CORS Annual Conference 2021

Regulation and Access to Medicines in Orphan and Vulnerable Populations – Who Benefits, Who Doesn’t, and Why?

Regulations that are designed specifically to address market failure through incentives and/or obligations such as orphan and pediatric regulations, have had a substantial impact on the development of new treatments in specific therapeutic areas and target populations. However, they still have not done enough to direct the development in areas of greatest unmet medical need. There is a special need to foster the development and authorization of medicines for orphan diseases and vulnerable groups, including children, elderly adults, and poly-pharmacy patients. Yet, no clear strategy for addressing the unmet medical needs of vulnerable populations exists. Furthermore, while regulation has increased the availability of medicines, it has fallen short in assuring affordability and accessibility, which are also strongly influenced by external factors, such as the commercial strategies of individual companies, and the pricing and reimbursement decisions of national governments. In addition, the evidence base about how to use potential new medications is of central importance in drug development and treatment. Yet, vulnerable populations are often left out of clinical trials, which informs decisions about which patients to treat, at what dose, and with what other treatments, estimates of the benefits and risks of appropriate use of the drug, and further research in the field. This year, the CORS conference will focus on the question, who benefits and who does not from existing regulation, and why? Through discussions and presentations from invited speakers, we will identify and discuss the benefits and costs, the winners and losers in the current regulatory framework and explore the reasons for this from a multi-disciplinary perspective.

Conference Programme (Preliminary)

9:30 Registration and coffee
10:00 WelcomeAssociate Professor Christine E. Hallgreen, CORS, KU

The COMP experience: current and future challenges

Violeta Stoyanova-Beninska, Chair of COMP at EMA
11:00 Coffee Break
Session 1: Orphan regulation and access to medicines for rare disease patients, Chair Nina Christiansen, Leo-Pharma 

Review of the OD regulation: Unmet needs and challenges to solve them

Terkel Andersen, President of the Board, EURORDIS-Rare Diseases Europe

The EU legislation on rare diseases medicines: What is the way ahead to address the unmet needs of rare disease patients?

Vittoria Carraro, EUCOPE

Topic: Impact of the EU Orphan drug regulation, opportunities and challenges 

Anneloes de Ruiter, technopolis-group (TBC)

12:30 Panel discussion
13:00 Lunch
Session 2: Vulnerable patients, clinical trials and increasing diversity in the development of medicinal products, Chair Prof. Theis Lange, KU

The Imperative of Inclusion in Clinical Research

Prof. Barbara Bierer, Harvard

14:40 Coffee Break

Is there a legal requirement to address underrepresentation? – considerations on the “new” Clinical trial regulation and EMA strategy for regulatory science

Jakob Wested, Postdoc DKMA/JUR, KU

Paediatric regulations to foster the development and authorization of medicines for the paediatric population

Helle Christiansen, PhD candidate CORS, KU

Understanding cause and effect of pharmaceuticals through data science - title TBC

Kajsa Kvist, Scientific Vice President at Novo Nordisk

Topic: RWE in regulatory decision making

Nikolai C Brun, CORS Affiliate Professor, KU

16:20 Panel discussion
16:50 Closing remarks - Associate Professor Christine E. Hallgreen, CORS, KU
17:00 Refreshments

Sign up for the conference here.

Click here for livestreaming of the conference from the Adolf Hannover Auditorium.