$120m - will it help, and a look at the greater issues

The last few months have consisted of a blur of meetings, round table discussions, presentations, and impromptu conversations on the train and at universities - all largely revolving around a few key topics. There's been the open science and knowledge sharing in the sciences dialogue, data policy and drafting codes of conduct, and innovation and access models for rare and neglected disease research.

The last of those topics surfaced this week, after catching an item on Scientific American's 60-Second Science Blog. The post deals with drug development and NIH's recent commitment of $120 million over five years for drugs and therapies for rare and neglected diseases. Woo hoo. I support that. But the real question, surfaced in this post as well - will that make a difference?

One of these meetings I attended over the last 3+ months of travel was a recent summit on innovation and access by the National Organization of Rare Diseases (apparently "neglected" is implied? :) ) The crowd - independent disease foundations that were members of NORD, FDA representatives, folks from the Office of Orphan Products, biopharma, and other medical professionals, among others. It was their annual meeting, and as somewhat expected, included an initial 1.5 hour "congratulations" and offering of thanks to one another for their "contributions to rare disease research" as a kickoff to the meeting.

Excuse me here for being cynical and a bit brash, but were they congratulating one another for a drug pipeline that a) is insanely costly, b) takes approx. 17 years to get a drug to market IF it succeeds, c) doesn't work in favor of the community they're serving? The fact that a significant portion of patients afflicted with the 6,800 + diseases characterized as "rare" and/or "neglected" use off-label drugs or find other ways to circumnavigate not only the prices of these therapeutics but also the FDA and approval process doesn't seem like a win. Good for finding a way to alleviate symptoms, helping people potentially live longer, but not a win for bringing the price of these treatments down and getting them into the hands of those that desperately need them.

Trust me, even as I sat there in the audience, I was able to find out about a FDA approved hypertension drug that has shown positive results in Marfans patients (which a dear friend of mine has) to maintain muscle growth and lower / maintain TGF-β signaling levels. Some are getting this through basic hypertension diagnoses and prescriptions, whether they have it or not. Talk about working around red tape.

Now, I fully realize that drug companies need to make money, and that a portion of that comes from selling drugs for diseases that affect a large subset of people. The money and logic just isn't there, strictly thinking of $$$, for those diseases that instead of affecting 500,000 a year may only affect 100. I get that. Clinical trials are difficult to conduct with a small sample size. It's difficult, with how expensive it is to bring a drug to market (approx $1 billion), to get any of that cost back. But not admitting that there's a problem with the pipeline? Really?

After the first set of speakers and a keynote by Gov. Tommy Thompson (WI), the moderator opened the floor to a few comments from the audience, giving us each a few minutes to make remarks on the day. At this point, it was two hours into the event ... without mention of "innovation" or "access" outside of "access to $200,000 therapeutics".

For those familiar with our work at Science Commons (and the above opinions are certainly influenced by that work, but are still my own), you know that the concepts of "innovation" and "access" are integral parts of our mantra, our raison d'être, our everyday work. But it seemed like somehow, in the midst of all of the congratulating for thousands of people in their "network" not having any sort of drug or treatment, these two words - the themes of the event - went unmentioned.

I raised my hand to have a turn, puzzled by all of this and made a comment, which was later backed and echoed by Janet Woodcock of the FDA and Francis Collins, the famous geneticist (thanks to both). I talked about the reasons we were all there - to talk about "innovation" and "access" in terms of access to research, accelerating scientific discovery, new "innovative" models to help fix this broken pipeline we all were dancing around, and get therapeutics and results to patients faster, cheaper and more successfully. It was astonishing a) how many people were nodding and smiling when I brought this to the forum and b) the fact that if not said, it may have gone unmentioned for the rest of the meeting. All of a sudden, the tone changed - with Francis Collins emphasizing the importance of Open Access and Janet Woodcock even saying "Put information into the public domain".

Small wins in an area that still needs a bit of coaching (like others, certainly) on making better use of a poorly funded wing of disease research.

Will $120 million over 5 years make a difference? Certainly, in some respect. How large of a difference depends on what model is constructed to hopefully better share the scientific knowledge we're pumping tens of hundreds of thousands of dollars into, the funding model, etc. Perpetuating the "walled garden" approach does not "fix" the system. Offering therapeutics that patients cannot afford does not solve the problem. And 17 years (at a minimum) and $1 billion per drug is just not acceptable. All need to be further addressed, because at the end of the day, in terms of drug development, $120 million is barely a blip on the radar screen.