s the exciting Expanded Access Program (EAP) results creeps closer for us, I thought it was timely for a brief post on some of the merits of the EAP.
A very good evening to you Paradigmers....
The thing I like about PAR is that we do have 'a few irons in the fire' at the one time. This isn't a company that is just undertaking
one indication and we have to wait and wait and wait for weeks...for months before any news arrives and then again after that we wait again for the next lot of news. There are multiple things happening specially over the next year...overlapping events...overlapping milestones. And in amongst the somewhat known events are unknowns, like a peer reviewed paper, further data and always a looming possibility of a distribution deal. Don't forget it may not just be a deal on one of the big ones like OA and MPS, it could be one jurisdictional right/tie up...it could be Ross River Fever deal with perhaps with the DOD or some other entity.
We really don't know what's just around the corner in terms of positive news but the potential for overlapping news excites me, specially going forward when we get to the meatier part of the year (Q3/Q4).
In regards to the EAP, we don't know exactly the timing of the said result, it might be just a few short days away, it might be a few weeks away... .
Let's tonight investigate this program a little more.
DEFINITIONThe FDA have a compassionate use program. It's a program that allows a potential patient to access a promising drug that hasn't yet been officially sanctioned by the FDA outside of clinical trials. Also known as the Expanded Access Program, EAP for short.
High level of the EPA process we have just been through.CRITERIAEssentially to gain access to such a program the following criteria should be met:
- The patient has a serious disease or condition
- There is no comparable therapy to treat the patient
- The patient themselves cannot enrol themselves in a clinical trial
- The benefit justifies access for the patient to the EAP program
- There should be no interference with this program and the ensuing concurrent investigational trial that will occur.
Much work has already been done before this program was approved by the FDA. We find ourselves finally at the tail end of this process, the data is being compiled and cleaned I suspect as I type this post...PAR will now figure out how best to present it, who should it be presented by and in what format? Appropriate media channels will be involved, an exciting process to present our material? You bet.MERITSSo what's in it for us? Probably in my view the best way to think of at least one of the advantages is that it is like a Dress Rehearsal.
The dress rehearsal? Well it's still important to get this right...When actors and actresses have practiced and have memorised their lines, when they all know how and when to say their dialogues, the dress rehearsal serves to go through the motions simulating the actual production as close as possible. It's not just them, it's the whole team...Lighting...Sound...SFX...all the crew... In the same way I think of this like a dress rehearsal for our up-coming trial.
Yes our 'performance' just involves 10 patients..but the repercussions are still broad. It really is like the final night of practice that still counts before the big live performance of a Phase III. Butterflies? Kinda...I mean I know it will go well, the data will be the same or better as what we already know...it's just that there is a fair bit riding on it and it will be interesting to see how it all plays out.
DATAWell PAR collects valuable data on these patients. Yes we do have the SAS program but this also is additive. Not only that we get a feel for how things operate over in the USA, there can be differences and a lot of learning can happen with U.S. protocols, how the patients react and the best approach for things like recruitment and typical questions that are asked by the patient. There have been examples of this data being used to augment actual clinical data, "sponsor Bayer presented data not only from the actual clinical trial but also the company reported on data from 11 patients who received S
elitrectinib (aka LOXO-195) under the FDA expanded access single-patient protocol."
3PLANNING, MARKETING AND LABELLINGA program like the EAP can give PAR insight in terms of starting to think about marketing and labelling. EAP feedback and data will also give PAR an insight on global market access and ideas and strategies and would garner them feedback from local sources in the US. Think of it as a head start in drawing insights of just how the a global launch may play out for the PAR staff.
BRAND ADVOCATES Top Doctors are always researching the latest breakthroughs, once we go public with the data, and if the data matches what we have experienced so far, we will be picked up on the radars of these Docs. In fact the two leading Docs, namely Dr Mitch Marrow and Dr John Michels will have contacts and networks....these networks of Docs could very well become brand advocates and early adopters with this theme being supported by a white paper on the topic back in 2016.
4 Key Opinion Leaders (KOL's) will also be watching for these results; it is an important test for us. Don't forget once Docs are on board this will help immensely in the quicker roll out and material support for the usage of the drug and commendation to the patient.
FEEDBACKThe feedback obtained by these first ten American patients serves as real life feedback outside a strict clinical trial. The information can provide insights on the wider use of the drug by different patients and can also assist PAR in future planning for the eventual drug roll-out.
PUBLICITYWell the biggest one and probably the most obvious is the publicity we get as a result of the hopefully successful data. In this area we should attract the attention of quite a wide based profile, from potential future patients, to professional and amateur sports people through to the investment community.
EXAMPLEMozz, all very good in theory, but do you have any hard evidence? Ah Paradigmers, you guys ask the best questions: In a study conducted to determine the financial impact of the implementation of NPPs, it was found that the NPP had a considerable effect on the market share of the product during the 1st year after the launch. Not only that but this study found that the probability of spending $1 on the drug was an amazing 1.362 times greater for the drug supplied when the NPPs were implemented prior to commercial launch. A NPP stands for Named Patient Program and usually deals with the availability of a drug in a country where it hasn't been approved as yet.
MASTERSTROKEI don't know who came up with this idea of putting forward the idea of treating Aussie Footballers and the American Footballers as a targeted subset, but let me tell you, it was a
Masterstroke. These athletes are revered in society and they do put their bodies through much punishment for the entertainment of a lot of fans. Modern Day Gladiators? Quite possibly. But you can't deny that the focus is on them.....why do you think so many athletes and sports people are lucrative product ambassadors? At any rate it is the release of positive data from this subset of patients that should act as a draw card for us in terms of renewed interest and new larger funds having us on their radars. It will certainly bring new investors on to our books and demand for our shares should in theory, go upwards.
DYOR as per usual.
REFERENCES1]
https://www.fda.gov/media/119971/download2]
https://www.fda.gov/news-events/public-health-focus/expanded-access3]
https://www.trialsitenews.com/increased-use-of-expanded-access-program-data-drug-sponsors-capitalizing-on-real-word-evidence/4]
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4763516/
(20min delay)