Clinic launch guide
New Indications, INDs and Clinical Studies
Reviewed by ExaVeyra Sciences editorial team · Updated 16 August 2026
Practitioners are told regularly that an unapproved product may only be administered under an investigational new drug application, and are rarely told what that involves. It is a defined process with published steps, statutory timelines and a first move that costs nothing but preparation. Clinicians in private practice do run studies, and the regulations anticipate exactly that: an individual who both initiates and conducts an investigation is a sponsor-investigator, and the regulations name the role.
This page walks the pathway in the order you would actually meet it, from the pre-IND meeting through to the reporting that continues after a study opens. It is written for a clinician or a small sponsor deciding whether this is a realistic route, and for a practice that wants to understand what a partner sponsor is doing on its behalf.
Start with the pre-IND meeting
Before an IND exists, a prospective sponsor can request a formal meeting with the FDA review division that would handle it. This is the highest-value step in the whole sequence and it comes first for a reason: it is where you find out what the agency would want to see, from the people who would review it, before you have spent anything building the file.
The mechanics are straightforward. You submit a meeting request with a statement of the questions you want answered, and a briefing package containing the data and the proposal those questions rest on. FDA’s formal meetings guidance sets out the request format, the categories of meeting and the timelines the agency works to. Coming with specific, answerable questions is what separates a productive meeting from a general one, and vague questions get correspondingly general answers.
What an IND contains
The application is submitted on Form FDA 1571, which serves as the cover and the index. The substance of the submission falls into four areas, and their relative weight shifts with what is being studied.
| Section | What it establishes | Where the work usually sits |
|---|---|---|
| Chemistry, manufacturing and controls | What the material is, how it is made, and that each batch is the same as the last | The heaviest section for a biologic or a novel preparation |
| Pharmacology and toxicology | That there is a reasonable basis to conclude the study is safe to begin | Animal data, plus any prior human experience |
| Previous human experience | What is already known from any prior use in humans, including outside the US | Often thin for novel material, which is itself informative |
| Clinical protocol and investigator brochure | What will be done to whom, by whom, and what the investigators are told | Where a practising clinician contributes most directly |
For extracellular vesicle material the chemistry, manufacturing and controls section is usually the long pole, because identity, potency, particle characterisation, sterility, endotoxin and stability all have to be addressed with methods that are qualified for the purpose. Our dedicated reference on CMC requirements for exosome products goes through those expectations in detail.
The 30-day clock
Once an IND is submitted, the regulation is explicit about when dosing may begin. Under 21 CFR 312.40(b), an IND goes into effect thirty days after FDA receives it, unless FDA notifies the sponsor that the investigations described are subject to a clinical hold under 312.42, or on earlier notification by FDA that the clinical investigations may begin.
Two practical readings follow. Silence from the agency after thirty days means the IND is in effect, so no approval letter is coming and none is needed. And a clinical hold is a communication rather than a verdict: it identifies deficiencies, and the sponsor responds to them. Holds are a normal part of the process for novel material and are not the end of a programme.
The same section adds the condition that is easy to overlook. An investigational drug may be used in a clinical investigation only if the sponsor complies with all applicable requirements in part 312 and in parts 50 and 56, and each participating investigator does the same. Those two parts are informed consent and institutional review, and they run alongside the IND rather than after it.
IRB review and informed consent
Institutional review board review under 21 CFR part 56 and informed consent under 21 CFR part 50 apply to the study regardless of the sponsor’s size. A practice without an institutional affiliation uses an independent IRB, which is routine and is how most non-academic research is reviewed. Starting the IRB conversation in parallel with the IND rather than after it usually shortens the overall timeline, because protocol revisions the IRB asks for are cheaper before the protocol has been filed.
The consent document is worth unusual care in a practice setting, because the same clinician is often both the treating provider and the investigator. Making the distinction between clinical care and research explicit in the consent, and in how the study is described to patients, protects both roles.
