Executive summary

In 2018, Clinical Research Pathways conducted a physician needs assessment to help guide the development of educational activities to improve the use of expanded access to investigational drugs in the treatment of desperately ill patients. Here are some highlights:

  • Most physicians believe that investigational drugs that are still being tested should be made available to patients who have no other options, even if these patients are ineligible for a clinical trial.
  • A majority say they know “a lot” or “some” about the FDA expanded access program. A majority also say they have never used the program.
  • Few are aware of existing places to turn for help with the FDA expanded access program. Nearly half would be more likely to consider the program if support were available via a website with guidelines, forms, and a checklist.
  • Most are interested in learning more about expanded access to investigational drugs for patients and the FDA expanded access program.

A more detailed report follows.

Process

Clinical Research Pathways developed a needs assessment instrument and contracted with McKing Consulting Corporation to conduct the assessment. A total of 5,811 physicians were invited to participate. The response rate was approximately 2%.

A total of 107 physicians responded to the assessment by answering at least one question. Physicians were not required to answer all questions; thus, the number of responses varies per question. Four physicians never see patients who have serious or life-threatening conditions and are not responding to the standard treatment options, and two physicians would never consider using a nonapproved treatment for a patient. These six physicians were excluded from completing further questions.

Demographics

Approximately one-third of physicians are practicing neurologists (37), and another third are oncologists (36). The other third represents a combination of specialists in infectious disease (14), rheumatology (10), and other specialties and sub-specialties (7). Most physicians are employed by a university, hospital or clinic (84 [80%]). Physicians represent 26 states and all geographic regions of the United States. There were more male (61 [59%]) than female (40 [39%]) physicians. Most physicians (84 [82%]) have served as a principal investigator in a clinical trial and, of those who have not, over half (11 [61%]) have treated a patient in a clinical trial.

Most physicians (74 [73%]) report “very often” or “often” seeing patients who have a serious or life-threatening condition and are not responding well to the standard treatment options. These physicians report most often searching for a clinical trial (47 [49%]) or searching for nonstandard treatment options (33 [34%]) for these patients.

Expanded Access Opinions and Experiences

Most physicians (69 [73%]) believe that patients with no other options should be able to try investigational drugs that are still being tested even if these patients cannot get into a clinical trial. Of those who believe patients should only receive treatments that have been approved by the FDA (n=25), most would consider a nonapproved treatment for a patient (24 [93%]).

When asked whether they know about the FDA-regulated expanded access program, a majority (51 [57%]) of physicians reported knowing “a lot” or “some” about the program (see Figure 1). Approximately one-quarter (22 [25%]) of physicians have never considered using expanded access, and another quarter (24 [27%]) have considered using it but never started the application. Most (76 [85%]) physicians “very” or “somewhat closely” follow the progress of new drugs that might benefit patients. The most common source for this information was reported to be clinicaltrials.gov (48 [54%]).

Figure 1. Provider Knowledge Regarding the FDA Expanded Access program [Click image to enlarge]

Physicians were provided a list of all steps required for expanded access to drugs for non-emergency, single-patient use and were asked which they had not heard about before this assessment. Nearly half of physicians (40 [46%]) were familiar with all steps of the expanded access process. Physicians rated their unfamiliarity with each step of the process; this unfamiliarity ranged from 11-40% (see Figure 2). When asked to rate the difficulty of each step, the most difficult steps reported were submitting an application to the FDA, submitting an application to an IRB, receiving a timely IRB response, and getting reimbursed from the patient or insurance company.

Figure 2. Provider Familiarity with Expanded Access Program Steps [Click image to enlarge]

Barriers to Participation

Slightly over half (45 [53%]) of physicians reported they are “very” or “somewhat” confident that their practice has the resources to complete the application and reporting steps that are a part of the expanded access program. Nearly three-fourths (63 [72%]) of physicians reported having a staff member who is responsible for completing clinical research paperwork. Those who did not have a staff person responsible for completing clinical research paperwork almost always (96%) reported they were not confident they had the resources to complete the application and reporting parts of the expanded access program. Oncologists were most likely to report having a staff member responsible for clinical research paperwork (78%). Nearly all physicians (82 [94%]) have a relationship with a specific IRB; but nearly all of those that do not (80%, 4 of 5) feel it would be difficult to locate one.

There was also a split as to whether the physician’s practice had the time and resources available to enter an individual patient into the FDA’s expanded access program. Slightly more physicians (46 [53%] vs. 40 [47%]) report that this process will take too much time. Of those who report the expanded access program will take too much time (n=10), most also report they know nothing at all about the expanded access program (Table 2).

Table 1. Resources needed for expanded access by knowledge of the program [Click table to enlarge]

Physicians thought that the level of difficulty in completing the various steps in the process for expanded access varied between very easy to very difficult, as shown in Table 2.

Table 2. Perceived Level of Difficulty in completing the steps in the process for expanded access [Click image to enlarge]

Preferences for Assistance

Most physicians (67 [78%]) were unaware of any organization or individual that could be contacted for assistance with the aspects of the FDA’s program. Those who were aware of a resource (n=5) cite local IRBs, the Children’s Miracle Network Hospitals, a pharmacist, Kids v Cancer, and Reagan Udall Foundation for the FDA as possible resources.

Physicians were asked to rank the types of assistance that would make them more likely to consider expanded access programs. Having a “website with program guidelines, sample forms, and an online checklist of the steps to complete” was most often (27 [45%]) ranked as the most important. Table 3 lists the types of assistance physicians ranked the most and least important.

Table 3. Types of assistance that would make physicians likely to consider the expanded access program [Click table to enlarge]

When asked which groups are trusted for guidance, information, and assistance regarding the use of expanded access, most physicians reported trusting their specialty’s professional society (73 [88%]), a university or research institute (61 [73%]). Physicians were less likely to trust the AMA (20 [24%]), a pharmaceutical company (19 [23%]), a health-care advocacy organization (22 [27%]), and a health-care foundation (23 [28%]).

Most physicians (62 [74%]) reported an interest in learning more about expanded access for investigational drugs. Of those “very interested,” nearly all were likely to have participated as a principal investigator in a clinical trial.

Right-to-Try Legislation

Most physicians were unaware (74 [86%]) if their state has right-to-try legislation. A smaller percentage (50 [58%]) were unaware that federal right-to-try legislation exists; one-third of physicians (29 [34%]) responded there is federal right-to-try legislation.