Dare to meet the need in rare!

Coincident with the lengthening of daylight hours and the imminent forwarding of the clock, both signs that we are nearing the beginning of March, comes Rare Disease Day. This worldwide series of events occurs on the last day of February each year and is organised by EURORDIS, the European organisation for rare diseases. 

So who is EURORDIS and what is Rare Disease Day exactly? Well, EURORDIS is a non-governmental patient-centric alliance of almost 750 individual rare disease organisations; that number alone says a lot about the magnitude and scope of the problem and movement that is rare disease advocacy. It's underlined by the fact that some 50% of existing rare or orphan diseases affect children. 

In many ways, given the low prevalence of rare diseases (by definition, less than 200,000 patients in the USA), the best way to focus serious attention on the issue is to join forces and create a larger footprint between big pharma and their desire to fill their pipelines with blockbuster drugs for major diseases. Although there has been unquestionable progress in steering big pharma R&D in the direction towards rare disease drug discovery, the current controversy is primarily on how much those exciting new treatments should cost.

The political landscape for both big pharma and patient advocacy groups has been shaken (to see the least!) by recent administration changes south of the border, and given that Obama and Trump are not even as similar as chalk and cheese, no one is sure what will happen next. Apart from some occasional bleating, Obama was always pretty friendly to big business such as the big banks and big pharma, and it's far from clear how Trump is going to impact things. 

Some of these treatments for extremely rare diseases can cost exorbitant amounts while also being difficult to access here in Canada, which doesn't strictly have a rare/orphan disease policy or set of regulations. In fact, Health Canada doesn't even have a definition of rare diseases, even though they say they are working on developing an orphan drug regulatory framework that will encourage drug development and provide better access to existing drugs.

As stated above, the US-based criterion for orphan status is a current number of 200,000 patients at a given time. In Europe it is categorised differently, wherein a prevalence of less than 1 in 2,000 people is identified as a rare disease or disorder. The spectrum is pretty wide even inside that narrow-sounding category, such that in the EU one rare disorder could affect a mere handful of individuals while another may impact around 250,000 people. Further, it is estimated that in Europe as many as 30 million people suffer from one of a staggering 6,000 existing rare diseases. 

Canada's lower population in part explains lack of organisation around this issue, but by any measure Health Canada is lagging behind, badly, and needs to get some policy around this problem if patients are to have a chance at accessing such drugs at a remotely affordable price. In a recent example of the problem in Canada, the announcement by Marathon Pharmaceuticals that they were raising the price of a generic drug (deflazacort) for Duchenne Muscular Dystrophy (DMD) to almost $90,000 USD was only part of the problem. 

Marathon isn't even intending to market the drug in Canada, because the USA represents their target market, and it probably isn't worth their while promoting it's use north of the border. But $90,000 for a generic drug that costs mere hundreds for a year's supply, imported from elsewhere? So much for the era of Shkreli and Pearson being over! For their efforts, Marathon also received a voucher from the FDA, which inandof itself could be worth hundreds of millions of dollars. So it's (big) business as usual, in other words.

Donald Trump does have strong views on the whole pricing question, but he also loves big business and has no problem with savvy businessmen (himself included!) making bucketloads of money. The recent get-togethers with the CEOs of big pharma at the White House must have been fascinating in terms of such conversations, not least as essentially everyone is quaking at the prospect of Trump's take on not only big pharma drug pricing but also his choice of the next leader of the FDA. Moreover, tomorrow morning he sits down with the big healthcare insurers, such as Aetna, Signa and UnitedHealth, and that's another conversation with far-reaching implications.  

In any case, Tuesday is all about increasing global awareness of the rare disease phenomenon, and spreading the word on a cause that affects a relatively small group of people to the wider population at large. Improved access to existing medicines, at an honest cost, as well as stronger health policies and education around rare diseases, combined with increasing incentives  for focused R&D and drug development by pharma are all goals of the initiative. Last year's Rare Disease Day involved events in some 84 countries, and that's a pretty healthy level of participation. 

On that note, I will end this particular blog by sharing the video for this year's Rare Disease Day 2017, and wishing everyone involved their most successful event day yet!

Oak Island is a blessing, not a curse!

Although reality TV has become the scourge, if not the curse, of trends in television in recent years, every cloud indeed has a silver lining, and one can actually find some sapphires in the mud if one looks hard and digs deep enough. Speaking of curses and jewels and digging deep enough, one diamond in the rough (for me) is the fascinating "The Curse of Oak Island" on the History Channel.  

The story is essentially as much one of brotherly love as it is a treasure hunt, but the basic premise is that Marty and Rick Lagina set out to solve the 200-year-plus mystery of Oak Island in Nova Scotia, while hopefully becoming very wealthy in the process. For all of us who are still in touch with the younger boy (or girl!) inside of us, the story has much to offer - pirates, buried treasure, gold bullion, the awe-inspiring Knights Templar - and all this with a dreaded curse thrown in as the icing on the cake! 

I actually have my own frustrations regarding Oak Island because I drove from downtown Montreal to St. Margaret's Bay in Nova Scotia for a family vacation, and spent a wonderful time there, but sadly without any knowledge of such an intriguing mystery a few miles down the road from our oceanfront repurposed fish store. We drove through the Mahone Bay area several times, and must have seen Oak Island, but it was before the current reawakening of the mystery, so nothing was evident to the uneducated eye. It is a given that I would have taken the Jeep onto Oak Island to explore it, had I known!

