CMT Blog

Faster, Less Predictable: What the FDA’s Turbulent Years Mean for CMT

Dan Knauss

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Line drawing of a teal stopwatch on the left and a dotted route that loops back on itself before reaching a violet and teal capsule, with two question marks beneath the capsule.

For people living with Charcot-Marie-Tooth disease, the US Food and Drug Administration (FDA) has always been a distant gatekeeper. There is still no cure for any form of CMT. The treatments that exist, such as physical therapy, braces, surgery and medication, manage symptoms rather than slowing the disease.1 What we have instead is a pipeline of experimental treatments: antisense oligonucleotides (short, manufactured strands of genetic material designed to change how a gene’s message is used), gene therapies, small-molecule drugs, and a growing list of orphan drug and rare pediatric disease designations. Whether those programs ever reach patients depends heavily on how the FDA works, and on whether the research that feeds the pipeline keeps getting funded.

That matters in Canada too. Most companies developing CMT treatments design their trials around what the FDA will accept, because the US is the largest market. But FDA approval isn’t Canadian approval: Health Canada makes its own decision, and whether a public drug plan pays for a treatment is decided separately again.

Both the FDA and US research funding have changed a lot in the past two years. Some of the changes could help people with rare diseases. Others create real risks. Often it’s the same change, and which way it cuts depends on the evidence behind each decision and how consistently the rules are applied.

The Promise: Trial Designs That Fit Small Populations

The administration’s message to the rare disease community has been consistent: evidence standards built around common diseases don’t fit small patient populations. There’s real truth in that. By the FDA’s own count, only about 5 percent of rare conditions have an approved treatment,2 and recruiting a large randomized trial for an uncommon subtype such as SORD neuropathy is hard.

It helps to distinguish two approval routes—traditional and accelerated approval—from the draft plausible mechanism framework, which can operate within either route.

  • Traditional approval requires “substantial evidence” that a drug works. The FDA has long accepted one adequate and well-controlled trial plus confirmatory evidence as enough in some cases. A revised draft guidance in June 2026 explains how sponsors can rely on that approach.3 In a New England Journal of Medicine piece in February, Marty Makary, then FDA commissioner, and Vinay Prasad, then its top biologics regulator, said this one-trial approach should become the default.4 One trial doesn’t have to mean a small or uncontrolled trial.
  • Accelerated approval lets a drug for a serious condition be approved on an endpoint that is reasonably likely to predict a real benefit, such as a blood or imaging marker, before that benefit has been shown. The company then has to run confirmatory trials, and the FDA can withdraw the approval if they don’t confirm the benefit.5 That later confirmatory trial is a different thing from the “confirmatory evidence” that supports a single-trial approval.
  • The plausible mechanism framework, set out in draft guidance in February 2026, is meant for individualized therapies, such as gene editing or RNA-based drugs, for very rare genetic conditions where a randomized trial isn’t practical. It asks a developer to identify a specific genetic cause, target it, rely on well-characterized natural history data, confirm the target was reached, and demonstrate improvement in clinical outcomes or course. When a disease progresses slowly, it says robust natural history data will likely be needed as an external control. Approval would still go through the traditional or accelerated route.6 Despite early talk of a new “pathway,” the draft doesn’t create a new approval standard.7

The FDA has added a broader effort on top of these. The Rare Disease Framework Initiative, launched on October 14, 2026, proposes a “fit-for-purpose” approach covering everything from early meetings with developers through post-marketing requirements and manufacturing review. Its stated goals include clearer, more consistent decisions, earlier patient involvement and more use of outside experts.8

Several of these ideas could help CMT. Many CMT subtypes are caused by single, well-characterized genes, which is the situation a mechanism-based framework is designed for. Vanda’s VCA-894A, an antisense oligonucleotide aimed at one specific variant in IGHMBP2 that causes CMT2S, received a rare pediatric disease designation in July 2026. That’s a designation, not an approval; if the drug is eventually approved and meets the program’s conditions, Vanda could qualify for a priority review voucher.9

The Concern: Flexibility Without Predictability

The FDA’s stated goals are good ones. The trouble is that its decisions over the past two years have been hard to predict, and in rare disease that unpredictability has costs.

