World Orphan Drug Congress LIVE: Drugs and Sustainability

In Advocacy, Clinical Development, Market Access, Orphan Drug Congress, Orphan Drugs Live by Freya Smale

Our keynote speaker Stephanie Okey, Senior Vice President and Head of North America, Genetic Diseases and US General Manager, Genzyme started her 11am session on how good medicine equals good business. An effort is being made to transform lives and meet unmet needs. During her presentation, she pointed out a couple of issues including:

  • How can we sustain the rare disease business taking into consideration the payers’ perspective
  • Developing medicines for and with the patients as a focal point
  • Articulating the clinical value of orphan drugs

Genzyme’s enterprise is formed by a patient center focus. The biggest concern is the level of sustainable care in the US. It’s all about getting the right medicine to the right patient at the right time with the right dose. If every patient was able to get an early diagnosis and overall access to information needed to support their issues, we could generate sustainability in the rare disease business. The question Stephanie also points out is how can we partner even more closely so there is truly sustainable care? She gave an example of when Genzyme received an email from a family dealing with their child’s diagnosis of NPS 1. The family was not given any options of paths to take by health care providers because the disease is so rare and resorted to contacting Genzyme. Genzyme immediately appointed them to a local patient advocacy group and gave resources to them. The process starts by learning from patients and being positive in this rare and scarce area. Stephanie mentions using social media, big data, and technology to bring these issues together and spread awareness and information.   Recap on The Great Debate: Is the price too high? AGAINST: First there was rare, now there’s ultra-rare. And it seems every other disease is rare and every other drug orphan. We won’t be able to afford it – period. Ed Pezalla from Aetna is concerned with whether or not there will be continued access to medication. Who is paying for these costs? It’s not a terrible thing that we are spending money on healthcare, but it just means we won’t be able to spend that money on other parts of our lives. He is talking about the taxpayers at this point. “Wages are static and healthcare is not,” Ed quotes. It is not just a matter of profits between companies, but it’s how we are spending our money. As a community, we ant to pay for life improving drugs, but the plan is mainly to let everyone have access to it. Complicated (rare disease) drugs take up 1% of produced drugs, however it’s 50% of the costs of overall drugs. We must think of better ways of funding and add a little creativity to it. Jeff Myers of Medicaid Health Plans of America mentions that the generic side of old drugs are increasing in price. There are products out there that are 100, 200, 300 thousands of dollars and the prices are just increasing. It’s completely unsustainable because the prices never goes down, only up, and it brings the automatic reaction to, “we can’t afford it.” No other market in the world functions like this. On the other side of the debate, Douglas Paul, PharmD, PhD Vice President and Partner, Medical Marketing Economics, LLC, argues. FORThe orphan population is so small the rest of the population can more than cover the cost. The prices of these drugs are what makes it sustainable and attractive. The high costs are necessary for innovation. Solutions in cutting 50%  of prices won’t help in this industry. Doug quotes, “Are we going to fund innovation or not?” These panelists were each given one minute to come up with a solution for their sides taken. Doug mentions to take on better planning steps, for instance when an orphan drug comes in with a great discount, payers should take advantage of it before the prices increase. Ed suggests to have development of drugs to come with some sort of equity that can be owned. Jeff quickly mentions that drug companies should start taking risks in drug development just like every other segment in healthcare. As a side note, this is the first time a payer has mentioned “ultra orphan.” Overall a great morning session of keynote speakers.