Thread by @biosectinvestor

Some quick thoughts on $NWBO and #DCVaxL. DCVaxL is designated an #OrphanDrug, for an #OrphanDisease, #Glioblastoma. This provides a bunch of benefits which are off topic for this thread.

My main point is to suggest that patients needing #DCVaxL may likely qualify for either of two funds in the UK, the #InnovativeMedicinesFund or the #CancerDrugsFund, which together have £640M allocated to provide early coverage to patients in the UK's NHS.

The UK charity, BrainTumorResearch.org recently put out a PR about the new program in the UK for rare diseases, which is modeled on an already existing program called the #CancerDrugsFund.

The #InnovativeMedicinesFund seeks to fast track new treatments to patients in the UK ASAP, once they are approved but before they have been evaluated as to pricing and coverage by the UK's NHS via the National Institute for Health and Care Excellence (NICE).

Quoting the PR: "Health and Social Care Secretary Sajid Javid said: “I want NHS patients to be the first in the world to access the most promising and revolutionary treatments that could extend or save their lives.”

"The fund will support NHS England in offering patients potentially transformative new drugs while further real-world evidence is collected to inform a final decision by the National Institute for Health and Care Excellence (NICE) on whether the treatment is clinically and cost…

…effective, reducing delays and boosting patient outcomes in the interim." braintumourresearch.org/media/news/new…

More relevantly, the charity went on to state: "Our Director of Research, Policy and Innovation, Dr Karen Noble said: “Accelerating access to potentially life-saving treatments is particularly critical for brain tumour patients, many of whom do not have the luxury of time.

We welcome the launch of this fund and hope that brain tumour patients will be amongst those benefitting from quicker access to novel treatments.

“The fact remains, though, that for brain tumour patients these new therapeutic options just haven’t been available.

The route to clinical innovation and new therapeutics remains underpinned by appropriate funding of early-stage discovery research, such as that which Brain Tumour Research funds at its Centres of Excellence.

That begins the translational pipeline and that is how we will find a cure.”"

In another PR related thereto, the same UK charity indicated: "The Innovative Medicines Fund will allow patients to have early access to the most clinically promising treatments without the need for lengthy assessments.

It is an extension of the existing Cancer Drugs Fund and will support patients with any condition, including those with rare and genetic diseases. Across both funds, £640 million has been ring-fenced annually for therapies." #DCVaxL $NWBO

Moreover, "The Chair of the Brain Tumour Research Scientific and Medical Advisory Board (SMAB) Emeritus Professor of Neurosurgery, Garth Cruickshank feels that this IMF initiative appears to be very good news for brain tumour patients. braintumourresearch.org/media/news/new…

He said: “For glioblastoma multiforme (GBM) patients this a real step forward as their life expectancy is so poor that they may not live long enough to be well enough to take such a new drug or to benefit from it.

Importantly, this initiative starts to unravel the conventional wisdom of having a long runway of testing before access to patients in a situation where such an approach has not borne much fruit in the last 30 years.”

However promising this sounds though, and we would hope that brain tumour patients such as those with recurrent GBM are early accessors to this programme, the fact remains that the drugs must be available for early access to be of benefit and for brain tumour patients these new…

…therapeutic options just haven’t been available. The route to clinical innovation and new therapeutics remains underpinned by appropriate funding of the early-stage research that begins the translational pipeline."

So many of the posts regarding what funds #DCVaxL patients are eligible for only seem to count the Innovative Medicines Fund, and not the Cancer Drugs Fund, and as indicated above, the TWO funds have a total of £640M available or approximately $785M USD at today's exchange…

…rate, for patients to access a variety of cancer drugs and drugs for rare diseases.

#DCVaxL has orphan drug designation in the US, EU and the UK, and I suspect Canada. Orphan drug designation is for rare diseases. Glioblastoma is also, obviously, a cancer.

Therefore it likely qualifies for either fund, once it receives initial approval but before it is approved by NICE, as a drug for the UK NHS, which has to determine separately from approval as to whether DCVax-L is cost effective to cover under the UK NHS.

"An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States." cancer.gov/publications/d… $NWBO #DCVaxL #Glioblastoma

"The Innovative Medicines Fund supports the government’s Rare Diseases Action Plan to ensure people living with rare conditions benefit from faster diagnosis, treatments and support to manage their conditions." gov.uk/government/new…
#Glioblastoma #DCVaxL $NWBO

"[The Innovative Medicines Fund] builds on the success of the reformed Cancer Drugs Fund which, in the past 5 years, has provided more than 80,000 people access to life-extending or potentially life-saving drugs which might otherwise not have been available for years."

gov.uk/government/new…

Note also, according to the BBC, in addition to the UK's £640M program, "A similar fund for innovative treatments - the New Medicines Fund - already exists in Scotland.

Wales has a New Treatments Fund that helps pay for high-cost drugs which have been recommended as cost-effective by NICE." bbc.com/news/health-61…

The BBC writes further, "The Innovative Medicines Fund (IMF) works like the existing Cancer Drugs Fund (CDF), fast-tracking promising treatments, even if they are expensive and have not yet been approved for routine NHS use.

It will cover potentially life-saving drugs for rare and genetic diseases.
The government has allocated up to £680m a year to be shared by the funds.

The IMF, like the CDF, will mean a newly approved medicine could be prescribed immediately, before final recommendations on it are drawn up by the advisory body that weighs the cost versus benefit of drugs used by the NHS - an organisation called NICE (the National Institute…

…for Health and Care Excellence).
Patients would be able to access the treatment while data is collected for NICE to determine whether the medicine is affordable and effective enough to offer more widely."

This thread should help clarify some of the confusion that limits the available funding to one program.