CHANGE!

[CPT_Tom]

CHANGE!

In the past year my Parkinson’s medication, L-dopa, and I have both celebrated our 40th birthdays. In some respects, I suppose that I am lucky that for the last 13 years, I have been able to swallow varying amounts of this drug everyday. It is fantastic. Without it I would not enjoy my life. Despite this, I do not celebrate its 40th anniversary because, in that time, there has been nothing to even come close to replacing it. It has remained the so-called ‘gold standard’ treatment for my illness.

How can this be when every week we hear stories in the media of new therapies, even potential cures for this chronic neurological condition?

The truth is that pioneering new treatments for Parkinson’s have become more of a breeding ground for breakdowns than breakthroughs. The current system of bringing new treatments to market is almost totally reliant on biotech and pharmaceutical companies whose first responsibility has to be to their shareholders, otherwise there would be no investment in this sector at all.

Despite this, these industries are charged with the responsibility of taking basic science and delivering it through an extremely costly, highly risky and prohibitively long regulatory and clinical trial process to ensure safety and efficacy for all new products. The likelihood of taking any product out of basic science and translating it into something for use in the clinic is slim. At best, only one in ten products which reach a Phase I trial actually make it onto the market. There is, therefore, an understandable reluctance to pioneer new products and a propensity towards adapting existing products rather than investing heavily in new science which is high risk and hugely expensive.

Ultimately it is the patients who suffer from these imperfections in the market and the shortage of the development of new treatments.

A further by-product of this lack of control is that pharmaceutical products which do reach the market tend to be disproportionately expensive compared to the real cost of manufacture. This stems again from the totally commercial nature of the development process. Given the frightening statistics on the future burden of healthcare for chronic conditions such as Parkinson’s, it seems likely that this situation will become unsustainable. National Health organisations such as the one in the UK will not be able to maintain the standard of care to which they aspire unless other methods and strategies for drug development are generated. In other countries the system would be far worse, increasing the polarisation of access to medicine between rich and poor.

The three principal stakeholders in the drug development process are the patient, the pharmaceutical industry and the state - which exercises control over the entire process through regulation, legislation e.g. patents and distribution. Each of these parties have different priorities all of which are positively influenced by innovation; patients feel better, pharma company profits increase and the costs to the state will be reduced dramatically if the new therapies are disease-modifying in nature.

It seems obvious therefore that the duty of encouraging innovation should be a collective responsibility between all three stakeholders. An organisation which pooled the expertise and interests of these stakeholders could act as a hub through which exciting new products could be provided. These prospective treatments could have a structured plan for their development and the resource to accelerate the process. The hub could also provide much needed governance to early stage drug development, making it more risk averse, quicker, cheaper and generally more focussed and effective.

Above all else a culture of teamwork would be created by this new structure. Organisationally, it would sit mid-way between the commercial and public sectors; commercially run but with the profits being reinvested into the business thus retaining its charitable, moral and ethical status. The goal would be to deliver therapies to a stage where the three critical pre-cursors to medical progress would be satisfied; therapeutic benefit, value to the state both in terms of cost and benefit and thirdly, commercial viability.

In this way, therapeutic benefit to patients would be the principal focus but without compromising the required outputs of state and industry.

There is sufficient evidence to suggest that there is science out there to make a real impact on the lives of people living with Parkinson’s – not just in the future – but now! Structural change to the system is needed to convert this science into tangible treatments and such change can be achieved to the advantage of all of those involved in the process – not just the patients.

Is this relevant in the States? I think more than anywhere. Even in 1988 it was cited that by slowing the progress of PD by a mere 10% would save US$327 million p.a. in direct and indirect costs to central Government. Given that a more recent figure suggests that the total socioeconomic burden of PD is projected in the region of US$23 billion p.a. and given that due to simple demographics this figure is set to rise dramatically, there is a pretty good reason for everyone to support the speedy development of potentially disease modifying treatments.

So just to recap:

We need to minimise the hazards involved in the drug development process by spreading risk between the three stakeholders.

We need a critical path upon which good prospective treatments cannot deviate due to commercial frailties

We need a sense of teamwork and “can do” rather than the existing culture of barriers and individual agendas.

