Haffner Associates is a Globally Recognized Expert in Orphan Product Development
What is an Orphan Disease?
Defined by law in the US as a disease affecting
patients in the US
Defined by regulation in the EU as a disease affecting
patients in the EU
What is an Orphan Product?
A drug or biologic used to treat, prevent, or diagnose an orphan (rare) disease.
Our Services
Haffner Associates' recognized expertise in policy and procedures relative to products for rare diseases provides the following capabilities:
Advising on Orphan Product Development
Regulatory strategies, development issues, and FDA/EMA communications
Developing FDA and EMA Applications and Meeting Packages
Orphan Drug, Breakthrough Therapy, Fast Track Designations, and more
Evaluating Applications and Correspondence with FDA and EMA
Sponsor authored applications, questions from the FDA or EMA, deficiency letters, and other issues
Our full suite of services includes:
- Considering the client’s strategic goals, project pipeline gaps, and potential regulatory hurdles
- Advising on orphan product development regulatory strategies, regulatory and development issues, and communications with the FDA.
Developing applications and meeting packages for the FDA including (but not limited to):
- Orphan Drug (and biologic) Designations (ODD)
- Rare Pediatric Disease (RPD) Designations (pending reauthorization)
- Breakthrough Therapy Designations (BTD)
- Fast Track Therapy Designations (FTD)
- Regenerative Medicines Advanced Therapy (RMAT) Designations
- Qualified Infectious Disease Products (QIDP) Designations
- Meeting requests, briefing packages, meeting preparing/coaching, and attendance at INTERACT and Types A, B, C and D meetings
- Orphan Products Grants
Developing applications for the EMA including (but not limited to):
- Orphan Drug Designations
- PRIME designations
Evaluating Applications and Correspondence with FDA and EMA
- Including all the above applications and communications authored by the sponsor and other agents
- Deficiency and denial letters from all the above applications and communications
About Us
Haffner Associates was founded in 2009 by Marlene E. Haffner, Director of the FDA Office of Orphan Products Development (1986-2007). Our team assists companies developing drugs and biologics for the treatment of rare diseases. Haffner Associates is well regarded globally for their expertise, high standards, unique understanding of “FDA think,” and commitment to its clients developing orphan products along all stages of the regulatory pathway.
Marlene E. Haffner, MD, MPH
Principal and Founder
Dr. Marlene Haffner is a leading expert in orphan product development. She brought the term “orphan products” to the minds of many in the pharmaceutical world. As Director of the FDA’s Office of Orphan Products Development for more than 20 years, she helped advance more than 300 treatments and establish global programs in countries including the EU, Japan, Australia, the Philippines and beyond.
As founder of Haffner Associates, she continues to advise biotech and pharmaceutical companies, patient advocacy groups, and government agencies concerning orphan products.
Sandra Heibel, PhD
Chief of Science and Regulatory Affairs
Dr. Sandra Heibel is a scientist and regulatory expert with extensive knowledge of drug development. She has expertise in rare disease regulatory strategy, preclinical and clinical research, and scientific writing in the US and EU. Sandra joined Haffner Associates in 2015. Prior to working in the regulatory affairs field, she spent 13 years conducting preclinical and clinical research in a laboratory setting with a focus on rare, heritable diseases. She is a passionate advocate for rare disease therapy development and research.
Marlene E. Haffner, MD, MPH
Principal and Founder
Dr. Marlene Haffner is a leading expert in orphan product development. She brought the term “orphan products” to the minds of many in the pharmaceutical world. As Director of the FDA’s Office of Orphan Products Development for more than 20 years, she helped advance more than 300 treatments and establish global programs in countries including the EU, Japan, Australia, the Philippines and beyond. As founder of Haffner Associates, she continues to advise biotech and pharmaceutical companies, patient advocacy groups, and government agencies concerning orphan products.
Sandra Heibel, PhD
Chief of Science and Regulatory Affairs
Dr. Sandra Heibel is a scientist and regulatory expert with extensive knowledge of drug development. She has expertise in rare disease regulatory strategy, preclinical and clinical research, and scientific writing in the US and EU. Sandra joined Haffner Associates in 2015. Prior to working in the regulatory affairs field, she spent 13 years conducting preclinical and clinical research in a laboratory setting with a focus on rare, heritable diseases. She is a passionate advocate for rare disease therapy development and research.
