HDA Media And Political Bulletin – 22 July 2016

David Mowat appointed pharmacy minister

21 July, The Pharmaceutical Journal

David Mowat has been announced as the new Parliamentary Under Secretary of State for Community and Healthcare. His portfolio includes pharmacy among others. He joins the Department for Health along with Philip Dunne, who is now Minister of State for Health, and Nicola Blackwood as Parliamentary Under Secretary of State for Public Health and Innovation.

MHRA data integrity guidance: 18 months on

21 July, MHRA Inspectorate, David Churchward

Eighteen months have passed since the MHRA published its GMP data integrity guidance. Following this the pharmaceutical industry has been seen to make significant effort to implement the guidance globally. The MHRA is supporting the World Health Organisation and the European Medicines Agency for the preparation of regulatory guidelines, as well as working to raise awareness of data integrity challenges that face the industry and its regulators. The MHRA are also working towards the next phase of guidance, with a good practice (GxP) focus building on the past 18 months, to address gaps and clarify expectations.

Multi-indication pricing: no longer mission impossible?

              Pharmaphorum: Deep Dive, Leela Barham,

Economic consultant Leela Barham investigates the potential for the use of multi-indication pricing (MIP) to becoming more widespread as we see a push for a value-based approach to drug pricing coupled with a smarter use of data. Under MIP, the prices of drugs with more than one indication differ depending on value in each use. The concept of MIP has appeal because it aims to send a clear signal to the market about the different values in use for a single product.

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Full Coverage

David Mowat appointed pharmacy minister

21 July, The Pharmaceutical Journal

David Mowat, Conservative MP for Warrington South, has been announced as the new Parliamentary Under Secretary of State for Community Health and Care.

Mr Mowat’s portfolio includes pharmacy and all other aspects of primary care, as well as adult social care, community services, cancer, dementia and learning disabilities.

He succeeds Alistair Burt MP, former Minister of State for Community and Social Care, who announced his resignation from the post after the EU referendum. Burt had previously stated he intended to carry on his duties until September but Mowat’s appointment to the role was announced during the finalisation of Theresa May’s cabinet reshuffle since taking over as prime minister.

Mr Mowat, who grew up in the Midlands, was elected to Parliament at the 2010 general election, gaining the seat from Labour. He previously trained as a chartered accountant and worked at the consulting firm Accenture for a number of years, going on to become one of its leading partners. He previously sat on the Scottish Affairs Select Committee, and has also served as private secretary to Greg Clark MP.

He joins the Department of Health alongside two other newly appointed junior ministers: Philip Dunne MP for Ludlow, who is now Minister of State for Health, and Nicola Blackwood MP for Oxford West and Abingdon, who is Parliamentary Under Secretary of State for Public Health and Innovation.

In a message posted on Twitter, Mowat said he was “delighted and honoured” to be joining the department.

MHRA data integrity guidance: 18 months on

21 July, MHRA Inspectorate, David Churchward

Eighteen months have passed since the MHRA published its GMP data integrity guidance. Since then we have seen pharmaceutical industry make significant effort to implement the guidance globally. It has been encouraging to see industry stakeholders and regulatory partners adopting a common approach in their own publications and the messages and themes being echoed in online discussions, workshops and conference presentations.

International convergence

Convergence is important in facilitating common expectations for industry. It also enables regulators to work together more effectively by facilitating information sharing and supervision of the global pharmaceutical development and supply chains.

The international hot topic of data integrity continues to develop. In the past year MHRA has supported the preparation of regulatory guidance from the World Health Organisation and the European Medicines Agency inspectors working group.

Together with colleagues in the Australian Therapeutic Goods Administration, MHRA is co-chairing a data integrity working group within PIC/S. This group of representatives from fourteen authorities spread across four continents has been working to produce guidance for participating inspectorates. This is developing existing convergent requirements into a harmonised approach to data integrity in the context of GMP and GDP inspections.

Our team has also been working with stakeholders in the international GLP and GCP networks to raise awareness of the data integrity challenges facing industry and regulators.

But we haven’t stopped there.

Revision of data integrity guidance with GxP focus

During this busy period we have also been working on the next phase of MHRA guidance, which has a GxP focus (good laboratory practice, good clinical practice, good manufacturing practice, good distribution practice and good pharmacovigilence practice). Data integrity is important throughout the pharmaceutical lifecycle, and GxP regulatory requirements have a common focus of requiring confidence in the quality and the integrity of the data used for decision-making.

