Physicians

As a part of the ROADMAP project participation, we asked the rare disease nonprofits to forward the survey to their US-based physician network and invite them to take the survey. Our focus in the physician section of the survey was on off-label drug prescription practices, the factors that physicians consider when deciding to prescribe a drug off-label, as well as their involvement in clinical trials for rare diseases. Although the sample size of participating physicians was small (21), we present the data as an initial reference for understanding physician involvement in different aspects of drug repurposing initiatives. Making broad generalizations as to physicians that treat rare disease based on this data may be challenging based on the small sample size.

*Note that most of the survey questions were not required, so the total number of responses is noted for each graph, as they vary.

Prescription of off-label drugs

Off-label drug use for rare diseases can offer a viable treatment option for patients when approved therapies are not available. The fact that a significant proportion of physicians in the dataset (70.6%) are aware of off-label drug options for the rare diseases they treat is encouraging and highlights the importance of staying up-to-date with the latest research and treatment options. However, it is important to note that off-label drug use should be closely monitored and that additional research is needed to better understand the potential benefits and risks of using these drugs in the context of rare diseases.

n (physicians): 17

Off-label drugs: patient’s symptom reduction effectiveness

When asked how effective these off-label treatments are at reducing overall symptoms in their rare disease patients, physicians reported that 18 (69.23%) of the drugs are somewhat effective while only 5 drugs are very effective.

n (drugs): 26
n (physicians): 9

Off-label drugs: preventing patient’s disease worsening

When asked how effective these off-label treatments are at preventing overall disease worsening for their rare disease patients, physicians described 17 (65.38%) drugs as somewhat effective, 4 as very effective and 5 as neutral or ineffective.

n (drugs): 26
n (physicians): 9

Off-label drugs: effectiveness at improving patient’s overall state of health

When asked how effective these off-label treatments are at improving the rare disease patients’ overall state of health, physicians listed 20 (76.92%) drugs as somewhat effective, and 3 drugs each as very effective and neutral as to the effects, highlighting the need for continued research and monitoring of off-label drug use.

n (drugs): 26
n (physicians): 9

Off-label drugs: effectiveness at addressing all of patient’s rare disease challenges

Physicians in our data reported that 80% of the current off-label options do not completely address all of the challenges associated with their patients’ rare disease. The statement emphasizes the importance of providing more choices for patients with rare diseases and monitoring the usage of off-label medications over time. This can help identify which patients could potentially benefit from a specific drug, along with information about appropriate dosage and treatment to maximize benefit.

n (drugs): 25
n (physicians): 9

Off-label drugs: patient’s access roadblocks

The most common roadblock in access to the off-label treatments was insurance coverage (12 drugs), while accessibility and affordability were reported for 9 drugs each, suggesting that addressing financial barriers to treatment access could greatly benefit many patients with rare diseases.

Note: respondents were able to select multiple answer choices.

n (drugs): 26
n (physicians): 9

Off-label drugs: frequency of prescription

Almost half of the physicians who took the survey (10, 47.62%) said that they prescribe specific off-label treatments to their rare disease patients often, while 2 (12%) stated that they prescribe them for every rare disease patient they treat.

n (physicians): 17

Off-label drugs: factors affecting prescription decisions

Among the various factors a physician can consider when deciding whether to prescribe an off-label drug, the top three reported were recommendations by physician-experts (reported by 12 physicians), the side effect profile of the drug (10 physicians) and clinical research evidence (8 physicians).

Note: respondents were able to select multiple answer choices.

n (physicians): 17

Off-label drugs: sharing clinical response

Sharing a patient's clinical response to off-label drug use with other physicians can be incredibly valuable for several reasons. It allows for collaboration and the exchange of ideas among medical professionals, which can ultimately lead to better patient outcomes.

In our data, 53% of physicians reported to regularly engage in sharing information about their patient’s clinical response to treatment with off-label drugs.

n (physicians): 17

Off-label drugs: format of sharing clinical response

Physicians who reported regularly sharing information about their patients' clinical response to off-label drug treatment indicated a preference for sharing through published literature, and less so through medical forums, registries, WOM and through serving on an SAB/MAB.

n (physicians): 9


Drug repurposing

For purposes of this project, we utilized a broad definition of drug repurposing, as a process of research to identify potential treatments that are already FDA-approved or in development for one disease, for use in another disease by gathering data and analyzing efficacy in order to improve treatment guidelines and access.

Drug repurposing: level of familiarity

More than half of physicians in our data reported being somewhat or highly familiar with drug repurposing (12, 70.6%). This is promising, as it suggests that physicians are aware of ongoing research for alternative treatment options beyond their approved uses, which could potentially lead to the development of new treatments for rare diseases and other conditions.

n (physicians): 17

Drug repurposing: awareness of clinical trials

It is encouraging to see that the majority (65%) of physicians reported being aware of ongoing clinical trials. This suggests that physicians are keeping up-to-date with the latest research in their field, which can inform their treatment decisions and ultimately improve patient outcomes.

n (physicians): 17

Drug repurposing: clinical trial referrals

Of those aware of ongoing clinical trials, all but two physicians (9, 81.82%) said that they have referred patients of theirs to these trials. This high percentage is a positive sign of their commitment to providing their patients with the most up-to-date and effective treatment options available.

n (physicians): 11

Drug repurposing: willingness to serve as a clinical trial co-investigator

100% of our participating physicians stated that, if the opportunity presented itself, they would be willing to be a co-investigator on a clinical trial to assess the efficacy of a drug that is being repurposed for a rare disease that they treat. This willingness to participate in clinical trials can facilitate the development of new treatment options for rare diseases.

n (physicians): 17

Drug repurposing: factors affecting willingness to serve as a clinical trial co-investigator

Among the various factors physicians would consider when deciding whether to be a co-investigator on a clinical trial, the top ones reported were the fact that clinical trials are vital tools in rare disease research and being able to bring a direct benefit to their patients.

n (physicians): 17