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Hormonal Therapy for Post-menopausal Women with Early Stage Breast Cancer

Anna Mazzucco, PhD, Brandel France de Bravo, MPH, Caroline Halsted, Danielle Shapiro, MD, MPH, and Diana Zuckerman, PhD, Cancer Prevention and Treatment Fund

Breast cancer is the most common type of cancer in women around the world, and the second leading cause of cancer deaths among U.S. women. The survival rate for early-stage breast cancer is very high.  For women whose cancer is diagnosed when it is only in the breast, the 5-year survival rate is 99%.  For women whose breast cancer has spread to the lymph nodes, the 5-year survival rate is 85%.

Women who are diagnosed with early-stage breast cancer almost always undergo surgery to remove the cancer (either lumpectomy/partial mastectomy or mastectomy). Most will also choose at least one other treatment in addition to surgery:

1) If they have a lumpectomy, they often undergo radiation to either shrink the tumor before surgery or to kill any cancer cells in the breast that were missed during surgery.

2) If their cancer is estrogen receptor positive (about 84% of breast cancers), many women will try to take hormonal therapy for at least five years after surgery to lower the chance of cancer coming back in either breast the future. For pre-menopausal women, the standard treatment is tamoxifen. For women who have completed menopause (post-menopausal breast cancer), tamoxifen and/or an aromatase inhibitor can be used.[1]

Types of Hormonal Therapies for Early-Stage Breast Cancers

Hormonal therapy (also called endocrine therapy or anti-estrogen therapy) is the opposite of the type of hormones women sometimes take to reduce the symptoms of menopause. It lowers your estrogen levels instead of increasing them.[1]

Hormonal therapy is recommended for most women with breast cancer, and sometimes it is taken by women who have not been diagnosed with breast cancer but are at high risk for it based on their genes or family history. When hormonal therapy is used before breast cancer develops, it is called “primary prevention” or “chemoprevention.” Chemoprevention is completely different from the drugs used in chemotherapy to treat breast cancer.[1]  See our article on breast cancer prevention.

Four types of hormonal therapy are FDA-approved for early-stage breast cancer treatment for post-menopausal women: tamoxifen, exemestane, letrozole, and anastrozole.[3]

For post-menopausal women, there are many recommended ways to take hormone therapy, including: [4]

  • Taking tamoxifen for 5 to 10 years
  • Taking an aromatase inhibitor for 5 to 10 years
  • Taking tamoxifen for 5 years, then switching to an aromatase inhibitor for the next 5 years
  • Taking tamoxifen for 2-3 years, then switching to an aromatase inhibitor for the next 5 years

How Does Hormonal Therapy Work?

Tamoxifen:

Tamoxifen is a selective estrogen receptor modulator (SERM), which means it blocks estrogen activity in breast tissue, but promotes estrogen activity in other tissues (such as in bone and the inner lining of the uterus). Tamoxifen is only effective in breast cancers that are estrogen receptor positive.[3,4]

Aromatase inhibitors:

In post-menopausal women, the ovaries slow down the production of estrogen, but the body uses an enzyme called aromatase to make estrogen from other hormones. Aromatase inhibitors block aromatase, which lowers the amount of estrogen in the body. By taking away the supply of estrogen, aromatase inhibitors help to stop the growth of cancer cells.[3,4]

How Effective are the Treatments?

The effectiveness of treatments is often reported in terms of risk or risk reduction. Risk is another word for chance–what is the chance that something will happen, such as cancer returning or the patient dying? Risk can be reported in terms of relative risk or absolute risk. Let’s use simple numbers to show what we mean: In a study 100 women are given a new drug and 100 other women are given an older drug.  What if the study showed that 4 patients (4%) taking the older drug became nauseous compared to only 2 patients (2%) taking the new drug.  The relative risk of patients getting nauseous is 50% lower for patients taking the new drug, and that sounds impressive.  But the absolute difference is only 2% – when you subtract 2% taking the new drug compared to 4% taking the old drug.

Based on the statistics, the odds may favor taking the new drug. But if the new drug costs much more or has other side effects, a patient might decide she is willing to take the 2% greater risk of becoming nauseous. We prefer to use the absolute difference in risk as it is more informative for patients than the relative risk.

Tamoxifen

Over decades of clinical trials in hundreds of thousands of women, tamoxifen has been shown to reduce the chances of breast cancer recurrence and breast cancer death. But it is important to consider exactly what the benefits are likely to be for you.

Breast cancer recurrence:

A landmark report showed that about 23% of women aged 55 and older who took tamoxifen for 5 years after their cancer was removed had a breast cancer recurrence within 10 years compared to about 42% of women 55-69 and 44% of women 70 and older who did not take tamoxifen.  In that study, women with early-stage breast cancer included women with Stage 1, Stage 2, and Stage 3A; in other words, it ranges from a very tiny breast cancer to a large cancer that has spread to several lymph nodes. The researchers defined breast cancer recurrence as the first appearance of any breast cancer including, cancer in the same breast, cancer in the opposite breast, or distant spread of cancer.[6]

Breast cancer deaths

Among women who were 55-69 years old at the time of diagnosis, about 90% who did not have a breast cancer recurrence were alive for at least 10 years, regardless of whether they took tamoxifen or not. For women in that age group, about 16% of women who took tamoxifen and had a recurrence died from breast cancer within 10 years of the initial diagnosis, compared to 26% of women who did not take tamoxifen and had a recurrence.[6]

Among women who were 70 years old or more at the time of their diagnosis and did not have a breast cancer recurrence, 80% who took tamoxifen were alive 10 years later, compared to 70% of women who did not take tamoxifen. Importantly, this difference was not statistically significant, meaning it could have happened by chance and is unlikely to be related to tamoxifen therapy.  Among women in this age group who had a breast cancer recurrence, about 20% who took tamoxifen and had a recurrence died from breast cancer within 10 years of the initial diagnosis compared to 37% of women who did not take tamoxifen and had a recurrence. [6]

Living Longer

Most women who are diagnosed with breast cancer do not die from breast cancer.  Of course, eventually they die of something else.  For women who were 55-69 years old at the time of their diagnosis, 5 years of tamoxifen helped women live longer.  More than 90% of women who did not have a breast cancer recurrence were alive 10 years later, regardless of whether they took tamoxifen or not. In contrast, about 76% of women who took tamoxifen and had a breast cancer recurrence were alive 10 years after the initial diagnosis, compared to 67% of women who did not take tamoxifen.[6]

By the time women are over 70, they are likely to have health issues whether or not they are diagnosed with breast cancer. Among those who did not have a breast cancer recurrence, about 80% of women taking tamoxifen and 70% of women not taking tamoxifen were alive 10 years later.  However, this apparent 10% difference was not statistically significant and therefore is probably not related to tamoxifen. In contrast, among women who had a recurrence, about 67% of women who took tamoxifen were alive 10 years later compared to 47% of women who did not take tamoxifen.[6]

As you can see, the benefits of tamoxifen depend on how old and healthy a woman is when she is diagnosed In addition, certain characteristics of early-stage breast cancers, including size of the tumor, types of cancer cells, and how many lymph nodes the cancer had spread to prior to surgery, can help doctors predict the chances of breast cancer recurrence. Therefore, it is important for you to talk with your doctor about these specific issues and which treatment options may be right for you.  Remember that some benefits (such as survival) might be more important to you than others (such as recurrence) – or not!

