Patient recruitment will be 100% online as patients are recruited through internet advertisements (see our explanation on using Google Adwords for patient recruitment) directly through the study-website.
Patients will need to have their blood drawn at either a local clinic or at home. Once enrolled, the investigators will send an electronic diary and the blinded study drugs directly to the Patients. So patients will not have to travel to clinics. Which is an advantage to the patients. While on the other hand, also allows Pfizer to attract a much large population into this trial, as there is no requirement anymore that patients should live in the vicinity of one of the participating sites. So again, a win-win for both sites. And as a side-effect, the population might be different (more diverse ?) than the ‘regular’ trial population.
By reducing the burden for Patients, Pfizer also hopes to get better compliance.

And once the Study is completed, patients will be send their study results. Surprisingly enough, this still seems to be an exception.

It goes without saying that a 100% internet-based trials will not be the final solution for all studies. However, if this first step is successful, and I can’t think of a reason why it won’t be, it will change the landscape for Clinical Sites.
So far, and partly thanks to regulations, Sites were an indispensable factor in recruiting patients. They had access to patients and were the only party allowed to have direct communication with patients. Moving to internet-based trials will partly erase the need to include Clinical Sites.

You can read the full article in the Washington Post.

Study-Portal will exhibit at the ‘Clinical Trial Education and Awareness days’ on June 16-18 in Berlin.
Please drop by and discuss with our team how we can assist you with ‘engaging’ Clinical Trial Subjects.

Personally, I was most struck by a presentation on a very rare disease, the Marshall Smith Syndrome (MSS). What made it is so special was that it was presented jointly by the physician, as well as the father of the young patient. It was clear in this case why they were operating jointly, none could do without the other. The parents were dependent on the treating physician as there is hardly any knowledge available on this rare disease. And the phyisican couldn’t do without the parents as there are less than a handful of patients in The Netherlands.
Nevertheless, I think their way of operating could be an example for many other diseases.

There was also an interesting observation related to community management. The father of the diseased child has created an online community with a patient Forum, a Wiki, etc. Normally, as a rule of thumb, you would expect at least hundreds of participants in order to have a successful community with sufficient number of activity.
In this case, the community consisted of (only) 31 families, and it was very succesful! Which reminded me that there are two key variables to a successful community: Critical Mass and Participation. As parents of these diseased children felt so isolated (one mother had desperately looked for over 15 years to find another parent of a MSS child), that the level of participation more than covered for the lack of critical mass.

And another important lesson I have taken back home is that we are sometimes creating a kind of virtual word around a clinical trial. I will give two examples to illustrate this.

In a presentation by Jeana Frost, it was mentioned that about 25% patients on PatientsLikeMe who use anti-depressants report sexual problems or side-effects. When you compare this rate to the ‘official’ Adverse Events reported sexual related problems, which is about 4%, there is huge gap. Sure, not every mentioning of sexual problems should be regarded an AE. On the other hand, 4% or 25% is a huge difference. So perhaps the whole process of patient screening in combination with the official procedure for AE reporting result in a bit skewed figures on side-effects.
Personally, I would argue that this is another argument to tap into the richness of patient experiences, rather than relying solely on the ‘clinical’ results of a clinical trial.

And last not least, valuable lessons were given by a number of presenters favoring a more prominent role of Patient Organisation in deciding on Trial outcomes or endpoints. The endpoints decided by Pharma are not always matching those by patients.
A good example of this was provided by Michael Rutgers of the Astma Fonds. In a previous role he was heading the Burns Foundation. Based on a survey among the treating physicians, the top-10 of aspects to study were all related to cosmetic surgery or treating the burned skin. While for the actual patients, itching was felt as one of the major problems. In other words, researcher were looking for answers to problems, which were felt as less relevant to patients. And problems which were felt as important by the patients were not investigated.

I guess I am repeating myself when I say that it in order to advance medicine, the voice of the patient needs to be heard.

It is clear that these three opportunities also exist for Pharma 2.0.

• In almost every clinical trial a lot of communication needs to take place between different stakeholders (CRO employees, site investigators, sponsor, or even a trial subject). Social media tools lower the barriers to communication and collaboration between people that are geographically dispersed or would normally not even meet.
• Collaboration leads to creativity, and creativity in its turn leads to innovation. So the use of social media tools, which lowers barriers to communication and collaboration, can lead to interesting insights for Pharma companies on new ideas and feedback on studies.
• The use of social media provide opportunities for Pharma companies to offer better service to patients participating in clinical trials. Nowadays, patients participating in a clinical study expect to have online access to all relevant trial information. And not only one-way communication from the Pharmaceutical company, but also online communication and feedback with other trial participants. Social media tools can provide this need.
  

So, I would say: after the rise of Web 2.0 and Enterprise 2.0 it is now time for Pharma 2.0! What do you think?

What is a ‘Site-centric’ approach?

Using multiple pathways, Sites are identified which are expected to be able to enroll an adequate number of trial subjects. Then a time- and money consuming process is started to qualify each clinical site. It is not untypical for the complete regulatory review and qualification process to take months. Not sure if anybody has ever been ‘daring’ to calculate the cost for qualification per site.
And does this always pay off? No. In some studies, up to about 20% of the ‘enrolled’ sites fail to deliver a single patient. With the percentage of Sites not being able to supply the expected number of Patients being even higher. In fact, patient recruitment issues has been ranked as one of the major causes for trial delays.

What is a ‘Patient-centric’ approach?

A patient-centric approach starts with finding the right patient for a clinical trial. A possible method for finding a suitable trial patient has been detailed in our whitepaper on ‘Using Google Adwords for Patient recruitment’.
If a qualified patient is found, the patient is either directed to one of the existing qualified sites, or, and this is a new avenue, a new site is opened.

