The 2017 Deal and Trikafta: A Preliminary Analysis by CFI

A few CFI members have asked us to help clarify the ‘pipeline aspect’ of the HSE/Vertex agreement OF 2017 and how this relates to the forthcoming triple combination drug Trikafta (which might be renamed for the European market) and in particular the position of those outside the pipeline/portfolio deal of 2017.

Please note: We give this information with some caution as we are currently seeking definitive clarification and updates from Vertex and the HSE. CFI does not have access to the full agreement, so this is our best understanding of the 2017 deal. We would welcome comments or corrections if your read is different.

The Pipeline deal of 2017 (also called the portfolio deal) not only resulted in the reimbursement of Orkambi for 12 year olds and over and the extension of Kalydeco to 2-5 year olds, but also covered possible future extensions of Orkambi to younger ages (which has happened in the interim) and it also covered Symkevi (subsequently reimbursed in 2018, but not specifically named at the time of the deal in 2017). In short the 2017 deal provided access to Orkambi and it extended Kalydeco but it also provided some important future proofing of CFTR drugs that were likely to developed over the lifetime of the 2017 deal (10 years).

CFI pushed for and supported the pipeline dimension during the negotiations and we acknowledge the positive role of Vertex, the HSE and Minister Harris in this development. 

However, it is important to recognise that the pipeline was very positive and innovative development, but it is not (as we have stated previously) a panacea for resolving all future CF drug therapy reimbursements. 

CFI understands the HSE/Vertex agreement currently covers:

• Orkambi for all CF patients 12 years and older who have two copies of the F508del alteration
• Expands access to Kalydeco for children aged 2 to 5 with any approved gating alteration (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D)
• Access to Kalydeco for people aged 18 and older who have an R117H mutation
• Rapid access for people with all the above alterations (ie: two copies of the F508del mutation, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D, R117H) if the labels of the existing medicines are expanded to cover additional age groups and if new Vertex medicines are approved for these populations [NOTE: FURTHER GENE ALERATIONS HAVE BEEN ADDED SINCE 2017- CFI IS SEEKING COMPREHENSIVE UP TO DATE INFORMATION ON THIS]

From our understanding, under the 2017 deal If a drug therapy is developed for the gene alterations not listed above (or subsequently added in), there would have to be further discussions between Vertex and the HSE as and when required. In effect this means that those who are presently entitled to Kalydeco, Orkambi and Smykevi should also be entitled to access to Trikafta, or whatever it will be called in Europe when it is approved by the EMA.

CFI estimates that as a result of the 2017 deal and the previous Kalydeco agreement of 2013 and the subsequent access to Symkevi and predicted future access to Trikafta that as much as 90% of the CF population in Ireland (to be confirmed) will possibly have access to a bespoke CFTR drug therapy within the next year.

CFI’s approach and commitment to those without a CFTR Drug: No one Left Behind

CFI is currently seeking further clarification and confirmation on all the above and we will keep you updated and will amend this information if necessary. Just to stress that this is our understanding which needs to be confirmed with Vertex and the HSE.

Needless to say, CFI will continue to adopt a robust approach to potential reimbursement of future drug therapies including in particular for those who do not have a bespoke CFTR drug at present, provided CFI is convinced on the efficacy, safety and impact of such drugs. In short, CF patients without a bespoke CFTR drug in Ireland will remain an absolute priority for CFI.

It should be noted that for some rarer gene mutations/combinations of mutations there might never be a CFTR drug that impacts positively on their condition. However as you know there is much investment in research for other possible CF therapies such as gene editing/CRISPR and Ireland is making a significant contribution to such research. It is also important to note the participation of specialised CF centres in Ireland in the clinical trials for most of the Vertex suite of drugs, including follow up/post market research on CFTR drugs that are already available.  

It is worth noting the indirect impact of the reimbursement of Orkambi and Smykevi (and hopefully Trikafta) is to encourage further research into ground breaking CF drugs. Further, the CFF in the US continues to invest millions of dollars into other therapies, including for those without a bespoke therapy at present. In short the CF drug therapy agreements in many countries, including Ireland, have had an important positive impact on the investment/search for the inclusion of all and indeed in possible future cures.  These are major steps forward but there remains so much more to do, including improving our services in Ireland, building on achievements to date.

Timescale for Trikafta

Before Trikafta can be considered for use/reimbursement in Ireland it will need to be approved by the European Medicines Agency. This will take some months. Following on from the experience in the US where the FDA fast tracked Trikafta 5 months ahead of schedule, it is likely the EMA will consider fast tracking for Europe, but we have no precise timescale at present as to when it will be available in Ireland for this reason.

Incidentally the 2017 pipeline/portfolio deal for Ireland is also sometimes referred to as the ‘Netflix deal’ by some Pharma analysts as it refers to access to suite of existing and forthcoming drugs based on genotype, rather than just a named, existing drug. This deal has thus featured in many recent worldwide conferences on access to orphan (rare disease) drugs. The Irish pipeline/portfolio deal has also helped gain access to CFTR drugs in other countries.

As with the introduction of Kalydeco, Orkambi and Symkevi, it is vital that patients seek the advice of their clinicians about possible changes in medications to enable informed choices to be made if and when the time arises.