Insights from the 6th annual World Orphan Drug Congress 2015

By: Pritha Kuchaculla, MPH

On November 12 – 13, over 300 individuals representing pharmaceutical companies, biotechs, and payers and patient advocates gathered in Geneva, Switzerland. Over 100 attendees represented patient advocacy organizations, a record for the Congress.

On day one, following the opening remarks, we heard from Ségolène Aymé, coordinator and scientific secretariat for the International Rare Diseases Research Consortium (IRDiRC) a global consortium that was founded to address the need for global collaboration for research of rare diseases.  Dr. Aymé was excited to report that the patient voice is being heard and IRDiRC may hit its goal of delivering 200 rare disease therapies by 2020, earlier than it thought.

We then heard from, Henri Termer, former, chief executive officer, Genzyme who emphasized the need for companies to do more than “just put a vial up on the shelf”.  He stated “the rare disease movement is unstoppable” patients are becoming more involved than ever in conversations about the value of rare disease therapies.  In order to be responsive to the changing environment it is essential for companies to create a more effective and sustainable model in the development of rare disease therapies.  This model will involve understanding each patient, taking responsibility for addressing current unmet medical needs and acting in a transparent manner.

During the “How can payers, industry, academics and patient groups work together to create real dialogue and move away from transactional relationships to improve outcomes” panel we learned that NICE is considering new ways of evaluating value of rare disease therapeutics. One approach is to weigh the severity of a given rare disease instead of concentrating solely on its rarity.  Approaches like this one – and others that were discussed at the meeting by the experts – reflect increasingly constrained national health care budgets and rising orphan drug prices.  One panelist indicated that rare disease funding as currently constructed in England is broken.  For example, a Duchenne drug that was approved 18 months ago has yet to be given a decision by NICE, showing inability to deliver an appropriate – if any– decisions for patients in a timely fashion.

In addition, industry needs to take responsibility for initiating conversations with payers early – discussions after clinical trials are complete and data reported do not benefit anyone.  Industry also must include patient perspectives from the onset of clinical trials; too often outcomes are defined solely by investigators and the opportunity to collect meaningful PROs is lost.

Finally, when speaking to HTA bodies, industry needs to be honest about how it arrives at the price of its drug – giving an honest rationale is often times better than not addressing the issue at all.

We welcome your comments.

About the Author:

Pritha Kuchaculla, MPH

Pritha Kuchaculla, MPH, is an associate vice president at MK&A.  She can be reached at +1 212 620 2770 or at

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