More Than 25% of New Molecular Entities Approved in 2016 Are Personalized Medicines


The Personalized Medicine Coalition (PMC) recently reported that for the third year in a row, personalized medicines accounted for more than 25% of all new molecular entities (NMEs) approved by the US Food & Drug Administration (FDA). Personalized Medicine at FDA, 2016 Progress Report (Report). Personalized medicines are new drugs, agents or therapeutic biologics where a diagnostic test is used to determine which medical treatments will work best for a specific patient. For purposes of the Report, the PMC categorized personalized medicines as those therapeutic products for which the label includes reference to specific biological markers.

PMCs Report specifically indicates that of the 22 NMEs approved by the FDA in 2016, 6 of them are personalized medicines, half of which are oncology drugs. This continues the 3-year trend that the PMC first documented in 2014 when it reported that 9 of the 41 approved NMEs were personalized medicines.

The 6 new FDA-approved drugs are:

  1. Rubraca (rucaparib) for the treatment of advanced ovarian cancer;
  2. Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy;
  3. Epclusa (sofosbuvir and velpatasvir) for the treatment of chronic hepatitis C infection;
  4. Tecentriq (atezolizumab) for the treatment of advanced or metastatic urotherlian cancer and metastatic non-small cell lung cancer;
  5. Venclexta (venetoclax) for the treatment of chronic lymphocytic leukemia; and
  6. Zepatier (elbasvir and grazoprevir) for the treatment of chronic hepatitis C infection.

The Report concludes that while challenges in the areas of diagnostic regulatory policy, reimbursement, and clinical adoption remain, the FDAs approval of these new drugs that rely on diagnostic medicine signals a shift toward a more personalized health care system.


Precision Medicine - Obama’s Health Care Legacy


As we come to the end of President Obama’s administration, it is time to look back on the past eight years and the administration’s impact on the delivery of the nation’s health care. While the Affordable Care Act (signed into law early in the President’s administration on March 23rd, 2010) is a significant achievement in that it provided many Americans access to health care, one cannot dismiss the President’s transformative commitment to precision or personalized medicine.

In 2006 as a then junior senator, President Obama introduced the Genomics and Personalized Medicine Act to improve access to and utilization of valid, reliable genetic tests and to secure the promise of personalized medicine for all Americans. While neither President Obama’s bill nor subsequent versions were enacted into law, many of the elements of the bills were incorporated into, and expanded upon, in the recently enacted 21st Century Cures Act (the “Act”).  Signed into law by the President on December 13th, 2016, the Act passed the House of Representatives (392-26) and Senate (94-5) with overwhelming bipartisan support.

The Act secures $4.8 billion in funding for the necessary infrastructure to advance precision medicine. This includes funding to the National Institutes of Health to support the President’s Precision Medicine Initiative ($1.5 billion) and to Vice President Biden’s “Cancer Moonshot” initiative ($1.8 billion). Spending under the Act will advance precision medicine more specifically by developing:

  • New approaches for addressing scientific, medical, public health and regulatory issues;
  • New genomic-based technologies;
  • New private-public partnerships to better leverage data sources and interpretation;
  • New expanded sources of genetic and clinical information; and
  • Improved regulations and laws to protect human participants and their private health care information.

We can all look back with gratitude to President Obama’s leadership in the advancement of personalized medicine and the modernization of the tools necessary to advance its promise.

A copy of the Bill and a concise summary provided by The Energy and Commerce Committee can be found here.

New Report Outlines 5 Year Plan to Achieve Cancer Moonshot


The Cancer Moonshot Task Force has just released its 5 year plan for implementing President Obama’s “Cancer Moonshot” program (“Report”). The initiative, announced during President Obama’s 2016 State of the Union Address, is a national effort to dramatically accelerate efforts to prevent, diagnose and treat cancer that take advantage of recent advances in molecular medicine and information sciences. This initiative is unique in its emphasis on public and private collaboration:

“The Cancer Moonshot aims to realize this promise by leveraging public and private efforts focused on building a system in which patients, researchers, and clinicians can seamlessly share information on treatments and outcomes to accelerate research, guide treatment decisions, and improve cancer outcomes for people across the Nation, and ultimately the world.”

