Voices from the front line in rare disease


Online affected person communities have develop into an important useful resource for rare disease sufferers, and pharma is coming spherical to the concept of partaking with them for real-world information. Now these communities may also help present us rare disease sufferers’ ideas on scientific trials throughout COVID-19. Raremark’s Julie Walters tells us extra.

For almost a century, gross sales reps have been targeted on clinicians. After all, they’re the ones who write the prescription. But with 4 billion folks now on-line, greater than half the world’s inhabitants, is it time to re-evaluate the sufferers’ position in the prescribing choice?

Pew Research reviews that 52 million American adults have used the net to get well being or medical info – and 47% of those that sought well being info for themselves throughout their final on-line search say the materials affected their selections about remedy and care.

The want to tell your self as a affected person or caregiver is much more acute relating to a rare disease, in accordance with members of Raremark’s on-line affected person communities.

One of our greatest communities is in the rare disease myasthenia gravis (MG), with almost 3,000 members round the English-speaking world. MG is a rare autoimmune situation, typically misdiagnosed as tiredness or despair.

“There is hope that the next generation of patient communities will go even further, using artificial intelligence to predict which treatments will work best for which patients”

During MG consciousness month, we requested our group what they’d like folks to find out about this rare disease. Here are 10 typical replies:

  • “My doctor said I look just fine! I’m looking for a new primary care doctor. I have given him plenty of time to do some research!”
  • “You need to find a very good specialist.”
  • “Don’t be afraid to be your own best advocate. Speak up the best you can until somebody hears you – and don’t back down.”
  • “No one knows what to do with you! It’s a pure guessing game!”
  • “I’m not lazy or drunk.”
  • “One day you feel decent. The next day you feel awful. There’s no rhyme or reason to it.”
  • “It’s unpredictable for my husband. I am always scared.”
  • “You don’t have to look handicapped to be handicapped.”
  • “It’s kicking my butt right now. Treatment not working.”
  • “It’s not made up. It’s not a figment of my imagination. I am not a hypochondriac. I am not making it up for attention. I do hurt. I do stumble. I do struggle. I am scared.”

Pharma reps then, reliant on clinicians as a path to market, could also be lacking an vital channel to sufferers in want of a second opinion or who’re prepared to change remedies.

Online affected person communities have emerged in the latest decade as an moral interface between pharma corporations and sufferers. The first affected person connector, PatientsLikeMe (PLM), was formally launched in 2006. CNNMoney described PLM as one in every of the 15 corporations that may change the world. For the first time, PLM allowed sufferers to share info with and study from different sufferers with comparable illnesses.

About 11% of members switched their physicians primarily based on their learnings at PLM.

At first, business was suspicious of sufferers speaking to one another. What if they’ve an hostile occasion? What in the event that they don’t like our remedy and inform others? What if a competitor’s drug is extra common with sufferers?

Early innovators in the area used affected person communities to run real-world security and efficacy research. Many of the outcomes had been revealed in peer-reviewed papers to tell medical observe.

Through greater than a decade’s exhausting work out of Boston, PLM proved you can get dependable, real-world proof direct from sufferers.

So the place are we now in 2020? Clinical trials are benefiting from the rising paradigm of going direct to sufferers, and accelerating recruitment timelines.

“Thinking about trials of the future, a majority of respondents preferred trials that didn’t require them to go to the clinic for every appointment”

And industrial groups are studying the place sufferers are misplaced in the system; extra are ready now to reply questions resembling: What kinds of clinicians are they seeing? What lastly satisfied a clinician to do the proper diagnostic check?

Depending on native rules, pharma can even educate sufferers straight on their remedy choices.

There is hope that the subsequent era of affected person communities will go even additional, utilizing synthetic intelligence to foretell which remedies will work greatest for which sufferers.

As one affected person instructed us when requested if something optimistic had come out of their MG analysis: “Absolutely. Proof that it wasn’t ‘in my mind’, wasn’t ‘normal tired’ and (it) provided a therapy, which works for me.”

COVID adjustments

As the COVID-19 pandemic grips the world and thrusts healthcare techniques into unprecedented conditions, affected person communities may give us vital insights into how folks with a rare disease are responding.

We additionally requested our affected person communities how their opinions and behaviours are altering throughout the pandemic. We have almost 2,000 sufferers and caregivers affected by idiopathic pulmonary fibrosis (IPF), a rare lung situation, engaged in an internet group.

IPF is a troublesome, long-term progressive situation that causes everlasting scarring of the lungs. Patients more and more depend on exterior sources of oxygen. Currently, medical doctors don’t know what causes IPF. It normally impacts folks between the ages of 50-70, is barely extra frequent in males than ladies, and is extra frequent in folks with a historical past of smoking.

IPF sufferers are a weak group at the better of instances, not to mention throughout a pandemic when medical appointments are delayed or cancelled. In June, we requested round 50 of our IPF group for his or her ideas on present trials and trials of the future.

Surprisingly, 85% had been nonetheless wanting to participate in scientific trials; now or in the future.

For these seeking to be a part of a trial, the most vital extra security measures throughout a pandemic had been seen as: limiting the variety of folks in the ready room, requiring all of the clinic’s employees to make use of PPE, temperature checks for all sufferers who come to the clinic, hand sanitiser in the ready room and different elements of the clinic, and distancing in the ready room.

For these not on the lookout for trials, some cited the COVID-19 pandemic as the motive, or that they didn’t need to journey proper now.

For the group of sufferers not on the lookout for trials now, a pre-visit guidelines for all sufferers was an vital further security measure that may should be in place to make them really feel snug in collaborating in one.

Thinking about trials of the future, a majority of respondents (each in the trial seeker and non-trial seeker teams) most popular trials that didn’t require them to go to the clinic for each appointment.

In phrases of receiving their remedy, the most votes went to a trial design that included a mixture of clinic visits, at-home visits and common appointments at a hospital nearer to their dwelling, or with their common physician or specialist, to obtain their remedy.

Patients perceived an a variety of benefits from this sort of decentralised trial design, together with the sensible benefits of not having to ask others for assist in attending to the clinic, and fewer time travelling.

IPF sufferers would additionally admire seeing their common physician or specialist throughout a trial as an alternative of medical professionals they don’t know. Using expertise resembling FaceTime or Skype to talk to the analysis employees would even be appreciated.

For some, the boundaries to becoming a member of a trial had been the identical as ever: they don’t know the place to begin on the lookout for related info. Others would first ask their physician or specialist for details about trials obtainable, or a affected person advocacy group. Overwhelmingly, info from their physician or specialist was the most vital issue in serving to them to determine whether or not to truly take part in a trial.

Overall, the excellent news is that, each for industrial and for scientific groups, sufferers are greater than prepared to be lively contributors in their healthcare, significantly in rare disease – pandemic or no pandemic.