Empiric therapies

Provider rosterDiagnostic supportSeeking a lead#cohort-empiric-therapies PDF (members)

The anchor question: once a care map says treat, who infuses it, dispenses it, compounds it, or gets the patient onto the trial or the expanded access protocol, at a price stated in advance? Several maps end in a therapy that an anchor can prescribe but can't deliver alone: enzyme replacement infusions, a silencer infusion for ATTR, a formulation nobody sells, an investigational drug when nothing approved fits.

What the care maps need

Service	Serves	What the care maps need from it
Infusion centers and home infusion	Lysosomal storage disorders, transthyretin amyloidosis, inherited metabolic disease	The lysosomal map's enzyme replacement runs every one or two weeks. The center runs the anchor's orders and reaction protocol, with a physician on call. Home infusion works with the right setup: in 121 adults with Pompe disease, 73% of 18,380 alglucosidase alfa infusions were given at home after a reaction-free start in hospital, with infusion reactions in 0.8% of home and 2.9% of hospital infusions.¹
Specialty pharmacy	Transthyretin amyloidosis, hypophosphatasia, neurogenetic, lysosomal storage disorders, inherited metabolic disease	Dispenses the disease-specific drugs the maps name: asfotase alfa and burosumab in the hypophosphatasia map; hemin, givosiran, augmentation therapy and pegvaliase, through infusion centers and specialty pharmacy, in the inherited metabolic map. Quotes the cash price, and works the manufacturer-assistance paperwork with the anchor, who coordinates the start as the ATTR map describes.
Compounding: 503A pharmacies and 503B outsourcing facilities	Any map that needs a dose or formulation not sold commercially; none of the current maps names one yet	Two different things. The 2013 Drug Quality and Security Act, passed after contaminated compounded drugs caused a fungal meningitis outbreak, tightened oversight of patient-specific compounding in 503A pharmacies and created 503B outsourcing facilities.² 503B facilities make sterile drugs at scale under FDA oversight, and registration isn't inspection: of 48 facilities newly registered from January 2020 to April 2025, 11 had been inspected.³
Expanded access	Neurogenetic, transthyretin amyloidosis, lysosomal storage disorders, hypophosphatasia, inherited metabolic disease, when approved therapy doesn't fit and no trial will take the patient	FDA can authorize expanded access to an investigational drug for a serious or immediately life-threatening disease when there is no comparable alternative, the benefit justifies the risk, and access won't compromise the drug's development (21 CFR 312, subpart I). The manufacturer has to agree to supply the drug and provides a letter of authorization. From 2005 to 2014, FDA's drug center (CDER) received 10,939 new expanded access applications, mostly for single patients, and allowed 99.7% to proceed.⁴ Outside emergencies, the treating physician also needs IRB approval.⁵
Right to Try	The same cohorts	A separate federal pathway for drugs not approved for any indication that have completed phase I and have an ongoing pivotal trial. It needs the manufacturer's agreement and obliges no one to supply or pay for the drug.⁶
Trial enrollment	Neurogenetic, transthyretin amyloidosis, progressive monogenic disease, lysosomal storage disorders, hypophosphatasia, inherited metabolic disease	A site coordinator who takes a referral from an anchor and tells the anchor whether the patient is eligible. The maps call for trial screening and referral where no approved therapy exists or the approved one doesn't fit.
Off-label and empiric prescribing	Nonspecific symptoms, connective tissue	Off-label use has long been common: in a 2001 national sample of 160 commonly prescribed drugs, 21% of use was off-label, and 73% of that had little or no scientific support.⁷ Every POTS drug was used off-label, because none was FDA-approved for POTS as of the 2019 NIH consensus.⁸ The roster's job here is to make each empiric course deliberate and written down.

Vetting criteria

Every entry is checked against these before listing and reviewed yearly.

Publishes cash prices: per infusion with chair time and drug quoted separately, per prescription, per compounded formulation.
Infusion centers and home infusion services run the anchor's orders and reaction protocol, with trained nurses and a physician on call.¹
Pharmacies hold an active state license. A 503B outsourcing facility is FDA-registered and shares the outcome of its most recent FDA inspection.³
For expanded access and trials: has filed a single-patient expanded access request before, or will walk an anchor through the first one, including the IRB step.⁵
Empiric and off-label courses are documented with the target symptom, the measure and the stop date, and every dose change or reaction goes back to the anchor.
Clinician entries meet the independent practice test: founded or co-founded the practice, with more than half of its revenue from patients rather than payers.

How the roster is built

Year one: one infusion center or home infusion service on the Front Range that will run enzyme replacement and ATTR infusions on an anchor's orders at a posted price, one specialty pharmacy, and one 503A compounding pharmacy. A one-page expanded access checklist for anchors.
Year two: a second entry for each, a 503B source for sterile products, and trial-site contacts for the neurogenetic and ATTR maps. Members log each expanded access request, de-identified, so the next anchor knows the real timeline.
Year three: track price, infusion reactions reported back, time from request to first dose under expanded access, and whether the anchor heard about every change. Prune and add.

Roster

No entries yet. The convener drafted this roster and it's looking for a member to lead it. Members propose centers, pharmacies and trial sites in the cohort channel; the convener and one member check the criteria before listing. Each entry will show the center, pharmacy or site, what it delivers (infusion, home infusion, specialty pharmacy, 503A or 503B compounding, expanded access support, trial enrollment), its license or registration, the cash price or how to get a quote, the area it covers, and the member who vouched for it.

Propose a center or pharmacy in Slack

References

Ditters IAM, van Kooten HA, van der Beek NAME, et al. Home-based infusion of alglucosidase alfa can safely be implemented in adults with late-onset Pompe disease: lessons learned from 18,380 infusions. BioDrugs. 2023;37(5):685-698. doi:10.1007/s40259-023-00609-2
Quertermous J, Desai S, Harper J, et al. The practice of compounding, associated compounding regulations, and the impact on dermatologists. J Drugs Dermatol. 2018;17(7):s17-s22. PMID: 30005109
McCall KL, Lynch SE, Hageman EA, Williams KG. A quantitative and qualitative study of US FDA inspection reports of 503B outsourcing facilities. J Am Pharm Assoc (2003). 2026;66(5):103156. doi:10.1016/j.japh.2026.103156
Jarow JP, Lemery S, Bugin K, Khozin S, Moscicki R. Expanded access of investigational drugs: the experience of the Center of Drug Evaluation and Research over a 10-year period. Ther Innov Regul Sci. 2016;50(6):705-709. doi:10.1177/2168479016656030
Chapman CR, Shearston JA, Folkers KM, et al. Single-patient expanded access requests: IRB professionals' experiences and perspectives. AJOB Empir Bioeth. 2019;10(2):88-99. doi:10.1080/23294515.2019.1577192
Agarwal R, Saltz LB. Understanding the Right to Try Act. Clin Cancer Res. 2020;26(2):340-343. doi:10.1158/1078-0432.CCR-19-2015
Radley DC, Finkelstein SN, Stafford RS. Off-label prescribing among office-based physicians. Arch Intern Med. 2006;166(9):1021-1026. doi:10.1001/archinte.166.9.1021
Vernino S, Bourne KM, Stiles LE, et al. Postural orthostatic tachycardia syndrome (POTS): state of the science and clinical care from a 2019 National Institutes of Health expert consensus meeting, part 1. Auton Neurosci. 2021;235:102828. doi:10.1016/j.autneu.2021.102828

References retrieved from PubMed on 2026-10-30. Rosters are reviewed at each yearly cycle.

Colorado Care Network. Empiric therapies. Version 2026-10-30.