Estimated reading time: 4 minutes

Turns out being self-employed means making difficult choices, even about which streaming platforms survive the inevitable belt-tightening of the early business years. With fewer options, I’ve finally been binging shows I missed the first time around. Currently I’m on The X-Files.

When I was at university, The X-Files were a Sunday night ritual in my residence hall, even if one that I declined to participate in, being a pseudo intellectual then who pretentiously tried to shun pop culture. Now I’m making up for lost time.

The series is not without its flaws (a whole separate article could be written there). I was surprised to find rare diseases and gene therapies cropping up as storylines in the first season, and not least in the episode,Young at Heart.

Here’s the gist of the episode (warning, spoiler alert!).  A rogue doctor is working on an experimental gene therapy, and he thinks he can reverse the aging process. He is unethically experimenting on children with Progeria (an extremely rare genetic disorder that causes rapid aging) and later, he extends this to prisoners, in a quest to refine his treatment. He ultimately succeeds in reversing the aging process! But (there is a big but) this makes the adults he successfully made younger now vulnerable to childhood diseases, and they develop, er, rare cancers, for some implausible pseudo-sciency-sounding reason. 

One of these prisoner-patients somehow manages to steal the research from this unethical doctor and makes his escape from prison—and it seems the doctor can’t reproduce his technique without the stolen papers. The government gets involved (of course) because they want to control the secret gene therapy fountain of youth themselves. Lots of people are pointlessly shot—new characters are introduced just to be killed off. Etc. etc—you get the picture.

Photo credit: istock.com/PepeLaguarda

As implausible as the plotline is, it raises a good question about the extent to which science fiction like this has unconsciously primed us to be sceptical, and even afraid, of the processes that lead to biomedical advances. This question is especially relevant now, when gene therapies are once again in the headlines at a moment when rare disease therapy development already faces significant headwinds. Our sector badly needs some good news. And maybe we have found it with theEuropean Life Sciences Strategy.

Positioned as part of the EU’s competitiveness agenda, the strategy is essentially a plan to re-establish Europe as a global leader in life sciences, broadly defined to include health, biotech, agriculture, food and environmental science. Encouragingly, it explicitly recognises rare diseases as drivers of innovation. This strategy, while not unexpected, feels re-energised in the context of what many see as a ‘hostile environment’ for life sciences investment in the United States under the current administration.

At its core, the strategy has three prongs:

1. optimising the research and innovation ecosystem, including funding multi-country clinical trials and establishing ATMP (advanced therapy medicinal product) centres of excellence.
   
   
2. enabling rapid market access for innovations, with a new EU Biotech Act to create a more ‘innovation-friendly regulatory framework’.
   
   
3. boosting trust, uptake and use of innovation, by tackling misinformation and increasing public trust. (For those who grew up on The X-Files, this last one feels particularly important!)

If you want to dive deeper into what this means for rare diseases,EURORDISandEFPIAhave generally been excellent in articulating the opportunity.

For our community, this feels like a real chance to unite behind something concrete. I remain a believer in the European Action Plan for Rare Diseases, but the reality is that it may be dormant for the foreseeable future. The Life Sciences Strategy gives us a way to keep momentum alive by repositioning our advocacy around a framework, where, at least for now, we’re pushing at an open door.

To make sure that the potential benefits for rare diseases are realised, we will need to be unified and visible as an ecosystem and develop effective ways to make sure the necessary things happen.

There’s reason for hope: the future of life sciences is not only out there, it is within our reach.

Connect with Michael

• LinkedIn

in the know brings you the latest conversations from the RARE think tank. To access more in the know articles click below.