Expanded Access

Expanded Access is a way for sufferers of deadly unsolved diseases to try late-stage investigational therapeutics that are not yet fully approved for marketing.

Where the purpose of clinical trials is research (statistical evaluation of a drug’s performance), the purpose of Expanded Access is treatment; specifically, treatment for patients who have no other options for improving their own outcomes of the disease.

It’s our job to make these treatment programs an option for ALS patients who are unable to receive treatment via clinical trials. It is estimated that over 60% of ALS patients do not meet the enrollment criteria of any open clinical trials. Add to that the limited duration of each trial and the difficulties many families face in getting to research clinics, and you have an enormous opportunity to address unmet medical need for near term treatment options.

How quickly can a new therapeutic become a treatment option for current ALS sufferers?

Usually, not quickly at all. And, in the case of ALS, where patients have a pretty short average remaining lifetime, the majority of current people with ALS (PALS) face the prospect of dying of their disease before any drug now in trial is approved in their country for use. Once a new therapeutic shows preliminary evidence of safety and efficacy in clinical trials, it can still take years for that product to be approved for use in the particular disease.

The U.S. regulatory code has a few different mechanisms to speed this up for rare and life-threatening diseases with unmet medical need, but you’re usually still looking at years, not months, between the time proof-of-concept is established in Phase 2 trial and the time FDA approves the drug for the indication.

Without some kind of treatment program for late-stage unapproved drugs, patients who are unable to take part in clinical trials face death without even the chance to access existing and potentially disease-modifying therapy.

This is why there are exemptions to allow for the *treatment* use of drugs still in the approval process, in the cases life threatening conditions where no approved treatment is available.

Since 1962, Investigational New Drug (IND) exemptions have been filed to allow emergency treatment access to patients. IND’s for treatment purposes (not research) have gone by many names. In FDA’s code they are now classified as “Treatment IND’s”, but the term “Expanded Access Program” is fitting when talking about the treatment programs.

The Evolution of the Treatment IND

Expanded Access for AIDS and Cancer

Regulatory Criteria


Tropical Disease IND

Orphan Drug IND

Open Label IND

Group-C IND

Compassionate IND

Emergency IND

Treatment IND

1987 Promulgation

  • Emergency IND
  • Treatment IND
  • +Group-C
  • +Open Label

1992 FDA / PHS Statement

  • +Parallel Track IND

2009 Final Rule
Treatment IND:

  • Individual
  • Intermediate
  • Protocol IND

+(Group-C, Open-Label, Parallel, Emergency)

Required FDA to encourage submission of a Treatment IND for patients not in clinical trials for any investigational drug where there is

“preliminary evidence that the drug has effectiveness in the prevention or treatment of AIDS.”

Drug Dates # Enrolled
AZT 1986-87 4,804
trimetrexate 1988-94 753
pentamidine 1989-89 728
ddI 1989-91 >21,000
ddC 1990-92 6,705
atovaquone 1991-93 1,054
rifabutin 1992-93 2,506
d4T 1992-94 12,551
3TC 1993-95 29,430
saquinavir 1995 2,200
indinavir 1995 1,500
d4T 1992-94 12,551

About 75,000 AIDS sufferers had access to investigational drugs through Expanded Access Programs.

Since then, about the same number of cancer patients have been enrolled in Expanded Access Programs, including 500 multiple myeloma patients in the CMAP program for Kyprolis, which was approved in 2012.

Treatment IND, basic requirements:

  1. The Drug is intended to treat a serious or life-threatening disease.
  2. There is no satisfactory alternative treatment available.
  3. The drug is already under investigation, or trials have been completed.
  4. The trial sponsor is actively pursuing marketing approval.

The programs we pursue meet these criteria.

PLUS, the EAP must not interfere with clinical development of drugs for the disease.

This is a manageable factor.

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