Celebrating 30 Years of the Orphan Drug Act & the Evolution of Drug Development for Therapies in Rare Diseases

Friday, February 28 2014
9:00 am – 11:00 am

Location: Massachusetts State House, Room 222 (2nd Floor)

The total number of patients in the United States and Europe suffering from a rare disease is estimated at 25 and 30 million, respectively. There are millions more around the globe. Specific orphan drug legislations across the globe have been introduced to stimulate the pharmaceutical industry to further develop and bring much needed rare disease therapies to the market.  Please join us for this dynamic panel discussion during which we will discuss past, present and future of the orphan drug development.

This session will be held at the Massachusetts State House. Please note that the room has limited seating - arrive early to reserve your spot.

The all-star panel of experts in the rare disease arena includes:

  • Phil Vickers, PhD, Head of Research and Development, Shire
  • Marlene E. Haffner, MD, MPH, President & CEO, Haffner Associates, LLC
  • Patricia Weltin, Executive Director at the Rare Disease United Foundation
  • Senator Karen E. Spilka, Majority Whip, Massachusetts State Senate


  • John Crowley, JD, MBA, President and CEO, Amicus Therapeutics


After the Forum, join MassBio, VHL Alliance, member companies, patient groups, patients and legislators to recognize Rare Disease Day in the Nurses Hall.


This event is organized by the MassBio Legal and Regulatory Forum Working Group.


Speaker Bios

John F. Crowley, J.D., M.B.A. , President and Chief Executive Officer, Amicus Therapeutics

Mr. Crowley became president and CEO of Amicus in January 2005, having served as a director since 2004. Previously he was founding president and CEO of Orexigen Therapeutics. Preceding Orexigen, Mr. Crowley was senior vice president at Genzyme Therapeutics, a position he assumed after overseeing the sale of Novazyme Pharmaceuticals to Genzyme in September 2001. Mr. Crowley was the founding president and CEO of Novazyme that was developing a novel treatment for Pompe disease. He previously served in several senior management roles with the Bristol-Myers Squibb Company (BMS), including director of the Executive Committee for the U.S. Medicines Group, director of Business Strategy for the U.S. Pharmaceuticals Group, and director of U.S. Area Marketing for the Neuroscience and Infectious Disease Division. Preceding his experience at BMS, Mr. Crowley worked as a business strategy consultant for Marakon Associates. Mr. Crowley began his professional career as a litigation associate in the Health Care Practice Group of the Indianapolis-based law firm of Bingham, Summers, Welsh & Spilman.

Mr. Crowley is involved with several charitable and community organizations, including serving as president of the National Tay-Sachs and Allied Diseases Association. He is also on the Research Advisory Board of the national Muscular Dystrophy Association and the Board of Directors of St. Peter’s University Hospital. Mr. Crowley’s involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease - a fatal neuromuscular disorder. Mr. Crowley and his family have been featured on the cover of The Wall Street Journal and on The Today Show, CNBC and The Paula Zahn Show.

Mr. Crowley is a commissioned officer in the United States Navy Reserve (active).

Mr. Crowley earned his B.S. degree in Foreign Service from Georgetown University's School of Foreign Service, his J.D. from the University of Notre Dame Law School and his M.B.A. from Harvard Business School.


Marlene E. Haffner, MD, MPH, President & CEO, Haffner Associates, LLC

Marlene E. Haffner, MD, MPH, is  the CEO of Haffner Associates, LLC a firm dedicated to the strategy, development and policy of drug development with a special emphasis on rare diseases and the products that treatment them.  Prior to establishing her own company, in March 2009, she was for two years Executive Director, Global Regulatory Policy and Intelligence at Amgen, Inc.   

For 20 years, Dr. Haffner served as Director of the Office of Orphan Products Development (OOPD) of the Food and Drug Administration (FDA). As OOPD Director she was responsible for the leadership and management of the FDA orphan products development program, the first Orphan Products program in the world.  In addition to her FDA responsibilities, Dr. Haffner was instrumental in the development of Orphan Drug programs in the EU, Japan, Australia and beyond.  She is well known as an expert in orphan drug development and is a sought after speaker and consultant in that area of regulatory science.