AND

We need a Parkinson’s specific hub made up of representatives of those living with the condition, industry and Government (NIH, FDA) to bring focus, drive and ethical and moral status that is incontrovertible. This hub can be the conductor. With help and resource, it can assemble the finest orchestra with the finest instruments and it can re-create the most wonderful harmonious movement ever written – our own.

We have strong argument for change – we are costing a fortune and we don’t look too good either! Let’s use these two arguments and make the case for change. ENOUGH TALK LET’S DO!!!

That is my view. Anyone with me?

Tom Isaacs

The Cure Parkinson’s Trust

cureparkinsons.org.uk

[Lindylanka]

I have watched and waited for some great replies to this blog to appear, and even though it has been up a while - nothing. Which is disappointing, because I read this as not a rhetorical article, but a real challenge to move for change

Is the absence of comment because most of us know that change is needed, and have already identified the obstacles, or because the answers are much harder to find?

The idea of a PD hub is wonderful - though I still feel as though out of the trio of stakeholders that Tom has identified, patients are sitting under the table, while the others are at it, talking to each other, and not hearing what is said down below. So either the music has got to get louder, or it has to be so compelling that it cannot be ignored.

Talking to someone in an exceptional position to push for change, it became clear that not just for us, but for other players too, one of the biggest obstacle is the absence of of truly effective strong patient voice. By that I don't mean that it is not there, just that it is not yet being listened to in the right way. So long as we are a background murmur we will not be heard. Our voice needs in some way to be integrated into the whole process, we are a bit like the missing link!

Identifying OUR priorities for action to bring about change has to be urgent. We have the wonderful harmonious movement, it is here, right now.

I have loads of things I would like to happen, and I am sure that each of the people here has a list of their own, but until we get them out and start sharing them visibly we will not be able to move towards that change.

My 'urgent priority' is that patients are involved in helping to defining the subtypes of PD, without which it is unlikely that better treatments will emerge.

The reason I believe this is incredibly important is that drug trials often do not meet their endpoints because there are flaws in trial design and the way the trial cohorts are recruited. So this is a complex ask, with patient advisory input from the outset.

The two most compelling pieces of research that I know of that that approach this are MJFF's PPMI study, for biomarkers, and the much less conventional 23&me genetics initiative for PD. Both need more recruitment. The PPMI study, as with many requires untreated 'de novo' patients with a lot of commitment. Hard to find.....

And of course many patients around the world are looking at the various GDNF studies that have either started, or are about to start...... and supporting them in every way they can......

How can we help with these and other initiatives. Sometimes I feel as though patients are well regarded as potential fund-raisers, and that support is well respected. More is needed though...... Having a strong voice is not just about shouting louder, or even in the right direction. There has to be somebody listening too. Just as in the video that Sara posted, about Health 2........ if you haven't watched it yet, give it a go, it is all about the need for communication, and a bottom up approach....

There has been a long standing debate in the PD community about the need for a database of patient supplied information. With a huge cohort, of many thousands. This has not happened, though Sweden recently became, I believe, the first country in the world to have a national database for PD. As PwP we can push for things like this.

It's a matter of setting out our priorities and becoming an orchestra for change........ and not playing under the table, but being at it, and heard.

Others will have different priorities - these are just my thoughts - would love to hear what they are. Tom has set out the stage beautifully, how we need to inhabit it and bring it to life......

So what do we need to do?

[PeggyT]

I just read both of the above comments, and I must admit, I feel a bit (OK, a whole lot) intimidated by your knowledge and insight. I feel as if I'm just one PwP, and I'm trudging along on this journey alone. I've felt more connection through this website in the past couple of weeks since I discovered it than I have in the 9 1/2 years since I've been diagnosed.What can I do? Where do I start? Most of the time, following a doctor visit, I've felt like I know more about my condition than my doctor, and I agree that a strong, united, patient voice is lacking. So, to reiterate Lindylanka's question, "What do we need to do?" The priorities of the US pharmaceutical companies are totally skewed; research is inhibited by government regulations; and the patients are supposed to be just that--patient. The fact is, during the time we're being patient, and waiting...and waiting...for breakthroughs in research, we're losing quality life time, and the quality is diminishing every moment. Tell us, oh wise Tom, where do we go from here?