Rejections, changed advice and holds. Bloomberg counted at least 23 complete response letters for rare disease therapies between the start of 2025 and August 2026.10 A complete response letter means the FDA can’t approve an application as it stands. It isn’t always final: companies often fix the problems and resubmit. The count is of letters, not products, and on its own it doesn’t show that decisions were arbitrary. Individual cases show more:

  • REGENXBIO’s RGX-121, a gene therapy for Hunter syndrome. After an earlier rejection that cited trial design, the FDA said in June 2026 that existing data could support an application for accelerated approval.11 In August the FDA placed the trial on clinical hold after spine MRI scans found small, symptomless nodules or cysts in five participants, and the company said it doesn’t expect to resubmit soon.12
  • uniQure’s AMT-130, a gene therapy for Huntington’s disease. This was changed advice, not a rejection. The FDA had earlier asked for another trial, which the company described as a reversal of its position.13 In June 2026 the company announced that the FDA had agreed a three-year analysis of its Phase 1/2 study could be the main basis of an application for accelerated approval.14
  • Sarepta’s Elevidys, a gene therapy for Duchenne muscular dystrophy. In July 2025, amid reports of patient deaths, the FDA asked Sarepta to pause shipments, then allowed them to resume for patients who could still walk.15 Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research (CBER), which reviews gene and cell therapies, resigned the day after shipments resumed and returned less than two weeks later.16 In November 2025 the FDA added a boxed warning, its strongest, about serious and fatal liver injury, and limited Elevidys to patients four and older who can walk.17 New safety findings are a legitimate reason to change course. The concern is with how decisions are made and explained, not with acting on safety signals. And a CMT gene therapy wouldn’t necessarily carry the same risks.

A former CBER deputy director has said some sponsors believe the reasons in recent rejection letters conflict with the FDA’s own earlier advice.18 That is a financing risk for small CMT companies. Rare disease programs are paid for on the expectation that a trial designed with the FDA’s agreement will be reviewed against that agreement. If advice seems to change with the people in charge, raising money for the next trial gets harder.

Leadership churn. Prasad left CBER for good at the end of April 2026.19 Makary resigned as commissioner in May, and Kyle Diamantas, the deputy commissioner for food, became acting commissioner.20 The president’s nominee, Heidi Overton, had her Senate hearing in September. RAPS reported on October 2 that a Senate confirmation vote was not expected until after November 9.21 The FDA’s Center for Drug Evaluation and Research (CDER), which reviews most drugs, had five directors between January 2025 and April 2026. Over the same period, officials commented publicly on specific products under review and convened fewer advisory committees, the public meetings where outside experts weigh in on new drugs.22 New frameworks are only as durable as the people who interpret them.

Staffing. The FDA cut more than 3,000 jobs in 2025 and by June 2026 was planning to hire 2,200 people back.23 Reviewers still met nearly all of their deadlines for the 53 novel drugs and biologics approved in 2025, though that count includes only approvals.24 Rare disease reviews depend on experienced reviewers who know a disease area, and that expertise is the hardest thing to rebuild.

What CMT Has Already Learned About Surrogates

Applied Therapeutics’ govorestat, for neuropathy caused by SORD variants, shows why the evidence question matters. In the Phase 2/3 INSPIRE trial, the drug missed its primary endpoint, the main outcome the trial was designed to measure: the 10-metre walk-run test. That means the trial didn’t establish its prespecified main result. It doesn’t prove the drug has no effect, but it means positive secondary results need careful interpretation. The company pointed to a self-reported mobility measure and to lower blood sorbitol levels.25

A placebo-controlled confirmatory study of 155 patients was registered in September 2025.26 Then, in February 2026, Cycle Group Holdings acquired Applied for 8.8 cents a share, plus payments that depend on milestones such as approval in CMT-SORD.27 In April, Applied stopped all of its govorestat studies to evaluate new development strategies, saying the decision wasn’t based on new safety findings.28 The confirmatory study was withdrawn before it enrolled anyone.29

Govorestat is a conventional drug program, not an example of the plausible mechanism framework. But it shows the gap any flexible approach has to cross. Lowering sorbitol is a change in a biomarker; walking better is a change in a person’s life. A surrogate endpoint is only useful if it reliably predicts the second from the first, and in a slowly progressive neuropathy that link is hard to establish.