We are now onto the fourth season of the show, and the elusive treasure remains, well, as elusive as ever. The brothers have been furiously digging deep into Oak Island areas with evocative names such as the Money Pit, 10-X, and the Swamp, among others. But as one false hope has turned into another real disappointment, it has become clearer and clearer that the brothers (and other team members) are having to dig deep within themselves, as much as into Oak Island soil. 

As predictable as the show can be in terms of producers ramping up drama with a potentially explosive find, the camera zooming rapidly down a dark, watery hole to hopefully reveal the discovery, and then, right into a commercial about Tide detergent! As appropriate as that is given the dirt all over the brothers, so far, the tension built up is inevitably evaporated within seconds of return to the show and one sees it was yet another false alarm. This trick might work the first few times, but with repetition it totally loses power, and one just heads to the kitchen to put the kettle on. 

Where am I going with this? Well, the thing that probably keeps me watching is both the human aspect of the production, and the relationship and (occasionally wavering) belief between the brothers, and team members, as well as the investment in both money and time that is being poured down 10-X or the Money Pit. No, I didn't say being poured down the drain!

Investment is investment, almost irrespective of the endeavour being invested in; nothing is ever certain and considerable risk is taken in placing money in harm's way, with no guarantee nor even the faintest signs of a return. And yet, in this case the two brothers continue on with pervasive passion, endless enthusiasm, tireless travails and a brotherhood borne of belief. This is what investors do on a daily basis, and at AmorChem we are not so different from the Lagina brothers. 

We are also a tightly knit team founded on belief; and while drug discovery might not quite equal a 200-year-old hunt for the truth, well, the 10-20 year hunt for new drugs is no sprint either! And even if we don't have to believe in any actual curse around our endeavours, it is an unquestionable truth that drug discovery and development is littered with failures, and many, many tombstones lie in the so-called "valley of death" between discovery and late stage clinical trials and subsequent marketing. Drug discovery percentage success rates are not that much better than sea turtle survival from egg to adulthood, and that's not even 1%! 

In parallel with Oak Island entrepreneurs, in many ways we are also investing time and money in what can appear to be deep, black holes, but there are jewels in there amongst the rocks and mud, and we believe that we can find one or two of them. This takes both belief and teamwork not only within, but also and primarily with our talented researchers in whose laboratories we invest in; we are all aligned in our very own quest to find new truth - truth that benefits mankind - and yes, to share in the profits of our collective labours if we get there. 

Notwithstanding the fact that we do and will face failures, we remain optimistic - why would one invest if one had a pessimistic view of the outcome? - and patient. Patience is a key word among investors, but my version of that characteristic would perhaps best be described (somewhat oxymoronically) as a patient impatience! There's nothing wrong with being impatient, when it comes to success we all are to some extent, but it must be tempered with a solid dose of Rome-not-being-built-in-a-day. Easy to say, harder to live by! 

I think what's important is acknowledging from the get-go that something is going to take a few years (big picture) to mature, and accepting that, but being pushy and even a little impatient on a daily/weekly basis (small picture) by trying to achieve the maximum possible. It's always surprising how quickly a blank canvas can evolve into something so much bigger when it is diligently worked on each and every day. Let the head deal with the big picture, while the heart pumps out daily energy and lifeblood for the small picture tasks and challenges. 

Perhaps surprisingly, one overarching take-home lesson learnt from watching Oak Island or while working at AmorChem is actually one and the same: as cliched as it sounds, it is the journey that matters. Success and wealth are great driving forces, of course, but if one is miserable for the several years (decades?!) it took to get there, was it worth it? When I watch the brothers on Oak Island face flooded digs due to the legendary booby trap flood tunnels laid down centuries ago, it remains clear that everyone is still enjoying themselves! 

The only way to be able to get out of bed and go get cold, wet and muddy on a frigid, desolate island is because you are filled with a passion; one that is not diluted by small defeats, and by realising that you wouldn't want to be anywhere else. Ditto investing in life science projects that will take many years to get there; frankly, the only realistic way of doing it is because you love what you do, on the daily basis, not just because of some defrayed dream of wealth in say 13 years. The journey may be a frustrating one but nothing worth having ever comes easy, as my Dad used to say, and the journey just has to be valued to its maximum. All the real fun is in the getting there! 

The journey is where all the shiny, bejeweled lessons reside, and one must take the time to see them and treasure them, even if they are obscured by the cold black earth of defeat and failure. Embracing failure may appear to be such a contradictory concept, but how many winners have never lived defeat? So many winners have stated that it was the bitter taste of failure that drove them ever harder towards the sweet smell of success. Ergo, failure can (or should) be seen as instructive. It's all about the attitude in the end!

Now then, it is Sunday today, which means tonight I get to escape for an hour with my team at Oak Island, and let the boy in me go off on an East Coast treasure hunt with the Lagina brothers; it certainly is therapeutic before another week in the office. I am fully behind the Laginas in their hunt for the truth about Oak Island; this is Season 4 now, so I am hoping that they find that truth some day soon and then maybe can return to warm offices instead of frigid swamps on Monday mornings. 

Ironically, I can only imagine how romantically they are all destined to look back on their days in the trenches of Oak Island if/when that day finally comes. Maybe that will be the real curse of Oak Island in the end - the solving of the mystery - leaving behind a hole almost as deep as the Money Pit or 10-X that fascinated them all so much!

Repatha Will Test the NewRepatha Will Test the New Drug Pricing Realiax Nisen Drug Pricing Reality

By Max Nisen