No one has all the answers

Realising the benefits of medical innovation to patients, healthcare systems and society is a collaborative effort.

Rapid advances in biology, biotechnology and medicine, increased focus on big data and predictive analytics, complemented by impressive progress in genomics and personalised medicine, are reshaping the way we deliver healthcare to patients.

Unprecedented advances in R&D have created an exciting new wave of medical innovation, with over 7,000 medicines in development for a wide range of diseases, many of which will play a key role in addressing the challenges faced by both patients and healthcare systems. These innovative therapies are capable of improving overall patients’ survival and quality of life, while saving resources for health services when old treatment methods are taken out of commission. This brings wider benefits to society as well, in terms of a healthier workforce.

Let’s take a closer look at just a handful of these ground-breaking therapies:

CAR-T cell therapies allow scientists to engineer immune cells to target specific types of blood cancer, using the body’s own immune defences to tackle the cancer. This innovation could replace lengthy treatments with aggressive chemotherapy.

Cell therapies employ living cells to replace or repair damaged cells in the human body. Cell therapy hold the potential to help patients with Type 1 diabetes control their blood-glucose levels through implanting healthy pancreatic cells, diminishing any ongoing dependency on insulin injections.

Gene therapy goes even further, introducing genes (DNA) to correct or modify abnormal or mutated genes in a patient. This establishes a potential arsenal against genetic diseases, including haemophilia (an inherited condition that affects the body’s ability to clot effectively).

Antibacterial monoclonal antibodies (mAbs) provide new and more effective ways of addressing antibiotic resistance. They offer targeted treatments, in contrast with existing antibiotics which act against a wide spectrum of both disease-causing and beneficial bacteria.

Treatments are being developed to combat Alzheimer’s disease, which is on the verge of creating one of Europe’s most pressing public health crises. Whilst existing therapies only treat the symptoms of the disease, new treatments focus on delaying the onset and / or progression of the disease by preventing or reversing the build up of the plaques in the brain that are believed to cause Alzheimer’s. Serious challenges to developing these new therapies have resulted in some trial setbacks, but the pharmaceutical industry is nevertheless determined to help resolve these issues.

This exciting wave of medical innovation is set to change the outlook for many patients and their families in Europe, bringing value to European healthcare systems and societies. It is vital to acknowledge the role of patients in generating this innovation: without patient volunteers there would be no clinical trials – and without clinical trials there would be no new medicines.

Innovation is only innovation if patients can access it and there a number of potential hurdles that are slowing the adoption of new treatments by our healthcare systems.

For example, as medicine becomes increasingly personalised new therapies are often carefully targeted at small patient populations, using completely new technologies. It can mean there is less evidence available to regulators when a medicine is ready to start making a difference to patients. It delays access for patients and increases costs to innovators, discouraging new advances in these areas.

Regulatory guidance can help manufactures generate the necessary information for regulators and health technology assessment (HTA) bodies to make rapid, informed decisions and minimise access delays. Early and on-going consultation on clinical trial design and manufacturing processes can build our collective experience of introducing novel therapies and guide evidence development for approval. Finally, adaptive licensing could provide temporary access to new treatments, in spite of potential uncertainty, allowing the product label indication to be updated as more data becomes available through the capture and analysis of real world evidence (RWE).

Increased dialogue between industry, HTA bodies and regulators, through a joint scientific advice process, would ensure that the right data is produced at each key decision point in the life-cycle of a medicine. Adoption of flexible HTA assessment processes would allow for provisional access to be provided where benefit at launch is uncertain, by using methods such as predictive modelling. Increased access can then be updated post-launch and/or evolve over time based on RWE.

New technologies often require new processes to deliver it to patients, these are sometimes outside the traditional pharmaceutical delivery pathway. It will require new or upgraded infrastructure, data capture and analysis systems and expert input from patients, clinicians, industry and payers.

There are many examples of new approaches such as establishing integrated care and manufacturing facilities, allowing cell and gene therapies to be produced and delivered at centres of excellence in order to reduce turn-around time and enable collection of reliable patient outcomes data. Early-diagnosis centres with diagnosis labs could be launched to realise fully the value of certain therapies, such as disease-modifying therapies in Alzheimer’s or diagnostic tools for cancer, before patients and healthcare systems are burdened by the long-term consequences.

But with an ageing population and increased prevalence in chronic disease, our healthcare systems are under increasing strain. Add in the costs of all this innovation and payers are left wondering where to turn. The benefits of a new treatment may be realised by patients, by healthcare systems and by society over many years but the reality for payers is the costs are upfront. Holding back medical innovation is no answer, but how we introduce new treatments takes dialogue, new, flexible models and a longer-term, less-siloed perspective.

Healthcare policymakers, authorities, clinicians, patients and the industry will need to work together to ensure that the wave of medical innovation translates in to better outcomes for patients across Europe. That means a shared ambition of sustainable access to new medicines. There are some concrete steps we can take such as: agreeing on a sustainable framework of value that reflects the opportunity for patient outcome focused payment models, ensuring the development of interoperable outcomes data capture mechanisms that can support outcomes-focused pricing models at scale, establishing an agreed approach to horizon scanning at country level providing health planners with 5-year forward visibility of emerging technologies and of course, showcasing examples of best practice.

The challenges faced by our healthcare systems are well documented but despite these challenges there are many reasons to be optimistic about the future of our health in Europe. Whether its evolving the regulatory system to keep pace with the rapidly advancing science or developing flexible financing models, the answers will not be defined by any one group but lie in increasing partnership and collaboration.

Andy Powrie-Smith

Andy Powrie-Smith is Communications Director at EFPIA, the European Federation of Pharmaceutical Industries and...
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