4/15/2011

A Great Advocate and Leader in the Alpha-1 Antitrypsin Deficiency Community

Genetic Alliance Honors Dennis Pollock with the Art of Advocacy Award

On June 25, 2011, during the Genetic Alliance's 25th Anniversary Annual Conference, Dennis Pollock will be honored with the annual Art of Advocacy Award.

Dennis Pollock was a great advocate and leader in the Alpha-1 antitrypsin deficiency community who lost his battle with Alpha-1 on October 4, 2010. Pollock trained countless others on how to be effective advocates.

Pollock was a tireless champion within the Alpha-1 community and wore many hats as part of the Alpha-1 Association: peer guide, fundraiser, support group leader, board member, and advocate. The latter was his greatest passion. He stressed to others that “every single person can make a difference” and proved this when he played a critical role in the passage of the Genetic Information Nondiscrimination Act (GINA) of 2008.

Though Pollock was a double lung transplant recipient in 2004, he would jump on a plane to Washington, DC, whenever needed to advocate for legislation affecting Alphas. On one day’s notice, he traveled to DC from Oklahoma, learned quickly about GINA, and marched to Capitol Hill with a heart as big as his state, a steady gaze, and a frank story of living with a genetic disorder.

3/18/2011

Bipartisan Bill to Help Develop Treatments for Rare Diseases

WASHINGTON, DC— U.S. Senator Bob Casey (D-PA) today introduced the Creating Hope Act of 2011, a bipartisan bill that would encourage the development of new treatments for rare and neglected diseases that disproportionately affect children. The bill, which also has broad support among the medical community, patient advocates and biopharmaceutical companies, would strengthen a cost-neutral FDA program giving biopharmaceutical companies an incentive to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions.

“Millions of Americans are affected by rare diseases and neglected conditions for which there is currently no hope because there is no treatment,” said Senator Casey. “The Creating Hope Act brings light where there is now only darkness by providing an incentive, at no cost to taxpayers, to develop treatments for these illnesses. The broad support for this legislation speaks to the need to solve this problem.”

The Creating Hope Act is cosponsored by Senators Scott Brown (R-MA), Sherrod Brown (D-OH), Al Franken (D-MN) and Johnny Isakson (R-GA).

Rare diseases, such as pediatric cancers, and neglected tropical diseases, such as malaria, affect significant numbers of Americans and people around the globe. More than 20 million Americans have a rare disease, according to the National Organization for Rare Disorders. An estimated one billion people worldwide suffer from a neglected tropical disease according to the World Health Organization, and children are most frequently and profoundly affected by these conditions.

“I applaud Senator Casey’s leadership in introducing the Creating Hope Act,” said Nancy Goodman, Executive Director of Kids v. Cancer. “I want to ensure that one day, children like my son, Jacob, who are diagnosed with brain cancer and other serious and rare diseases, will have drugs available for safe and effective treatments.”

Peter C. Adamson, M.D., Chair of the Children’s Oncology Group at the Children’s Hospital of Philadelphia, said: “I commend Senator Casey and his colleagues for introducing the Creating Hope Act of 2011. As a pediatric oncologist who leads the Children’s Oncology Group, a nationwide team of physicians, scientists, nurses and others dedicated to treating children with cancer, I know that improving the outcome for children with cancer will require development of new, more effective anti-cancer drugs. With cancer remaining the leading cause of disease related death in children in the United States, the Creating Hope Act of 2011 will provide a critically important new way to help engage scientists in the biopharmaceutical industry to help develop better medicines for the children we care for.”

“Long a pioneer in pediatric medical research, Children’s Hospital of Pittsburgh of UPMC is committed to seeking new and innovative therapies for the cure of childhood diseases and disability – no matter how rare,’’ said David H. Perlmutter, MD, Physician-in-Chief and Scientific Director. “Creating incentives for companies to invest in new treatments will help ensure our continued advancement in providing the most exceptional medical care for the most vulnerable children.”

“Of the nearly one in 10 Americans with rare diseases, approximately two-thirds are children,” said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD). “Many of these rare pediatric diseases are very serious and treatments are desperately needed. By expanding priority review vouchers to include pediatric rare diseases, this legislation would encourage the development of treatments for children with serious rare diseases.”

“Pennsylvania Bio thanks Senator Casey for his continued commitment to the life sciences industry and the thousands of patients in the United States who suffer from rare diseases,” stated Christopher P. Molineaux, President, Pennsylvania Bio. “With nearly 7,000 rare diseases, defined as diseases affecting 200,000 or fewer Americans, few treatment options exist for these small patient populations because it is often prohibitive for companies to enter the rare disease marketplace. Pennsylvania Bio encourages members of the 112th Congress to follow Senator Casey’s lead and take the necessary steps to support corporations who are conducting research into treatments for these rare diseases, many of which affect children.”

Despite this significant unmet medical need, private companies seldom pursue new therapies for tropical illnesses or rare diseases because it requires making an investment in products that will likely not recoup the high costs associated with their research, development, marketing and distribution. Developing products for children is particularly challenging because of the difficulties associated with conducting clinical trials on this population.

Currently, pharmaceutical companies can receive a priority review voucher (PRV) if they develop novel treatments for neglected tropical diseases, entitling the company to a priority six month review of another new drug application that would otherwise be reviewed under the FDA’s standard ten month review period. This shortened review time, which can lead to earlier market entry, is estimated to be worth hundreds of millions of dollars.

The Creating Hope Act of 2011 will build on the PRV program to create a stronger incentive for companies to invest in new treatments for rare diseases affecting children and neglected tropical diseases. This legislation will:

•Expand priority review vouchers to include treatments for pediatric rare diseases, so that there is greater incentive to close the devastating treatment gap for kids.

•Close a loophole in current law to prevent companies from receiving a voucher for products that they already market in other countries.

•Offer unlimited transferability of vouchers to create a more easily-traded asset.

•Provide greater certainty to sponsors by permitting them to seek a designation from FDA before they submit their new drug application that the drug, if approved, will qualify for a voucher.

•Strengthen reporting and marketing requirements by requiring that the sponsor submit a statement of good faith intent to market the eligible drug.

•Add Chagas disease, which is responsible for more deaths in Central and South America than every other parasite-borne disease, to FDA’s list of neglected tropical diseases.

The following groups and businesses have endorsed the legislation: Aeras Global TB Vaccine Foundation, Association of Pediatric Oncology Social Workers, American Society of Clinical Oncology, American Society of Pediatric Hematology and Oncology, American Society for Tropical Medicine and Hygiene (ASTMH), Bio Ventures for Global Health, Chai Lifeline, Children's Brain Tumor Foundation, Children’s Hospital of Philadelphia, Children’s Hospital of Pittsburgh of UPMC, CureSearch, GlaxoSmithKline, Genzyme, Kids v Cancer, Leukemia & Lymphoma Society, The National Association of Children’s Hospitals, The National Children's Cancer Society, National Organization for Rare Disorders, Novartis, Medicines for Malaria Venture (MMV), Merck, Metronomix, Sarcoma Foundation of America, Pennsylvania BIO, Shire, CSL Behring, TB Alliance.

2010 Genetics Day on the Hill, AARC's Virtual Lobby Day

Genetics Day On July 15, 2010, Dennis Pollock, Vice Chair of the Alpha-1 Association's Board of Directors and Linda Rodriguez, staff liaison of the joint Public Policy Working Group, represented the Alpha-1 Community at the Fifth Annual Genetics Day on the Hill. For this one day, the genetics and health community came together as one harmonious voice to initiate and build momentum for change in policy issues that impact the community as a whole.