Are HTA & payment decisions impeding access to rare disease therapies?

jeannie joughin, csl behring, orphan drugs, rare disease, market accessIn a return to our Speaker Interview Series, where we spoke to all 60+ confirmed speakers of the 2012 World Orphan Drug Congress in Geneva, we are delighted to report on the insight of Jeannie Joughin, VP Business Development, CSL Behring and to also share her perspectives on the pressing issues currently facing the orphan drug industry at large and set to be debated at the congress in November.

"The most important areas of debate are how to appropriately differentiate the treatment of rare disease therapies from regulatory policies through to payment and market access for patients. It is recognized that there is a need to recognize the unique challenges in rare disease therapy development in policies (orphan drug acts, accelerated approvals, etc.). However, the spread of health technology assessment (HTA) and payment decisions can impede access to the therapies. Payment regimes need to address the special needs for rare disease therapies in developing HTA and reimbursement decision, such as the seriousness of the conditions treated, the small populations and the societal benefit of treatment, not strictly a quality adjusted life year model."

It is always intriguing to try some more outlandish thinking, so when I posed the hypothetical question of if one thing could be changed/improved in order to speed the development of rare disease treatments to reach patients in need, what would it be?…

"Allowing more patient input on the risk they would be willing to tolerate with a therapy versus no treatment at all. The Safety and Innovation act signed into law in the US in July 2012 formally requires greater ability to patient input into these decisions. Patients are the key stakeholders in all of this and have a need to be heard."

As VP of Business Development at CSL Behring, Jeannie Joughin will be speaking in the Corporate Development & Partnerships Track and presenting specifically on "How larger organisations are viewing small biotech as innovative co-developers".

The brochure can now be downloaded using this link and if you have any comments to add in terms of the most most debatable point of the industry, join the discussion below.

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