August 2015





Sunday, August 16, 2015
20th Annual ELLEN’s RUN
Southampton Hospital, Southampton, New York
9:00 a.m. sharp, rain or shine
Sponsored by The Ellen Hermanson Foundation


Sunday, September 13, 2015
25th Annual Komen Greater NYC Race for the Cure
Run with Team JALBCA or cheer us on!
Central Park – NYC

Wednesday, September 16, 2015
Cocktails and Conversation
A deliciously artful JALBCA  fundraiser with art historian Tanya Bastianich Manuali, PhD, co-author of the fascinating and beautiful book “Reflections of the Breast: Breast Cancer in Art Through the Centuries.”
5:30 p.m. @ four-star restaurant Felidia, 243 East 58th Street, NY, NY
(Attendance limited to 50 persons. Ticket price includes a copy of the book.)
Click here for tickets


October 26, 2015
JALBCA’s Annual Symposium
New York City Bar Association
44 West 44th Street, NY, NY


This month we look at:

  • Obama, Medicare, and extremely expensive specialty drugs
  • New lawsuit challenging the FDA’s stance on promotion off-label drug usage
  • The latest in new anti-hormone therapy research

Pharmaceutical Initiatives of the Obama Administration

In its April 2015 budget request, the Obama Administration reportedly requested Congress to fund an initiative to develop drugs tailored to the genetic characteristics of individual patients. Congress was also asked to permit Medicare officials to negotiate prices with the pharmaceutical industry, which is currently prohibited. And, a “precision medicine” initiative is being pursued, which has support among scientists. However, personalized medicines which are already on the market are costly, and their expense may not be covered by health insurance.

While companies may have a patient assistance program or a “patient access scheme” (a.k.a. discount) to provide financial help or free medicine to people who cannot afford the drugs, many patients would find these specialty drugs cost-prohibitive. The cost of analyzing a person’s genes, by using DNA sequencing, has declined sharply in recent years, but the cost of medicines that target specific genes or mutations is high and climbing higher. A few examples show how expensive these drugs have become:

  • For a standard course of treatment with blinatumomab (BLINCYTO®, Amgen Inc.), a new leukemia medicine, the price is about $178,000, the company said. On December 3, 2014, the FDA granted accelerated approval and breakthrough therapy designation for BLINCYTO for the treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
  • For crizotinib (XALKORI®, Pfizer, Inc.), a targeted lung cancer drug, therapy costs approximately $12,000 a month in the U.S. Treatments typically last seven months, Pfizer has said. On April 21, 2015, Pfizer announced that XALKORI® received breakthrough therapy designation by the FDA for the potential treatment of patients with ROS1-positive non-small cell lung cancer.
  • For imatinib mesylate (GLEEVEC®, Novartis), a targeted therapy to treat a certain type of chronic myeloid leukemia, the company charges $9,210 a month, and most patients will need lifelong therapy.
  • For ivacaftor (KALYDECO®, Vertex Pharmaceuticals Incorporated), used to treat cystic fibrosis, the list price for a one-year supply would cost $311,000 according to Vertex, its manufacturer.

More than 40 million people have prescription drug coverage through Medicare. A federal panel, the Medicare Payment Advisory Commission, told Congress in March 2015 that the use of high-cost drugs poses a big challenge for the government and for Medicare beneficiaries, who typically pay 25-33% of the cost of specialty drugs. For more, see The New York Times article, “Obama Proposes That Medicare Be Given the Right to Negotiate the Cost of Drugs.”

Amarin Lawsuits Against the FDA Regarding Off-Label Drug Use

On May 7, 2015, Amarin Pharma, Inc., a wholly-owned subsidiary of Amarin Corporation plc, and four independent physicians, in support of improved patient care, filed a lawsuit challenging the U.S. Food and Drug Administration’s regulations that prohibit the company from disseminating “truthful and non-misleading statements” to healthcare providers about a drug’s off-label use. The lawsuit, captioned Amarin Pharma, Inc., et al. v. Food & Drug Administration, et al., was filed in the United States District Court for the Southern District of New York. The Complaint alleges that doctors need this information in order to make informed medical decisions but do not have access to it because of the FDA restrictive regulations. The suit is premised on the principle that better informed physicians make better treatment decisions.

The drug at issue is Vascepa®, a fish oil product indicated as an adjunct to diet to reduce triglyceride levels in certain adults; it consists of pure EPA (eicosapentaenoic acid), an omega-3 fatty acid. This is FDA-approved to reduce triglyceride levels only in patients with “very high” levels. The plaintiffs allege that doctors also prescribe it for off-label uses, i.e., for patients with “persistently high” triglycerides to lower those patients’ triglyceride levels and/or non-HDL cholesterol. The FDA has not approved the promotion of the drug for such off-label use and, therefore, Amarin is not permitted to disseminate information about the drug’s benefits for such use without facing possible criminal prosecution and civil liability.

