Patients are the platform
Patients are the true limiting reagent in the crucible of drug development. It's time we recognize this reality, and build a care-to-cure system that rewards patients with both cures and cash.
When the audience is the general public - think Super Bowl spot, People magazine ad, or NYT op-ed - the biopharma industry highlights patients: putting patients at the center, leaving no patients behind, and other marketing me-too formulations.
But when the audience is other biopharma insiders - think the annual JPM Healthcare conference in San Francisco or a STAT article about a VC-backed NewCo Series A announcement - the keyword shifts to platforms: discovery engines, pipelines-in-a-product, and other variations of the “multiple shots on goal” paradigm that has dominated industry practice for decades.
On the surface, both messages sound like corporate-speak. The patient-first messaging feels like overcompensation for opaque drug pricing practices and runaway inflationary healthcare costs that drive the public berserk, while the platform-first messaging glorifies whiz-bang tech over patient access to affordable medicines in the real world.
Neither message touches the industry’s third rail: financial compensation and upside for patients whose participation in clinical trials creates the data packages that lead to regulatory approvals and the ability of commercial sponsors to sell new medicines. It’s simple business math: no patients, no medicines.
But there’s a chance to unite this house too-long divided by recognizing that both messages are really saying the same thing:
The Patients Are The Platform.
Patients have always been the platform. That deep insight has been obscured by a top-down business model that was never designed by patients, but takes patient participation and lasting buy-in for granted. After all, they reason, patients can’t possibly create cures by themselves, they will have to work with the professionals.
But what happens when your disease is too rare for Big Pharma to invest in? The hard truth is that if your patient community is too small, you’re on your own. Not enough patients, no medicines.
Biopharma talks a big game about patients in the streets, but in the term sheets, it seems that the industry sees opportunities through a dichotomous lens that never really mattered to patients: platform OR asset.
Many VC-backed platform companies are formed and raise tens of millions of capital before committing to a specific disease indication. Why would investors purposefully curtail optionality when patients have no choice but to line up for whatever the industry is offering? Funding for new biotech companies is raised from investors who are driven by returns; they’re not personally affected by the diseases that their portfolio companies work on.
A multi-decade transition is imperceptibly underway. The biopharma industry is transitioning from a top-down B2B operating system, where assets and platforms discovered mostly in publicly funded labs in academia are transacted between companies without patient community ownership, to a bottom-up DTC operating system where patient communities own all of their data, fund their own research and are empowered by AI, software tools and marketplaces to advance new medicines to the clinic without intermediaries.
New platform technologies (sometimes called modalities) are discovered in labs across the world daily. Platforms are so ubiquitous that they’ve become assets. The oncoming waves of ever-improving platforms means we are no longer limited by the pace of innovation.
However, the inescapable truth is that the ultimate rate-limiting step for any new medicine will always be a pivotal Phase 3 study approval, which is in the hands of 30-40 volunteer study participants, if you’re the average rare disease.
Regulatory agencies will need to embrace 1-to-N clinical trial design if we’re ever going to get to a velocity of hundreds of new medicine approvals each year. Single-patient INDs coordinated by pioneer families will become the crucible from which to spin up placebo-controlled crossover studies with highly motivated participants and the flexibility of telemedicine visits.
Patients are the platform is another way of recognizing that patients are living laboratories on the frontiers of precision medicine. Patients are the platform because they take as many shots on goals as long as they have breath in their bodies. Patients are the platform because they will decide which asset or modality works best for them, and then they push faster and spend less to get into the clinic than any traditionally financed and staffed company.
If patients are living laboratories, then contact registries are just scratching the surface. We know it’s possible for one patient to discover novel clinical biomarkers and then rapidly validate the results in a handful of other patients — the clinical trial equivalent of multi-factor authentication.
Single-patient studies can inform which endpoints will be both great pharmacodynamic markers and meaningful to patients and their families and caregivers. This is what I would call the Fajgenbaum approach, which is poised to scale. We also did it with Maggie and PMM2-CDG. We’re doing it now with a second CDG. I believe we will do it for every disease we work on.
Accelerating innovation has caused platforms to become assets, and over time assets become commodities, i.e. low-cost generics. That’s a deflationary force that puts more power in the hands of patients. Cost Plus Drugs is a branded generics company and they understand that patients are the platform better than any biopharma company out there today.
Throughout the history of medicine, leaps and bounds have been made by consenting medical pioneers, individuals who were shaped by their environment but in the end acted as determined individuals, venturing into the darkness with the other hope of a brighter horizon. The Hempels — tragically, ahead of their time — are an example that continue to inspire patient communities.
By the time biopharma industry starts to really understand that patients are the platform, patient-led companies and nonprofit biotechs will be commonplace, and over time, competition. It will also become more commonplace to trace a new medicine back to a single patient.
To repeat, patients are the discovery platform: from a pathogenic variant in a gene in a whole exome report, to disease models/patient avatars engineered with CRISPR; from unbiased phenotypic drug screens to discovering a drug class targeting a disease modifying pathway; from a clinical biomarker to elucidation of pathomechanism and the ability to create targeted cures.
Patients are also the clinical platform: from one single-patient study to multiple single-patient studies; from longitudinal data in a natural history study that has an associated biobank to a synthetic placebo group that obviates the need for a placebo-control.
And finally, patients are the commercial platform. Patients self organize into communities long before a clinical trial starts recruiting. New medicines will have to convince patient communities that they would be superior to standard of care, ensuring fierce competition between experimental medicines for attention in the patient economy.
It all flows from the patients, because the patients are the platform.