We know all too well that, unfortunately, having a new medicine developed for a condition isn’t the end of the story. Just because a medicine exists, doesn’t mean everyone who needs it gets access straightaway.
Take Kalydeco. After Kalydeco was discovered, manufactured, and found safe and effective by the European Medicines Agency, we had to work hard to get it made available for use on the NHS. And we’re still working to widen access, be it for two to five-year-olds with cystic fibrosis (CF), with the currently licenced mutations, or people with the R117H mutation.
Then there’s Orkambi. Orkambi still isn’t routinely available for people with CF in the UK. Over a year ago, NICE found the cost of Orkambi put forward by manufacturer Vertex to be too high for its benefit. Since then, we’ve all been calling for negotiations and a deal. Because everyone who needs Orkambi, needs Orkambi now.
We’ve pushed and elbowed our way into the offices of people we hope can make a difference. We’ve put forward our own idea for sustainable access using our amazing UK CF Registry. We’ve emailed MPs, appeared in newspaper stories and on TV, written open letters, petitions, and held a simultaneous protest in all four UK nations. We need negotiations and a deal for Orkambi and Kalydeco, which are available right now, and for medicines that will be produced in the future.
Today, the Department of Health announced new ways that access to medicines could and should be speedier. The changes are based on the Accelerated Access Review (AAR), an influential government report. The AAR was also the subject of our Westminster debate in December 2016 where we highlighted the unacceptable delays people with CF are currently facing.
The changes announced today are themselves complicated, and we don’t know all the ins and outs of which medicines will be covered and when. We still need to keep elbowing our way into offices, emailing, protesting, and writing blog posts, vlogs, and open letters for CF treatments.
However, we are definitely pleased that there will be a new Strategic Commercial Unit to ensure the government has the best people to negotiate for fair and sustainable prices. We’re also hopeful for the Accelerated Access Pathway, because there is an exciting pipeline of new treatments for CF being developed, and we’ll need access to them as soon as possible too.
For now, we will remain optimistic but also slightly cautious. We won't stop until we have access to all medicines for everyone who needs them.
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