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Events

RARE Rev-inar Episode 001 Early Access Programmes


A multi-stakeholder look at early access pathways

Excited to welcome Tanner Pharma as co-hosts of our very first RARE Rev-inar. This live event will guide attendees through A multi-stakeholder look at early access pathways.

We’ll look to answers questions such as:

Early access plays an important role in getting treatments to patients, but what does an early access pathway look like?

What role can patient groups play in designing early access programmes?

What is an early access pathway and how do we design one that expands product access for manufacturers and meets the needs of patients? 

Joining us on the panel are several speakers with experience of different role within early access.

Speakers

John Lagus

John is the executive vice president of business development at Tanner Pharma. John spent more than a decade at Orphan Medical where he held various roles in development, commercial, corporate development, and oversight of international. Prior to joining Tanner Pharma, John worked for 12 years at Idis (and subsequently Clinigen) with roles in commercial, operations, and general management. John has a BA in mathematics from St Olaf College and a MS in statistics from Iowa State University.


Professor Das

Prof. Das is head of Paediatric Metabolic Medicine at the Department of Paediatrics, Hannover Medical School.

After undergraduate studies of medicine at Göttingen University he was a scholar of the ‘Max-Planck Society’ in Göttingen (Germany) where he worked in respiratory physiology, followed by a postdoctoral fellowship at the Department of Biochemistry, University of Oxford (UK) where his focus of research was the regulation of cardiac energy metabolism. Prof. Das trained in Paediatrics at Hannover Medical School and University Clinics Hamburg-Eppendorf (Germany). His special interest is in Inborn Errors of Metabolism and Neuropaediatrics with a research focus on energy metabolism, amino acid metabolism and lysosomal storage diseases. He has also obtained experience in Newborn screening at the ‘Screening Laboratory Hannover’. Prof. Das is adjunct visiting professor at the Department of Biochemistry at Kasturba Medical College, Manipal University (India).

Prof. Das is member of the scientific advisory board of the ‘German Society for Muscle Diseases’, the ‘Centre for Systems Neurosciences’ in Hannover and the ’Centre for Rare Diseases’ as well as the ‘Ethical Review Board’ at Hannover Medical School.


Roberta Smith

Roberta is President of the Alagille Syndrome Alliance, a Nuclear Medicine Technologist, Small Business and Nonprofit Consultant, and mom to 18-year-old twins. Roberta is deeply involved in the rare disease community. As a parent to a daughter with Alagille Syndrome, she is very passionate about helping parents and patients become better advocates. Along with that, Roberta supports and works to knowledge share, mentor, and assist other nonprofit leaders to help them achieve their goals with encouragement and compassion. She is currently working on her Master’s degree in Organizational Leadership. 


Dr Femida Gwadry-Sridhar

Dr. Femida Gwadry-Sridhar, founder & chief executive officer of Pulse Infoframe. Femida is a pharmacist, clinical epidemiologist, and methodologist with over 25 years of experience in clinical trials, patient registries, knowledge translation, health analytics, and clinical disease outcomes.  It was the challenges she witnessed in academia & research that encouraged her to start Pulse Infoframe. With a focus on oncology and rare diseases for the last 10 years, Femida and her colleagues took a collaborative approach to research, building networks of stakeholders from patient groups, sponsors, and researchers to solve the mysteries around drug development in a way that focused on evidence generation. Pulse Infoframe’s evidence generation platform healthie™ 2.0, is a scalable, centralized solution where registries are linked by commonalities across shared data elements. Recently recognized as a fellow of CIHR for her contributions to global research with impact. Femida has published over 200 manuscripts and abstracts in top tier journals and continues to support research to improve the lives of patients. 


Dave Caponera

Dave is currently the executive director, patient advocacy at Novo Nordisk.  Previously he was the executive director of patient advocacy and patient services at Dicerna Pharmaceutical which was acquired by Novo Nordisk in December 2021. He has worked for nearly 25 years as a specialist in patient advocacy and engagement both within the Bio-pharmaceutical industry with a focus on ultra-rare disorders and as an independent consultant.  He had been involved in developing programs for patients to access rare disease products in early access programs as well as approved product. His industry experience includes four years at Catalyst Pharmaceuticals, where, as vice president, patient engagement and access support, he developed a unique support program for a rare disease patient community that had previously limited access to treatment and educational resources. He has also held senior positions in patient advocacy, reimbursement and access services at Aegerion Pharmaceuticals, Pfizer, Amicus Therapeutics, and Genzyme. Dave is also a licensed respiratory therapist providing patient therapy.




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