Hansard

First, I thank the clerks, the Scottish Parliament information centre and all those who contributed written evidence and gave of their time to participate in the committee’s public sessions. It is just as important to thank all those people who came forward over the period of the committee’s inquiry at a difficult and vulnerable time in their life and spoke out about the failings in terms of the patient experience. I am speaking on behalf of the Juszczak family in my constituency and the family of Anne Fisher, who is sadly deceased, who spoke out at a very difficult time. All those contributions ensured that the Health and Sport Committee approached the whole issue with sensitivity and consensus.

Access to medicines is a big, complex and deeply emotive issue. The committee first looked at the matter in March 2012 when we received a trio of petitions on orphan medicines. Now, as they say, for the science bit: orphan medicines are those used to treat very rare diseases. We have learned a lot in the past 18 months and I hope to share some of it with members in the next 13 minutes.

I want to retrace our steps as a reminder of why we are discussing the issue today. Along with the usual who, what and when, I will set out the main findings of the report that we published in early July, which is the how. I shall also offer some thoughts on yesterday’s statement from the cabinet secretary in the where-we-are-headed bit.

My colleagues on the Public Petitions Committee deserve credit for their role in this story. It was with their committee that the petitioners first raised their concerns. The petitioners were Alastair Kent, Allan Muir, Lesley Loeliger and Professor Peter Hillmen, individuals working on behalf of Rare Disease UK, the Association for Glycogen Storage Disease, and PNH Scotland, respectively. I am sure that they would all acknowledge the work on an earlier petition of January 2008 by Tina McGeever, on behalf of the late Mike Gray, which resulted in revision of the guidelines to the end-to-end process, which is the licensing of medicines through to individual patient treatment requests, or IPTRs.

The petitioners argued that the revision had not resulted in improved access to orphan medicines for patients with rare diseases. The committee took evidence last March from the petitioners and then from the Scottish Medicines Consortium, the SMC, and the Association of the British Pharmaceutical Industry, the ABPI. I apologise for all the acronyms—it is like a secret services convention.

We followed up the earlier evidence with evidence from clinicians and patient representative bodies. On 14 November 2012, the cabinet secretary announced the Routledge and Swainson reviews. The committee heard from the authors of the reviews and from the cabinet secretary and the chief pharmaceutical officer on 7 May 2013. On 21 May, we held a further round-table session with interested parties to gauge reaction to the twin reviews. The report of the committee’s findings was published on 3 July. The committee found that there is enthusiasm from all quarters to work together to improve the system for accessing new medicines and create a system that enables a wider assessment of their value with more of what might be termed a societal dimension.

Our report welcomes the recommendations from Swainson and Routledge, but we want both the IPTR and the SMC processes to be improved to ensure that we have a more transparent system for accessing new medicines. In short, we want more yeses. Many of the suggestions in the reviews are welcome, including those on meetings being held in public, the standardisation of paperwork, the monitoring of applications and the publication of decisions, but they are about process and would do little to improve access.

One of the difficulties with the IPTR system lies in establishing the exceptionality of the patient’s circumstances. We said that the Scottish Government must outline the steps that it will take to improve the system. Decisions on whether to recommend a medicine for use in Scotland depend on the cost of the additional quality-adjusted life years—a system known as QALY. I hope that the official reporters have a glossary, because there are a lot of acronyms in this area. Nobody told us of a better system than QALY for assessing the value of competing treatments. Who knew that an equation could be as brutal as cost divided by the number of weeks for which a life might be extended?

However, the way in which so-called modifiers are applied is crucial in determining the cost effectiveness of medicines. We asked the SMC and the Scottish Government to review how modifiers and thresholds are applied to take better account of orphan and ultra-orphan conditions, end of life and innovation. After all, our work began with the petitions on orphan and ultra-orphan medicines.

We welcomed the interim £21 million rare conditions medicines fund, but questions remain about the extent to which it can improve access to medicines. The committee said that clear guidance should be published and that decisions about specific cancer medicines should be made on the same basis as decisions on medicines for other conditions. We said that cancer should not be singled out in comparison with other life-shortening conditions. The committee accepted that this was a difficult issue. Nevertheless, we did not believe that a cancer drugs fund was the Scottish answer.

The committee recognised that there were concerns about the impact of innovative medicines not routinely being available in Scotland and we asked the Scottish Government to investigate. Likewise, we said that developments with value-based pricing, or VBP, should be monitored.

