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New plan on how pharmaceutical science can create better medicines

Elizabeth Sukkar finds that every pharmacist and pharmaceutical scientist can do their bit to promote the science underpinning pharmaceutical practice.

As a pharmacist, when I counsel a patient about their medicine, I cannot help but think about the work that went on before it arrived neatly as a package in the pharmacy. The work of pharmaceutical scientists, formulation pharmacists, researchers, clinicians, pharmaceutical companies, drug regulators and patients themselves has all contributed to create a product that could improve health outcomes. In short, a multidisciplinary effort.

After a two-year process, the Royal Pharmaceutical Society has launched “New medicines, better medicines, better use of medicines”, a 119-page report which covers the staggering breadth of work that goes on behind the scenes to develop new medicines and improve how they are used.

It contains seven recommendations for action (see below), which in essence call for a new covenant to be developed between the pharmaceutical industry and wider society to accelerate the development of new and better treatments. I struggled to think of the right word to describe the document, and “celebration” is the one that seemed most appropriate — a celebration of the science underlying pharmaceutical practice. It is dotted with anecdotes of pharmacists’ and pharmaceutical scientists’ achievements.

Action plan for better use of medicine

The seven recommendations for action

“We did not set out for it to be a celebration, but it is inevitable that it may have that tone,” Theo Raynor, professor of pharmacy practice at the University of Leeds, tells me. He is one of the 17 experts on a relatively new body, the Pharmaceutical Science Expert Advisory Panel, that the RPS brought together to create the report.

But why has the RPS created this “guide” and are there similar documents already out there? The recommendations seem broad, ranging from ensuring the safe use of medicines, the adoption of new technologies and supporting the pharmaceutical science base in the UK, through to improving access to medicines in developing countries.

“It is very broad and that is the beauty of the RPS, and it reflects the membership,” says Professor Raynor. Jayne Lawrence, RPS chief scientist and professor of biophysical pharmaceutics at King’s College London, who worked closely on the guide, tells me: “There was no document that detailed what pharmacists and pharmaceutical scientists did across the whole spectrum of science.”

Another panellist, Janet Halliday, who is associate vice-president of special delivery technology at Ferring Controlled Therapeutics, adds: “There was nothing out there to look at new compounds right through to the impact on patients. You will see other reports look at certain aspects [of this], but not from the perspective of the pharmacist.” Others panellists think the RPS must shape the way in which medicines benefit society.

“RPS members are positioned at all stations along the route taken by a molecule as it progresses from first synthesis to the patient. As a Society, we have a unique position to view the value added at each stage of drug discovery, development and clinical use. An appraisal is long overdue,” says Steve Wicks, director of research and enterprise at the department of pharmaceutical, chemical and environmental sciences, University of Greenwich.

Challenges in drug development

It is no secret that the pharmaceutical industry has been struggling for years to deal with increased regulatory burdens, cost-conscious payers, a patent cliff for major selling drugs, as well as responding to the demise of the “blockbuster” model, which worked well in previous decades but now is being increasingly replaced by more niche, personalised medicines.

The guide stresses that the high rate of drug attrition is often not understood, with only a few of the many thousands of molecules making it to the development stage. Taking a medicine from concept to market takes about 12 years and over £1bn. One of the guide’s key recommendations is to encourage the development of appropriate models of reimbursement to support the use of new technologies and encourage further development of these types of medicines.

The guide looks at the clinical trial process, noting that more than 50 per cent of drug failures are at the phase II stage due to lack of efficacy.

When a drug reaches the phase III stage, the failure rate drops to 2–5 per cent, but this still leaves the company with considerable unrecoverable expenses. Research is a circle, the guide states, explaining that patients’ perceptions and use of a medicine can feed back into the drug development process to influence the development of new drugs.

Some important statistics are set out in the document: that around 20 per cent of today’s top 100 medicines originate from research performed in the UK, and that the industry invested £4.9bn in UK research and development in 2011.

The industry employs some 72,000 people, with 27,000 in research and development, but there have been changes in where they work. The number of employees in large companies is decreasing, while the number in small- to medium-sized enterprises is on the rise. This month alone, there are signs that megamergers in the industry are back on the agenda; Pfizer’s £63bn bid for AstraZeneca has opened up concerns among politicians about how the US company may alter Britain’s pharmaceutical science base. Government initiatives, such as the “patent box” and “innovation platform”, need to be regularly monitored to see if they are achieving their aims and, if necessary, modified to ensure reinvigoration of the UK industry, the guide says.

Yet the industry is advised to develop new innovative models and an understanding of how best to share knowledge and costs among collaborators. The guide turns to the issue of pharmaceutical science workforce planning, stating that a constant supply of knowledgeable pharmaceutical scientists is required. The panel that worked on the guide would like to see the UK’s position as a major player in the global sector strengthened. This is what the industry itself has been saying for years.