Report at page 2.

Five Goal Approach

The Report, authored by the Moonshot Task Force, noted the need to have an organizational framework for addressing and uniting efforts to reach the goal of improving cancer diagnosis and therapy. Five goals emerged, each of which is noted to be critical to the overall mission, but also intended to build on and augment the success of the other goals. Report at page 5.

Strategic Goal 1: Catalyze New Scientific Breakthroughs

New scientific breakthroughs will be accelerated by: 1) promoting interdisciplinary approaches for elucidating the biological mechanism underlying cancer onset and treatment; 2) aligning research and care as a seamless and iterative process; and 3) maximizing the collection and research use of longitudinal data and biospecimens.

Strategic Goal 2: Unleash the Power of Data

A stated goal of the Task Force is to maximize access to and usability of data now available due to advances in genomic information and health record information.  This will be achieved by: 1) enabling a seamless data environment; 2) supporting open publication and storage platforms; and 3) developing a workforce that can take advantage of the open and connected environment.

Strategic Goal 3: Accelerate Bringing New Therapies to Patients

New effective therapies are the ultimate goal of the President’s initiative. The Task Force seeks to remove impediments by: 1) finding efficiencies in regulatory review and licensing processes; 2) enhance data sharing and incentivize pre-competitive collaborations; and 3) strengthen oncology clinical research.

Strategic Goal 4: Strengthen Prevention and Diagnosis

Cancer prevention and diagnosis can be strengthened by: 1) promoting public access to health programs, policies and outreach; 2) gaining a better understanding of the environmental determinants of cancer; and 3) enhancing cancer screening and testing.

Strategic Goal 5: Improve Patient Access and Care

Strategic goal 5 seeks to build on the improved access to health care provided by the Affordable Care Act. The Task Force seeks to identify areas with the greatest potential for meaningful patient impact by: 1) improve efficiencies of current programs; 2) improve cancer prevention, treatment and quality of care by applying current knowledge into workable policies; and 3) ensuring each patient receives quality care.

Creating New Paradigms

The Report sets out a blueprint with a series of activities to accelerate gains for cancer diagnosis and therapy – that is, to achieve ten years of progress in five. The Report summarizes the aspirations, conceptual and practical, that can be achieved:

“Ultimately, through the creation of new paradigms for generating, sharing, and integrating research and clinical data to enhance patient care, the Cancer Moonshot can accelerate the delivery of effective cancer prevention strategies, diagnostics, and treatments to patients in communities around the world.”

Report at page 29.

Inherent Disclosure Satisfied Written Description

In Yeda Research and Development Co., Ltd. v. Abbott GMBH & Co. KG, Slip Op. 2015-1662 (Fed. Cir. 2016), the Federal Circuit held that a claim to an isolated protein described by its partial amino acid sequence satisfies 35 U.S.C. § 112 (written description) when the partial sequence is combined with other identifying characteristics of the protein.

A Purified and Isolated TNFα Binding Protein

Yeda Research and Development Co., Ltd. (Yeda) appealed two decisions by the U.S. District Court for the District of Columbia, that were in turn appeals of two decisions by the USPTO Board of Patent Appeals and Interferences, regarding Yeda’s allegation that Abbott Gmbh & Co. KG’s (Abbott) U.S. Patent No. 5,344,915 entitled “Proteins and Preparations Thereof” (‘915 Patent) is invalid as anticipated by an intervening prior art reference. Yeda and Abbott did not dispute that the reference anticipated the claims if the claims were entitled to its earliest priority.

The ‘915 Patent, was filed on September 26, 1991 and claims priority to two German applications, P39 15 072 (the ‘072 application), filed on May 9, 1989 and P39 22 089 (the ‘089 application), filed on July 5, 1989.

