count, the Cystic Fibrosis Foundation’s annual national advocacy campaign.

    By participating in Make Every Breath Count, volunteers hope to build support in Congress for vital cystic fibrosis research and care.

    “You have to stand up. You have to visit your congressman,” says Rep. Edward Markey (D-MA), co-chair of the Congressional CF Caucus, emphasizing the importance of advocacy in the fight against CF.

    Between now and Labor Day, Foundation volunteers plan to hold at least 75 meetings with representatives — one or more meeting in every state.

    Go to the CFF website to find out more about Make Every Breath Count and to schedule a meeting with your representative in your hometown.

  • HHS Reduces Insurance Premiums for Pre-Existing Condition Plan

    The U.S. Department of Health and Human Services (HHS) on May 31 announced new steps to reduce premiums and make it easier for Americans to enroll in the Pre-Existing Condition Insurance Plan.

    Premiums for the Federally-administered Pre-Existing Condition Insurance Plan (PCIP) will drop as much as 40 percent in 18 States, and eligibility standards will be eased in 23 States and the District of Columbia to ensure more Americans with pre-existing conditions have access to affordable health insurance.

    The Pre-Existing Condition Insurance Plan was created under the Affordable Care Act and serves as a bridge to 2014 when insurers will no longer be allowed to deny coverage to people with any pre-existing condition, like cancer, diabetes, and asthma.

    “The Pre-Existing Condition Insurance Plan changes lives, and in many cases, literally saves lives,” said HHS Secretary Kathleen Sebelius. “These changes will decrease costs and help insure more Americans.”

    In 23 States and the District of Columbia, the PCIP program is Federally-administered. The remaining States operate their own PCIP programs using Federal funds provided by the Affordable Care Act.

    Under the changes announced today, PCIP premiums will drop as much as 40 percent in 18 States where the Federally administered PCIP operates. These premium decreases help bring PCIP premiums closer to the rates in each State’s individual insurance market; in the six States where PCIP premiums were already well-aligned with State premiums, premiums will remain the same.

    The changes announced today will make enrolling in the Federally-administered PCIP in 23 States and the District of Columbia easier. Starting July 1, 2011, people applying for coverage can simply provide a letter from a doctor, physician assistant, or nurse practitioner dated within the past 12 months stating that they have or, at any time in the past, had a medical condition, disability, or illness. Applicants will no longer have to wait on an insurance company to send them a denial letter. This option became available to children under age 19 in February, and this pathway is being extended to all applicants regardless of age. Applicants will still need to meet other eligibility criteria, including that they are U.S. citizens or residing in the U.S. legally and that they have been without health coverage for six months.

    HHS also sent letters today to the 27 States running their own programs to inform them of the opportunity to modify their current PCIP premiums.

    To further enhance the program, beginning this fall, HHS will begin paying agents and brokers for successfully connecting eligible people with the PCIP program. This step will help reach those who are eligible but un-enrolled. Several States have experimented with such payments with good success. This is a part of continuing HHS outreach efforts with States, insurers, providers, and agents and brokers to reach more eligible people and let them know that coverage is available. HHS is also working with insurers to notify people about the PCIP option in their State when their application for health insurance is denied.

    Congress created the temporary PCIP program as part of the Affordable Care Act to help uninsured Americans with a variety of medical conditions get affordable coverage rather than be locked out of the system by insurance companies. In 2014 and beyond, insurers will be prohibited from denying coverage to anyone with a pre-existing condition and new competitive marketplaces called Health Insurance Exchanges will give people the opportunity to shop for the policy that best suits their needs. Millions of Americans also will receive tax credits to help make coverage affordable.

    Enrollment in PCIP programs has begun to grow rapidly. In the period between November 2010 and March 2011, enrollment in all programs rose 129 percent to more than 18,000 Americans enrolled in PCIP.

    “These changes will get more people covered,” said Steven Larsen, the Director of the Center for Consumer Information and Insurance Oversight. “We’re encouraged by recent increases in enrollment and we’re excited to build on these efforts and reach even more people.”

    PCIP provides comprehensive health coverage, including primary and specialty care, hospital care, prescription drugs, home health and hospice care, skilled nursing care and preventive health and maternity care. It limits annual out-of-pocket spending and does not carve out benefits the people need. Eligibility is not based on income and people who enroll are not charged a higher premium because of their medical condition.

