Orphan Drugs Asia Live: Significance of early access to orphan drugs

In Orphan Drug Congress, Orphan Drugs Live by rachel

Mark Corbett, Senior Vice President , Clinigen Global Access programs opened proceedings at the second half of day 1 of World Orphan Drug Congress Asia 2014 by touching on the significance of early access to orphan drugs

Clinigen is a fast growing specialty pharmaceuticals and services business with offices in the UK, US and Japan, serving more than 50 markets globally with an extensive customer base of pharmaceutical companies, biotechnology companies and contract research organizations.

In his keynote presentation, he brought us through an in depth discussion on the rationales, challenges and options available behind early access to orphan drugs.  What is early access? It is the process or mechanism that exists for patients with unmet medical needs and whereby treatments or medicine are  not commercially available in the market or approved in their country. These patients are usually classified under rare disease patients who require prompt treatments  and access to medicine.

Some market access challenges and considerations he raised during his presentation include:

  • Control
  • Ethical issues
  • Complex regulatory mechanisms
  • Uncertainty of demand
  • Resourcing
  • Collection of data

All these factors affect early access to orphan drugs as patients and physicians will have greater information towards rare diseases and orphan drugs respectively. Patient can better understand their condition and types of treatments available. Informed physicians will know of medicine coming through clinical developments before they become commercialised and this will hopefully spur more uptake of these orphan drugs as viable treamtent options.