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.
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?
The Cure Parkinson’s Trust