Drug discovery processes have changed, too. Highlighted is the important role played by high throughput screening, structure-based drug discovery, microarray analyses, proteonomics, systems biology and systems pharmacology. The guide adds that, although there has been an expectation that mapping the human genome would revolutionise the diagnosis and treatment of disease, only now is this promise starting to be realised in terms of new medicines.

One of the reasons why genomics has yet to have its full impact on patient therapy is because of the mistaken premise that all illness is solely genetic, neglecting the major role played in some diseases by environment and lifestyle factors, such as diet and smoking, the guide says.

When medicine gets personal

The guide acknowledges that a “major change” is occurring in drug discovery, in that typical low molecular weight drugs are being increasingly supplemented by large complex biological products. Well-known examples include insulin, monoclonal antibodies, blood proteins, cytokines, vaccines, cells and tissues.

It also looks at the promising role that could be played by regenerative medicine and stem cell therapies.

The role of stratified (or personalised) medicines, it says, has only become a realistic proposition for the treatment of many diseases because of the recent advances made in pharmacogenetics and pharmacogenomics.

The role of these medicines —to improve the efficacy of a drug while reducing side effects — is “extremely exciting”. But for stratified medicines to become a reality, a significant number of scientific, ethical and social barriers need to be overcome, as well as a consideration of the likely cost and whether it is feasible for all patients to access stratified medicines for their particular medical condition, the guide says.

The report also notes that stratified medicines pose challenges because the genetic causes of diseases are often complex, with only a small number of conditions having a single gene defect responsible for the disease.

In the vast majority of cases the disease is polygenic, and this is further complicated by the role of epigenetics —that is, environmental and lifestyle factors.

For a long time now, commentators have been predicting that the industry’s blockbuster model is dead. So are stratified medicines the next big thing and what are the risks to patients and payers?

“It is a big thing in that medicines are getting more tuned to patients,” says Dr Halliday. “They are more expensive to the NHS and payers because the recovery costs for developing them are based on fewer numbers of patients requiring them.” She also acknowledges that their safety profile is different to traditional small molecules. You may see immunological responses or dormant tuberculosis developing in users of tumour necrosis factor inhibitors, for instance.

Professor Raynor says: “These new medicines will be very expensive and will have novel ways of being administered, so patients’ understanding is crucial. So pharmacists are at the front line of explaining these medicines.”

Particularly their risks and benefits, he adds. Pharmacists need to keep up their competencies to deal with all medicines, including stratified medicines, says Gillian Hawksworth, a community pharmacist and senior pharmacy lecturer at the University of Huddersfield, who also worked on the guide.

Professor Lawrence says that stratified medicines are still “very expensive” and that they won’t be used to treat headaches, for instance, only serious diseases. Moreover, she believes that society needs to think about creative funding models to pay for them. She is also concerned that some patients may not understand that stratified medicines will only suit a small group of patients. “Over time, we hope to get medicines to all patients.”

The panel behind the guide tried to look beyond “trends” in science and pharmacy practice to identify enduring problems that require careful research, effort and funding to overcome, according to Professor Wicks.

He says: “The stratified medicines idea, as applied to cancer therapy, is very similar to the 30 year journey of discovery in HIV/AIDS — the biology of the disease, the biology of diagnostics and, of course, the drugs are different but the research philosophy is the same.”

The dilemma around funding new costly anticancer drugs like Kadcyla (trastuzumab emtansine; Roche) is highlighted as an example by Professor Wicks. The £90,000- per-breast-cancer-patient drug was provisionally rejected by the National Institute for Health and Care Excellence last month (April 2014) because it “does not work well enough to justify its high cost”.

We are living in an incredibly exciting age, where the human genome is being decoded and scientists are examining how we can cure diseases rather than just offer symptomatic relief. However, the market is not set up to incentivise the production of new types of medicines.

Professor Wicks says: “Plug £90,000 per patient per year for 5.8 month extension to life into the NICE QALY [quality-adjusted life years gained] model and it is not difficult to conclude that, in the current economic climate, the drug is unaffordable. There is now a dilemma. The patient cannot access the drug unless the cancer drugs fund supports his or her application; the drug company cannot, or will not, set a reasonable price that the NHS is willing to pay. The RPS can and should enter and show leadership in this debate.”

Professor Lawrence adds: “We are living in an incredibly exciting age, where the human genome is being decoded and scientists are examining how we can cure diseases rather than just offer symptomatic relief.

However, the market is not set up to incentivise the production of new types of medicines.” She continues: “We need the NHS to be able to cope with funding treatments which have a high initial cost but may not require a lifetime of use by patients. Unless we find a way to develop treatments that cure illnesses in months, rather than treat symptoms for years, we will not see the breakthroughs that both scientists and patients want.”

