Interview: Surmounting barriers to patient access

In Market Access, Partnering & Investment by Freya Smale


As 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 Dr. Durhane Wong-Rieger, president, Canadian Organization for Rare Disorders. Total Orphan Drugs would like to take this opportunity to thank Dr. Durhane Wong-Rieger for speaking to us.

Focusing on the most appropriate care for the patient also means talking about value, according to Dr. Durhane Wong-Rieger, president, Canadian Organization for Rare Disorders

Getting patients access to orphan drugs involves surmounting a number of barriers, according to Dr. Durhane Wong-Rieger. Producing these drugs for small patient populations is very costly, from research and development to finding patients to participate in clinical trials, dealing with challenging endpoints and unknowns in terms of safety and natural progression of disease.

“Given all that, we have to deal with the fact that many of those drugs are coming in with very challenging individual price tags.” said Wong-Rieger. As more drugs are developed for different diseases and introduced into the Canadian drug plans, it makes it more difficult to build the rationale or make the case that these prices are sustainable.

“Success breeds its own challenges.” said Wong-Rieger. “As development transitions from start-up biotechs to larger pharma, I think there is the expectation that these bigger companies are going to be introducing these drugs at lower prices. We all know you are not trying to make your entire overhead out of this one drug. The expectation is that the drugs should become a lot more affordable.

I think the big question everyone is asking is where are these prices based on?

Canada does not subsidize drug development or provide tax incentives and loans. “You do have government sponsored research programs in the academic institutions but as we all know, it is a big step from identifying a gene or even a viable candidate molecule in an academic setting and actually translating it into a drug to market.” said Wong-Rieger. “It is a pretty tenuous kind of a chain to suggest that we are funding the academic institutions and therefore we are indirectly subsidizing a drug even if it is in that same disease category.”

The value of a drug has to be seen a broad economic perspective, according to Wong-Rieger. Unfortunately, we do not offset or subsidize the cost of drug development even though it may create other industries or employment opportunities or tax revenues.  “Moreover, just because [the healthcare systems] is not funding the drug does not mean that we are not paying patient costs.” said Wong-Rieger. “The real question is what is the value of a patient being able to work and take care of his/her family. Ethically, do we penalize the person if he has a disease that is less common?” The value of allowing a patient to work is incalculable. “As a strict “return-on-investment”, nobody is worth $300,000 a year.” Wong-Rieger said. “No one would deny that is a lot of money to pay for one person. But as a society we don’t discriminate on that basis.  Do we really want to go down that path for patients with rare diseases?”

1. Patients have responsibility to be informed partners

To achieve appropriate responsible use the community needs long-term progressive access programs including post-market monitoring, outcome monitoring, and informed agreements with patients that they should not take drugs if they don’t achieve planned outcomes. “In order to give each patient the most efficient and appropriate care available.” said Wong-Rieger, “we must always start with the individual patient. The patient must come on board as a responsible partner. We need to have this discussion with the patient community.”

2. Infrastructure for secure patient registries is needed.

Wong-Rieger says that patients are willing to contribute their DNA, blood samples and personal information, but they must be assured confidentiality and privacy protections.  “Currently participation by the patient community is limited by the lack of infrastructure.” said Wong-Rieger. “We need common platforms to share data securely across jurisdictions. We also the right legal protections if we want patients, especially children,  contribute their information.”

3.  Transparency about goals for use of data is important.

“Patients give you their samples, trust, information.” said Wong-Rieger.  In return, they expect the research goals and use of data to be transparent and to be informed about progress or roadblocks.”

imageSpecial 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

Read more interviews in this orphan drug series
Partnering from the pharma perspective
How to deliver value to patients
Perspective from patient advocates in Sweden
Interview: Surmounting barriers to patient access
Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked
Where do the current and future challenges lie in bringing new orphan drugs to market?
Bringing Biotech Spirit to a Non-Profit Environment
Paving the way for a whole new medical paradigm
Tailoring your reimbursement strategy, step by step, country by country
An Interview With Pfizer on the Orphan Drug Industry: Part I
An Interview With Pfizer on the Orphan Drug Industry: Part II
An Interview with the Chair of the IRDiRC
An Interview with an Orphan Drug Payer