Interview: Paving the way for a whole new medical paradigm

In Market Access by Freya Smale

imageAs part of a series of interviews conducted with leading industry experts in the run up to the World Orphan Drug Congress in Geneva, Total Orphan Drugs was delighted to sit down with Hans Christian Rohde, Chief Commercial Officer, uniQure. Total Orphan Drugs would like to take this opportunity to thank Hans Christian Rohde for speaking to us.

Debuting a truly game-changing therapy means thinking beyond the orphan drug box,
according to uniQure Chief Commercial Officer Hans Christian Rohde

As the first company to get a gene therapy approved in the Western world, Netherlands-based uniQure is a pioneer on multiple fronts. In October 2012, the European Commission approved the marketing authorization for uniQure’s lead product, Glybera for lipoprotein lipase deficiency (LPLD), a rare, inherited disorder. Patients with LPLD cannot metabolize large fat-carrying particles called chylomicrons, which can lead to painful inflammation of the pancreas (pancreatitis), a potentially lethal condition.

Market adoption of a novel treatment paradigm requires a focus on educating patients and health care providers. As Glybera is a single intervention of intramuscular injections, according to Rohde, it should be weighed differently by the healthcare system and regulators than a drug that involves repeated, ongoing treatments. The final cost of the drug is under negotiation. “We need to think outside of the orphan drug box in determining value.” said Rohde. “As Glybera involves a one-time administration, it is not expected to use the same mechanisms of cost effectiveness. A number of patients will not be coming back to the hospital.”

The regulatory basis consisted of three open-label clinical trials in which uniQure administered Glybera to a total of 27 adult LPLD patients. “The patients experienced a significant reduction in newly formed chylomicrons, as well as reductions in acute pancreatitis resulting in a significant reduction in the number of hospitalizations.” said Rohde.

The drug was well tolerated, even after patients were followed for up to 3 years. The most common adverse event was general muscle pain at the injection site occurring in approximately one third of patients.

That is just the beginning. uniQure is expanding its gene therapy platform to other conditions. “Imagine if you have people in perhaps 10 indications, what does that mean for society, for our healthcare structure?” said Rohde. “Finally there may be a treatment for many diseases that would not otherwise have a treatment at all or a treatment which provides the patient with a potentially lifelong benefit. It will have a major impact on how our primary and secondary healthcare will look like going forward.” uniQure is also in the process of building a patient registry to collect ongoing data as part of the EMA approval of Glybera under exceptional circumstances.

Rohde notes that European and US markets are somewhat different in terms of the role patients play in orphan drug development and approval. In Europe, patient advocacy organizations are much less organized than those in the U.S. The U.S. Food and Drug Administration frequently solicits feedback from patient advocacy organizations when evaluating orphan drugs. This is less the case in Europe.

I think you should never forget the patient,.” said Rohde. “Gene therapy has so far changed the course of the lives of those patients who participated in the clinical trials.” According to Rohde, it is important that patients and patient groups understand the safety and efficacy of the drug as a one-time treatment, given that no other treatment for this condition exists today. “Apart from the administrative injectable side effects there haven’t been observed any serious side effects.” said Rohde. “However, the patient has to agree that this is a treatment that they want to have.”

1. Your drug may be rolled out globally but all reimbursement is local

The pricing and reimbursement process can vary tremendously from country to country in Europe. Obtaining P&R approval in Europe has potentially a different set of conditions than approvals in the U.S.

2. Communicate with regulators early and often

It is important to talk with pricing- and reimbursement authorities early on to ensure a constructive dialog as this paradigm is new for both parties,- it is also clear that there is as yet no structured regulatory path for gene therapy. “We had a bumpy road for the approval of Glybera.” said Rohde. “This is the first approved gene therapy treatment in the West and was recognized as a learning experience for both parties, potentially paving the way for approval of similarly medicines in future.” We are pioneering the path with the regulatory authorities, and doing the same with the price and reimbursement.”

3. Get strong support from Key Opinion Leaders

As this is breaking new ground it needs strong support from Key Opinion Leaders (KOLs) – the researchers who are driving knowledge acquisition and dissemination in the field. This is particularly critical in orphan diseases, where small populations can be scattered around the world.

Company Profile

uniQure is a world leader in the development of human gene based therapies. uniQure’s pipeline of gene therapy products in development, in-house or with collaborators, comprises hemophilia B, acute intermittent porphyria, Parkinson’s disease and Sanfilippo B. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what it believes to be the world’s first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases.


image_thumb4_thumb_thumbSpecial thanks go to Wendy Wolfson who conducted the interview on behalf of Total Orphan Drugs.

Wendy Wolfson covers innovations at the intersection of medicine, science and healthcare as a columnist for Chemistry & Biology, a publication of Cell Press. She has contributed to magazines including Science, Nature Biotechnology, the Lancet, and Red Herring. Her work can be found at and she can be contacted at