When an IND is not required
There is a genuine exemption, and knowing its shape saves time. Under 21 CFR 312.2(b)(1), the clinical investigation of a drug product that is lawfully marketed in the United States is exempt from part 312 if all of the following apply:
- The investigation is not intended to be reported to FDA as a well-controlled study in support of a new indication, nor to support any other significant change in the labeling.
- If the drug is lawfully marketed as a prescription drug product, the investigation is not intended to support a significant change in its advertising.
- The investigation does not involve a route of administration, dosage level, patient population or other factor that significantly increases the risks, or decreases the acceptability of the risks, associated with use of the product.
- The investigation is conducted in compliance with the requirements for institutional review in part 56 and informed consent in part 50.
- The investigation is conducted in compliance with the requirements of 21 CFR 312.7.
Determining whether a study meets the exemption is the sponsor’s responsibility in the first instance. Where it is genuinely unclear, FDA will accept a request for an exemption determination, and asking is cheaper than being wrong.
Registering the study, and reporting results
Applicable clinical trials are registered on ClinicalTrials.gov under 42 CFR part 11. The registration deadline is set by 42 CFR 11.24: registration information must be submitted not later than 21 calendar days after the first human subject is enrolled. Results information follows under 42 CFR 11.44, which sets the standard deadline at no later than one year after the primary completion date, with a certification route for delayed submission in defined circumstances.
Registration is worth treating as an asset rather than an obligation. A registered protocol with a stated endpoint is the record that a study was designed before the data arrived, and it is what makes the eventual result citable by anyone else.
What continues after the study opens
- Safety reporting to FDA and to participating investigators, on the timelines in part 312, for serious and unexpected suspected adverse reactions.
- An annual report on the progress of the investigation.
- Protocol amendments and new protocols submitted to the IND as the programme develops.
- Continuing IRB review at intervals the board sets.
- Records and retention obligations that outlast the study itself.
These are the obligations that turn a study into a programme, and they are the part most often underestimated when a small sponsor plans. Deciding who owns each of them, by name, before the first patient is enrolled is the single most useful piece of planning available.
Expanded access is a different route
Expanded access, sometimes called compassionate use, allows a patient with a serious or immediately life-threatening condition to receive an investigational product outside a clinical trial when there is no comparable alternative. It has its own subpart in part 312 and its own criteria, and it is a treatment route rather than a research route. It is not a way to offer an unapproved product as a service, and it is worth distinguishing clearly from a study because the two are sometimes conflated.
What this realistically takes
Published cost estimates for an IND vary by orders of magnitude depending on what they count, so a specific figure here would be misleading. What can be said usefully is what drives the number: the state of the manufacturing package for the material, whether the nonclinical work already exists or has to be run, the size and duration of the proposed study, and how much regulatory work is done in-house.
For most practices the realistic route is not filing alone. It is participating as a site under a sponsor who already holds the IND, or contributing clinical design to a programme a manufacturer is running. Both give a practice genuine involvement in generating evidence without carrying the whole regulatory burden, and both start with the same conversation.
Sources
- FDA, Investigational New Drug (IND) Application (accessed 16 August 2026)
- 21 CFR 312.40, General requirements for use of an investigational new drug (accessed 16 August 2026)
- 21 CFR 312.2, Applicability and exemptions (accessed 16 August 2026)
- 21 CFR 312.3, Definitions and interpretations (accessed 16 August 2026)
- 21 CFR 312.23, IND content and format (accessed 16 August 2026)
- FDA, IND Application Procedures: Clinical Hold (accessed 16 August 2026)
- 21 CFR part 50, Protection of Human Subjects (informed consent) (accessed 16 August 2026)
- 21 CFR part 56, Institutional Review Boards (accessed 16 August 2026)
- 42 CFR 11.24, When must clinical trial registration information be submitted (accessed 16 August 2026)
- 42 CFR 11.44, When must clinical trial results information be submitted (accessed 16 August 2026)
- FDA, Expanded Access to Investigational Drugs for Treatment Use (accessed 16 August 2026)
- FDA, Form FDA 1571, Investigational New Drug Application (accessed 16 August 2026)
- ClinicalTrials.gov, submit studies and reporting policy (accessed 16 August 2026)