I am afraid that I do not have time to tell you about ADTCs, which are area drug and therapeutics committees, about PPRS, which is the pharmaceutical price regulation scheme or about NICE, which is the National Institute for Health and Care Excellence. I could say “NICE but naughty”—speaking of which, I note that it was only yesterday morning when we received a copy of the Scottish Government’s response to our report. That is a shame, as we would have liked to have fully considered it and come to a view as a committee.

What I can say is that I appreciate the language and the intention of what is proposed. The Cabinet Secretary for Health and Wellbeing wants to move to a more flexible approach to the evaluation of medicines for end-of-life care and rare conditions and he wants to increase access to new medicines, which is good. The committee and the cabinet secretary are on the same page, but we need to see the detail, which is where the devil lurks, as always.

The Scottish Government says that value-based pricing will not be delivered. It believes that the pricing element of pharmaceutical price regulation is a reserved matter but medicines assessment is devolved, and it will develop a new value-based assessment, or VBA, process for Scotland.

As a first step, the SMC has begun to look at the evaluation of orphan, ultra-orphan and end-of-life medicines. That is to include a review of the wider aspects of value and QALYs to increase access to those medicines. The report states that the SMC is due to report its findings to the cabinet secretary before Christmas. As somebody once said,

“I love deadlines. I like the whooshing sound they make as they fly by”.

Let this please be one of those deadlines that we are able to stick to. That is particularly important for those who have been diagnosed with these conditions last week or today, or who will be diagnosed with them tomorrow or next week. Some consideration should be given to how the system operates in the transitional phase, but we need to stick to the deadline.

The IPTR system is being replaced with a new peer approved clinical system—PACS. We are told that guidance will be published shortly and I seek assurances that the old double act of “postcode” and “lottery” are not reunited by PACS.

In all honesty, it is hard to tell whether the committee’s recommendations will be matched by the new systems, as we are short of information. I would be grateful if the cabinet secretary could offer some clues about timescales.

We are told that the rare conditions medicines fund will continue until 2016, but it is still unclear whether the £20 million is an annual budget or total funding until 2016. I would be obliged if the cabinet secretary could elaborate.

The Scottish Government agrees that there should be scope for a temporary pause in the appraisal process to permit further dialogue with the manufacturer. That was a recommendation from Routledge and the committee welcomed it. The Scottish Government says that a pause would allow a confidential discussion with the manufacturer about cost through a new or improved patient access scheme. However, no picture has yet emerged of how the scheme will look.

I have another one for the cabinet secretary, who I appreciate is listening patiently—I hope that he is not getting writer’s cramp as a result of taking notes.

There is to be £1 million of funding for the SMC’s engagement with the public and the pharmaceutical industry, but it is not clear whether more money would be required following the development of VBA.

Scepticism is a good thing, but let us give credit where credit is due. I sense that the direction in which we are heading is the right one. The frustration, for me, is that we have yet to arrive.

I want to say something more about the cost side. It is clear that the Scottish Government considers there to be a devolved element in that regard, and I want to make a couple of observations. In March, the chief executive officer of GlaxoSmithKline described the often-mentioned $1 billion research and development price tag as

“one of the great myths of the industry”.

I think that that is significant and interesting. Doctors Without Borders said:

“It is true that innovative new drugs can change the way we treat people and we need more of them. But innovation is of little use if people cannot access new treatments because they are so expensive.”

The pricing of medicines is, in many ways, a global issue. The issue is big, complex and deeply emotive—we had better believe it.

I commend the inclusive and listening approach of the person who will speak next in the debate. We have come a long way, policy-wise, in the past 18 months. Things have moved relatively quickly since the committee took evidence, and we welcome announcements. However, things can never move quickly enough for people who are diagnosed with rare conditions and terminal diseases. Three months is a lifetime to such people.

I hope that the cabinet secretary will report back to us on progress by December. Perhaps he can give us that undertaking. The committee believes that we can improve the processes. We can remove some of the bumps in what clinicians call the patient journey, and we can devise a system that is fair, objective, transparent, robust and within our means.

We need to ensure that there is access for all people who have orphan and ultra-orphan and life-threatening diseases. That is what people petitioned the Parliament for. We owe them nothing less and they deserve nothing less.

14:55