Aromatase Inhibitors:

Aromatase inhibitors have also been shown to benefit post-menopausal breast cancer patients.  An international breast cancer study showed that about 84% of women taking letrozole were alive and cancer free at 5 years compared to about 81% of women taking tamoxifen. Despite the difference in dying from breast cancer, however, there was no difference in terms of the percentage of women who were alive 5 years after diagnosis.

The benefits of aromatase inhibitors continued 10 years after diagnosis, with about 19% of women who took letrozole having any cancer recurrence compared to about 23% of women who took tamoxifen. Breast cancer recurrence included any breast cancer in the same breast, the opposite breast, or distant spread of cancer.[3]

When considering your treatment options, talk with your doctor about your overall health and your heart health, because all women (including women with breast cancer) are more likely to die from heart disease than breast cancer. And some treatments for breast cancer can harm your heart. Read more about heart health and breast cancer in our article.

Treatment Considerations

There are many ways to take hormone therapy, including switching between the types of therapy, and taking the hormone therapy for additional years. The choices can be confusing. Here are some questions to think about: 

What are the benefits of extending therapy? Studies show that extending therapy after an initial 5-year period can lower a woman’s chances of cancer recurrence (including any breast cancer in the same breast, the opposite breast, or distant spread) by a small, but statistically significant 2% to 4%. The study looked at different hormone regimens including “primary” AI (AI for 5 years), “sequential” AI (tamoxifen for 2 years followed by 3 years of AI), and extended AI (5 years of AI after 5 years of tamoxifen). For example, about 7% to 12% of women who took any hormone therapy for 5 years had a recurrence compared to 5% to 8% of women who extended their therapy.[10]

For how long should women extend therapy? A 2018 study found that women adding on 2.5 extra years of letrozole had the same chances of surviving without their breast cancer returning as women adding letrozole for 5 years. However, about 1% of women who took letrozole for an extra 5 years had a new breast cancer develop in the opposite breast compared to 3% in the 2.5 year group. That difference is small but it is a way to lower your already small risk of developing a new cancer in your other breast and making it even smaller.[11,12]

As you consider your treatment options,  it is important to consider the risks as well, as described later in the next section of this article.

Side Effects and Risks of Treatment

Tamoxifen increases the chances of a woman developing endometrial cancer and blood clots in legs and lungs, especially for women over 50 years of age.[3,16] Women taking tamoxifen are also more likely to develop endometrial cancer, although the overall risk is still low; about 2 women out of 1,000 taking tamoxifen will get endometrial cancer each year.[16] In a Danish study, the 5-year risk of developing blood clots was about 1.2% in women with breast cancer taking tamoxifen compared to 0.5% in women with breast cancer who were not taking tamoxifen. Moreover, tamoxifen therapy often causes side effects similar to those experienced in menopause, including hot flashes and irregular periods.[16] In one study, 41% of women taking tamoxifen experienced hot flashes, and 10% experienced abnormal vaginal bleeding.[19]

Aromatase inhibitors increase the risk for osteoporosis compared with tamoxifen or taking no hormonal therapy at all.  Exemestane, a commonly used aromatase inhibitor (brand name Aromasin), also increases the risk for visual disturbances, allergic reaction, or diarrhea.  In one study, about 36% of women experienced joint problems while taking anastrozole, (brand name Arimidex) compared to about 30% of women taking tamoxifen.

The most common complications from hormonal therapy are listed above.  In addition, in very rare cases, other side effects of hormonal therapy can be fatal or can harm a patient’s quality of life.  Close monitoring of women for symptoms, such as abnormal uterine bleeding, is needed, and women taking tamoxifen should receive annual pelvic exams.[3]

Different women respond differently to the various forms of hormonal therapy, which is why it is not uncommon for women to switch to different hormonal treatments after starting.

The Bottom Line

There are many ways to treat early-stage breast cancer in post-menopausal women, in addition to surgery. A woman’s age, tumor characteristics, overall health, and personal wishes/goals may impact her decision. Talk with your doctor about which treatment options may be right for you by asking about the exact benefits of specific treatments on recurrence and overall survival, and considering these specific issues and not just what is best for cancer patients on average.

Footnotes:

  1. American Cancer Society. Cancer Treatment and Survivorship: Facts and Figures 2016-2017. Available online: https://www.cancer.org
  2. Burstein HJ. et al. Adjuvant Endocrine Therapy for Women With Hormone Receptor–Positive Breast Cancer: American Society of Clinical Oncology Clinical Practice Guideline Update on Ovarian Suppression. Journal of Clinical Oncology. 2017;34(14): 1689-1701. Doi: 1200/JCO.2015.65.9573
  3. National Cancer Institute. Breast Cancer Treatment (PDQ). (Nov. 2017). Available online: https://www.cancer.gov/types/breast/patient/breast-treatment-pdq#section/_125
  4. Adjuvant Therapy for Breast Cancer. (Aug. 2017). Available online: https://emedicine.medscape.com/article/1946040-overview#showall
  5. Colleoni M. et al. Annual Hazard Rates of Recurrence for Breast Cancer During 24 Years of Follow-Up: Results From the International Breast Cancer Study Group Trials I to V. J Clin Oncol. 2016;34(9): 927-935. doi: 1200/JCO.2015.62.3504
  6. Early Breast Cancer Trialists’ Collaborative Group (EBCTCG). Relevance of breast cancer hormone receptors and other factors to the efficacy of adjuvant tamoxifen: patient-level meta-analysis of randomised trials. Lancet. 2011;378(9793): 771-784. doi:1016/S0140-6736(11)60993-8
  7. Gierach GL, Curtis RE, Pfeiffer RM, et al. Association of Adjuvant Tamoxifen and Aromatase Inhibitor Therapy With Contralateral Breast Cancer Risk Among US Women With Breast Cancer in a General Community Setting. JAMA Oncol. 2017;3(2): 186–193. doi:1001/jamaoncol.2016.3340
  8. Early Breast Cancer Trialists’ Collaborative Group (EBCTCG). Aromatase inhibitors versus tamoxifen in early breast cancer: patient-level meta-analysis of the randomised trials. Lancet. 2015;386(10001): 1341 – 1352. doi: http://dx.doi.org/10.1016/S0140-6736(15)61074-1
  9. Pohlmann PR and Isaacs C. Extended Adjuvant Endocrine Therapy for Postmenopausal Women: Treating Many to Benefit a Few, JNCI: Journal of the National Cancer Institute. 2018;110(1): djx142, doi: https://doi.org/10.1093/jnci/djx142
  10. Burstein HJ, Prestrud AA, Seidenfeld J, et al. American Society of Clinical Oncology Clinical Practice Guideline: Update on Adjuvant Endocrine Therapy for Women With Hormone Receptor–Positive Breast Cancer. Journal of Clinical Oncology. 2010;28(23):3784-3796. doi:10.1200/JCO.2009.26.3756.
  11. Blok EJ, et al. Optimal Duration of Extended Adjuvant Endocrine Therapy for Early Breast Cancer; Results of the IDEAL Trial (BOOG 2006-05). JNCI: Journal of the National Cancer Institute. 2018; 110(1): djx134, https://doi.org/10.1093/jnci/djx134
  12. Van de Velde, C.J.H. et al. Optimal duration of extended letrozole treatment after 5 years of adjuvant endocrine therapy; results of the randomized phase III IDEAL trial (BOOG 2006–05). European Journal of Cancer. 2017;72(Supp1):S9. doi: http://dx.doi.org/10.1016/S0959-8049(17)30108-9
  13. Gianni L. et al. Treatment with trastuzumab for 1 year after adjuvant chemotherapy in patients with HER2-positive early breast cancer: a 4-year follow-up of a randomised controlled trial. Lancet Oncology. 2011;12(3): 236-44. doi: https://doi.org/10.1016/S1470-2045(11)70033-X
  14. Cameron D. et al. 11 years’ follow-up of trastuzumab after adjuvant chemotherapy in HER2-positive early breast cancer: final analysis of the HERceptin Adjuvant (HERA) trial. Lancet. 2017;389(10075): 1195-1205. doi: https://doi.org/10.1016/S0140-6736(16)32616-2
  15. Stenger M. ASCO Post: 11-Year Follow-up of Adjuvant Trastuzumab in the HERA Trial. (March 2017). Available online: http://www.ascopost.com/News/48405
  16. Gogas H, Markopoulos C, Blamey R. Should women be advised to take prophylactic endocrine treatment outside of a clinical trial setting? Ann Oncol. 2005;16:1861-1866. Available online: https://watermark.silverchair.com
  17. Fisher B, et al. J Natl Cancer Inst. 1994; 86:527-537.
  18. Bonneterre, et al. J Clin Oncol. 2000; 18:3748-3757.
  19. Howell A, et al. Results of the ATAC (Arimidex, Tamoxifen, Alone or in Combination) trial after completion of 5 years’ adjuvant treatment for breast cancer. Lancet. 2005;365(9453): 60-2. doi: 1016/S0140-6736(04)17666-6
  20. Bliss JM. et al. Disease-Related Outcomes With Long-Term Follow-Up: An Updated Analysis of the Intergroup Exemestane Study. Journal of Clinical Oncology. 2012;30(7): 709-717. doi: 1200/JCO.2010.33.7899
  21. Drugs and Diseases: Gosarelin. Available online: https://reference.medscape.com/drug/zoladex-la-goserelin-342129
  22. Drugs and Diseases: Trastuzumab. Available online: https://reference.medscape.com/drug/herceptin-ogivri-trastuzumab-342231#5