Smaller trials and more flexible designs may make treatment development possible for some CMT populations. The important question is what evidence each design can reliably provide, and whether the FDA and sponsors make those expectations clear, follow through on safety monitoring, and complete the studies needed to resolve what’s still uncertain.

The Funding Side: Where CMT Research Starts

Most CMT therapies start in academic labs: gene discovery, natural history studies, outcome measures, animal models. Much of that work depends on the US National Institutes of Health (NIH), the main federal funder of medical research, and on its neurology institute, the National Institute of Neurological Disorders and Stroke (NINDS). Funding for CMT research is a mix of public and charitable money. The Inherited Neuropathy Consortium is funded by the CMTA, and it is part of an NIH research network whose data coordinating centre is funded by NINDS and another NIH centre.30

NIH funding has been under pressure, and Congress has mostly protected it so far:

  • Budget. The administration’s budget for fiscal 2026 proposed cutting NIH by about 40 percent.31 Congress rejected the cuts. The spending bill signed on February 3, 2026, raised NIH’s budget by $415 million.32
  • Indirect costs. On January 5, 2026, a federal appeals court upheld an injunction blocking NIH’s attempt to cap the indirect costs it pays universities, which cover facilities and administration, at 15 percent.33
  • This year. Fiscal 2027 has no full budget yet. NIH is operating under a stopgap spending law through December 11, 2026, at fiscal 2026 levels, and its institutes may pay continuing grants at less than the amounts they committed until a budget is passed.34

A new emphasis on AI-driven science. In July 2026 the administration announced more than $5 billion in federal commitments to the Energy Department-led Genesis Mission, an AI-for-science effort that names drug discovery among its goals.35 An accompanying White House science policy report called for shifting federal research support toward individual scientists and AI, and away from universities. It declared that “federal support for science must be politically accountable.”36 The announcement doesn’t show money being taken from NIH or from CMT research, but it does signal changing priorities.

AI tools may well help with rare disease research, from interpreting genetic variants to designing trials. They can’t replace what CMT research needs most: groups of patients whose symptoms and progression have been carefully measured, outcome measures sensitive enough to detect change in a slowly progressive disease, and long-running natural history data. Those are slow, people-intensive projects, and they’re what university-based, peer-reviewed funding was built to support.

The FDA named its first Deputy Commissioner for Technology and Artificial Intelligence, Jared Seehafer, in September 2026.37 Nothing so far shows computer models standing in for clinical data in rare disease reviews. If they start to, the CMT community will want to know how those models are validated, and by whom.

The Incentive Question

There’s an older debate underneath all this. The 1983 Orphan Drug Act gives companies fee waivers, research tax credits and seven years of market exclusivity in exchange for developing drugs for small populations.38 Government auditors found that between 2008 and 2017, about 38.5 percent of orphan approvals were for drugs already approved for other uses.39 That number alone doesn’t show abuse: approving an existing drug for a new rare condition can be valuable, and the auditors’ report sets out both critics’ and patient advocates’ views.40 Orphan exclusivity also doesn’t block other treatments for a disease; since February 2026 the law limits it to the same drug for the same approved use.41

The voucher program raises the stakes. A company that gets a qualifying rare pediatric disease drug approved can receive a priority review voucher, which it can use for a faster review of another drug or sell.42 Vouchers often sell for $100 million or more, and Congress has extended the program through September 2029.43 If more flexible evidence standards are applied without adequate supporting evidence, a company could earn those rewards for a drug whose benefit is still uncertain. For patients, the risk isn’t only paying for treatments that don’t work. An approved but ineffective therapy can become the standard of care, which makes it harder to run placebo-controlled trials of the next candidate.

None of this means faster approvals are wrong for CMT. It means flexibility needs guardrails: confirmatory studies that actually get done, accelerated approvals withdrawn when the evidence doesn’t hold up, and the reasoning behind decisions made public.