The Complaint alleges that the Second Circuit recently explained that the FDCA cannot “criminaliz[e] the simple promotion of a drug’s off-label use,” because such a construction of the FDCA would “raise First Amendment concerns.” [See United States v. Caronia, 703 F.3d 149, 160 (2d Cir. 2012).]

Yet, given FDA’s interpretation of the FDCA and the complex regulatory regime built up around it, the threat of criminal prosecution for simple promotion of a drug’s off-label use remains very real. In addition, they allege, the Government’s interpretation of the False Claims Act, 31 U.S.C. §§ 3729- 3733, raises the specter of enormous civil liability for simple promotion of a drug’s off-label use. The plaintiffs further contend that although the FDA’s written (non-binding) guidance for treatment of off-label use allows manufacturers to “respond to unsolicited requests for information” it does not allow manufacturers to initiate exchanges regarding off-label uses. Moreover, they allege that the guidance limits the information a manufacturer can provide in response and prevents doctors from engaging in free dialogue with manufacturers about off-label uses.

The plaintiffs seek a judicial declaration that FDA regulations limiting off-label promotion of such truthful and non-misleading information are unconstitutional under the First Amendment (freedom of speech) or Fifth Amendment(restriction against vague laws) as applied to Amarin’s proposed promotion of Vascepa. They also seek a declaration that Amarin may communicate to healthcare professionals – not the general public- certain information about Vascepa. The lawsuit does not:

  • seek to compel the FDA to approve an expanded indication for Vascepa based on Amarin’s ANCHOR clinical trial results for Vascepa;
  • require the court to evaluate whether FDA’s scientific conclusions about Vascepa are right or wrong;
  • seek to strike down off-label promotion laws and regulations as facially unconstitutional; or
  • challenge the government’s ability to prohibit pharmaceutical companies, including Amarin, from disseminating false or misleading information.

Oral arguments were heard in July 2015 before U.S. District Judge Paul A. Engelmayer. To see the Amarin Corporation plc SEC Form 8-K filing, click here.

Pasireotide – A Promising Alternative for Breast Cancer Chemoprevention Treatment?

Numerous clinical trials have demonstrated the preventative effects of selective estrogen receptor modulators (SERMs), such as tamoxifen and raloxifene, in women at increased risk for estrogen receptor-positive (ER+) breast cancer. However, SERMs have a number of side effects linked to their anti-estrogenic properties, including hot flashes in premenopausal women, increased risk of blood clots, and endometrial hyperplasia and carcinoma, which contribute to reduced compliance among women treated with anti-estrogen therapy. Therefore, new anti-hormone therapies without negative side effects are a significant need in breast cancer clinical care.

To address this need, Dr. David Kleinberg of the New York University School of Medicine received a Fiscal Year 2006 (FY06) Breast Cancer Research Program (BCRP) Synergistic Idea Award to examine the efficacy of a somatostatin analog (pasireotide) that had been shown to inhibit the effects of insulin-like growth factor I (IGF-I) and estrogen in the mammary glands of rats despite the presence of estrogen. Together with Drs. Julia Smith, Deborah Axelrod, and Baljit Singh, Dr. Kleinberg enrolled 15 women volunteers with atypical hyperplasia (AH) of the breast to conduct the study in order to assess the effects of pasireotide on IGF-I and breast tissue in humans.

The women were treated with the compound for 10 days in the study. By comparing tissue samples before and after the treatment phase, it was found that pasireotide inhibited cell proliferation and stimulated cell death in hyper plastic breast lesions by inhibiting IGF-I action and also reducing growth hormone secretion from the pituitary. Furthermore, the side effects of pasireotide were mild or moderate. The most notable was an increase in blood glucose during treatment.

Based on these promising results, Dr. Kleinberg proposed to further assess the potential clinical utility of pasireotide in a larger cohort of women with ductal carcinoma in situ (DCIS) through a FY09 BCRP Idea Expansion Award. Additionally, the treatment phase was extended to 20 days to both study the direct effects on DCIS over a longer time period and examine the persistence of altered blood sugar and growth hormone levels. The study is ongoing at this time.

Preliminary imaging results have revealed that two patients with low nuclear grade DCIS experienced complete disappearance of DCIS at excision, two patients with high nuclear grade disease had apparent tumor shrinkage, and three with high nuclear grade disease had no apparent tumor shrinkage. Also, as with the pilot study, it appears that serum glucose levels return to normal following the treatment phase. The results from these two studies represent a major step toward the identification of a treatment that can prevent the development of breast cancer or reverse premalignant lesions of the breast, while maintaining intact circulating estrogen levels.
This article was provided courtesy of Deborah Axelrod, M.D., a member of JALBCA’s Scientific Advisory Committee. The original post, with links to studies referenced, can be found here.


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