    To find a chart showing changes to PCIP premiums in the States with Federally-administered PCIP programs, visit www.HealthCare.gov/news/factsheets/pcip05312011a.html.

    For more information, including eligibility, plan benefits and rates, as well as information on how to apply, visit www.pcip.gov and click on “Find Your State.” Then select your State from a map of the United States or from the drop-down menu. The PCIP Call Center is open from 8 a.m. to 11 p.m. Eastern Time. Call toll-free 1-866-717-5826 (TTY 1-866-561-1604).

    Source: U.S. Department of Health and Human Services

  • Clinical Trials Legislation Takes Effect

    A new law that allows patients with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits went into effect yesterday. The bill, known as the “Improving Access to Clinical Trials Act” (IACT), was signed into law in October 2010.

    Because of the new law, patients receiving federal assistance will no longer have to choose between access to healthcare benefits and participating in a clinical trial that could result in a new treatment for CF. 

    Previously, many people who received Supplemental Security Income (SSI) were prevented from accepting research compensation because it made them ineligible to receive government medical benefits. This penalty stopped significant numbers of people with rare diseases from participating in clinical studies. 

    The legislation was introduced by Senator Ron Wyden, D-Ore., in the Senate, and by Cystic Fibrosis Caucus Co-Chairs Reps. Edward Markey, D-Mass., and Cliff Stearns, R-Fla., in the House.

    Source: Cystic Fibrosis Foundation

  • FDA Panel Not Sold on Personal Genetic Testing

    A Food and Drug Administration advisory panel said March 8 that genetic tests directly marketed to consumers should be allowed only under a doctor’s supervision.

    Personal testing, which is mainly available online from firms operating outside traditional medical institutions, can produce ambiguous or misleading results without proper analysis, panel members said.

    “I would suggest that we are not ready yet to put this completely in the consumer’s hands,” said panelist Joann Boughman of the American Society of Human Genetics. “Each test is complex, and when you have each provider doing slightly different tests, it complicates it even more.”

    For example, a consumer test to determine whether someone is a carrier for cystic fibrosis might not screen for all the genetic permutations that trigger the disease, meaning that a negative result could promote a false sense of security.

    “It’s very dangerous to get a false reassurance when you don’t know about environmental and other risk factors,” said panel member George Netto of the Johns Hopkins School of Medicine.

    The 21-member panel, predominantly a mix of physicians and academics, did not vote on specific questions during the first day of a two-day hearing that concludes Wednesday. But members expressed general agreement that doctors should be in charge of ordering and interpreting the tests.

    The panel’s consensus on new regulations is not binding on the FDA, but the agency usually follows them.

    Unlike genetic tests ordered by doctors, which are processed by a laboratory and delivered to the physician for review with the patient, direct-to-consumer testing allows individuals to get genetic information directly from a lab without involving a healthcare provider.

    Advocates of testing say it allows consumers to take a better-informed role in their medical care.

    “We’re not trying to substitute for a physician — we’re simply providing a service that doesn’t exist otherwise,” said Jeff Gulcher, cofounder of testing company deCODE Genetics Inc. of Reykjavik, Iceland.

    Critics argue that personal testing still lacks the precision to be an effective mass-market healthcare tool.  

    Doubts were fueled in July by a Government Accountability Office report that found that different companies came to different conclusions about the meaning of the same DNA sample.

    In one case cited by GAO investigators, four companies evaluating the same DNA reported variously that the person had a below-average, average and above-average risk for prostate cancer and hypertension.

    Direct-to-consumer tests have been available online for several years, but assumed a higher public profile in May when Walgreen Co. announced that it would sell one brand of tests in its drugstores.

    That prompted the FDA to declare that they needed to meet regulatory standards as medical devices.

    Walgreen has shelved plans to sell the test in its stores until the regulatory uncertainty is cleared up, a spokesman said.

    Source: Andrew Zajac, Los Angeles Times

  • ‘Improving Access to Clinical Trials Act’ Passes U.S. House; Heads to President for Signatur

    The U.S. House of Representatives passed the “Improving Access to Clinical Trials Act” (I-ACT), in a victory for the Cystic Fibrosis Foundation, its advocates and 120 other health advocacy organizations.