What the CMT Community Can Do Now

  • Weigh in on the Rare Disease Framework Initiative. The FDA’s public meeting is on October 28, 2026, from 1 to 5 p.m. Eastern (11 a.m. to 3 p.m. in Edmonton). In-person seats are full, but anyone can register to watch online. Requests to speak have closed. Written comments go to docket FDA-2026-N-12214 on Regulations.gov and are due December 11, 2026.44 Patient groups and researchers can write about outcome measures for slowly progressive neuropathies, the use of natural history controls, and how surrogate endpoints get validated.
  • Hold the FDA to its own goals. The initiative already promises consistency, patient involvement and outside expertise. Ask how those commitments will be put into practice and measured, and ask the FDA to keep publishing the reasoning behind rare disease approvals and rejections.
  • Defend the research base. If you live in the US, tell your members of Congress that NIH and NINDS funding, and the networks built with it, are what make CMT therapies possible in the first place.
  • Ask hard questions of sponsors. When a company announces a CMT program, ask what clinical benefit it expects to show, how it will measure it, and what happens if the confirmatory trial fails.

The rare disease community spent decades asking the FDA to take small populations seriously. In some ways, it finally is. Workable trial designs, predictable decisions and reliable evidence have to advance together. If any one of them is missing, the next CMT treatment will be slower to arrive, or less likely to help.