Statement of National Center for Health Research Supporting A CDC Review to Save Women’s Lives


The National Center for Health Research strongly supports the announcement that the Centers for Disease Control and Prevention may conduct a review of the potential benefits of biopsies for women scheduled for hysterectomy or fibroid removal.  The review is essential because a recent article, published in the medical journal Obstetrics & Gynecology reports that the rate of unsuspected cancer is dangerously high in women undergoing hysterectomy.  When those women undergo surgery, particularly procedures involving a medical device called a power morcellator, the cancer can spread inside the woman’s abdomen, resulting in an early-stage cancer being upstaged to a much more dangerous metastatic (stage 4) cancer.  The risks are especially high for women ages 55 and older, reaching almost 10% for undiagnosed uterine cancer.

The study authors, from Yale Medical School and the Columbia University College of Physicians and Surgeons, studied more than 26,000 women who underwent hysterectomy or fibroid removal, and found that undetected cancers were much higher than previously estimated.  The implications are that the FDA’s warnings regarding the risk of power morcellation, which estimated one hidden cancer in every 352 women, are inadequate to provide informed consent for patients considering these surgical procedures.

We thank Dr. Redfield for his attention to this matter and urge the CDC leadership to move forward as soon as possible to develop guidelines pertaining to tissue biopsy methods that will identify women with gynecological cancer before the women undergo a surgical procedure to remove the uterus or fibroid.  Such testing could save thousands of women’s lives.

Diana Zuckerman, PhD
President
See our related letter to CDC Dirtector Dr. Robert Redfield here.

Letter to Board of Education of Montgomery County on Synthetic Playgrounds

Diana Zuckerman, PhD, National Center for Health Research, April 23, 2018

April 23, 2018

Dear Board of Education members,

As a long-time resident of Montgomery County and as president of the National Center for Health Research, I am shocked by the misinformation I have seen circulating about all types of artificial turf and rubber playgrounds in Montgomery County.

The recent plan to use Zeolite indicates a dangerous lack of understanding of the need for safety testing before using materials that our children will be exposed to day after day.  Zeolite is a mined volcanic mineral.  Its use in artificial turf has not been tested for human health,  but there is every reason to be concerned that inhaling the dust from Zeolite could cause lung damage, similar to the damage from asbestos.

In addition, it is my understanding that synthetic rubber play surfaces are now being proposed for MCPS outdoor play spaces.  These “poured in place” and other types of rubber playground surfaces have many of the same risks as tire crumb and other rubber infill that the County Council realized were too dangerous to use in 2015.  These rubber playgrounds look very attractive and feel good when they are new, but you can’t see the chemicals that are in them.  As a result, these synthetic rubber playgrounds (like artificial turf playing fields) are often over 140-160 degrees Fahrenheit 50-60 degrees on a sunny day – even on days when the air temperature is only 60 degrees!.  In addition, the rubber (which is made from petroleum and other products) release toxins that can cause cancer and also contribute to early puberty, obesity, attention deficit disorder, asthma, and rashes.  The playground surfaces also start to tear, and very small pieces of colorful materials are very tempting for young children to eat because they look like candy.

Please stop these dangerous installations from going forward.

As president of the National Center for Health Research, I testified about the risks of these materials at the U.S. Consumer Product Safety Commission this past week, and have previously testified before the Maryland House of Delegates Appropriations Committee  and the D.C. City Council.  I am sorry to say that I have repeatedly seen and heard scientists from the turf industry and other turf industry lobbyists say things that are absolutely false.  They claim that these products are proven safe (not true) and that federal agencies have stated there are no health risks (also not true).  They also give misleading assurances such as “there is no evidence of a child getting cancer from these products.” The problem with that type of statement is that it is virtually impossible to prove that a chemical exposure causes one specific individual to develop cancer.  What we do know is that many of the materials being used contain carcinogens, and when children are exposed to those carcinogens day after day, week after week, and year after year, they increase the chances of our children developing cancer, either now or as they get older.  That should be adequate reason to not install them in Montgomery County.  Add to that the short-term risk of asthma, obesity, attention problems, and early puberty, and it is time  for the County to educate its opinion leaders and stop spending millions of dollars on fields and playgrounds that are less safe and more expensive than well designed natural grass fields and ADA-compliant engineered wood fiber.

For more information, please read our user-friendly footnoted summary at http://www.center4research.org/children-athletes-play-toxic-turf-playgrounds/

Sincerely,

Diana Zuckerman, Ph.D.
President
National Center for Health Research

2018 Foremother and Health Policy Hero Awards Luncheon

National Center for Health Research, May 4, 2018

Friday May 4 is our Annual Awards luncheon at the Mayflower Hotel!

Every year, we take time off from our research and public education to thank women and men who have improved our lives.

The Foremother Lifetime Achievement Award recognizes women who expanded women’s horizons, improved our communities, and made remarkable contributions to our country.  We let them know what an honor it is to follow in their formidable footsteps.

