Bioinformatics and Beyond: July 2024

Welcome to the July Bioinformatics and Beyond newsletter!

I hope you are having a good summer so far, or if you are in Scotland like me, I hope you have a summer at some point.

For those of you in the Southern hemisphere, I should wish you a pleasant winter, but that feels odd considering some of your winter days are warmer than our summer days! I'm looking at you Australia, It's 17° Celsius in Sydney at time of writing (I write this newsletter in advance of its publish date), while it is 12° in Glasgow. Yes, 12°.

Anyway, rant over, let's get on with the newsletter. Today we are going to look at why gene therapies are so expensive, and how bioinformatics can help.

Why do Gene Therapies Cost so Much?

(And how to reduce gene therapy development costs with bioinformatics)

Gene therapies are expensive. So expensive in fact, that the world's most expensive drug is a gene therapy. It's called Lenmeldy and it is used to treat children with metachromatic leukodystrophy (MLD). Its wholesale cost is US$4.25 million.

You might be thinking that Lenmeldy is the world's most expensive drug, so surely other gene therapies cost a lot less, but it's completely normal for gene therapy costs to be in the millions.

So, why do they cost so much?

The answer isn't straightforward, there are many factors that influence gene therapy pricing. These include: research and development costs, manufacturing costs and market demand.

First of all, let's take a look at research and development costs.

R&D costs comprise of:

• Preclinical Research Costs
• Costs Associated with IND Applications
• Clinical Trial Costs
• Regulatory Approval Costs
• Recouping the Cost of Failures

Recouping the Cost of Failures

Are some of those surprising? When considering R&D costs, recouping the cost of failures may not be an initial thought, but it is a crucial one for drug developers. If a drug developer goes through preclinical research, the IND application process and even just Phase 1 clinical trials - they are incurring significant costs. Then, if during the clinical trial stage, their treatment fails for whatever reason, they have to start all over again - incurring more and more costs to, hopefully, finally succeed in developing a safe and effective drug.

All of those costs need to be recouped, so they are taken into account when it is time to determine the price of new gene therapy.

And that's only one element of the R&D costs, I haven't even touched on the others.

Market Demand

Then, after R&D there are manufacturing costs and market demand to consider. Let's take a quick look at how market demand impacts gene therapy pricing.

You already know that gene therapies typically cost millions of dollars, while drugs like paracetamol cost $5 or less for a pack. So why the huge difference?

Well, a lot of the difference can be explained by market demand. Paracetamol is one of the most widely used drugs in the world. In America alone, more than 60 million people consume it on a weekly basis. In short, it has high market demand. This means that when paracetamol is being priced, companies can divide the costs of developing, manufacturing, and marketing paracetamol, between the millions of people who will use it, resulting in a low price.

On the other hand, individual gene therapies tend to have low market demand. They usually cater to small patient populations with rare diseases. In fact, the total market for a gene therapy is usually only a few hundred patients at the high end of the scale. Meanwhile, some gene therapies are developed for just a single patient. So all the costs of developing the gene therapy can only be recouped from a low number of patients, resulting in a much higher price.

You may now be starting to understand why gene therapies cost so much, but I've barely scratched the surface of all the factors that influence their pricing. If you are interested, you can read a more in-depth overview of factors influencing gene therapy pricing on the Fios Genomics blog, here.

How Bioinformatics Can Help Reduce Gene Therapy Costs

For now however, let's take a look at how bioinformatics can help bring down the cost of gene therapies.

Since bioinformatic analyses support everything from target identification to optimising therapeutic efficacy, they can be used throughout the gene therapy development process to reduce costs. This is the case as, by using bioinformatics strategically, developers can speed up timelines and identify potential failures as quickly as possible, which helps minimise R&D costs. Also, when used early in the development process, bioinformatics can highlight new development paths which, in turn, helps with avoiding setbacks and increases the likelihood of developing successful therapies.

You can discover many more ways bioinformatics supports gene therapy development in our latest whitepaper: Advancing Cell and Gene Therapy Research with Bioinformatics, but for now, it is suffice to say that bioinformatics is a must-have tool in the tool box of gene therapy developers.

We have now come to the part that keeps me in a job writing these informative newsletters??: If you are involved in gene therapy development and would like to learn more about how bioinformatic analyses can improve your company’s gene therapy research, Fios Genomics can help. Our bioinformatics specialists are excellent at advising on the most suitable analyses to advance different gene therapy research projects. Contact us now and we'll be happy to help!

(My boss will be happy too - I'm going on holiday (vacation) soon and she'll be picking up my slack, so scoring some brownie points would be great right now ??)

Well, that's almost the end of this newsletter. But, while we are on the topic of holidays, did you know that going on holiday is good for your heart?

Women studied in the Framingham Heart Study who took the least vacation time - no more than once every 6 years- were 8 times more likely to have a heart attack than women who took 2 vacations a year.

Time for a holiday?

Thanks for reading!

-Breige McBride,?Content and Social Media Manager, Fios Genomics

Thanks for reading!

-Breige McBride,?Content and Social Media Manager, Fios Genomics