    The bill, which passed the Senate Aug. 5, now goes to President Obama’s desk for his signature. He is expected to sign it.

    This legislation enables patients with rare diseases to participate in clinical trials without losing eligibility for public health care benefits.

    Passage of this legislation is particularly important for people with CF, a rare genetic disease. A limited patient population makes it challenging to find enough people to participate in research studies evaluating the effectiveness of promising new drugs.

    “Because of this groundbreaking legislation, people with CF and other rare diseases will no longer be forced to choose between critical health care coverage and participation in research that could lead to the development of a cure for our most serious illnesses,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We are grateful to our champions in Congress for approving this bill, which will help move new treatments more swiftly from the lab to the patients who need them most.”

    Congressional Cystic Fibrosis Caucus Co-Chairs, Reps. Edward Markey, D-Mass., and Cliff Stearns, R-Fla., led the effort to pass the bill in the House. The House version of the legislation, H.R. 2866, has 141 co-sponsors.

    “No one should have to choose between participating in a clinical trial and accessing the essential benefits they need. Today’s bill will open doors of hope and offer the possibility of better health to those with rare diseases like cystic fibrosis. I am proud to partner with my friend and co-chairman of the Congressional Cystic Fibrosis Caucus, Congressman Cliff Stearns, in the passage of this bi-partisan bill, which now will be signed into law by President Obama. I also want to commend the Cystic Fibrosis Foundation for its incredible work on this vital issue. Today represents an important and hopeful milestone in the battle to beat devastating rare diseases that afflict millions of Americans around the country,” Markey said.

    Added Stearns: “As co-chair and co-founder of the Congressional Cystic Fibrosis Caucus, I commend my colleagues for approving this legislation allowing people with rare diseases such as cystic fibrosis to participate in life-saving clinical trials that provide nominal compensation without the risk of losing their health care coverage. I also deeply appreciate the work of the Cystic Fibrosis Foundation in supporting my legislation.”

    The Senate version of the legislation, S. 1674, was introduced by Sen. Ron Wyden, D-Ore., with Sens. Chris Dodd, D-Conn., James Inhofe, R-Okla., Richard Shelby, R-Ala., and Richard Durbin, D-Ill., as original co-sponsors. An additional 17 co-sponsors signed on.

    Current law prevents many people who receive Supplemental Security Income (SSI) from accepting research compensation because it makes them ineligible to receive government medical benefits. This penalty has stopped significant numbers of people with rare diseases from participating in clinical studies.

    Source: Cystic Fibrosis Foundation

  • ‘Improving Access to Clinical Trials Act’ Passes U.S. Senate in Victory for CF Advocates

    The U.S. Senate last night passed the “Improving Access to Clinical Trials Act” (I-ACT), a bipartisan piece of legislation championed by the Cystic Fibrosis Foundation, its advocates and 120 other health advocacy organizations.

    The legislation enables patients with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits.

    “We are one step closer to breaking down a serious barrier to participation in clinical trials, which one day could deliver a cure for cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This legislation represents an important opportunity for people with CF to take part in groundbreaking research that was previously out of their reach. We are elated that this billhas been approved by the Senate.”

    The legislation was introduced by Senator Ron Wyden, D-Ore., with Senators Chris Dodd, D-Conn., James Inhofe, R-Okla., Richard Shelby, R-Ala., Dick Durbin, D-Ill. as original co-sponsors and an additional 14 co-sponsors also signed on.

    Current law prevents many people who receive Supplemental Security Income (SSI) from accepting research compensation because it makes them ineligible to receive government medical benefits. This penalty has stopped significant numbers of people with rare diseases from participating in clinical studies.

    Following Senate approval, the bill now awaits consideration by the U.S. House of Representatives. Reps. Edward Markey, D-Mass., and Cliff Stearns, R-Fla., are leading the effort to pass the bill in the House. The legislation, HR 2866, is co-sponsored by 135 members.

    Passage of this legislation is particularly important for people with CF, a rare genetic disease that affects 30,000 people in the United States. A limited patient population makes it challenging to find enough people to participate in research studies evaluating the effectiveness of promising new drugs.

    Source: Cystic Fibrosis Foundation