The ‘915 Patent issued with three claims. Claim 1 was determined to be representative:

  1. A purified and isolated TNFα-binding protein which has a molecular weight of about 42,000 daltons and has at the N terminus the amino acid sequence

Xaa Thr Pro Tyr Ala Pro Glu Pro Gly Set Thr Cys Arg Leu Arg Glu

where Xaa is hydrogen, a phenylalanine residue (Phe) or the amino acid sequences Ala Phe, Val Ala Phe, Gln Val Ala Phe, Ala Gln Val Ala Phe, Pro Ala Gln Val Ala Phe or Leu Pro Ala Gln Val Ala Phe.

Inherent Disclosure Can Provide Adequate Written Description

Prior to addressing the substance of the challenge, the court first reviewed the legal standard for written description. Citing its en banc decision Ariad Pharmaceuticals, Inc. v. Eli Lilly & Co., 598 F.3d 1336 (Fed. Cir. 2010), the court held that for the claims of an application to benefit from a priority application’s filing date, the claimed invention must be “disclosed in a way that clearly allows a person of ordinary skill to recognize that the inventor invented what is claimed and possessed the claimed subject matter at the filing date.” Id. at 1351.

Yeda argued that to gain the benefit of the earlier application’s filing date, Abbott must show that at the time of filing the priority application (here, the ‘072 application), a person of ordinary skill in the art would have understood that the claimed protein contained more than the partial sequence disclosed in the ‘072 application, i.e., the additional amino acids described in the ‘915 Patent specification. Abbott disagreed and argued that the ‘072 application need only describe and enable the claimed protein, and that a protein can be adequately described when a partial amino acid sequence is disclosed along with other biological characteristics.

The court agreed with Abbott, explaining that under the doctrine of inherent disclosure, “when a specification describes an invention that has certain undisclosed yet inherent properties, that specification serves as adequate written description to support a subsequent patent application that explicitly recites the invention’s inherent properties.” Slip Op. at 6. The court determined that the doctrine applied because it was “undisputed that the invention described in an earlier application was the exact invention claimed by the later patent.” Slip Op. at 7.

Finally, the court rejected Yeda’s argument that Abbott cannot rely on inherent disclosure on the ground that the inherent limitation cannot be material to the patentability of the invention. Yeda argued that the amino acids absent from the priority application are material because Abbott relied upon their absence to distinguish prior art during examination. During examination, the claims were rejected on prior art that disclosed a protein with the same source, weight, and function of the protein claimed in the ‘915 Patent. The court disagreed, noting that Abbott’s response did not rely on only the information absent from the priority application and that the sequence disclosed in the priority application was sufficient to distinguish the prior art.

Adequate Written Description

The court’s decision is a reminder that there is more than one way to describe an invention to satisfy written description, and, as here, to obtain the benefit of an earlier filing date. Automated DNA sequencers have simplified the sequencing of proteins such that it is common for an application disclosing a protein to disclose its complete amino acid sequence. Nevertheless, where a prior application discloses the same protein as the later filed application, and the prior art application provides sufficient description to characterize the protein and distinguish the prior art, along with a description of how to obtain it from its biological source, the prior application supports the later filed application. Of course, should Abbott assert the claim against an alleged infringer, the separate question of whether the claim is patent eligible as required by 35 U.S.C. § 101, would be an primary issued for consideration.

Personalized Medicine: Insights Into Current Legal Issues


Guest Post by:  Nate Beaver, Esq.; Gary Koch, Esq.; Antoinette Konski, Esq.; and Judy Waltz, Esq., Foley & Lardner, LLP.

In his January 20, 2015 State of the Union address, President Obama brought to the nation’s attention the promise of personalized medicine when he announced the Precision Medicine Initiative (the “PMI”). In announcing this initiative during his address, which was to be funded with $215 million in the President’s 2016 budget, the President gave many in the nation their first hint of the tremendous benefits to be gained by physicians being better able to tailor medical treatments to individual patients. This focus on the unique attributes of each patient – the patient’s environment, lifestyle, and, most notably, genetic characteristics – allow targeted therapies to be deployed that are more likely to be efficacious, less likely to lead to adverse side effects, and, in many circumstances, more cost-effective for both the patient and society at large than current approaches to many different illnesses. Continue reading this entry