We also recognize Health Policy Heroes. This award honors men and women (and in this case, boys and girls) who have changed the public debate and public policies in ways that help to improve the lives of adults and children nationwide.

 

Please join our wonderful Emcee Maureen Bunyan as we celebrate

the National Center for Health Research’s

2018 Foremothers Lifetime Achievement and Health Policy Heroes Awards Luncheon

Friday, May 4, 2018 at Noon
The Mayflower Hotel
1127 Connecticut Ave NW
Washington, DC 20036
~~~
We hope you will join us at the elegant Mayflower Hotel in Washington, D.C. as we celebrate these inspiring honorees.

Help us celebrate the amazing women who are our 2018 Foremother Lifetime Achievement honorees for careers that made all our lives better and broke down barriers for other women:

Dr. Rita Colwell is an extraordinary scientist whose work has successfully fought cholera and created safer water supplies around the world, saving lives while breaking down many barriers for women in science. She was the first woman to serve as Director of the National Science Foundation (NSF), presiding over a doubling of the NSF budget. She has won numerous other scientific awards over more than 40 years, including the National Medal of Science presented by then-President George W. Bush, and the Medal of Distinction from Columbia University. She previously served as the President of the American Association for the Advancement of Science (AAAS) and is a member of the prestigious National Academy of Sciences (NAS).

Lynn Povich is an award-winning journalist who began her career as a secretary at Newsweek magazine. She was one of 46 women who filed sex discrimination charges against the magazine in 1970, an experience that 40+ years later inspired her book THE GOOD GIRLS REVOLT, and the Amazon TV series that was soon followed by #MeToo. The law suit was groundbreaking, and she subsequently became the first woman appointed Senior Editor at Newsweek. Ms. Povich later became Editor-in-Chief of Working Woman magazine and then joined MSNBC.com as East Coast Managing Editor. She also edited a book of columns by her father, famous sports writer Shirley Povich.  She serves on the Advisory Boards of the International Women’s Media Foundation, the Women’s Rights Division of Human Rights Watch, and the CUNY Graduate Center Foundation Board.

Our Health Policy Heroes are the students, teachers, and parents of Parkland, Florida; Washington, D.C. metro area; and across the country who are successfully fighting for effective policies to prevent gun violence. MSD High School student Kai Koerber and Parkland teacher Susan Rioux will be two of the heroes accepting the award on their behalf. Kai has been actively lobbying for better gun policies since the tragic shooting on February 14. Ms. Rioux is credited with having taught Parkland students how to find safety in violent situations, thus saving their lives during the February 14 shooting. She is now one of those helping the children cope with PTSD and fear in the aftermath and supporting their efforts to reduce gun violence. March for Our Lives Lodging, a group of mothers who organized free housing, meals, and other necessities for thousands of students and families traveling to D.C., will also be honored.

~~~

We hope you will take advantage of this great opportunity to meet these inspiring women, previous honorees, and many of D.C.’s other movers and shakers. Lunch is from noon to 1:30, preceded by a 11:30 champagne reception for honorees, patron guests, and sponsors only.

Prices below are valid through April 28.

Seats are limited and tickets are not available at the door.

Regular lunch tickets are available for a donation of $110 each. Patron Tickets ($175 per ticket) include a champagne reception with honorees at 11:30, priority seating, and a listing in the program. A Patron table for 10 is $1,750. Sponsorships are also available, from $1,800-$6,000.

The National Center for Health Research is the leading national organization dedicated to improving the health and safety of all adults and children. Donations for this event support our Cancer Prevention and Treatment Fund helpline.

To reserve a ticket, you may donate online here. Or, send a check payable to “NCHR,” to 1001 Connecticut Ave, Suite 1100, Washington, DC 20036.Be sure to indicate it is for the Awards Luncheon.

For more info, contact Alex Pew at ap@center4research.org or (202)223-4000.

US Regulators Float Ideas for Boosting Medical Device Safety

Matthew Perrone, The Associated Press: April 17, 2018

U.S. health officials on Tuesday proposed steps to improve the government’s system for overseeing medical devices, which has been criticized for years for failing to catch problems with risky implants and medical instruments.

The plan from the Food and Drug Administration includes few immediate changes, but lists a number of ideas and proposals with the goal of improving safeguards on pacemakers, artificial joints, medical scanners and other devices.

Among other measures, the FDA will consider requiring more training for doctors who implant certain high-risk devices. But that step, like others floated by the agency, might require new guidelines or regulations. Other proposals may require additional money from Congress.

The FDA has repeatedly been forced to issue safety alerts about unexpected problems with devices that only appeared years after they were approved for use in patients. In the last decade, those have included hip replacements that failed prematurely, faulty wiring in implanted defibrillators, surgical mesh linked to pain and bleeding and a surgical instrument that inadvertently spread uterine cancer.

“We want to have better tools for detecting issues that occur post-approval,” FDA Commissioner Scott Gottlieb said Tuesday. “But we also want to have better policies to quickly intervene and better inform patients and providers if we see adverse events happening.”

An agency critic said the report contains few concrete changes and “many sections that will please the device industry.”

“FDA’s safety strategies for medical devices are still years away from effective implementation,” said Diana Zuckerman, president of the National Center for Health Research, a consumer advocacy group. “Overall, the report indicates that the FDA’s approval standards for medical devices remain completely inadequate.” […]

Among other proposals laid out in the FDA’s “Medical Device Safety Action Plan,” the FDA will consider:

— How to quickly require additional safety requirements for certain devices, including training for doctors who work with the complex devices.

— Extra scrutiny of devices for women, following recent problems with vaginal mesh, the birth control implant Essure and surgical tools.

— New ways to encourage manufacturers to improve safety, including quicker approval for devices that appear safer than what’s available.

— Requiring cybersecurity features for electronic devices like implantable heart pacemakers and defibrillators.

The agency will also ask Congress for more money for a public-private system intended to monitor insurance claims, electronic health records and other data sources for early signs of device problems. The project is estimated to cost $250 million over five years to become operational; it is now slated to receive $30 million from device manufacturers.

Read the original article here.

Comments by Diana Zuckerman, Ph.D. on the U.S. Consumer Product Safety Commission Agenda and Priorities for FY2019/2020

Diana Zuckerman, PhD, National Center for Health Research,April 11, 2018

The National Center for Health Research is a nonprofit research center staffed by scientists, medical professionals, and health experts who analyze and review research on a range of health issues. We conduct studies, we scrutinize research done by others, and we try to make sense of conflicting research findings.  Our goal is to explain that information so it can be used to improve policies, programs, services, and products.  Thank you for the opportunity to share our views concerning the Consumer Product Safety Commission’s (CPSC) priorities for fiscal year 2019 and 2020. We respect the essential role of the CPSC, as well as the challenges you face in selecting the most important priorities.

I’m trained as an epidemiologist at Yale Medical School, and I was on the faculty at Yale and Vassar and a researcher at Harvard before moving to Washington, D.C. as a Congressional Fellow in the program sponsored by the American Association of the Advancement of Science (AAAS).  While our Center’s mission overlaps with much of the work of the CPSC, today I will talk as a scientist about safety risks that you don’t hear as much about – the ones that we can’t see.