Availability of GxP guidance is an important step forward, as media reports and regulator websites continue to describe serious data integrity failures in clinical trial conduct and laboratory practice as well as in medicines manufacture.

The revised MHRA guidance builds on experience over the past 18 months; addressing some gaps in our previous guidance, and clarifying expectations where inspections have shown an unexpected interpretation of the requirements. In common with other regulatory partners, we have cross referenced to other relevant guidance, to avoid repetition.

Good manufacturing practice stakeholders may note that the revision contains less GMP-specific examples than the 2015 guidance. In the GxP version we have used examples applicable to more than one regulated area where illustration was important. This also demonstrates how the principles of data governance, and the way in which they can be implemented, can be very similar across all areas of the pharmaceutical lifecycle. This is important for organisations participating in research and commercial supply to consider when reviewing risks and control measures. Shared learning and common systems can save resources and avoid common failures.

Three month consultation on GxP data integrity guidance

Data integrity is everyone’s responsibility. There will therefore be a three-month public consultation on the GxP guidance which is your opportunity to engage with MHRA in developing guidance.  The consultation documents can be found on our website, with a closing date of 31 October 2016. Our data integrity team looks forward to your contribution to driving forward this important initiative.

Multi-indication pricing: no longer mission impossible?

                Pharmaphorum: Deep Dive, Leela Barham,

Will the push for value-based approaches to pricing, coupled with smarter use of data, mean multi-indication pricing becomes more widespread? Economic consultant Leela Barham examines the question.

MIP could help deliver more access for patients and incentivise more indications that offer value

If a single price reflects a higher-value indication, reimbursement agencies may not recommend use in a lower-value indication, restricting patient access

Italy illustrates that the infrastructure can be put in place for multi indication pricing – but it can take years to embed

Multi-indication pricing (MIP), also known as indication-specific pricing, is the simple idea that prices for drugs with more than one indication differ depending on value in each use.

However, though the idea may be simple, in practice having different prices for the same branded drug has proved less than straightforward.

More multi-indication drugs coming

Interest in the concept of MIP has been around for some time. It ties in with the general debate about how best to pay for new drugs. The difficulty lies in determining what is a reasonable price to pay in light of what the drug can really do, avoiding paying too much when times are tough, yet still paying enough to ensure incentives for more new drugs come through. All this has to be considered in the context of maintaining access for patients. This is hard enough when a product is for one indication; it is even harder when it is for more indications and where its value can differ markedly, yet there is a single price.

It is likely that many future drug launches will be for drugs with several indications. In oncology, for example, there could be a rise from 2014 levels, when just over half of products had more than one indication, to reach more than 75 per cent by 2020 (Figure 1). Not only that, but many will have more than three indications (Figure 2).

The appeal of MIP

The idea of MIP has appeal. For economists, price is a signal to the market place about a product. In multi-indication products with different values in use with the same price, that signal is not clear; those differences in value can be significant too. For example, according to the media, Roche’s Tarceva generates a 3.5-month extension in life versus chemotherapy in lung cancer. In pancreatic cancer, however, it is less than two weeks versus placebo.

If the single price reflects the higher-value indication, countries’ reimbursement agencies may not recommend use in a lower-value indication. Patients could be missing out. As the Organisation for Economic Co-operation and Development (OECD) noted back in 2013, ‘When medicines are approved for several indications which display very different cost-effectiveness ratios, most often the price of a medicine is unique, set at market entry and countries make a ‘yes or no’ decision for each new indication.’

If the single price reflects the lower-value indication that, in turn, may discourage the manufacturer from developing further, potentially higher-value, indications. Companies may also add that having a lower price than the value being delivered does little to reward innovation.

Future uses

The underlying issue for companies is the absence of perfect foresight in markets that only allow a single price; companies won’t necessarily know the full range of indications, nor specific timings, when they bring the first indication to market. They must try their best to determine a price that is likely to work across all indications over time, at launch of the first indication. They are, in essence, trying to set an average price across indications that will optimise access and maximise revenue at a single point in time.

On the other side, reimbursement agencies tend to assess use in a single indication (which is hard enough in itself, let alone trying to do that across multiple indications). When they look at price and value they are not, typically, concerned with the broader uses of a drug and whether, overall, the price makes sense ‘on average’ across all indications.

Of course companies have a lot of pricing freedom (buffered by much market intervention) but, understandably, they are seeking a commercial return. The solution of low prices for every indication isn’t particularly attractive for them. Nor is seeking separate marketing authorisations using different brand names, given the time, cost and inconvenience.