In addition to her consulting activities Marlene is Adjunct Professor, Department of Preventive Medicine and Biometrics, and Clinical Professor, Department of Medicine, at the F. Edward Hébert School of Medicine, Uniformed Services University of the Health Sciences (USUHS) in Bethesda, Maryland.  For 36 years she served in the United States Public Health Service beginning her career with the Indian Health Service in Gallup, New Mexico.  She received her MD from the George Washington University School of Medicine where she then interned in Internal Medicine. She received further training in internal medicine, dermatology and hematology at the Presbyterian Hospital, New York and that the Albert Einstein College of Medicine, New York.  She received an MPH from the Johns Hopkins University Bloomberg School of Public Health. During her Public Health career, she rose to the rank of Rear Admiral in the USPHS.

A sought after speaker and consultant, Dr. Haffner  has received many awards for her work in drug development including The Outstanding Contributions to Pharmaceutical Medicine Award from the American Academy of Pharmaceutical Physicians, and  the Woodrow Wilson Award for Outstanding Government Service from the Johns Hopkins University.   On February 25 of this year she received the Lifetime Achievement Award from EURORDIS – the European Organization for Rare Diseases.  She is the author of multiple articles in peer reviewed literature concerning issues of orphan product development.


Senator Karen E. Spilka, Majority Whip, Massachusetts State Senate

Through hard work, a keen ability to navigate complex issues, and a demonstrated commitment to advocating for her constituents, Senator Karen E. Spilka has established herself as a respected leader in state government.

First elected to the House of Representatives in a special election in the fall of 2001, Senator Spilka served three years in the House before her election to the Senate. She was officially sworn in as the State Senator for the 2nd Middlesex and Norfolk district in January 2005. As such, she represents the MetroWest, one of the most important economic regions in Massachusetts.

In January 2013, Senator Spilka was promoted by Senate President Therese Murray to serve as Majority Whip after previously serving as Assistant Majority Whip since January 2012. With this position, Senator Spilka is charged with developing and actively supporting the Senate’s legislative agenda. With these appointments, Spilka has joined a select group of members in the Senate President’s leadership team chosen for their commitment, ability to lead in the legislature, and their track record of being able to deliver on important issues for the Commonwealth. Senator Spilka currently serves as the Senate Chair of the Massachusetts Biotech Caucus.


Phil Vickers, PhD, Head of Research and Development, Shire

Philip J. Vickers, Ph.D., is Head of Research and Development at Shire.  Dr. Vickers is responsible for overseeing preclinical research and development, clinical research, regulatory affairs, and medical affairs at Shire. He oversees the organization’sgrowing product portfolio and plays a key role indeveloping and executing Shire’s global business strategy. 

Dr. Vickers has over 23 years in the pharmaceutical industry experience. He joined Shire from Resolvyx Pharmaceuticals, where he was Chief Scientific Officer and President. While at Resolvyx, he was a member of the Board of Directors, with accountability for all preclinical and clinical research, as well as partnering with investors, external business development partners, and establishing external collaborations.

Prior to this, he was Senior Vice President and US Head of Research at Boehringer-Ingelheim Pharmaceuticals, where he had responsibility for the Ridgefield CT research site, which focused on therapeutic areas of cardiovascular and immunology & inflammation. Before BI, he spent over 13 years with Pfizer in a number of R&D leadership positions of increasing responsibility in the US and UK. These roles included drug discovery leadership across therapeutic areas, leading the Pfizer Research and Technology Center in Cambridge, MA. He started his career with Merck Frosst in Canada, where he played a key role in the successful leukotriene and cyclooxygenase programs.

Dr. Vickers holds a Ph.D. in Biochemistry from the University of Toronto, and a Bachelor of Science degree in Applied Biochemistry from the University of Salford, Manchester. He was also a Visiting Fellow at the National Cancer Institute in Bethesda, Maryland.  


Patricia Weltin, Executive Director/Founder at Rare Disease United Foundation

Patricia Weltin is the Executive Director and Founder of the Rare Disease United Foundation, a state-based, non-disease specific organization with communities in Rhode Island and Massachusetts. As a rare disease mom with a background in Finance, Patricia started working in the rare disease space to help raise awareness for all people living with a rare disease. In just a little over two years, the RDUF has had two of the largest World Rare Disease Day events in the country, garnered support from legislators in both RI and MA, successfully advocated on both state and national legislation and provided support for rare patients. Patricia and her team are innovators in the rare disease space and have used their own rare disease journeys to positively affect change.

Patricia’s most recent accomplishments include:

  • Development of Rhode Island Rare Disease Community
  • Development of Massachusetts Rare Disease Community
  • Two World Rare Disease Day Events in Rhode Island with 150+ attendees
  • Actively advocating for rare disease legislation in both RI & MA
  • Worked on EXPERRT Act with U.S. Senator Sheldon Whitehouse, signed by President Obama into law in July of 2012.


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