We are surrounded by chemicals in the air we breathe, the table in front of me, and the dust in the room.  Today I will focus on three issues involving chemicals in products that affect our health and our children’s health. These issues are clearly consistent with the CPSC priorities. We are very concerned about flame retardants and phthalates, both of which migrate out of products and into the dust we breathe and touch. We’re also very concerned about artificial turf fields and playgrounds, which contain a range of endocrine-disrupting chemicals and other toxic materials that can harm children’s development and possibly increase risk for cancer as these children grow up.

Organohalogen Flame Retardants

Thank you for voting to initiate rulemaking on non-polymeric organohalogen flame retardants (OFRs) and to provide guidance for manufacturers, distributors, and retailers to avoid OFRs.1 We urge you to convene a Chronic Hazard Advisory Panel (CHAP) as soon as possible and develop regulations to address OFRs in children’s products, upholstered residential furniture, mattresses/mattress pads, and the plastic casing of electronic devices. In addition, it is essential to consider current flammability standards to determine if there are changes that would improve their safety from both chemical exposures and potential fire.

Since OFRs are not bound to products, they migrate out of products and into dust, and thus get onto our skin and food as well as into the air we breathe. Because so many products are made with these chemicals and because they are so long-lasting, consumers are repeatedly exposed day after day.2,3 In addition, many OFRs bioaccumulate in our food supply.4,5,6,7 As a result, nearly all people in the U.S. have OFRs in their bodies. 8

OFRs have been associated with various health problems, including disrupting hormones, altering brain development, and harming reproductive health, such as reduced ability to get and stay pregnant and the timing of puberty.5,9,10,11 While not all OFRs have been sufficiently studied to determine whether all are unsafe, those that have been sufficiently studied have proved to be harmful to health.

While we recognize that the Commission must be concerned about fire hazards as well, it seems that these flame retardants may not be effective at preventing deaths in real world situations.12,13 When the chemicals burn during a fire, the inhaled smoke is more toxic to humans, and exposures could result in serious harms, including death.

Artificial Turf and Playground Surfaces

We appreciate the CPSC’s ongoing efforts to investigate the safety of crumb rubber on playgrounds and playing fields. This requires your immediate attention, because artificial turf fields are becoming increasingly popular surfaces for fields and playgrounds where children are exposed day after day, year after year. And yet, the materials used are often treated as “trade secrets” making it impossible to know exactly what they are, which ones are safer, and which ones are more dangerous. We encourage you to closely evaluate the research that has been done, focusing on independently funded research rather than industry claims. We also urge you to carefully examine the EPA/CDCs studies when they are completed, and to develop rules that will protect our children from harm. We urge you to convene a Chronic Hazard Advisory Panel (CHAP) to examine the short-term and long-term risks of different types of artificial turf used in playing fields and children’s playgrounds.

Crumb rubber contains chemicals with known health concerns, which are released into the air and onto skin and clothing and even into children’s ears and noses. This is inevitable for a product that is outdoors and in constant use. The chemicals include endocrine disruptors such as phthalates, heavy metals such as lead and zinc, as well as other carcinogens and skin irritants such as some polycyclic aromatic hydrocarbons (PAHs) and volatile organic compounds (VOCs).14,15,16,17,18,19 While one time or sporadic exposures are unlikely to cause long-term harm, children’s repeated exposures over the years, especially during critical developmental periods, raise the likelihood of serious harm.

These fields can also cause short-term harms. Artificial turf generates dust which may exacerbate children’s asthma.20,21 Fields heat up to temperatures far higher than ambient temperature, reaching temperatures that are more than 70 degrees warmer than nearby grass; for example, 180 degrees when the temperature is in the high 90’s and 150-170 degrees on a sunny day when the air temperature is only in the 70’s. 22,23,24 This can cause heat stress and burns.

Fields made of crumb rubber have been marketed as reducing injuries compared to grass. However, research has shown that this is not the case. We have spoken to students harmed by turf burn, and some studies have indicated increased risk for joint injuries and brain injury.25,26

We need to know more about the risks of “virgin rubber” compared to “recycled tires.” However, we already know that “virgin” rubber is made from many of the same chemicals that have these health concerns.27,28

Phthalates in Children’s and Household Products

CPSC has helped millions of American children by finalizing the phthalate rule to ban five additional phthalates (DINP, DPENP DHEXP, DCHP, DIBP) in children’s toys and care products.

The next priority should be for CPSC to expand its work on phthalates to include other household products. Children are exposed to many products with the same phthalates as those that are banned in toys and products specifically for children. Restricting the use of phthalates in common household products would reduce exposure for young children and also older children, pregnant women and other adults. Phthalates in household dust can be harmful regardless of what products it comes from and prenatal exposure is of particular concern.

Phthalate exposure has been associated with an increased risk for early puberty and reproductive problems.29,30,31 In utero exposure or exposure through breast milk puts the developing fetus, neonate, or infant at serious risk of abnormal neurological and reproductive development.32

In conclusion, endocrine disruptors and chemicals in common consumer products that do not stay bound to those products get into the air and dust and thus into our bodies. These chemicals tend to pose greater risks to fetuses and children. There are large gaps in our knowledge about the chemicals in the products on the market. Ideally, all of these chemicals would be evaluated in the final product for health concerns before it was sold. Since that is not happening, we must constantly play catch-up as health concerns are identified. Too often this leads to cases of false claims regarding the safety of new products that we later learn are as harmful or even more harmful that the ones they are replacing. While research is lacking regarding the exact extent of the dangers of many of these products, there is already sufficient evidence to cause concern. We need CPSC to address those as soon as possible.

References:

  1. Consumer Product Safety Commission. (2017) Guidance document on hazardous additive, non-polymeric organohalogen flame retardants in certain consumer products. https://www.federalregister.gov/documents/2017/09/28/2017-20733/guidance-document-on-hazardous-additive-non-polymeric-organohalogen-flame-retardants-in-certain
  2. Gramatica P, Cassani S, Sangion A. (2016) Are some “safer alternatives” hazardous as PBTs? The case study of new flame retardants. J Hazard Mater. 306:237-246.
  3. Allgood JM, Vahid KS, Jeeva K, Tang IW, Ogunseitan OA. (2017) Spatiotemporal analysis of human exposure to halogenated flame retardant chemicals. Sci Total Environ. 609:272-276.
  4. Lupton SJ, Hakk H. (2017) Polybrominated diphenyl ethers (PBDEs) in US meat and poultry: 2012-13 levels, trends and estimated consumer exposures. Food Addit Contam Part A Chem Anal Control Expo Risk Assess. 34(9):1584-1595.
  5. Lyche JL, Rosseland C, Berge G, Polder A. (2014) Human health risk associated with brominated flame-retardants (BFRs). Environ Int. 74:170-180.
  6. Schecter A, Colacino J, Patel K, Kannan K, Yun SH, Haffner D, Harris TR, Birnbaum L. (2010) Polybrominated diphenyl ether levels in foodstuffs collected from three locations from the United States. Toxicol Appl Pharmacol. 243(2):217-24.
  7. Widelka M, Lydy MJ, Wu Y, Chen D. (2016) Statewide surveillance of halogenated flame retardants in fish in Illinois, USA. Environ Pollut. 214:627-634.
  8. Centers for Disease Control and Prevention (2015) Fourth national report on human exposure to environmental chemicals, updated tables. http:/www.cdc.gov/exposurereport/.
  9. Dishaw L, Macaulay L, Roberts SC, Stapleton HM. (2014) Exposures, mechanisms, and impacts of endocrine-active flame retardants. Curr Opin Pharmacol. 0:125-133.
  10. Hendriks HS, Westerink RHS. (2015) Neurotoxicity and risk assessment of brominated and alternative flame retardants. Neurotoxicol Teratol. 52:248-269.
  11. Kim YR, Harden FA, Toms LM, Norman RE. (2014) Health consequences of exposure to brominated flame retardants: A systematic review. Chemosphere 106:1-19.
  12. McKenna S, Birtles R, Dickens K, Walker R, Spearpoint M, Stec AA, Hull TR. (2018) Flame retardants in UK furniture increase smoke toxicity more than they reduce fire growth rate. Chemosphere. 196:429-439.
  13. Shaw SD, Blum A, Weber R, Kannan K, Rich D, Lucas D, Koshland CP, Dobraca D, Hanson S, Birnbaum LS. (2010) Halogenated flame retardants: Do the fire safety benefits justify the risks? Rev Environ Health 25:261-305.
  14. Llompart M, Sanchez-Prado L, Lamas JP, Garcia-Jares C, et al. (2013) Hazardous organic chemicals in rubber recycled tire playgrounds and pavers. Chemosphere. 90(2):423-431.
  15. Marsili L, Coppola D, Bianchi N, Maltese S, Bianchi M, Fossi MC. (2014) Release of polycyclic aromatic hydrocarbons and heavy metals from rubber crumb in synthetic turf fields: Preliminary hazard assessment for athletes. Journal of Environmental and Analytical Toxicology. 5:(2).
  16. California Office of Environmental Health Hazard Assessment (OEHHA). (2007) Evaluation of health effects of recycled waste wires in playground and track products. Prepared for the California Integrated Waste Management Board. http://www.calrecycle.ca.gov/publications/Detail.aspx?PublicationID=1206
  17. Kim S, Yang J-Y, Kim H-H, Yeo I-Y, Shin D-C, and Lim Y-W. (2012) Health risk assessment of lead ingestion exposure by particle sizes in crumb rubber on artificial turf considering bioavailability. Environmental Health and Toxicology. 27, e2012005. http://doi.org/10.5620/eht.2012.27.e2012005
  18. S. National Library of Medicine, National Institutes of Health. (2017) Tox Town (Environmental health concerns and toxic chemicals where you live, work, and play): Polycyclic aromatic hydrocarbons (PAHs). https://toxtown.nlm.nih.gov/text_version/chemicals.php?id=80
  19. Armstrong B, Hutchinson E, Unwin J, and Fletcher T. (2004) Lung cancer risk after exposure to polycyclic aromatic hydrocarbons: a review and meta-analysis. Environmental Health Perspectives, 112(9), 970.
  20. Shalat SL. (2011) An evaluation of potential exposures to lead and other metals as the result of aerosolized particulate matter from artificial turf playing fields. Submitted to the New Jersey Department of Environmental Protection. http://www.nj.gov/dep/dsr/publications/artificial-turf-report.pdf
  21. Mount Sinai Children’s Environmental Health Center. (2017) Artificial turf: A health-based consumer guide. http://icahn.mssm.edu/files/ISMMS/Assets/Departments/Environmental%20Medicine%20and%20Public%20Health/CEHC%20Consumer%20Guide%20to%20Artificial%20Turf%20May%202017.pdf
  22. Thoms AW, Brosnana JT, Zidekb JM, Sorochana JC. (2014) Models for predicting surface temperatures on synthetic turf playing surfaces. Procedia Engineering. 72:895-900. http://www.sciencedirect.com/science/article/pii/S1877705814006699
  23. Penn State’s Center for Sports Surface Research. (2012) Synthetic turf heat evaluation- progress report. http://plantscience.psu.edu/research/centers/ssrc/documents/heat-progress-report.pdf
  24. Serensits TJ, McNitt AS, Petrunak DM. (2011) Human health issues on synthetic turf in the USA. Proceedings of the Institution of Mechanical Engineers, Part P: Journal of Sports Engineering and Technology. 225(3), 139-146.
  25. Balazs GC, Pavey GJ, Brelin AM, Pickett A, Keblish DJ, Rue JP. (2015) Risk of anterior cruciate ligament injury in athletes on synthetic playing surfaces: A systematic review. American Journal of Sports Medicine. 43(7):1798-804.
  26. Theobald P, Whitelegg L, Nokes LD, Jones MD. (2010) The predicted risk of head injury from fall-related impacts on to third-generation artificial turf and grass soccer surfaces: a comparative biomechanical analysis. Sports Biomechanics. 9(1):29-37.
  27. Canepari S, Castellano P, Astolfi ML, Materazzi S, Ferrante R, Fiorini D, Curini R. (2018) Release of particles, organic compounds, and metals from crumb rubber used in synthetic turf under chemical and physical stress. Environ Sci Pollut Res Int. 25(2):1448-1459.
  28. Kim S, Yang JY, Kim HH, Yeo IY, Shin DC, Lim YW. (2012) Health risk assessment of lead ingestion exposure by particle sizes in crumb rubber on artificial turf considering bioavailability. Environ Health Toxicol. 27:e2012005. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3278598/
  29. Chen Q, Yang H, Zhou N, Sun L, et al. (2017) Phthalate exposure, even below US EPA reference doses, was associated with semen quality and reproductive hormones: Prospective MARHCS study in general population. Environ Int. 104:58-68.
  30. Mariana M, Feiteiro J, Verde I, Cairrao E. (2016) The effects of phthalates in the cardiovascular and reproductive systems: A review. Environ Int. 94:758-776.
  31. Yi Wen, Shu-Dan Liu, Xun Lei, Yu-Shuang Ling, Yan Luo, and Qin Liu.(2015) Association of PAEs with precocious puberty in children: A systematic review and meta-analysis. Int J Environ Res Public Health. 12(12): 15254–15268. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4690910/
  32. Consumer Product Safety Commission. (2014) Chronic Hazard Advisory Panel on Phthalates and Phthalate Alternatives. https://www.cpsc.gov/PageFiles/169876/CHAP-REPORT-FINAL.pdf

CMS Payment Rule Seen as Bad for Some Patients

Joyce Frieden, MedPage Today, April 10, 2018

New final regulations on the Affordable Care Act (ACA) health insurance exchanges issued by the Centers for Medicare & Medicaid Services (CMS) have drawn mixed reactions from health policy experts and others.

The rule makes a number of changes to the exchanges, including:

  • Expanding the number of “benchmark” plans from which states can choose to model their coverage of the 10 “essential health benefit” (EHB) categories included in the ACA, potentially allowing states to choose plans with more generous or skimpier coverage than is currently offered on their exchanges.
  • Adding several new “hardship exemptions” to allow consumers to avoid paying a penalty for not buying health insurance. One exemption is for consumers who live in an area in which there are no health plans offered for them on the exchange, or only a single plan offered which is unaffordable. Another exemption is for consumers who live in an area in which the only health plans offered provide coverage of abortions, in cases where that conflicts with the consumer’s personal beliefs.
  • Allowing states to adjust the “medical loss ratio,” which determines what percentage of a health insurer’s revenue must be used for paying healthcare costs. Currently, according to the ACA, health insurers must spend at least 80% of their revenue on healthcare claims and quality improvement, with the rest going toward overhead and profit.
  • Increasing the percentage premium increase which requires review by insurance regulators. Under current ACA rules, review is triggered if an insurer requests to increase rates by an average of 10% or more; the new regulations increase that threshold to 15%.