The first option, routing a patient to an existing Study Site, is pretty standard and does not require many adjustments to the Site approval process. In fact, the only requirement is that you have your Sites ‘in place’ before you start recruiting patients.

The second option is a novel approach to Patient recruitment and requires a re-design of current workflow and business processes. It expects a system is in place which allows Sites to be reviewed and qualified in a much shorter timeframe, say 2 weeks as a maximum. As another implication might be that you end up having only a single patient at a Site, the Site qualification process will need to be as cost-effective as possible. Moving from the current paper-based, sending documents back-and-forth, approach to a more online approach, will be among the steps which have to be taken.

Which approach is the best?

There is no best method in this. It is clear that the Site-centric approach has proven its value over time. However, if we take again oncology as an example, the trend is that cancer treatments become more and more personalized, making the patient recruitment process more challenging. If you are looking to recruit patients with a rare condition, starting with finding those patients and then finding treatment centers within reach of the patient, might be a more optimal pathway. Compared to qualifying treatment centers first and then wait until a proper patient shows up.
It is clear that this redesign of the recruitment process might bring value to everybody in the process. For the patient as more patients with the rare condition will be able to choose if they want to participate in the trial. For the Site, as no needless time is spent on the qualification process. And for the Sponsor, as they might see higher recruitment rates.

A more detailed discussion of the ins and outs of the Patient-centric approach is part of our ‘Workshop on Patient recruitment and retention’

If you are interesting to learn more about this DataFax interface, we are happy to provide you with Login details to our Demo Portal which provides access to the DataFax reports and graphs. Alternatively, we are happy to arrange an online and web-based Demo of this DataFax interface.

Let’s face it, most clinical trials face the problem of actual patient accrual falling well below expected goals. And with the ever growing number of clinical trials (at any point in time, there are at least 40,000 trials active) and the increasing number of patient per clinical trial, this problem is not going to fade away easily.

So time for some new tactics. Let’s start by looking at this problem from the point of view of the subject. Hmm, that’s new isn’t? From the Subject’s angle, participating in a trial is usually not to the best of his/her personal interest. Okay, there is a chance that he/she might get a novel treatment. But can we guarantee to the best of our knowledge that this novel treatment is any better than the arm(s) with existing treatment(s)? No, we can’t, that’s why we call it a trial.
Thus we need to convince people in order for them to participate in a trial. A basic rule in marketing says: “To succeed in getting people to do something you want them to do, you must offer them something they value in return.”
In a commercial transaction, this is easy to accomplish. But how about clinical trials? The key part in our marketing rule is the last part of the line ‘offer something they value in return’.
First of all, we must establish what people perceive as ‘value’ in the context of a clinical trial. Although we can make some educated guesses (like ‘I might get a new treatment which might cure my disease’ or ‘It helps to improve medical treatment’), the best thing to do would be to ask the actual stakeholder, yes, the patient himself (herself).
Once you know what the reason is for participation, you could invite some study participants for a video interview elaborating their trial participation/motivation. And post these video’s on your Study website. What would be more convincing than hearing another patient explain why they participate?

Now that we know what constitutes ‘value’, how do we supply ‘value’? In my belief, they best of value you can supply is information. If patients participate because they believe it helps to improve treatment, you could supply ‘value’ to them by informing them on the current status of treatment (for their disease), and more importantly, on the results of the clinical study they are (have been) participating. Even if the outcome is negative, provide feedback. Create a secure web environment where only participating patients have access to the study results and give them regular updates. Similar, though in easier-to-understand language, to the study results you supply to the Clinical Sites (you are doing that aren’t you ?).

Next week another Blog post on Patient retention in clinical trials. So stay tuned.

Step 1: The Clinical Study Portal is a secured environment, which requires user authorization before Users can logon. So it is like a gated community. Everything said within this ‘trial community’ will only be ‘heard’ by other group members. So this limits your problem to a ‘known’ group of people.
Step 2: Having said that, we still have our question “Do we, as a Bio-Pharmaceutical (or Medical Device) company, carry responsibility for anything said by a Patient on ‘our’ Forum as made available within a clinical trial setting?” The answer is ‘No’ and ‘Yes’.

‘No’ to the extent that it should be made perfectly clear to all participants that the intent of the Forum is to create a platform which allows more ‘openness’ and sharing of ideas and opinions within all trial participants. And that you, as a Sponsor, carry no responsibility for anything said or listed. In fact, Patients should feel free to discuss anything they want on a Forum.

‘Yes’ to the extent that this doesn’t alleviate you from your legal responsibility to report Adverse Events to FDA or EMEA. So does this imply that you as, as a Bio-Pharmaceutical company, should monitor all what’s being said on a Forum? Time for a next Step.
Step 3: So where do the previous two Frameworks lead us? We know we are dealing with a ‘known’ group of users, we have stated that patients participating in a Trial Forum should feel free to discuss anything they want, and we have confirmed that you, as a Bio-Pharmaceutical company, have a legal responsibility to report any Adverse Event.
The solution then, is to set a couple of simple ‘rules of engagement’ before you allow Patients access to the Forum or other Social Media tool. These rules can be simple and straightforward:

• All Patients should feel free to discuss anything they want on the Forum, including any side-effects or other complaints they might experience.
• You, as a Bio-Pharmaceutical company, have a legal responsibility to report any Adverse Event.
• You don’t want to review everything what is being said on the Forum to check if there is any reference to an Adverse Event.
• Patients are free to discuss any side-effect or complaints, as long as they have also reported that side-effect or complaints to the responsible trial investigator.

In other words, the responsibility for proper reporting of any Adverse Event rests at the patient. Is this new? No, because even without the additional communication tool of using a clinical trial Forum, it is the patient’s decision whether or not to report a side-effect to an investigator.