Interview: How to deliver value to patients

In Advocacy by Freya Smale

pharmaceutical value (ragesoss)

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 Bradley Campbell, Chief Business Officer at Amicus Therapeutics. Total Orphan Drugs would like to take this opportunity to thank Bradley Campbell for speaking to us.

Fostering open communication with patients helps usher cures to the clinic

“In the orphan space the most important criteria is if you are truly meeting an unmet need and delivering value to the patient.”  said Bradley Campbell, chief business officer of Amicus Therapeutics, a company that develops small molecules called pharmacological chaperones to treat lysosomal storage diseases such as Pompe and Fabry disease.

According to Campbell, one way to measure value is if you are meeting the design of your clinical studies. Unlike a major cardiovascular indication with well-defined endpoints and a clearly defined path to the FDA, rare diseases have no roadmap but can involve ongoing dialogue with physicians, patients, regulators and the manufacturer.

For example, instead of using the traditional endpoint of a six-minute walk test, biotech Biomarin negotiated with the FDA the new primary endpoints maximal inspiratory pressure (MIP) and maximal expiratory pressure (MAP) for its enzyme replacement therapy for Pompe disease. “I think that is an example of a manufacturer working closely with the agency and with investigators to come up with a way to measure the value that they bring to the Pompe patient disease course.” Campbell said.

According to Campbell, when Amicus was working on phase 2 of its migalastat monotherapy program, the company bolstered the traditional regulatory team with a medical affairs and advocacy team to work closely with physicians and patients to better design studies. Likewise, along with the usual medical advisory boards, Amicus established patient advisory boards to work with patients and voluntary patient organization leaders. When Amicus conducted phase 2 studies in Pompe disease using their small molecule chaperone to stabilize and improve the properties of enzyme replacement therapy, they needed to do muscle biopsies on patients. But as this was a single administration study, this meant patients would undergo an invasive procedure without direct benefit to them.

Amicus consulted the patient advisory board and the patient organizations.  “We were able to get a really nice handle from people who represented the community on how to operationalize that study.” said Campbell. “Without that trust established over time, we would not have been able to educate them on the importance of the scientific information nor would we have gotten that feedback.”  Patients agreed to undergo three muscle biopsies over the course of the study. “It is a benefit of it being small patient population that you can ask those questions, as long as the process is transparent and there is a give and take between patients and physicians and regulators.” said Campbell.

Using that feedback, Amicus used a needle core biopsy—still a large needle, but less invasive than the traditional muscle biopsy that the Pompe community was used to.  “Part of the discussion was that this is not the invasive biopsy that you are used to and that helped make them feel more comfortable.” said Campbell. “We walked patients through the protocol as if they were members of the medical community. While they wouldn’t benefit from the study from an efficacy perspective, they would certainly benefit as a community from a data perspective. We were then using that to design our next combinatiom study in Pompe patients that is scheduled to start at the end of this year.”

“For me it always comes back to unmet need.” said Campbell. “If you are truly delivering a product that meets an unmet need and can improve outcomes for patients I think the rest of it takes care of itself; agencies will walk with you down the road.”  Some other points Campbell made:

1. The relationship of price to value matters

Even though the individual price of an orphan drug is high relative to your average statin, the overall cost of any typical orphan drug is lower than most specialty pharmaceuticals, (such as non-orphan cancer drugs, human growth hormone). As long as you are delivering value and you get a label that is appropriate, as long as physicians are prescribing on-label then reimbursers are going to reimburse for that.

2.  To deliver value, build in access to pricing

While the reimbursement system in Europe is different from that of the US, the kind of traditional one-price model is you price it the same around the world and then ensure access through free drug or patient assitance programs for patients who can’t afford access otherwise.  Most orphan drug players out there charge orphan drug prices, but there are also robust patient access programs that go along with it.

3. Build dialogue in times of calm, for communication in times of trouble.

Aligning expectations is an ongoing process. Differing priorities and timelines between the drug developer and patients are an issue as well, as drug development never goes as fast as you need it to, and tomorrow is never soon enough. That being said, there are formal boxes you need to check in terms of the fundamental drug development process.


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