Notes

  1. National Institute of Neurological Disorders and Stroke, “Charcot-Marie-Tooth Disease,” last reviewed September 2, 2026, https://www.ninds.nih.gov/health-information/disorders/charcot-marie-tooth-disease. ↩︎
  2. U.S. Food and Drug Administration, “FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development,” press release, October 14, 2026, https://www.fda.gov/news-events/press-announcements/fda-launches-rare-disease-framework-initiative-develop-holistic-fit-purpose-framework-rare-disease. ↩︎
  3. U.S. Food and Drug Administration, “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products,” revised draft guidance for industry, June 2026, https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products. ↩︎
  4. Vinay Prasad and Martin A. Makary, “One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma,” New England Journal of Medicine 394, no. 8 (2026): 815–17, https://doi.org/10.1056/NEJMsb2517623, as summarized in Temkin et al., “FDA Advances a ‘Plausible Mechanism’ Framework.” ↩︎
  5. U.S. Food and Drug Administration, “Accelerated Approval Program,” https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program. ↩︎
  6. U.S. Food and Drug Administration, “Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause,” draft guidance for industry, February 2026, https://www.fda.gov/media/191247/download. ↩︎
  7. Eva Temkin, Daniel A. Kracov, Claire W. Dennis, and Jonathan Trinh, “FDA Advances a ‘Plausible Mechanism’ Framework for Rare Disease Drug Development and Signals a Shift to a Single Trial with Confirmatory Evidence Default for All Drugs,” Arnold & Porter, February 25, 2026, https://www.arnoldporter.com/en/perspectives/advisories/2026/02/fda-advances-a-plausible-mechanism-framework-for-rare-disease-drug-development-and-shifts-to. ↩︎
  8. Nicholas Jacobus, “FDA Launches Rare Disease Framework Initiative,” Pharmaceutical Executive, October 14, 2026, https://www.pharmexec.com/view/fda-launches-rare-disease-framework-initiative. ↩︎
  9. “FDA Grants Vanda Rare Pediatric Disease Designation for CMT Therapy,” RARE Daily, July 9, 2026, https://globalgenes.org/raredaily/fda-grants-vanda-rare-pediatric-disease-designation-for-cmt-therapy/. ↩︎
  10. Nyah Phengsitthy, “Rare Disease Drugmakers Watch FDA for Renewed Focus on Therapies,” Bloomberg Tax, August 10, 2026, https://news.bloombergtax.com/health-law-and-business/rare-disease-drugmakers-watch-fda-for-renewed-focus-on-therapies. ↩︎
  11. Phengsitthy, “Rare Disease Drugmakers Watch FDA.” ↩︎
  12. REGENXBIO Inc., “Form 8-K: Current Report,” U.S. Securities and Exchange Commission, August 24, 2026, including the press release “REGENXBIO Announces Regulatory Update on RGX-121 for MPS II,” https://ir.regenxbio.com/node/13911/html. ↩︎
  13. Jonathan Gardner and Shaun Lucas, “FDA, after Turbulent Year, Leaves Drugmakers Guessing on Its Direction,” BioPharma Dive, April 1, 2026, https://www.biopharmadive.com/news/fda-after-turbulent-year-leaves-drugmakers-guessing-on-its-direction/815482/. ↩︎
  14. uniQure, “uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease,” press release, June 17, 2026, https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-plan-bla-submission-amt-130-huntingtons. ↩︎
  15. Don Tracy, “Vinay Prasad’s FDA Departure Follows Controversy Over Drug Rejections,” Pharmaceutical Executive, July 30, 2025, https://www.pharmexec.com/view/vinay-prasad-fda-departure-controversy-drug-rejections. ↩︎
  16. Ben Fidler, “Vinay Prasad, in Surprise Reversal, to Rejoin FDA after Abrupt Departure,” BioPharma Dive, August 9, 2025, https://www.biopharmadive.com/news/vinay-prasad-cber-return-fda-vaccines-gene-therapy/757263/. ↩︎
  17. U.S. Food and Drug Administration, “FDA Approves New Safety Warning and Revised Indication that Limits Use for Elevidys Following Reports of Fatal Liver Injury,” press release, November 14, 2025, https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatal. ↩︎
  18. Jessica Karins, “Rare Disease, CGT Advocates Say CBER Needs Predictability After Prasad,” Inside Health Policy, May 7, 2026, via Friends of Cancer Research, https://friendsofcancerresearch.org/news/inside-health-policy-rare-disease-cgt-advocates-say-cber-needs-predictability-after-prasad/. ↩︎
  19. Heather McKenzie, “Through Flux and Fluidity, FDA Keeps Coming Back to Rare Disease,” BioSpace, July 8, 2026, https://www.biospace.com/fda/through-flux-and-fluidity-fda-keeps-coming-back-to-rare-disease. ↩︎
  20. Richard Payerchin, “FDA Leadership in Flux: Commissioner Makary Resigns after Rocky Tenure,” Medical Economics, May 12, 2026, https://www.medicaleconomics.com/view/fda-leadership-in-flux-commissioner-makary-resigns-after-rocky-tenure. ↩︎
  21. Ferdous Al-Faruque, “This Week at FDA: No Overton Vote until after Midterms, FDA Signals Advisory Committee Support, and More,” RAPS Regulatory Focus, October 2, 2026, https://www.raps.org/resource/this-week-at-fda-no-overton-vote-until-after-midterms-fda-signals-advisory-committee-support-and-more.html. ↩︎