“The final rule will mitigate the harmful impacts of Obamacare and empower states to regulate their insurance market,” CMS said Monday in a press release on the regulations. “The rule will do this by advancing the Administration’s goals to increase state flexibility, improve affordability, strengthen program integrity, empower consumers, promote stability, and reduce unnecessary regulatory burdens imposed by the Patient Protection and Affordable Care Act.” The release also asserted that the ACA “has led to higher premiums and fewer choices” and that the ACA “has priced many consumers out of the insurance market.”

Premium Increases for Comprehensive Plans

“The plan to allow the sale of policies with skimpier essential health benefits will inevitably cause premiums for good health insurance policies (the kind currently available through the ACA) to increase,” Diana Zuckerman, PhD, president of the National Center for Health Research, an organization that conducts, analyzes, and explains health-related research, wrote in an email.

“If very healthy people can buy skimpy health insurance policies, then people who know that they have health problems will be the only ones buying the better policies — resulting in an increase in costs. In other words, people with pre-existing health conditions such as cancer, heart disease, diabetes, and rare diseases, will be paying much more than anyone else — an outcome that most Americans do not want. The bottom line is that the result of these regulations will be exactly the opposite of the stated goal: rather than making healthcare more affordable, this would make health care much less affordable for the people who need it most.” […]

Read the original article here.

Patient Advocacy Groups Take In Millions From Drugmakers. Is There A Payback?

Emily Kopp, Sydney Lupkin, and Elizabeth Lucas, Kaiser Health News: April 6, 2018

Pharmaceutical companies gave at least $116 million to patient advocacy groups in a single year, reveals a new database logging 12,000 donations from large publicly traded drugmakers to such organizations.

Even as these patient groups grow in number and political influence, their funding and their relationships to drugmakers are little understood. Unlike payments to doctors and lobbying expenses, companies do not have to report payments to the groups.

The database, called “Pre$cription for Power,” shows that donations to patient advocacy groups tallied for 2015 — the most recent full year in which documents required by the Internal Revenue Service were available — dwarfed the total amount the companies spent on federal lobbying. The 14 companies that contributed $116 million to patient advocacy groups reported only about $63 million in lobbying activities that same year.

Though their primary missions are to focus attention on the needs of patients with a particular disease — such as arthritis, heart disease or various cancers — some groups effectively supplement the work lobbyists perform, providing patients to testify on Capitol Hill and organizing letter-writing and social media campaigns that are beneficial to pharmaceutical companies.

Six drugmakers, the data show, contributed a million dollars or more to individual groups that represent patients who rely on their drugs. The database identifies over 1,200 patient groups. Of those, 594 accepted money from the drugmakers in the database.

The financial ties are troubling if they cause even one patient group to act in a way that’s “not fully representing the interest of its constituents,” said Matthew McCoy, a medical ethics professor at the University of Pennsylvania who co-authored a 2017 study about patient advocacy groups’ influence and transparency.

Notably, such groups have been silent or slow to complain about high or escalating prices, a prime concern of patients.

“When so many patient organizations are being influenced in this way, it can shift our whole approach to health policy, taking away from the interests of patients and towards the interests of industry,” McCoy said. “That’s not just a problem for the patients and caregivers that particular patient organizations serve; that’s a problem for everyone.”

Bristol-Myers Squibb provides a stark example of how patient groups are valued. In 2015, it spent more than $20.5 million on patient groups, compared with $2.9 million on federal lobbying and less than $1 million on major trade associations, according to public records and company disclosures. The company said its decisions regarding lobbying and contributions to patient groups are “unrelated.”

“Bristol-Myers Squibb is focused on supporting a health care environment that rewards innovation and ensures access to medicines for patients,” said spokeswoman Laura Hortas. “The company supports patient organizations with this shared objective.”

The first-of-its-kind database, compiled by Kaiser Health News, tallies the money from Big Pharma to patient groups. KHN examined the 20 pharmaceutical firms included in the S&P 500, 14 of which were transparent — in varying degrees — about giving money to patient groups. Pre$cription for Power is based on information contained in charitable giving reports from company websites and federal 990 regulatory filings.

It spotlights donations pharma companies made to patient groups large and small. The recipients include well-known disease groups, like the American Diabetes Association, with revenues of hundreds of millions of dollars; high-profile foundations like Susan G. Komen, a patient group focused on breast cancer; and smaller, lesser-known groups, like the Caring Ambassadors Program, which focuses on lung cancer and hepatitis C.

The data show that 15 patient groups — with annual revenues as large as $3.6 million — relied on the pharmaceutical companies for at least 20 percent of their revenue, and some relied on them for more than half of their revenue. The database explores only a slice of the pharmaceutical industry’s giving overall and will be expanded with more companies and groups over time.

“It’s clear that more transparency in this space is vitally important,” said Sen. Claire McCaskill (D-Mo.), who has been investigating the links between patient advocates and opioid manufacturers and is considering legislation to track funding. “This database is one step forward in that effort, but we also need Congress to act.”

What Drives The Money Flow

The financial ties between drugmakers and the organizations that represent those who use or prescribe their blockbuster medicines have been of growing concern as drug prices escalate. The Senate investigated conflicts of interest in the run-up to the passage of the 2010 Physician Payments Sunshine Act — a law that required payments to physicians from makers of drugs and devices to be registered on a public website — but patient groups were not addressed in the bill.

Some of the patient groups with ties to trade groups echo industry talking points in media campaigns and letters to federal agencies, and do little else. And patients, supported by pharma, are dispatched to state capitals and Washington to support research funding. Some groups send patients updates on the newest drugs and industry products.

“It’s through groups like this that patients often learn about illnesses and treatments,” said Rick Claypool, a research director for Public Citizen, a consumer advocacy group that says it does not accept pharmaceutical funding.

For the patient group Caring Ambassadors Program, industry funds are needed to make up for a lack of public funding, said the group’s executive director, Lorren Sandt. According to IRS filings and published company reports, in 2015 the group received $413,000, the bulk of which came from one company, AbbVie, which makes a hepatitis C treatment and has been testing a new lung cancer drug, Rova-T, not yet approved. She said the money had no influence on the Caring Ambassadors Program’s priorities.

“There aren’t a lot of large pockets of funding outside of the pharmaceutical money,” Sandt said. “We take it where we can find it.”

Other patient groups such as The National Women’s Health Network, based in Washington, D.C., make sacrifices to avoid pharmaceutical funding. That includes operating with a small staff in a “modest” office building with few windows and outdated computers, according to executive director Cindy Pearson. “You can see the effect of our approach to funding as soon as you walk [in] the door.”

Pearson said it’s hard for patient groups not to be influenced by the funder, even if they proclaim independence. Patient groups “build relationships with their funders and feel in sync and have sympathy” for them. “It’s human nature. It’s not evil or weak, but it’s wrong.” […]

They Weren’t Always Backed By Pharma

Into the ’80s and early ’90s, patient lobbying was generally limited and self-funded with only one or two affluent patients from an organization traveling to Washington on a given day, said Diana Zuckerman, president of the nonprofit National Center for Health Research.