MIP could help deliver more access for patients and incentivise more indications that offer value

Moving to a system that permits MIP could help deliver more access for patients and incentivise more indications that offer value (even if lower value than existing higher-value indications). Systems would avoid over- or underpaying at the indication level.

The downside could be more money spent by the system overall (as more uses are reimbursed and brought to market) but, so the theory goes, it should offer value for money at the margin. This is the issue at a conceptual level; the practical issues of how to have many prices for the same drug are significant too.

Lack of clarity

What MIP would really mean for prices is unclear as it depends on how the price for each indication is worked out; prices could go down, or in some cases, up (Figure 4). As Peter Bach from the Memorial Sloan Kettering Cancer Centre pointed out, indication-specific pricing would “cut both ways. A relatively ineffective indication would not weigh down a value-based price if the drug were highly effective in another setting. Likewise a follow-on indication, for which the drug worked less well, would not drag down a value-based price set when the first indication was highly effective.”

The reality of MIP: limited

MIP may not be a widespread practice, but it has been done. For example, aflibercept is marketed as two separate products for use in ophthalmology and oncology. Perhaps having separate prices reflects differences in concentration and route of administration, so the product is somewhat different in physical terms.

Aside from ‘one-off’ examples of a handful of individual products, it is in Italy where MIP seems to have the infrastructure needed to operate.

Most innovative drugs used in Italian hospitals are included in web-based patient registries. They collect data on use by indication, not just by total volume. Over 100 registries cover thousands of patients – totalling over 350,000 in 2014 – so by now it must be more. In oncology alone, the registries covered 41 cancer drugs in 81 indications in 2014.

The registries were not set up to introduce MIP, but it seems a useful by-product; they are managed by the Italian Medicines Agency, so enable safety monitoring as well as managing reimbursement issues. The registries already mean that the transaction prices paid can change over time in light of results, since they include different performance-related schemes from the outset. There is no reason why these cannot be indication-specific.

The issue for companies is that they have to pay to add their product to the registry, and they may not get access to the data, as it is owned and analysed by the Italian Medicines Agency. There is also the obvious risk that pay-for-performance could hit their bottom line. However, it provides access (and revenue) where otherwise there would be none. The Italians set up the registries back in 2005 and the Agency stresses that time is needed to move from an administrative tool to an evidence-generating tool.

Mixed support

There are some who are in favour of MIP. The PharmaDiplomacy Group included the practicality of indication-specific pricing as part of an on-market price review in its proposed checklist for collaborative, mutually acceptable drug pricing in September 2015. IMS concluded, in its 2015 review of oncology, that ‘payment-by-use methodologies are required to match price with clinical value’, pointing out that ‘paying separate and distinct prices for patient groups, based on clinical value demonstrated by real-world evidence, may be preferred by payers and manufacturers’. There are reports that Germany could be interested in applying indication-specific pricing, partly to target use in appropriate sub-groups of patients.

Not everyone is convinced, though. The US-based Institute for Clinical and Economic Review’s (ICER) work, published in March 2016, outlines the challenges and risks and, on balance, suggests piloting the approach. US pharmacy benefit manager organisation Express Scripts seems to be doing just that.

In the UK, experts have highlighted that underpinning implementation of MIP would be data on use by indication and ways to reconcile the money. For example, rebates could be used and not necessarily different headline prices.

Although there are challenges, companies like Roche have projects in place to work through how MIP can be delivered in practice. The company has been focusing on using the Systemic Anti-Cancer Therapy (SACT) dataset and working with some English hospitals. That dataset should be collecting information on use by indication, as well as a host of other data across England. Not only would data challenges need to be overcome, but perhaps the attitudes of some working in the NHS too. When asked by Strategy&, PwC’s strategy consulting group, they thought indication-based pricing was one of the least attractive of the pricing and reimbursement options. However, the very small sample size – just 42 responses overall – should be taken into account (page 48 in linked document).

The future?

MIP could be one of a new wave of funding approaches to help respond to the myriad challenges for pricing and reimbursement for new drugs seen in health systems today. It has the potential to piggy-back on existing efforts to digitise health, and build on the long-running, value-based-pricing debate.

Perhaps the biggest threat to implementation is that of overall affordability; will payers accept an approach which could add to their drug spend, even if that offers value for money at the margin?

HDA Media And Political Bulletin – 22 July 2016

From Factory to Pharmacy

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