  22. Gardner and Lucas, “FDA, after Turbulent Year.” ↩︎
  23. Deena Beasley, “FDA to Hire 2,200 Workers after Cutting More than 3,000 Jobs,” Reuters, June 24, 2026, via Honolulu Star-Advertiser, https://www.staradvertiser.com/2026/06/24/breaking-news/fda-to-hire-2200-workers-after-cutting-more-than-3000-jobs/. ↩︎
  24. The FDA Group, “FDA Drug Reviews Held Steady in 2025 Despite Historic Staffing Losses,” January 8, 2026, summarizing Zachary Brennan’s analysis for Endpoints News, https://insider.thefdagroup.com/p/fda-drug-reviews-held-steady-in-2025. ↩︎
  25. Dan Samorodnitsky, “Applied Therapeutics’ Rare Disease Treatment Flunks in Late-Stage Trial,” BioSpace, May 19, 2025, https://www.biospace.com/drug-development/applied-therapeutics-rare-disease-treatment-flunks-in-late-stage-trial. ↩︎
  26. Tristan Manalac, “New Confirmatory Study Hints at ‘Alignment’ Between Applied Therapeutics, FDA for Rare Disease Drug,” BioSpace, September 29, 2025, https://www.biospace.com/drug-development/new-confirmatory-study-hints-at-alignment-between-applied-therapeutics-fda-for-rare-disease-drug. ↩︎
  27. Applied Therapeutics, Inc., “Form 8-K: Current Report,” U.S. Securities and Exchange Commission, February 3, 2026, https://www.sec.gov/Archives/edgar/data/1697532/000119312526034559/d93556d8k.htm. The milestone payments are described in Nasdaq’s report of the deal. ↩︎
  28. Applied Therapeutics, “Program Update: Govorestat Development and Clinical Studies,” April 17, 2026, https://www.appliedtherapeutics.com/. ↩︎
  29. ClinicalTrials.gov, “Confirmatory Study of Govorestat in CMT-SORD (NCT07191912),” status Withdrawn, last updated May 29, 2026, https://clinicaltrials.gov/study/NCT07191912. ↩︎
  30. Inherited Neuropathy Consortium, “Inherited Neuropathy Consortium,” Rare Diseases Clinical Research Network, https://inc.rarediseasesnetwork.org/. ↩︎
  31. Uli von Andrian, “Statement on House LHHS Appropriations Subcommittee Rejection of Deep Cuts to NIH Funding for FY 2026,” American Association of Immunologists, September 2, 2025, https://news.aai.org/2025/09/02/statement-on-house-lhhs-appropriations-subcommittee-rejection-of-deep-cuts-to-nih-funding-for-fy-2026/. ↩︎
  32. Monte Ward, “Updates on Government Funding, NIH Modernization Hearing,” American College of Sports Medicine, February 13, 2026, https://acsm.org/policy-corner-february-2026/. ↩︎
  33. Oregon Department of Justice, “NIH Indirect Costs: Massachusetts v. National Institutes of Health (D. Mass.),” Federal Litigation Tracker, https://www.doj.state.or.us/oregon-department-of-justice/federal-oversight/federal-litigation-tracker/nih-indirect-costs-massachusetts-v-national-institutes-of-health-d-mass/. ↩︎
  34. National Institutes of Health, “NIH Operates Under a Continuing Resolution,” notice NOT-OD-26-131, NIH Grants and Funding, September 28, 2026, https://www.grants.nih.gov/grants/guide/notice-files/NOT-OD-26-131.html. ↩︎
  35. Owen Dahlkamp, “Trump Administration Steers $5B toward AI Research,” E&E News, July 23, 2026, https://www.eenews.net/articles/trump-administration-steers-5b-toward-ai-research/. ↩︎
  36. Courtney Rozen, “US to Spend $5 Billion on Health, Construction Research Powered by AI,” Reuters, July 22, 2026, via The Star, https://www.thestar.com.my/tech/tech-news/2026/07/22/us-to-spend-5-billion-on-health-construction-research-powered-by-ai. ↩︎
  37. U.S. Food and Drug Administration, “Jared Seehafer,” content current as of September 9, 2026, https://www.fda.gov/about-fda/fda-organization/jared-seehafer. ↩︎
  38. Sarah Jane Tribble and Sydney Lupkin, “Government Investigation Finds Flaws in the FDA’s Orphan Drug Program,” KFF Health News, November 30, 2018, https://kffhealthnews.org/health-industry/government-investigation-finds-flaws-in-the-fdas-orphan-drug-program/. ↩︎
  39. Tribble and Lupkin, “Government Investigation Finds Flaws.” ↩︎
  40. U.S. Government Accountability Office, “Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue,” GAO-19-83, November 30, 2018, https://www.gao.gov/products/gao-19-83. ↩︎
  41. Komal Karnik Nigam, Gary Veron, Jason Conaty, Eva Marie Schifini, Katherine Kramer, and Noah Fisher, “Latest Congressional Spending Package Includes Important Updates for Orphan Disease and Pediatric Drug Development,” Hogan Lovells Cadwalader, February 10, 2026, https://hlc.com/en/publications/important-updates-for-orphan-disease-and-pediatric-drug-development. ↩︎
  42. U.S. Food and Drug Administration, “Rare Pediatric Disease Designation and Priority Review Voucher Programs,” https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs. ↩︎
  43. Nigam et al., “Latest Congressional Spending Package.” ↩︎
  44. Reagan-Udall Foundation for the FDA, “Rare Disease Framework Public Meeting,” https://www.reaganudall.org/news-and-events/events/rare-disease-framework-public-meeting. ↩︎

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