The Problem

Orkambi is a drug for cystic fibrosis (CF) created by Vertex. It is effective for approximately 40% of all CF patients. It was licensed in 2015 by the EMA and is available in 12 different countries including the United States. In Britain, patients can receive it based on compassionate use.

Orkambi is priced at 240,000 US dollars per year, per patient (or 105,000 pounds in the UK). There is also a follow-up drug currently in clinical trials which is expected to be priced even higher.

Just recently its come to light that seven executives working with Orkambi gave themselves a paycheck so exorbitant, it would have covered the cost of the treatment for 369 CF patients.

When pharma develops a novel treatment, they can practically put whatever price tag they want on the drug, especially when they’re the only one who has the therapy in their hands. Price tags have become astronomical, and it’s forming a new precedent for all pharma companies. Headlines in healthcare have been increasingly focused on increasing stock or investor tips as opposed to patient lives.

A recent article, written by a CF patient, argues that the model that pharma has adopted isn’t just bad for patients, its bad for business. Lower prices could lead to faster decisions by regulators, which would lead to faster access, and ultimately, faster returns for the manufacturers. As it is now, healthcare providers across the world are struggling to keep up and patients are struggling to receive access to sometimes life saving treatment.

Cure vs Treatment

In addition to high prices, some pharma companies seem to believe that if they withhold data which could lead to a cure, they will ensure they continue to make profit month to month on treatment. The problem with pharma companies is that they are businesses, not philanthropic nonprofits. While there are plenty of pharma employees that care about the patients they are serving, the company’s ultimate goal is still to make money. Some companies believe if they ever find a cure, their profit will be significantly reduced. This thought process is based on the observation of other companies such as Gilead who created a cure for hepatitis C and then faced a significant decrease in their profit.

The problem with this? Human lives are literally at stake.

The approach to costs needs to change- quickly.

Why Pharma Needs to Focus on the Patient

Focusing on the patient and lowering drug prices could provide benefits to everyone involved. Increased patient involvement in pharma processes could-

• Provide insight into the most useful primary endpoints for clinical trials
• Increase patient accessibility to treatment
• Increase ease of patient recruitment for clinical trials
• Increase understanding and empathy concerning reimbursement issues

Additionally, a better relationship between patients and pharma would lead to improved trust and adherence regarding the treatments themselves. In order for this relationship to form, empathy is needed for both parties.

Rare disease patients are motivated to be more involved in their care. But if collaboration is restricted, neither party will reap the benefits. And if prices continue to soar, things could become detrimental for everyone involved.

You can read more about this take on astronomical rare disease drug prices here.