Making patients part of the pharma conversation
*Why pharma should give patients a real voice in the drug-discovery process*
By on Oct 26, 2010Why pharma should give patients a real voice in the drug-discovery process
The relationship between pharma and the medical community is being shaken up. While commercial reasons may be the main cause, patients are also driving change.
Worldwide, they are demanding to be part of the conversation, but they start from the disadvantage that they understand very little about pharma and the medical community, or how each of these deals with the other.
We suspect that their interaction is first and foremost about drugs, their efficacy and safety.
Perhaps lurking somewhere in the background is also a discussion about price economics. Is your drug cheaper than his?
Of much more interest is the dialogue we hope takes place where no medicine exists for a specific illness, or where those that do exist have limitations.
Being part of this exchange would be of great interest to patients.
For some, it might be the only way that a particular disease therapy could move up the pharma agenda.
Much good might therefore come if patients had a fuller and more varied interaction with pharma and medical practice.
My own experience may illustrate how far we have to travel and also provide pointers to what could change.
Engaging with patients
I have the rare disease adrenomyeloneuropathy (AMN), which causes progressive neurological dysfunction.
Unlike common conditions, such as diabetes, cancer or heart disease, rare diseases mostly have no cures, and few therapeutic drug agents exist.
Medical professionals, perhaps because of their own inability to offer much, have been quick to encourage groups like ours to meet, share experiences and discuss what pharmaceutical agents or other therapies might offer relief.
I have organized an annual meeting between patients with AMN and medical practitioners for the last seven years. The response from both sides has been very positive.
It has been much harder to persuade pharmas to participate.
Idealism is fine, but pharmas need a compelling business reason to engage more fully with patients.
With an ironic twist, having a rare disease might now be a catalyst by which pharmas do just that.
GSK has noted the potential, seeing a higher probability of success in drug discovery: disease definitions are usually very clear, clinical trials tend to be small with robust endpoints, and the molecular target is often known.
Suddenly, perhaps, specific patient cohorts may have become desirable by pharmas.
Similar targeting also looks promising for common diseases such as cancer, as they are increasingly analyzed into gene-specific sub-types, each potentially amenable to targeted drug development.
So, might rarity, or disease sub-type, and the need to locate and analyze corresponding patient groups be a new stimulus for pharmas to engage?
Time to get involved
Finding such niche patient groupings will not be so straightforward.
Accessing patients through conventional state health providers may be too narrow a focus.
And the majority of the world population resides in regions without nationally organized health provision.
Fortunately, having productive conversations on a global basis with patients is getting easier.
Patients are exploiting new technologythe Internet and more recently its social media aspectto facilitate their conversations.
Currently, though, many of these conversations are random, unstructured and unfocused, from which pharmas must struggle to extract value.
Yet websites such as http://www.healthunlocked.com/ and others are flourishing examples of how patients are doing more than talking.
Useful data of value to scientists and pharmas about clinical symptoms, drug efficacy, or treatment regimes is starting to be gathered using quite sophisticated survey tools. (For more on patient data, see Adherence and the empowered patient.)
The Simons Foundation in the US has been a pioneer, having gathered data from approximately 3,000 families having an autistic child.
Pharma now needs to work more actively with these groups.
Rather than trying to re-invent the engagement model from the ground up, pharmas need to start small and see what works.
At this early stage, it should be less about projects, targeted outcomes, and cost/benefit analyses and more about mutual exploration and an open mind.
These technologies offer real opportunity for moving the drug-discovery process into radical new territory.
And its urgently needed. As one leading genetic scientist I meet regularly remarked, The way we recruit patients for drug trials is a disaster.
What lessons are there?
First, pharmas are not doing enough to find patient groups that could help them. This is not a public relations exercise; its about making the patient experience a real part of the drug-discovery process.
Second, pharmas could help educate patients about how drug discovery works.
Patients are effective lobbyists to health providers and government, but only if they understand more about the process.
Finally, for a pharma, this all takes time, effort and cash.
Patients often have a lot of the first two at least, so pharmas should think about how to use them.
Chris Ogden is a management consultant who also has a rare disease.