Kevin Shakesheff, professor of advanced drug delivery and tissue engineering at the University of Nottingham, thinks future medicines will be highly personalised, for example using stem cells and regenerative drugs to treat patients after a stroke, heart attack or spinal cord injury.

“These treatments will transform medicine but we face major hurdles getting them into widespread clinical use. They present new problems in manufacturing, safety and economics and require new ways of working
across disciplines in order to accelerate commercial and clinical success,” he adds.

Richard Bergström, director general of the European Federation of Pharmaceutical Industries and Associations, says there is a lot of “excitement” in the industry about all the targeted medicines that are in the pipeline.

However, he warns that there is also concern that the regulatory requirements and way forward for developing new drugs are not clear.

“We need the regulators to move with the science and update their guidelines as our understanding of disease changes.”

Drug discovery: do people understand it?

“New medicines, better medicines, better use of medicines” makes interesting

reading, but do pharmacists understand the drug discovery process?

Steve Wicks “suspects” that pharmacists working in the pharmaceutical

industry and academia, as well as early career and specialist pharmacists,

would have a comprehensive understanding of the process.

“It is important that pharmacists understand drug discovery and development, and our document seeks in part to educate. However, we also want to inspire early career pharmacists to become active participants in the drug discovery and development process. Science and practice should be complementary and not polar opposites whether you are in the high street, the hospital or the laboratory,” Professor Wicks says.

Janet Halliday says that to some extent pharmacists do understand the process, but not necessarily in the field of biologics, tailored or stratified medicines. “Some pharmacists are challenged on this topic, even if newly qualified.”

Theo Raynor says: “I think they [understand] as much as they need to. We have all got to keep up with new developments in pharmaceutical science, for example, stratified medicines.”

Yet Professor Lawrence remarks: “If you don’t understand the drug discovery process, you can’t be an effective pharmacist.”

This document is seeking wide appeal, so perhaps a better question is whether patients understand the drug discovery process. Professor Raynor believes they do not because there is “no real source to access this information for lay people”. However, he notes that the European Patient’s Academy on Therapeutic Innovation (EUPATI), which launched in 2012, aims to help citizens understand how medicines are tested and licensed. But where should the responsibility lie? With pharmacists, companies or both? “It is both and also the Medicines and Healthcare products Regulatory Agency,” says Professor Raynor.

Dr Halliday states: “If you listen to the radio, there is a new compound which will be a wonderful [treatment], but it will take at least 10 years before it is available. I don’t think patients understand this [concept]. I think pharmacists have a role in educating people on aspects of drug discovery.”

Patients seeking treatment for diseases where their needs are well met will probably take the discovery process for granted, Professor Wicks believes. But he points out: “I have always been amazed at the depth of knowledge shown by patients, and the families of patients, in cases where serious illness is not well treated or terminal. In this situation, the drug discovery process is a lifeline and the knowledge of the HIV patient in the 1990s or the breast cancer patient today is encyclopaedic.”

He believes pharmacists should be in a position to help patients navigate the drug discovery and development process when this becomes a need. “As we rightly claim to be experts in medicines, it does our professional credibility no good if our patients are more knowledgeable than we are,” he says.

Recommendations and next steps

Pharmacists and pharmaceutical scientists reading the guide may consider some recommendations to be more important than others, depending on their background and expertise.

Dr Halliday says: “To me, personally, recommendation 6 on supporting
pharmaceutical science in the UK is [important], because I work in industry, but other members of the panel may think another is important.”

Professor Raynor says: “I don’t think you can say which one is the most important because if a drug does not work, then it is end of story.

Personally, I want people to understand the risks and benefits of
medicines, which is covered in recommendation 1.”

Dr Hawksworth agrees: “Recommendation 1 is the most important to me because our [pharmacy] contract is about talking to patients about the safe use of medicines.”

The spirit of the document is to advance the seven recommendations as being equally important, according to Professor Wicks, who says that the political challenge is therefore the order of implementation, given scarce
resources. “Our aim is that RPS members embrace all recommendations and use their personal influence to promote clinical and scientific research to address these issues,” he suggests.

The next stage is to plan implementation of the recommendations. The RPS has committed action on recommendations 1, 2, 3 and 5, according to Professor Lawrence.

If acted upon, the recommendations will assure world leading status for the UK pharmaceutical sciences in the next 10 years, Professor Wicks believes. “If we shrink from the challenge, there are many others willing and able to step in.”

Ultimately, Professor Raynor says, the RPS cannot take the recommendations forward on its own; collaboration with others, including the scientific community and pharmaceutical industry, is needed.

Nevertheless, he adds: “I think the prime movers will need to be the RPS and its members.”

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2014.11138175

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