But the power of patient-lobbyists became apparent after a successful campaign by AIDS patients led to government action and a national push to find drugs to treat the then-terminal disease. Zuckerman said she will never forget when two women visited her office and asked how breast cancer patients could be as effective as the AIDS patients.

“At the time, there were no breast cancer patients advocating for money or anything else. It’s hard to believe,” she said. “I still remember that conversation, because it was really a turning point.”

Soon after, breast cancer patients started visiting the Hill more frequently. Patients with other diseases followed. Over time, patients’ voices became a potent force, often with industry support.

Even some wealthy, high-profile organizations take industry money: For example, $459,000 of Susan G. Komen’s $118 million in 2015 revenue came from drugmakers in the database, according to public disclosures. Asked about the pharma money, the foundation said it has institutional processes in place to ensure that “no corporate partner — pharma or otherwise — decides our mission priorities,” including a scientific advisory board — free of sponsor influence — that reviews its research program.

Today, patient advocacy groups flush with more industry dollars fly patients in for testimony and training about how to lobby for their drugs.

Some years ago, as the groups increased in number, Zuckerman said, she started getting email invitations from advocacy groups to attend so-called lobbying days explicitly sponsored by the pharmaceutical industry. The hosts often promised training and usually some kind of keynote speaker at a luncheon in Washington — plus a potential scholarship to cover travel. Now, lobbying days involving dozens of patients from a single group are part of the landscape.

Dan Boston, president of lobbying firm Health Policy Source, said, “It would be naive to think these people on a Tuesday afternoon just happen to turn up in XYZ places,” adding that the money isn’t necessarily a bad thing. Money tends to flow toward citizen groups that already have the same priorities as their funders, he said. […]

Read the original article here.

NCHR Letter to the Senate on Right To Try

National Center for Health Research, March 23, 2018

Dear Senators,

We are writing to urge you to vote against the Right to Try bill that recently passed the House of Representatives (HR 5247) despite strong opposition from the Democratic leadership. We agree with the idea that terminally ill patients should have access to potentially life-saving medical treatments, and understand that some terminally ill patients are willing to take big risks to have a chance to live longer. If they want access to experimental treatments that are undergoing clinical trials, they should be able to do so as long as they are well informed of the risks as well as the possible benefits.

That is supposed to be the goal of the federal Right to Try bill, but it fails. That is why four previous FDA Commissioners as well as the American Cancer Society; American Lung Association; National Physicians Alliance; American Society of Clinical Oncology (ASCO); Cystic Fibrosis Foundation; International Society for Stem Cell Research; National Consumers League; National Health Council; National Organization for Rare Disorders (NORD); Vietnam Veterans of America; and dozens of other patient and public health organizations oppose the bill, as we do.

We have spoken to families whose efforts to “try anything” made their loved ones’ remaining days miserable and left their families even more devastated. The House bill has a very loose definition of which patients would be eligible (since many patients with diabetes and heart disease have conditions that can cause irreversible damage that will cause premature death). In addition, it provides access to any drug that has passed Phase I clinical trials, which often don’t include even one patient. Instead Phase I trials can include healthy volunteers, such as college students, who are much less likely to be harmed by an experimental drug than a terminally ill patient.

Another problem is that these very preliminary (Phase I) clinical trials usually include very small numbers of people, and do not study whether or not a medical product has any benefit at all. They are designed to determine the immediate risks on just a few healthy volunteers or patients. That is why 85% of drugs that pass Phase I clinical trials are never proven safe and effective and never approved by the FDA.

Fortunately, the FDA’s current Expanded Access program requires at least some evidence that an experimental treatment could potentially be helpful. The FDA uses compassionate waivers when doctors request them for very ill patients, and FDA agrees to such requests 99% of the time.

The GAO’s July 2017 report on the FDA’s current Compassionate Use/Expanded Access program pointed out that most experimental drugs that pharmaceutical companies distribute under that program eventually obtain FDA approval. That shows that the program is working: It gives patients earlier (usually free) access to experimental drugs that will eventually be proven safe and effective.

Although HR 5247 includes some potentially useful requirements that the results of patients’ access to experimental drugs be made available to the FDA, so that they will be aware of serious harm that could be caused, there is no enforcement mechanism to make sure that information is made available. In other words, even if a drug was found to be extremely dangerous when used by patients through the Right to Try program, that information might not be available to FDA, patients, or physicians.

We have included our Center’s Right to Try Fact Sheet below, which we hope you will find useful.

Sincerely,

Jack Mitchell, Director of Health Policy

The Right to Try bill creates a program that is not as good as the existing FDA “Expanded Access” program, which has approved 99% of requests they received.

  • FDA’s Expanded Access program makes sure that there is some evidence that the experimental drug is safe and effective. Most of the drugs that go to patients through this program are eventually approved by the FDA.
  • Lowering the standards to drugs that completed “Phase 1 clinical trials” means that 85% of the drugs will never be proven safe and effective.
  • When standards are that low, desperate patients can die sooner and more painfully than they would have otherwise.
  • FDA physicians are available 24 hours a day to approve any emergency Expanded Access requests that the agency receives. They usually grant emergency requests immediately over the phone and non-emergency requests in an average of 4 days
  • Pharmaceutical companies may choose to deny patients access to experimental drugs if there is not enough of the drug available or they are concerned about dangerous side effects. When a patient is denied access to an experimental treatment, it is almost always because the company has said no, not the FDA.
  • State “right to try” laws do not give patients a “right” to try and have done little to expand access to investigational treatments. There is no evidence that anyone has obtained an investigational treatment via these laws that couldn’t have been obtained through FDA’s expanded access program.
  • Right to try laws do not require companies to provide patients access to an experimental treatment. They only give the right to request the treatment from the company. Patients already have that right.
  • The bills would weaken FDA’s ability to oversee dangerous side effects from the use of an experimental drug while protecting companies from law suits if the drugs are more harmful than the patients were informed.

Right To Try Fact Sheet

Cancer Prevention and Treatment Fund

The Right to Try bill creates a program that is not as good as the existing FDA “Expanded Access” program, which has approved 99% of requests they received.

  • FDA’s Expanded Access program makes sure that there is some evidence that the experimental drug is safe and effective. Most of the drugs that go to patients through this program are eventually approved by the FDA.
  • Lowering the standards to drugs that completed “Phase 1 clinical trials” means that 85% of the drugs will never be proven safe and effective.
  • When standards are that low, desperate patients can die sooner and more painfully than they would have otherwise.
  • FDA physicians are available 24 hours a day to approve any emergency Expanded Access requests that the agency receives. They usually grant emergency requests immediately over the phone and non-emergency requests in an average of 4 days
  • Pharmaceutical companies may choose to deny patients access to experimental drugs if there is not enough of the drug available or they are concerned about dangerous side effects. When a patient is denied access to an experimental treatment, it is almost always because the company has said no, not the FDA.
  • State “right to try” laws do not give patients a “right” to try and have done little to expand access to investigational treatments. There is no evidence that anyone has obtained an investigational treatment via these laws that couldn’t have been obtained through FDA’s expanded access program.
  • Right to try laws do not require companies to provide patients access to an experimental treatment. They only give the right to request the treatment from the company. Patients already have that right.
  • The bills would weaken FDA’s ability to oversee dangerous side effects from the use of an experimental drug while protecting companies from law suits if the drugs are more harmful than the patients were informed.